Autism Spectrum DisorderQ10ASDNCT04312152
Q10 Ubiquinol in Autism Spectrum Disorder and in Phelan-McDermid Syndrome.
A study testing Q10 Ubiquinol, Vitamin E and Multi-Vitamin B complex in people with Autism Spectrum Disorder and Phelan-McDermid Syndrome, run by Antonio Persico at 1 site in Italy.
Trial summary
This double-blind, cross-over, randomized, controlled trial (RCT) has the aim of evaluating the effectiveness of a metabolic support therapy in two cohorts of patients with idiopathic Autism Spectrum Disorder or Phelan-McDermid syndrome, commonly associated with syndromic autism. Read more about this study
- Who is it for?
- Ages 2 to 40 years
- Conditions the study is for
- Autism Spectrum Disorder, Phelan-McDermid Syndrome
- What is being tested?
- Q10 Ubiquinol, Vitamin E and Multi-Vitamin B complex
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 siteItaly
- Study dates
- 9 Mar 2019to 31 Aug 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Both parents or a legally authorized patient representative (LAR) must provide written informed consent. The parents and guardian must be able to understand and comply with the experimental protocol;
- Subjects of both sexes, aged between 2 and 40 years old, may be included in the study;
- The subject must meet DSM-5 criteria for a primary diagnosis of Autism Spectrum Disorder (idiopathic autism) or carry a documented deletion of human chromosome 22q13.33 or mutation in the SHANK3 gene (Phelan-McDermid Syndrome);
- Subjects with idiopathic autism must pass the threshold score for Autism of the Autism Diagnostic Observation Schedule;
- Baseline Children's Global Assessment Scale score must be between 45 and 59;
- Patients treated with psychoactive drugs (neuroleptics, antiepileptics, etc.) are enrolled only if the treatment and dosage of these drugs has been constant for at least 3 months prior to enrollment in the trial and is kept constant throughout the 8-month duration of the trial;
- Patients undergoing any kind of behavioral intervention must have must have started the intervention at least 3 months prior to enrollment and the intervention must remain unchanged throughout the 8-month duration of the trial;
- The patient is able to swallow the capsule or his/her parents are available to open it and administer immediately its content in a small quantity of juice or soft-drink.
Who cannot join
- Patients who meet any of the following criteria will not be recruited in the study:
- Patients with autism secondary to known genetic syndromes other than Phelan-McDermid syndrome (for example, Rett syndrome, fragile-X syndrome, etc.);
- Presence of brain malformations or major structural anomalies visible by magnetic resonance imaging;
- Patients with autism secondary to epileptic encephalopathy or with idiopathic autism comorbid with seizures more frequent than one episode every 6 months despite ongoing antiepileptic drug therapy;
- Patients with autism accompanied by marked facial dysmorphism and/or congenital malformations;
- Patients treated with anticoagulants;
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- Patients with serious medical illnesses (chronic renal disease, severe liver disease, cardiovascular disorders, uncontrolled hypertension with systolic pressure values> 170 and diastolic pressure> 100 mm Hg, malignant tumors, HIV infection);
- Patients with a history of acute cerebrovascular episodes;
- Patients with a history of stomach bleeding or active peptic ulcer;
- Patients with documented allergy, hypersensitivity or intolerance to one of the excipients of the experimental or comparative product.
- Trial interruption criteria:
- Patients whose medical conditions require starting treatment with anticoagulants.
- Patients with severe medical conditions starting during the 8-month duration of the trial.
- Patients who undergo a change in psychopharmacological or behavioral treatment during the 8-month duration of the trial.
Trial locations and contacts
1 site in 1 country.
Italy1 site
Interdipartimental Program "Autismo 0-90" at "G. Martino" Universitary Hospital
Messina, ME, I-98125
About this study
Official title: Randomized, Placebo-controlled, Cross-over, Double-blind Study of a Metabolic Support Therapy With Q10 Ubiquinol and a Multivitamin B and E Complex in Two Cohorts of Patients With Idiopathic and Syndromic Autism (Phelan-McDermid Syndrome)
This double-blind, cross-over, randomized, controlled trial (RCT) has the aim of evaluating the effectiveness of a metabolic support therapy in two cohorts of patients with idiopathic Autism Spectrum Disorder or Phelan-McDermid syndrome, commonly associated with syndromic autism. Each patient will receive Q10 ubiquinol + Vit. E and B for 4 months and only Vit. E and B for 4 months in a double-blind, cross-over design. Primary outcome measures of efficacy include Vineland Adaptive Behavior Scales, Childhood Autism Rating Scale, Clinical Global Impression-Improvement and Visual Analog Scales; secondary outcome measures include several questionnaires and tests of autism, cognitive function, problem behaviors, quality of life, communication and comorbid disorders, as well as measures of oxidative stress.
Read the detailed description
Autism Spectrum Disorder (ASD) is a clinically and genetically heterogeneous collection of different conditions, sharing socio-communicative deficits, repetitive behaviors, restricted interests, and dysfunctional sensory processing. Currently there are no pharmaceutical compounds effective on core ASD symptoms. Enhanced oxidative stress and mitochondrial dysfunction represent one of the most replicated abnormalities detected both systemically and in the Central Nervous System (CNS) of autistic individuals. Abnormalities in redox parameters are significantly correlated with the severity of autistic behaviors. Although oxidative stress usually represents the consequence and not the primary cause of ASD, reduced ATP production and oxidative damage can seemingly contribute an additional burden to the dysfunction directly produced by ASD-causing genetic or epigenetic defects. Importantly, redox abnormalities have been detected also in young autistic children and are not correlated with age. Therefore, enhanced oxidative stress and mitochondrial dysfunction represent an ASD-related "state-dependent" characteristic present in a consistent number of autistic individuals regardless of their age and of their specific underlying pathogenetic underpinnings. Sustaining mitochondrial function while controlling redox imbalance thus represents a viable "indirect" therapeutic approach, potentially able to ameliorate behavioral and neuropsychological deficits in many autistic individuals.
