Fragile X Associated Tremor/Ataxia Syndrome (Fxtas) (Diagnosis)NCT05233579
Open-Label Trial of Sulforaphane in Premutation Carriers With FXTAS
A study testing Sulforaphane in people with Fragile X Associated Tremor/Ataxia Syndrome (Fxtas) (Diagnosis), run by University of California, Davis at 1 site in United States.
Trial summary
FXTAS is a rare genetic progressive neurodegenerative disorder, linked to a trinucleotide repeat expansion in the FMR1 gene. FXTAS is characterized by tremor and ataxia in addition to atrophy and white matter disease in the central nervous system (CNS). Read more about this study
- Who is it for?
- Ages 50 to 85 years
- Conditions the study is for
- Fragile X Associated Tremor/Ataxia Syndrome (Fxtas) (Diagnosis)
- What is being tested?
- Sulforaphane
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Jun 2021to 1 Jan 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 50 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of FXTAS
- Presence of a FMR1 premutation (55 to 200 CGG repeats)
Who cannot join
- Individuals with severe renal failure (GFR is <60 ml/min/1.73 m^2)
- Significant and current reported substance abuse
- Individuals with substance use disorder (meets 6 or more symptoms of substance use disorder criteria)
- Any subject on hospice or on home oxygen
- Individuals who are pregnant
Trial locations and contacts
1 site in 1 country.
United States1 site
UC Davis MIND Institute
Sacramento, California, 95817
About this study
Official title: Open-Label Trial of Sulforaphane in Premutation Carriers With FXTAS to Find Biomarkers
FXTAS is a rare genetic progressive neurodegenerative disorder, linked to a trinucleotide repeat expansion in the FMR1 gene. FXTAS is characterized by tremor and ataxia in addition to atrophy and white matter disease in the central nervous system (CNS). In addition to the major clinical observations of intention tremor and gait dysfunction, minor symptoms of parkinsonism, neuropathy, and cognitive decline also significantly impact individuals with FXTAS.
The dietary supplement being tested in this study is called Sulforaphane. It is found in broccoli and similar cruciferous vegetables and may cause some gas and discomfort. This is not a study looking at clinical efficacy but instead a study of molecular outcome measures. Investigators want to get more information about how Sulforaphane affects specific biomolecular markers captured in blood.
In this study, participants will be taking an increasing amount of the Sulphoraphane supplement pills (238mg/tablet), starting at 1 and increasing to 6, every morning at breakfast for 6 months. In addition, there will be a total of 3 visits (Initial, 3-month and 6-month) to the MIND Institute where participants will be evaluated. At each visit (3 total) participants will undergo a battery of medical and neurologic exams which make take 2-3 days to complete each time. Participants and/or their caregivers will also be asked to fill out questionnaires/surveys. At the initial visit and at 6 months, we will collect blood for analysis. Two MRI scans will be done, also at the initial visit and at 6 months.
- Being tested
- Sulforaphane
- Keywords
- Sulforaphane
- Medical subject headings
- Fragile X Tremor Ataxia Syndrome, Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SulforaphaneDietary supplement
Also called Avmacol
Sulforaphane will be taken one tablet in the morning with breakfast and then every 2 weeks, the dose will be increased by one tablet until a total of 6 tablets are taken in the morning. The supplement should be taken around the same time each day.