Coenzyme Q10 (CoQ10, ubiquinone or ubiquinol) is a lipid soluble compound present in the majority of living cells. By increasing energy production and antioxidant capacity, CoQ10 is predicted to limit the damage generated by the neuroinflammation and excitotoxicity well documented in ASD brains, ultimately leading to excessive neuritic pruning and/or cell apoptosis. Administration of Q10 ubiquinol to autistic children, as frequently prescribed to children with mitochondrial disorders, yielded promising results with an extremely low incidence and minor impact of side effects in two open trials and in three RCTs involving numerous other active compounds. In the present RCT, each patient will receive Q10 ubiquinol (50-100 mg b.i.d.) + Vit. E (60 mg/die) and polyvitamin B for 4 months and only Vit. E and B for another 4 months (total duration 8 months) in a double-blind, cross-over design. The focused co-administration of Q10 ubiquinol with only two known antioxidants, vitamin E and a multivitamin B complex, is designed to synergistically boost the increase in energy production and cell protection viewed as deriving primarily from Q10 ubiquinol administration. This study was also designed to overcome two limitations present in previous RCTs evaluating the effects of Q10 Ubiquinone (precursor of Q10 ubiquinol) in ASD children and adults: (a) The administration of a very limited number of active compounds, as compared to cocktails containing many active substances, allows to focus here on the efficacy of Q10 ubiquinol; (b) the administration of Q10 ubiquinol, rather than its precursor Q10 ubiquinone, avoids the potential risk of reduced response due to pharmacokinetic interference with the biotransformation of the precursor into the active compound.
This trial addresses the efficacy of Q10 ubiquinol, paired with Vit. E and B, not only in "idiopathic" ASD, but also in "syndromic" ASD, using Phelan-McDermid syndrome (PMS) as a paradigm. PMS, also known as chromosome 22q13.3 deletion syndrome, represents one of the most studied syndromic forms of ASD. It is characterized by autism in as many as 70-80% of deletion carriers, in addition to early onset severe muscle hypotonia, developmental delay, facial dysmorphisms, absence of spoken language or severe language development disorder. Deletions or mutations of the SHANK3 gene, encoding a synaptic scaffold protein critical to glutamatergic synapse function, are primarily responsible for the syndrome, although larger 22q13.3 deletions encompass additional disease genes.
This study shall include up to 140 patients with idiopathic ASD and 60 patients with PMS. The study design of this RCT was balanced, so that half of the patients with ASD or PMS will receive Q10 ubiquinol during the first 4 months, and the remaining half will receive Q10 ubiquinol during the second 4 months. The purpose of this balancing is to observe not only whether Q10 ubiquinol produces and improvement in primary and secondary measures, but also if this improvement is sustained over time despite Q10 discontinuation or requires continued Q10 administration. In addition to clinical and psychometric parameters, oxidative stress will be measured at baseline and after 4 and 8 months, by drawing 8-10 ml of blood, isolating leukocytes by Ficoll gradient and assessing (a) protein carbonylation levels by oxyblot; (b) the activity of mitochondrial respiratory chain complexes normalized by citrate synthase activity; (c) the expression levels of mitochondrial respiratory chain complexes measured by Western-Blotting.
- Conditions
- Autism Spectrum Disorder, Phelan-McDermid Syndrome
- Being tested
- Q10 Ubiquinol, Vitamin E, Multi-Vitamin B complex
- Keywords
- Ubiquinol-10, Coenzyme Q10, Autism, Mitochondria, Oxidative stress, Cognitive symptoms, Chromosome 22q13 3 deletion syndrome
- Medical subject headings
- Autism Spectrum Disorder, Telomeric 22q13 Monosomy Syndrome, Autistic Disorder, Neurobehavioral Manifestations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Q10 UbiquinolDietary supplement
Q10 Ubiquinol (50 or 100 mg b.i.d. depending on body weight) in capsules containing also Vit. E and Vit. B complex, as described above. Capsules can be opened and the content drunk or chewed, if children have difficulties with swallowing capsules.
Vitamin EDietary supplement
Also called tocopherols
Vitamin E (30 mg b.i.d. regardless of body weight) in capsules containing also Vit. B complex, and, in the active arms, Q10 ubiquinol. Capsules can be opened and the content drunk or chewed, if children have difficulties with swallowing capsules.
Multi-Vitamin B complexDietary supplement
Multi-Vitamin B complex including Vit. B1, B2, B3, B5, B6, B8, B9, and B12, in capsules containing also Vit. E and, in the active arms, Q10 ubiquinol. Capsules can be opened and the content drunk or chewed, if children have difficulties with swallowing capsules.
| Study group | Role | What they receive |
|---|---|---|
| PMS Placebo | Active comparator | If body weight is up to 20 kg:
* Vitamin E 30 mg b.i.d.
* Multivitamin B complex b.i.d., including Vit. B1 (1.05 mg), Vit. B2 (1.2 mg), Niacin (9 mg), Pantothenic acid (4.5 mg), Vit. B6 (1.5 mg), Vit. B12 (4.5 mcg), Folic acid (0.1 mg), Biotin (0.1125 mg)
If body weight is above 20 kg:
* Vitamin E 30 mg b.i.d.
* Multivitamin B complex b.i.d., including Vit. B1 (2.1 mg), Vit. B2 (2.4 mg), Niacin (18 mg), Pantothenic acid (9 mg), Vit. B6 (3 mg), Vit. B12 (9 mcg), Folic acid (0.2 mg), Biotin (0.225 mg) Receives: Dietary Supplement: Vitamin E; Dietary Supplement: Multi-Vitamin B complex |
| ASD Placebo | Active comparator | If body weight is up to 20 kg:
* Vitamin E 30 mg b.i.d.