| Study group | Role | What they receive |
|---|---|---|
| Sulforaphane 1 Tablet | Experimental | Participants are taking 1 tablet per day. All participants will start with 1 tablet and continue with 1 tablet for 2 weeks before increasing dose. Receives: Dietary Supplement: Sulforaphane |
| Sulforaphane 2 Tablets | Experimental | Participants will increase dosage to 2 tablets per day after 2 weeks from the start of study participation. Participants will continue to take 2 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 1 tablet. Receives: Dietary Supplement: Sulforaphane |
| Sulforaphane 3 Tablets | Experimental | Participants will increase dosage to 3 tablets per day at 4 weeks from the start of study participation. Participants will continue to take 3 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 2 tablets. Receives: Dietary Supplement: Sulforaphane |
| Sulforaphane 4 Tablets | Experimental | Participants will increase dosage to 4 tablets per day at 6 weeks from the start of study participation. Participants will continue to take 4 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 3 tablets. Receives: Dietary Supplement: Sulforaphane |
| Sulforaphane 5 Tablets | Experimental | Participants will increase dosage to 5 tablets per day at 8 weeks from the start of study participation. Participants will continue to take 5 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 4 tablets. Receives: Dietary Supplement: Sulforaphane |
| Sulforaphane | Experimental | Participants will increase dosage to 6 tablets per day at 10 weeks from the start of study participation. Participants will continue to take 6 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 5 tablets. Receives: Dietary Supplement: Sulforaphane |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in mitochondrial function via mitochondrial membrane potential and mass.
Primary measures are performed on PBMCs. Mitochondrial mass and membrane potential will use selected floors and either plate readers or analytical flow cytometry.
Measured: Baseline, 6 months
Changes in mitochondrial function via ATP production
Using PMBCs we will observe in changes in ATP production of various segments of the electron transport chain by utilizing different substrates and coupling between electron transport and ATP production.
Measured: Baseline, 6 months
Observing changes in candidate molecular biomarkers in FXTAS and mitochondrial dysregulation: GRP78
Observing levels in potential molecular biomarkers that affect mitochondrial dysregulation. GRP78 is a master controller of the mitochondrial ER stress response. It helps improve Ca2+ transfer and mitochondrial function. Its level will be measured via ELISA
Measured: Baseline, 6 months
Observing changes in candidate molecular biomarkers in FXTAS and mitochondrial dysregulation: CHOP level
Observing levels in potential molecular biomarkers that affect mitochondrial dysregulation. CHOP is a common downstream indicator of mitochondrial ER stress signals. It helps improve Ca2+ transfer and mitochondrial function. Its level will be measured via ELISA
Measured: baseline, 6 months
Observing changes in candidate molecular biomarkers in FXTAS and mitochondrial dysregulation: Bax/Bcl-2 ratio
Observing levels in potential molecular biomarkers that affect mitochondrial dysregulation. Bax/Bcl-2 is a common measure of mitochondrial dysfunction. It helps improve Ca2+ transfer and mitochondrial function. It will be measure via Elisa.
Measured: baseline, 6 months
Secondary outcomes (18)
Changes in clinical staging of FXTAS
The standardized neurological examination including frontal release signs, snout, jaw jerk, and palmomental reflex; sensory examination of pinprick and vibration sense; and assessment of alternating movements and cranial nerves. These measures constitute the FXTAS Rating Scale (FXTAS-RS).
Measured: Baseline, 6 months
Changes in subcortical brain structures damaged in FXTAS through MRI imaging
The measurements will include anatomic volume for quantifying atrophy. We have developed a robust and efficient method for accurate segmentation of the cerebellum and brainstem and have applied this method to segment the hippocampus, amygdala, basal ganglia, and corpus callosum accurately. We will continue using this method for quantifying volumes of the white matter in individual lobes and subcortical structures, including the corpus callosum, midbrain, pons, and cerebellar white matter, and calibrating iron depositions in subcortical nuclei (i.e., dentate nucleus, substantia nigra, putamen, and globus pallidus)
Measured: Baseline, 6 months
Changes in FLAIR hypersensitivity volume for subcortical lesions
The measurements will include FLAIR hyperintensity volume for subcortical lesions. Subcortical hyperintensities will be segmented automatically using Lesion Prediction Algorithm (LPA) from SPM12, which also co-registers FLAIR images to T1 images. To calculate regional FLAIR hyperintensity volume, we will threshold the generated lesion probability maps at 0.7 and superimpose subcortical and white matter masks to the probability maps using fslmaths from FSL.