* Multivitamin B complex b.i.d., including Vit. B1 (1.05 mg), Vit. B2 (1.2 mg), Niacin (9 mg), Pantothenic acid (4.5 mg), Vit. B6 (1.5 mg), Vit. B12 (4.5 mcg), Folic acid (0.1 mg), Biotin (0.1125 mg)
If body weight is above 20 kg:
* Vitamin E 30 mg b.i.d.
* Multivitamin B complex b.i.d., including Vit. B1 (2.1 mg), Vit. B2 (2.4 mg), Niacin (18 mg), Pantothenic acid (9 mg), Vit. B6 (3 mg), Vit. B12 (9 mcg), Folic acid (0.2 mg), Biotin (0.225 mg) Receives: Dietary Supplement: Vitamin E; Dietary Supplement: Multi-Vitamin B complex |
| PMS Active compound | Experimental | If body weight is up to 20 kg:
* Q10 ubiquinol, 50 mg b.i.d.
* Vitamin E 30 mg b.i.d.
* Multivitamin B complex b.i.d., including Vit. B1 (1.05 mg), Vit. B2 (1.2 mg), Niacin (9 mg), Pantothenic acid (4.5 mg), Vit. B6 (1.5 mg), Vit. B12 (4.5 mcg), Folic acid (0.1 mg), Biotin (0.1125 mg)
If body weight is above 20 kg:
* Q10 ubiquinol, 100 mg b.i.d.
* Vitamin E 30 mg b.i.d.
* Multivitamin B complex b.i.d., including Vit. B1 (2.1 mg), Vit. B2 (2.4 mg), Niacin (18 mg), Pantothenic acid (9 mg), Vit. B6 (3 mg), Vit. B12 (9 mcg), Folic acid (0.2 mg), Biotin (0.225 mg) Receives: Dietary Supplement: Q10 Ubiquinol; Dietary Supplement: Vitamin E; Dietary Supplement: Multi-Vitamin B complex |
| ASD Active compound | Experimental | If body weight is up to 20 kg:
* Q10 ubiquinol, 50 mg b.i.d.
* Vitamin E 30 mg b.i.d.
* Multivitamin B complex b.i.d., including Vit. B1 (1.05 mg), Vit. B2 (1.2 mg), Niacin (9 mg), Pantothenic acid (4.5 mg), Vit. B6 (1.5 mg), Vit. B12 (4.5 mcg), Folic acid (0.1 mg), Biotin (0.1125 mg)
If body weight is above 20 kg:
* Q10 ubiquinol, 100 mg b.i.d.
* Vitamin E 30 mg b.i.d.
* Multivitamin B complex b.i.d., including Vit. B1 (2.1 mg), Vit. B2 (2.4 mg), Niacin (18 mg), Pantothenic acid (9 mg), Vit. B6 (3 mg), Vit. B12 (9 mcg), Folic acid (0.2 mg), Biotin (0.225 mg) Receives: Dietary Supplement: Q10 Ubiquinol; Dietary Supplement: Vitamin E; Dietary Supplement: Multi-Vitamin B complex |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Vineland Adaptive Behavior Scales scores
The Vineland Adaptive Behavior Scales are a standardized semi-structured interview to measure adaptive behavior, among the most sensitive to change in autism research. Standard scores have a mean of 100 and a standard deviation of 15.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Change in Childhood Autism Rating Scale score
The Childhood Autism Rating Scale is a clinical rating scale for the trained clinician to rate the presence and severity of signs and symptoms of ASD by direct observation of the child. Scores can range from 15 to 60: below 30, non-autistic; 30-36.5, mild to moderate autism; 37-60, severe autism.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Change in Clinical Global Impression of Improvement scale scores between experimental and active comparator arms.
The Clinical Global Impression of Improvement scale is a 7 point scale for the clinician to quantify illness severity, patient improvement/worsening and treatment side effects. Scores recorded at the end of the experimental and active comparator arms will be contrasted within-subject.
Measured: 4 and 8 months (record once at the end of each arm)
Change in Visual Analog Scales scores
16 visual analog scales have been created to measure all DSM-5 items included in the ASD diagnosis, as well as other cognitive and motor functions often affected in ASD. Scores measure the increasing severity of signs and symptoms on a 0-10 scale.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Secondary outcomes (13)
Children's Global Assessment Scale
The Children's Global Assessment Scale provides a global measure of level of functioning in children and adolescents. The measure provides a single rating on a 0-100 scale, with higher scores indicating better functioning.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Social Responsiveness Scale
65-item questionnaire used to assess social impairment, communication deficits and repetitive behaviors in children and adolescents 4-18 years old. Autism is severe, moderate, or mild when T-scores are above 75, 66-75, or 60-65, respectively. Scores below 60 are not clinically significant.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Repetitive Behaviors Scale - Revised
44-item questionnaire used to assess repetitive behaviors. Filled by parents for children 6-17 years old. Behaviors are rated on a 0-3 scale to measure increasing symptom severity, as does the overall global score ranging 0-100.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Aberrant Behavior Checklist
58-item questionnaire used to assess problem behaviors with parents or guardians. Behaviors are rated on a 0-3 scale to measure increasing symptom severity.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Short Sensory Profile
38-item questionnaire filled by caregivers to assess the sensory profile of patients, including sensory processing, modulation, and behavioral/emotional responses. The overall score ranges from 0 to 190, with lower scores reflecting greater symptom severity.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Conners' Parent Rating Scale-Revised
48-item rating scale used to evaluate through parental reports the presence and intensity of childhood hyperactivity/inattention, impulsivity and externalizing behaviors. Each item is rated on a 0-3 scale to reflect increasing symptom severity.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Child Behavior Checklist
The Child Behavior Checklist/6-18 provides ratings for 20 competence and 120 behavioral problem items of youth aged 6-18 years old by parental report. Each item is scored 0-2, to reflect symptom severity or frequency. Standard scores are scaled so that 50 is average for the youth's age and gender, with a standard deviation of 10 points.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Intellectual Quotient
Intellectual quotient measured as a standardized score with population mean 100 and standard deviation 15, using one cognitive test per subject, chosen depending on age and language development (either Griffiths Developmental Rating Scales, Wechsler Intelligence Scale for Children - Fourth Edition, or Leiter III).
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
The Quality of Life in Autism Questionnaire
A questionnaire used to assess parental quality of life either broadly (part A, 28 items, score range 28-140) or specifically related to the autism present in their offspring (part B, 20 items, score range 20-100). Total score range 48-240, with higher scores indicating better quality of life.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
The World Health Organization's Quality of Life Questionnaire
A questionnaire used to assess parental quality of life in four domains: physical, psychological, social, and environmental. The score can range from 15 to 105, with a higher score being indicative of a higher quality of life.
Measured: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Measurement of protein carbonylation level as a marker of oxidative stress in leukocytes.
Blood drawing, isolation of leukocytes by Ficoll gradient and measurement of protein carbonylation by oxyblot.
Measured: Blood drawn at 0, 4 and 8 months (pre-and post-treatment after each arm).
Measurement of the activity of mitochondrial respiratory chain complexes.
Blood drawing, isolation of leukocytes by Ficoll gradient and measurement of the activity of mitochondrial respiratory chain complexes normalized by citrate synthase activity.
Measured: Blood drawn at 0, 4 and 8 months (pre-and post-treatment after each arm).
Measurement of expression levels of mitochondrial respiratory chain complexes.
Blood drawing, isolation of leukocytes by Ficoll gradient and measurement of the expression levels of mitochondrial respiratory chain complexes by Western-Blotting.
Measured: Blood drawn at 0, 4 and 8 months (pre-and post-treatment after each arm).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2020
- Study start9 Mar 2019
- Primary completion31 Dec 2022 (estimated)
- Study completion31 Aug 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (17)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Each patient receives active compound \[Q10 ubiquinol + Vit. E and polyvitamin B\] for 4 months and placebo \[Vit. E and B\] for another 4 months. The total duration of the trial is 8 months. Half of the patients receive the active compound during months 1-4 and placebo during months 5-8, while the remaining half receives placebo during months 1-4 and active compound during months 5-8. Patients are assessed at T0, T4 and T8 mo. Administration of active compound and placebo is in double-blind.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Antonio Persico
- Sponsor type
- University, hospital or other
- Collaborators
- Associazione Italiana Sindrome di Phelan-McDermid (AISPHEM), University of Bari
- Responsible party
- Sponsor-investigator, Antonio Persico, University of Messina
- Investigators
- Antonio M. Persico, MD, Principal investigator, University of Messina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04312152
- Sponsor's protocol ID
- Q10_study_ASD
- Acronym
- Q10ASD
- Status last verified
- Mar 2020
- First submitted
- 9 Mar 2020
- Last updated
- 18 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
American Psychiatric Association. Diagnostic and Statistical Manual of Mental Disorders. In: Association AP, editor. Fifth Edition ed: American Psychiatric Publishing, Arlington; 2013.
Persico AM, Arango C, Buitelaar JK, Correll CU, Glennon JC, Hoekstra PJ, Moreno C, Vitiello B, Vorstman J, Zuddas A; European Child and Adolescent Clinical Psychopharmacology Network. Unmet needs in paediatric psychopharmacology: Present scenario and future perspectives. Eur Neuropsychopharmacol. 2015 Oct;25(10):1513-31. doi: 10.1016/j.euroneuro.2015.06.009. Epub 2015 Jun 20.
PubMed 26166453Kalayci M, Unal MM, Gul S, Acikgoz S, Kandemir N, Hanci V, Edebali N, Acikgoz B. Effect of coenzyme Q10 on ischemia and neuronal damage in an experimental traumatic brain-injury model in rats. BMC Neurosci. 2011 Jul 29;12:75. doi: 10.1186/1471-2202-12-75.
PubMed 21801363Kumari S, Mehta SL, Milledge GZ, Huang X, Li H, Li PA. Ubisol-Q10 Prevents Glutamate-Induced Cell Death by Blocking Mitochondrial Fragmentation and Permeability Transition Pore Opening. Int J Biol Sci. 2016 Apr 27;12(6):688-700. doi: 10.7150/ijbs.13589. eCollection 2016.
PubMed 27194946Duberley KE, Heales SJ, Abramov AY, Chalasani A, Land JM, Rahman S, Hargreaves IP. Effect of Coenzyme Q10 supplementation on mitochondrial electron transport chain activity and mitochondrial oxidative stress in Coenzyme Q10 deficient human neuronal cells. Int J Biochem Cell Biol. 2014 May;50:60-3. doi: 10.1016/j.biocel.2014.02.003. Epub 2014 Feb 15.
PubMed 24534273Rossignol DA, Frye RE. Evidence linking oxidative stress, mitochondrial dysfunction, and inflammation in the brain of individuals with autism. Front Physiol. 2014 Apr 22;5:150. doi: 10.3389/fphys.2014.00150. eCollection 2014.
PubMed 24795645Frustaci A, Neri M, Cesario A, Adams JB, Domenici E, Dalla Bernardina B, Bonassi S. Oxidative stress-related biomarkers in autism: systematic review and meta-analyses. Free Radic Biol Med. 2012 May 15;52(10):2128-41. doi: 10.1016/j.freeradbiomed.2012.03.011. Epub 2012 Apr 18.