Measured: Baseline, 6 months
Changes in Neurological Quality of Life using the Neuro-QoL upper extremity function fine motor scale
The upper extremity function fine motor scale measures the neurological quality of life related to one's ability to carry out various activities involving digital, manual, and reach-related functions ranging from fine motor to self-care. The assessment is administered as a self-report questionnaire and responses are recorded on a scale of 1 (unable to do) to 5 (without any difficulty). The assessment will be administered at baseline and at 6 months and any changes in scaled responses will measure changes in neurological quality of life related to the upper extremity functioning.
Measured: Baseline, 6 months
Changes in Neurological Quality of Life using the Neuro-QoL lower extremity functioning mobility scale
The lower extremity function mobility scale measures the neurological quality of life related to one's ability to carry out various activities involving the trunk region and increasing degrees of bodily movement, ambulation, balance, and endurance. The assessment is administered as a self-report questionnaire and response are recorded on a scale of 1 (unable to do/can't do) to 5 (without any difficulty/no difficulty). The assessment will be administered at baseline and 6 months and any changes in scaled responses will measure change in neurological quality of life related to the lower extremity functioning.
Measured: Baseline, 6 months
Changes in psychological Issues
The Symptom Checklist-90-Revised (SCL-90-R) is a 90 item self-report psychometric questionnaire created to capture a broad range of psychological problems and has been shown to be sensitive in capturing emotional issues in carriers both with and without FXTAS.
Measured: Baseline, 6 months
Changes in Grip Strength
For the NIH Toolbox Grip Strength Test, participants squeeze the Jamar Plus Digital dynamometer as hard as they can for a count of three. The dynamometer provides a digital reading of force in pounds.
Measured: Baseline, 6 months
Changes in gait variability
Gait variability will be measured using the GAITRite in which participants will traverse the GAITRite walkway across 6 walking trials for each condition with minimal interruption.
Measured: Baseline, 6 months
Changes in dexterity, tremor, and bradykinesia abnormalities
The Purdue Pegboard test will measure abnormalities in dexterity, tremor, and bradykinesia.
Measured: Baseline, 6 months
Changes in kinetic, postural, and rest tremor upper extremity bradykinesia, hypokinesia, dysrhythmia, and dyskinesias
Participants will perform pre-defined tasks for the Kinesia One test (e.g., finger to nose, finger tapping, wrist rotation, holding arms extended, arms resting on the legs, and hand grasps).
Measured: Baseline, 6 months
Changes in cognitive dysfunction using MoCA
The Montreal Cognitive Assessment (MoCA) will be administered at baseline and 6 months and the MoCA is scored on a scale of 0-30 points. Changes in the participant's score from the MoCA administered at baseline and the MoCA administered at 6 months will measure whether there are changes in cognitive dysfunction.
Measured: Baseline, 6 months
Changes in cognitive dysfunction using MMSE
The Mini Mental State Exam (MMSE) will be administered at baseline and 6 months. MMSE is scored on a scale of 0-30 points. Changes in participant's score from MMSE administered at baseline and MMSE administered at 6 months will measure whether there are changes in mild cognitive dysfunction.
Measured: Baseline, 6 months
Changes in executive function deficits using BDS-2
The Behavioral Dysfunction Scale 2 (BDS-2) is a 9-item assessment of simple motor tasks that scores the participant's completion of each item/task on a scale of 0 (patient demonstrates a complete lack of insight into the accuracy of his or her performance and fails to recognize that errors are being made) to 3 (patient demonstrates good performance with no errors). The BDS-2 is administered at baseline and 6 months and changes in participant scores will measure changes in executive function deficits.
Measured: Baseline, 6 months
Changes in executive function deficits using COWAT
The Controlled Oral Word Association Test (COWAT) measures executive functioning deficits using verbal fluency and word retrieval tasks. One's score is calculated by the total number of words listed (1 word = 1 point) Changes in the participant's score from the COWAT administered at baseline and the COWAT administered at 6 months will measure changes in executive function deficits.