PubMed 22542447Adams JB, Baral M, Geis E, Mitchell J, Ingram J, Hensley A, Zappia I, Newmark S, Gehn E, Rubin RA, Mitchell K, Bradstreet J, El-Dahr JM. The severity of autism is associated with toxic metal body burden and red blood cell glutathione levels. J Toxicol. 2009;2009:532640. doi: 10.1155/2009/532640. Epub 2009 Aug 26.
PubMed 20107587Ghezzo A, Visconti P, Abruzzo PM, Bolotta A, Ferreri C, Gobbi G, Malisardi G, Manfredini S, Marini M, Nanetti L, Pipitone E, Raffaelli F, Resca F, Vignini A, Mazzanti L. Oxidative Stress and Erythrocyte Membrane Alterations in Children with Autism: Correlation with Clinical Features. PLoS One. 2013 Jun 19;8(6):e66418. doi: 10.1371/journal.pone.0066418. Print 2013.
PubMed 23840462Adams JB, Holloway C. Pilot study of a moderate dose multivitamin/mineral supplement for children with autistic spectrum disorder. J Altern Complement Med. 2004 Dec;10(6):1033-9. doi: 10.1089/acm.2004.10.1033.
PubMed 15673999Adams JB, Audhya T, McDonough-Means S, Rubin RA, Quig D, Geis E, Gehn E, Loresto M, Mitchell J, Atwood S, Barnhouse S, Lee W. Effect of a vitamin/mineral supplement on children and adults with autism. BMC Pediatr. 2011 Dec 12;11:111. doi: 10.1186/1471-2431-11-111.
PubMed 22151477Adams JB, George F, Audhya T. Abnormally high plasma levels of vitamin B6 in children with autism not taking supplements compared to controls not taking supplements. J Altern Complement Med. 2006 Jan-Feb;12(1):59-63. doi: 10.1089/acm.2006.12.59.
PubMed 16494569Palmieri L, Persico AM. Mitochondrial dysfunction in autism spectrum disorders: cause or effect? Biochim Biophys Acta. 2010 Jun-Jul;1797(6-7):1130-7. doi: 10.1016/j.bbabio.2010.04.018. Epub 2010 May 9.
PubMed 20441769Gvozdjakova A, Kucharska J, Ostatnikova D, Babinska K, Nakladal D, Crane FL. Ubiquinol improves symptoms in children with autism. Oxid Med Cell Longev. 2014;2014:798957. doi: 10.1155/2014/798957. Epub 2014 Feb 23.
PubMed 24707344Mousavinejad E, Ghaffari MA, Riahi F, Hajmohammadi M, Tiznobeyk Z, Mousavinejad M. Coenzyme Q10 supplementation reduces oxidative stress and decreases antioxidant enzyme activity in children with autism spectrum disorders. Psychiatry Res. 2018 Jul;265:62-69. doi: 10.1016/j.psychres.2018.03.061. Epub 2018 Apr 4.
PubMed 29684771
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Placebo-controlled, Cross-over, Double-blind Study of a Metabolic Support Therapy With Q10 Ubiquinol and a Multivitamin B and E Complex in Two Cohorts of Patients With Idiopathic and Syndromic Autism (Phelan-McDermid Syndrome)
- ClinicalTrials.gov ID
- NCT04312152
- Acronym / sponsor ID
- Q10ASD / Q10_study_ASD
- Conditions
- Autism Spectrum Disorder, Phelan-McDermid Syndrome
- Keywords
- Ubiquinol-10, Coenzyme Q10, Autism, Mitochondria, Oxidative stress, Cognitive symptoms, Chromosome 22q13 3 deletion syndrome
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Antonio PersicoUniversity, hospital or other
- Collaborators
- Associazione Italiana Sindrome di Phelan-McDermid (AISPHEM), University of Bari
- Study start
- 9 Mar 2019 (actual)
- Primary completion
- 31 Dec 2022 (estimated)
- Study completion
- 31 Aug 2023 (estimated)
- First posted
- 18 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This double-blind, cross-over, randomized, controlled trial (RCT) has the aim of evaluating the effectiveness of a metabolic support therapy in two cohorts of patients with idiopathic Autism Spectrum Disorder or Phelan-McDermid syndrome, commonly associated with syndromic autism. Each patient will receive Q10 ubiquinol + Vit. E and B for 4 months and only Vit. E and B for 4 months in a double-blind, cross-over design. Primary outcome measures of efficacy include Vineland Adaptive Behavior Scales, Childhood Autism Rating Scale, Clinical Global Impression-Improvement and Visual Analog Scales; secondary outcome measures include several questionnaires and tests of autism, cognitive function, problem behaviors, quality of life, communication and comorbid disorders, as well as measures of oxidative stress.
Detailed description
Autism Spectrum Disorder (ASD) is a clinically and genetically heterogeneous collection of different conditions, sharing socio-communicative deficits, repetitive behaviors, restricted interests, and dysfunctional sensory processing. Currently there are no pharmaceutical compounds effective on core ASD symptoms. Enhanced oxidative stress and mitochondrial dysfunction represent one of the most replicated abnormalities detected both systemically and in the Central Nervous System (CNS) of autistic individuals. Abnormalities in redox parameters are significantly correlated with the severity of autistic behaviors. Although oxidative stress usually represents the consequence and not the primary cause of ASD, reduced ATP production and oxidative damage can seemingly contribute an additional burden to the dysfunction directly produced by ASD-causing genetic or epigenetic defects. Importantly, redox abnormalities have been detected also in young autistic children and are not correlated with age. Therefore, enhanced oxidative stress and mitochondrial dysfunction represent an ASD-related "state-dependent" characteristic present in a consistent number of autistic individuals regardless of their age and of their specific underlying pathogenetic underpinnings. Sustaining mitochondrial function while controlling redox imbalance thus represents a viable "indirect" therapeutic approach, potentially able to ameliorate behavioral and neuropsychological deficits in many autistic individuals.
Coenzyme Q10 (CoQ10, ubiquinone or ubiquinol) is a lipid soluble compound present in the majority of living cells. By increasing energy production and antioxidant capacity, CoQ10 is predicted to limit the damage generated by the neuroinflammation and excitotoxicity well documented in ASD brains, ultimately leading to excessive neuritic pruning and/or cell apoptosis. Administration of Q10 ubiquinol to autistic children, as frequently prescribed to children with mitochondrial disorders, yielded promising results with an extremely low incidence and minor impact of side effects in two open trials and in three RCTs involving numerous other active compounds. In the present RCT, each patient will receive Q10 ubiquinol (50-100 mg b.i.d.) + Vit. E (60 mg/die) and polyvitamin B for 4 months and only Vit. E and B for another 4 months (total duration 8 months) in a double-blind, cross-over design. The focused co-administration of Q10 ubiquinol with only two known antioxidants, vitamin E and a multivitamin B complex, is designed to synergistically boost the increase in energy production and cell protection viewed as deriving primarily from Q10 ubiquinol administration. This study was also designed to overcome two limitations present in previous RCTs evaluating the effects of Q10 Ubiquinone (precursor of Q10 ubiquinol) in ASD children and adults: (a) The administration of a very limited number of active compounds, as compared to cocktails containing many active substances, allows to focus here on the efficacy of Q10 ubiquinol; (b) the administration of Q10 ubiquinol, rather than its precursor Q10 ubiquinone, avoids the potential risk of reduced response due to pharmacokinetic interference with the biotransformation of the precursor into the active compound.
This trial addresses the efficacy of Q10 ubiquinol, paired with Vit. E and B, not only in "idiopathic" ASD, but also in "syndromic" ASD, using Phelan-McDermid syndrome (PMS) as a paradigm. PMS, also known as chromosome 22q13.3 deletion syndrome, represents one of the most studied syndromic forms of ASD. It is characterized by autism in as many as 70-80% of deletion carriers, in addition to early onset severe muscle hypotonia, developmental delay, facial dysmorphisms, absence of spoken language or severe language development disorder. Deletions or mutations of the SHANK3 gene, encoding a synaptic scaffold protein critical to glutamatergic synapse function, are primarily responsible for the syndrome, although larger 22q13.3 deletions encompass additional disease genes.
This study shall include up to 140 patients with idiopathic ASD and 60 patients with PMS. The study design of this RCT was balanced, so that half of the patients with ASD or PMS will receive Q10 ubiquinol during the first 4 months, and the remaining half will receive Q10 ubiquinol during the second 4 months. The purpose of this balancing is to observe not only whether Q10 ubiquinol produces and improvement in primary and secondary measures, but also if this improvement is sustained over time despite Q10 discontinuation or requires continued Q10 administration. In addition to clinical and psychometric parameters, oxidative stress will be measured at baseline and after 4 and 8 months, by drawing 8-10 ml of blood, isolating leukocytes by Ficoll gradient and assessing (a) protein carbonylation levels by oxyblot; (b) the activity of mitochondrial respiratory chain complexes normalized by citrate synthase activity; (c) the expression levels of mitochondrial respiratory chain complexes measured by Western-Blotting.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- Model description
- Each patient receives active compound [Q10 ubiquinol + Vit. E and polyvitamin B] for 4 months and placebo [Vit. E and B] for another 4 months. The total duration of the trial is 8 months. Half of the patients receive the active compound during months 1-4 and placebo during months 5-8, while the remaining half receives placebo during months 1-4 and active compound during months 5-8. Patients are assessed at T0, T4 and T8 mo. Administration of active compound and placebo is in double-blind.
| Arm | Type | Receives |
|---|---|---|
| PMS Placebo | Active comparator | Dietary Supplement: Vitamin E, Dietary Supplement: Multi-Vitamin B complexIf body weight is up to 20 kg: * Vitamin E 30 mg b.i.d. * Multivitamin B complex b.i.d., including Vit. B1 (1.05 mg), Vit. B2 (1.2 mg), Niacin (9 mg), Pantothenic acid (4.5 mg), Vit. B6 (1.5 mg), Vit. B12 (4.5 mcg), Folic acid (0.1 mg), Biotin (0.1125 mg) If body weight is above 20 kg: * Vitamin E 30 mg b.i.d. * Multivitamin B complex b.i.d., including Vit. B1 (2.1 mg), Vit. B2 (2.4 mg), Niacin (18 mg), Pantothenic acid (9 mg), Vit. B6 (3 mg), Vit. B12 (9 mcg), Folic acid (0.2 mg), Biotin (0.225 mg) |
| ASD Placebo | Active comparator | Dietary Supplement: Vitamin E, Dietary Supplement: Multi-Vitamin B complexIf body weight is up to 20 kg: * Vitamin E 30 mg b.i.d. * Multivitamin B complex b.i.d., including Vit. B1 (1.05 mg), Vit. B2 (1.2 mg), Niacin (9 mg), Pantothenic acid (4.5 mg), Vit. B6 (1.5 mg), Vit. B12 (4.5 mcg), Folic acid (0.1 mg), Biotin (0.1125 mg) If body weight is above 20 kg: * Vitamin E 30 mg b.i.d. * Multivitamin B complex b.i.d., including Vit. B1 (2.1 mg), Vit. B2 (2.4 mg), Niacin (18 mg), Pantothenic acid (9 mg), Vit. B6 (3 mg), Vit. B12 (9 mcg), Folic acid (0.2 mg), Biotin (0.225 mg) |