Measured: Baseline, 6 months
Changes in frontal executive planning
The Stocking of Cambridge subtest from the CANTAB assessment will be administered to measure deficits in frontal executive planning.
Measured: Baseline, 6 months
Changes in Hippocampus-mediated memory deficits
The Spatial Working Memory and Paired Associates Learning subtest from the CANTAB assessment will be used to measure hippocampus-mediated memory deficits
Measured: Baseline, 6 months
Changes in motor movements
The Choice Reaction Time subtest from the CANTAB assessment will be used to measure motor changes and bradykinesia.
Measured: Baseline, 6 months
Changes in cognitive ability
Cognitive ability will be measured using the Wechsler Adult Intelligence Scale, Fourth Edition (WAIS-IV).
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2022
- Study start25 Jun 2021
- Primary completion1 Jan 2023
- Study completion1 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Fragile X Associated Tremor/Ataxia Syndrome (Fxtas) (Diagnosis)Studies of the same condition, open and under way.
- More trials run by University of California, DavisThe sponsor's other registered studies.
- 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
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Davis
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05233579
- Sponsor's protocol ID
- 1743661
- Status last verified
- Jul 2023
- First submitted
- 20 Oct 2021
- Last updated
- 12 Jul 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Napoli E, Flores A, Mansuri Y, Hagerman RJ, Giulivi C. Sulforaphane improves mitochondrial metabolism in fibroblasts from patients with fragile X-associated tremor and ataxia syndrome. Neurobiol Dis. 2021 Sep;157:105427. doi: 10.1016/j.nbd.2021.105427. Epub 2021 Jun 19.
PubMed 34153466Uddin MS, Mamun AA, Jakaria M, Thangapandiyan S, Ahmad J, Rahman MA, Mathew B, Abdel-Daim MM, Aleya L. Emerging promise of sulforaphane-mediated Nrf2 signaling cascade against neurological disorders. Sci Total Environ. 2020 Mar 10;707:135624. doi: 10.1016/j.scitotenv.2019.135624. Epub 2019 Nov 21.
PubMed 31784171Berry-Kravis EM, Harnett MD, Reines SA, Reese MA, Ethridge LE, Outterson AH, Michalak C, Furman J, Gurney ME. Inhibition of phosphodiesterase-4D in adults with fragile X syndrome: a randomized, placebo-controlled, phase 2 clinical trial. Nat Med. 2021 May;27(5):862-870. doi: 10.1038/s41591-021-01321-w. Epub 2021 Apr 29.
PubMed 33927413
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Trial of Sulforaphane in Premutation Carriers With FXTAS to Find Biomarkers
- ClinicalTrials.gov ID
- NCT05233579
- Acronym / sponsor ID
- 1743661
- Keywords
- Sulforaphane
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- University of California, DavisUniversity, hospital or other
- Study start
- 25 Jun 2021 (actual)
- Primary completion
- 1 Jan 2023 (actual)
- Study completion
- 1 Jan 2023 (actual)
- First posted
- 10 Feb 2022 (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.
FXTAS is a rare genetic progressive neurodegenerative disorder, linked to a trinucleotide repeat expansion in the FMR1 gene. FXTAS is characterized by tremor and ataxia in addition to atrophy and white matter disease in the central nervous system (CNS). In addition to the major clinical observations of intention tremor and gait dysfunction, minor symptoms of parkinsonism, neuropathy, and cognitive decline also significantly impact individuals with FXTAS.
The dietary supplement being tested in this study is called Sulforaphane. It is found in broccoli and similar cruciferous vegetables and may cause some gas and discomfort. This is not a study looking at clinical efficacy but instead a study of molecular outcome measures. Investigators want to get more information about how Sulforaphane affects specific biomolecular markers captured in blood.
In this study, participants will be taking an increasing amount of the Sulphoraphane supplement pills (238mg/tablet), starting at 1 and increasing to 6, every morning at breakfast for 6 months. In addition, there will be a total of 3 visits (Initial, 3-month and 6-month) to the MIND Institute where participants will be evaluated. At each visit (3 total) participants will undergo a battery of medical and neurologic exams which make take 2-3 days to complete each time. Participants and/or their caregivers will also be asked to fill out questionnaires/surveys. At the initial visit and at 6 months, we will collect blood for analysis. Two MRI scans will be done, also at the initial visit and at 6 months.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Sulforaphane 1 Tablet | Experimental | Dietary Supplement: SulforaphaneParticipants are taking 1 tablet per day. All participants will start with 1 tablet and continue with 1 tablet for 2 weeks before increasing dose. |
| Sulforaphane 2 Tablets | Experimental | Dietary Supplement: SulforaphaneParticipants will increase dosage to 2 tablets per day after 2 weeks from the start of study participation. Participants will continue to take 2 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 1 tablet. |
| Sulforaphane 3 Tablets | Experimental | Dietary Supplement: SulforaphaneParticipants will increase dosage to 3 tablets per day at 4 weeks from the start of study participation. Participants will continue to take 3 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 2 tablets. |
| Sulforaphane 4 Tablets | Experimental | Dietary Supplement: SulforaphaneParticipants will increase dosage to 4 tablets per day at 6 weeks from the start of study participation. Participants will continue to take 4 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 3 tablets. |
| Sulforaphane 5 Tablets | Experimental | Dietary Supplement: SulforaphaneParticipants will increase dosage to 5 tablets per day at 8 weeks from the start of study participation. Participants will continue to take 5 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 4 tablets. |
| Sulforaphane | Experimental | Dietary Supplement: SulforaphaneParticipants will increase dosage to 6 tablets per day at 10 weeks from the start of study participation. Participants will continue to take 6 tablets for 2 addiitonal weeks before increasing dose. If participants experience adverse side effects, participants will decrease dosage to 5 tablets. |
Who may qualify
- Ages
- 50 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of FXTAS
- Presence of a FMR1 premutation (55 to 200 CGG repeats)
Key exclusion criteria (5 listed)
- Individuals with severe renal failure (GFR is <60 ml/min/1.73 m^2)
- Significant and current reported substance abuse
- Individuals with substance use disorder (meets 6 or more symptoms of substance use disorder criteria)
- Any subject on hospice or on home oxygen
- Individuals who are pregnant
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 |
|---|---|---|
| SulforaphaneAlso: AvmacolSulforaphane will be taken one tablet in the morning with breakfast and then every 2 weeks, the dose will be increased by one tablet until a total of 6 tablets are taken in the morning. The supplement should be taken around the same time each day. | Dietary supplement | Sulforaphane, Sulforaphane 1 Tablet, Sulforaphane 2 Tablets, Sulforaphane 3 Tablets, Sulforaphane 4 Tablets, Sulforaphane 5 Tablets |
Primary outcome
Changes in mitochondrial function via mitochondrial membrane potential and mass.
Time frame: Baseline, 6 months
Primary measures are performed on PBMCs. Mitochondrial mass and membrane potential will use selected floors and either plate readers or analytical flow cytometry.
Changes in mitochondrial function via ATP production
Time frame: Baseline, 6 months
Using PMBCs we will observe in changes in ATP production of various segments of the electron transport chain by utilizing different substrates and coupling between electron transport and ATP production.
Observing changes in candidate molecular biomarkers in FXTAS and mitochondrial dysregulation: GRP78
Time frame: Baseline, 6 months
Observing levels in potential molecular biomarkers that affect mitochondrial dysregulation. GRP78 is a master controller of the mitochondrial ER stress response. It helps improve Ca2+ transfer and mitochondrial function. Its level will be measured via ELISA
Observing changes in candidate molecular biomarkers in FXTAS and mitochondrial dysregulation: CHOP level
Time frame: baseline, 6 months
Observing levels in potential molecular biomarkers that affect mitochondrial dysregulation. CHOP is a common downstream indicator of mitochondrial ER stress signals. It helps improve Ca2+ transfer and mitochondrial function. Its level will be measured via ELISA
Observing changes in candidate molecular biomarkers in FXTAS and mitochondrial dysregulation: Bax/Bcl-2 ratio
Time frame: baseline, 6 months
Observing levels in potential molecular biomarkers that affect mitochondrial dysregulation. Bax/Bcl-2 is a common measure of mitochondrial dysfunction. It helps improve Ca2+ transfer and mitochondrial function. It will be measure via Elisa.