| PMS Active compound | Experimental | Dietary Supplement: Q10 Ubiquinol, Dietary Supplement: Vitamin E, Dietary Supplement: Multi-Vitamin B complexIf body weight is up to 20 kg: * Q10 ubiquinol, 50 mg b.i.d. * Vitamin E 30 mg b.i.d. * Multivitamin B complex b.i.d., including Vit. B1 (1.05 mg), Vit. B2 (1.2 mg), Niacin (9 mg), Pantothenic acid (4.5 mg), Vit. B6 (1.5 mg), Vit. B12 (4.5 mcg), Folic acid (0.1 mg), Biotin (0.1125 mg) If body weight is above 20 kg: * Q10 ubiquinol, 100 mg b.i.d. * Vitamin E 30 mg b.i.d. * Multivitamin B complex b.i.d., including Vit. B1 (2.1 mg), Vit. B2 (2.4 mg), Niacin (18 mg), Pantothenic acid (9 mg), Vit. B6 (3 mg), Vit. B12 (9 mcg), Folic acid (0.2 mg), Biotin (0.225 mg) |
| ASD Active compound | Experimental | Dietary Supplement: Q10 Ubiquinol, Dietary Supplement: Vitamin E, Dietary Supplement: Multi-Vitamin B complexIf body weight is up to 20 kg: * Q10 ubiquinol, 50 mg b.i.d. * Vitamin E 30 mg b.i.d. * Multivitamin B complex b.i.d., including Vit. B1 (1.05 mg), Vit. B2 (1.2 mg), Niacin (9 mg), Pantothenic acid (4.5 mg), Vit. B6 (1.5 mg), Vit. B12 (4.5 mcg), Folic acid (0.1 mg), Biotin (0.1125 mg) If body weight is above 20 kg: * Q10 ubiquinol, 100 mg b.i.d. * Vitamin E 30 mg b.i.d. * Multivitamin B complex b.i.d., including Vit. B1 (2.1 mg), Vit. B2 (2.4 mg), Niacin (18 mg), Pantothenic acid (9 mg), Vit. B6 (3 mg), Vit. B12 (9 mcg), Folic acid (0.2 mg), Biotin (0.225 mg) |
Who may qualify
- Ages
- 2 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Both parents or a legally authorized patient representative (LAR) must provide written informed consent. The parents and guardian must be able to understand and comply with the experimental protocol;
- Subjects of both sexes, aged between 2 and 40 years old, may be included in the study;
- The subject must meet DSM-5 criteria for a primary diagnosis of Autism Spectrum Disorder (idiopathic autism) or carry a documented deletion of human chromosome 22q13.33 or mutation in the SHANK3 gene (Phelan-McDermid Syndrome);
- Subjects with idiopathic autism must pass the threshold score for Autism of the Autism Diagnostic Observation Schedule;
- Baseline Children's Global Assessment Scale score must be between 45 and 59;
Key exclusion criteria (14 listed)
- Patients with autism secondary to known genetic syndromes other than Phelan-McDermid syndrome (for example, Rett syndrome, fragile-X syndrome, etc.);
- Presence of brain malformations or major structural anomalies visible by magnetic resonance imaging;
- Patients with autism secondary to epileptic encephalopathy or with idiopathic autism comorbid with seizures more frequent than one episode every 6 months despite ongoing antiepileptic drug therapy;
- Patients with autism accompanied by marked facial dysmorphism and/or congenital malformations;
- Patients treated with anticoagulants;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Q10 UbiquinolQ10 Ubiquinol (50 or 100 mg b.i.d. depending on body weight) in capsules containing also Vit. E and Vit. B complex, as described above. Capsules can be opened and the content drunk or chewed, if children have difficulties with swallowing capsules. | Dietary supplement | ASD Active compound, PMS Active compound |
| Vitamin EAlso: tocopherolsVitamin E (30 mg b.i.d. regardless of body weight) in capsules containing also Vit. B complex, and, in the active arms, Q10 ubiquinol. Capsules can be opened and the content drunk or chewed, if children have difficulties with swallowing capsules. | Dietary supplement | ASD Active compound, ASD Placebo, PMS Active compound, PMS Placebo |
| Multi-Vitamin B complexMulti-Vitamin B complex including Vit. B1, B2, B3, B5, B6, B8, B9, and B12, in capsules containing also Vit. E and, in the active arms, Q10 ubiquinol. Capsules can be opened and the content drunk or chewed, if children have difficulties with swallowing capsules. | Dietary supplement | ASD Active compound, ASD Placebo, PMS Active compound, PMS Placebo |
Primary outcome
Change in Vineland Adaptive Behavior Scales scores
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
The Vineland Adaptive Behavior Scales are a standardized semi-structured interview to measure adaptive behavior, among the most sensitive to change in autism research. Standard scores have a mean of 100 and a standard deviation of 15.
Change in Childhood Autism Rating Scale score
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
The Childhood Autism Rating Scale is a clinical rating scale for the trained clinician to rate the presence and severity of signs and symptoms of ASD by direct observation of the child. Scores can range from 15 to 60: below 30, non-autistic; 30-36.5, mild to moderate autism; 37-60, severe autism.
Change in Clinical Global Impression of Improvement scale scores between experimental and active comparator arms.
Time frame: 4 and 8 months (record once at the end of each arm)
The Clinical Global Impression of Improvement scale is a 7 point scale for the clinician to quantify illness severity, patient improvement/worsening and treatment side effects. Scores recorded at the end of the experimental and active comparator arms will be contrasted within-subject.
Change in Visual Analog Scales scores
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
16 visual analog scales have been created to measure all DSM-5 items included in the ASD diagnosis, as well as other cognitive and motor functions often affected in ASD. Scores measure the increasing severity of signs and symptoms on a 0-10 scale.
13 secondary outcomes
Children's Global Assessment Scale
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Social Responsiveness Scale
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Repetitive Behaviors Scale - Revised
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Aberrant Behavior Checklist
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Short Sensory Profile
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Conners' Parent Rating Scale-Revised
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Child Behavior Checklist
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Intellectual Quotient
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
The Quality of Life in Autism Questionnaire
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
The World Health Organization's Quality of Life Questionnaire
Time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)
Measurement of protein carbonylation level as a marker of oxidative stress in leukocytes.
Time frame: Blood drawn at 0, 4 and 8 months (pre-and post-treatment after each arm).
Measurement of the activity of mitochondrial respiratory chain complexes.
Time frame: Blood drawn at 0, 4 and 8 months (pre-and post-treatment after each arm).
Measurement of expression levels of mitochondrial respiratory chain complexes.
Time frame: Blood drawn at 0, 4 and 8 months (pre-and post-treatment after each arm).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2020.
- BackgroundAmerican Psychiatric Association. Diagnostic and Statistical Manual of Mental Disorders. In: Association AP, editor. Fifth Edition ed: American Psychiatric Publishing, Arlington; 2013.
- Background: PubMed 26166453Persico AM, Arango C, Buitelaar JK, Correll CU, Glennon JC, Hoekstra PJ, Moreno C, Vitiello B, Vorstman J, Zuddas A; European Child and Adolescent Clinical Psychopharmacology Network. Unmet needs in paediatric psychopharmacology: Present scenario and future perspectives. Eur Neuropsychopharmacol. 2015 Oct;25(10):1513-31. doi: 10.1016/j.euroneuro.2015.06.009. Epub 2015 Jun 20.
- Background: PubMed 21801363Kalayci M, Unal MM, Gul S, Acikgoz S, Kandemir N, Hanci V, Edebali N, Acikgoz B. Effect of coenzyme Q10 on ischemia and neuronal damage in an experimental traumatic brain-injury model in rats. BMC Neurosci. 2011 Jul 29;12:75. doi: 10.1186/1471-2202-12-75.
- Background: PubMed 27194946Kumari S, Mehta SL, Milledge GZ, Huang X, Li H, Li PA. Ubisol-Q10 Prevents Glutamate-Induced Cell Death by Blocking Mitochondrial Fragmentation and Permeability Transition Pore Opening. Int J Biol Sci. 2016 Apr 27;12(6):688-700. doi: 10.7150/ijbs.13589. eCollection 2016.
- Background: PubMed 24534273Duberley KE, Heales SJ, Abramov AY, Chalasani A, Land JM, Rahman S, Hargreaves IP. Effect of Coenzyme Q10 supplementation on mitochondrial electron transport chain activity and mitochondrial oxidative stress in Coenzyme Q10 deficient human neuronal cells. Int J Biochem Cell Biol. 2014 May;50:60-3. doi: 10.1016/j.biocel.2014.02.003. Epub 2014 Feb 15.
- Background: PubMed 24795645Rossignol DA, Frye RE. Evidence linking oxidative stress, mitochondrial dysfunction, and inflammation in the brain of individuals with autism. Front Physiol. 2014 Apr 22;5:150. doi: 10.3389/fphys.2014.00150. eCollection 2014.
- Background: PubMed 22542447Frustaci A, Neri M, Cesario A, Adams JB, Domenici E, Dalla Bernardina B, Bonassi S. Oxidative stress-related biomarkers in autism: systematic review and meta-analyses. Free Radic Biol Med. 2012 May 15;52(10):2128-41. doi: 10.1016/j.freeradbiomed.2012.03.011. Epub 2012 Apr 18.
- Background: PubMed 20107587Adams JB, Baral M, Geis E, Mitchell J, Ingram J, Hensley A, Zappia I, Newmark S, Gehn E, Rubin RA, Mitchell K, Bradstreet J, El-Dahr JM. The severity of autism is associated with toxic metal body burden and red blood cell glutathione levels. J Toxicol. 2009;2009:532640. doi: 10.1155/2009/532640. Epub 2009 Aug 26.
- Background: PubMed 23840462Ghezzo A, Visconti P, Abruzzo PM, Bolotta A, Ferreri C, Gobbi G, Malisardi G, Manfredini S, Marini M, Nanetti L, Pipitone E, Raffaelli F, Resca F, Vignini A, Mazzanti L. Oxidative Stress and Erythrocyte Membrane Alterations in Children with Autism: Correlation with Clinical Features. PLoS One. 2013 Jun 19;8(6):e66418. doi: 10.1371/journal.pone.0066418. Print 2013.
- Background: PubMed 15673999Adams JB, Holloway C. Pilot study of a moderate dose multivitamin/mineral supplement for children with autistic spectrum disorder. J Altern Complement Med. 2004 Dec;10(6):1033-9. doi: 10.1089/acm.2004.10.1033.
- Background: PubMed 22151477Adams JB, Audhya T, McDonough-Means S, Rubin RA, Quig D, Geis E, Gehn E, Loresto M, Mitchell J, Atwood S, Barnhouse S, Lee W. Effect of a vitamin/mineral supplement on children and adults with autism. BMC Pediatr. 2011 Dec 12;11:111. doi: 10.1186/1471-2431-11-111.
- Background: PubMed 16494569Adams JB, George F, Audhya T. Abnormally high plasma levels of vitamin B6 in children with autism not taking supplements compared to controls not taking supplements. J Altern Complement Med. 2006 Jan-Feb;12(1):59-63. doi: 10.1089/acm.2006.12.59.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Antonio M. Persico, MD principal investigator
University of Messina
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Q10 Ubiquinol, Vitamin E and Multi-Vitamin B complex in people with Autism Spectrum Disorder and Phelan-McDermid Syndrome, run by Antonio Persico at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Vineland Adaptive Behavior Scales scores (time frame: At 0, 4 and 8 months (pre- and post-treatment after each arm)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04312152, which also lists 17 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Mar 2020.