18 secondary outcomes
Changes in clinical staging of FXTAS
Time frame: Baseline, 6 months
Changes in subcortical brain structures damaged in FXTAS through MRI imaging
Time frame: Baseline, 6 months
Changes in FLAIR hypersensitivity volume for subcortical lesions
Time frame: Baseline, 6 months
Changes in Neurological Quality of Life using the Neuro-QoL upper extremity function fine motor scale
Time frame: Baseline, 6 months
Changes in Neurological Quality of Life using the Neuro-QoL lower extremity functioning mobility scale
Time frame: Baseline, 6 months
Changes in psychological Issues
Time frame: Baseline, 6 months
Changes in Grip Strength
Time frame: Baseline, 6 months
Changes in gait variability
Time frame: Baseline, 6 months
Changes in dexterity, tremor, and bradykinesia abnormalities
Time frame: Baseline, 6 months
Changes in kinetic, postural, and rest tremor upper extremity bradykinesia, hypokinesia, dysrhythmia, and dyskinesias
Time frame: Baseline, 6 months
Changes in cognitive dysfunction using MoCA
Time frame: Baseline, 6 months
Changes in cognitive dysfunction using MMSE
Time frame: Baseline, 6 months
Changes in executive function deficits using BDS-2
Time frame: Baseline, 6 months
Changes in executive function deficits using COWAT
Time frame: Baseline, 6 months
Changes in frontal executive planning
Time frame: Baseline, 6 months
Changes in Hippocampus-mediated memory deficits
Time frame: Baseline, 6 months
Changes in motor movements
Time frame: Baseline, 6 months
Changes in cognitive ability
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2023.
- Background: PubMed 34153466Napoli E, Flores A, Mansuri Y, Hagerman RJ, Giulivi C. Sulforaphane improves mitochondrial metabolism in fibroblasts from patients with fragile X-associated tremor and ataxia syndrome. Neurobiol Dis. 2021 Sep;157:105427. doi: 10.1016/j.nbd.2021.105427. Epub 2021 Jun 19.
- Background: PubMed 31784171Uddin MS, Mamun AA, Jakaria M, Thangapandiyan S, Ahmad J, Rahman MA, Mathew B, Abdel-Daim MM, Aleya L. Emerging promise of sulforaphane-mediated Nrf2 signaling cascade against neurological disorders. Sci Total Environ. 2020 Mar 10;707:135624. doi: 10.1016/j.scitotenv.2019.135624. Epub 2019 Nov 21.
- Background: PubMed 33927413Berry-Kravis EM, Harnett MD, Reines SA, Reese MA, Ethridge LE, Outterson AH, Michalak C, Furman J, Gurney ME. Inhibition of phosphodiesterase-4D in adults with fragile X syndrome: a randomized, placebo-controlled, phase 2 clinical trial. Nat Med. 2021 May;27(5):862-870. doi: 10.1038/s41591-021-01321-w. Epub 2021 Apr 29.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Sulforaphane in people with Fragile X Associated Tremor/Ataxia Syndrome (Fxtas) (Diagnosis), run by University of California, Davis at 1 site in United States.
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 2 inclusion and 5 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: finished, as of the sponsor's last update on 12 Jul 2023. 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 United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Changes in mitochondrial function via mitochondrial membrane potential and mass. (time frame: Baseline, 6 months). The study lists 5 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 NCT05233579, which also lists 3 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 12 Jul 2023.