Down SyndromePhase 2NCT00891917
Liq-NOL Efficacy in Pediatric Patients With Down Syndrome
A Phase 2 study testing Ubiquinol-10 Syrup in people with Down Syndrome, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The purpose of this study is to measure the effects of LiQ-NOL supplementation on language production using the Clinical Evaluation of Language Fundamentals test, language sampling using the mean length of utterance test, and speech articulation using the Goldman-Fristoe Test of Articulation. Read more about this study
- Who is it for?
- Ages 6 to 16 years
- Conditions the study is for
- Down Syndrome
- What is being tested?
- Ubiquinol-10 Syrup
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2005to Dec 2012
- Phase
- Phase 2
- 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 6 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients ranging from 6.0 years to 16 years of age.
- Patients will have proven Trisomy 21.
- Females, incapable of bearing children or capable of practicing adequate birth control methods. Abstinence will be acceptable.
- Written informed consent will be obtained from parents of all subjects prior to enrollment. Verbal assent will be obtained from all patients with DS who have sufficient decision making ability and are at least 11 years old.
Who cannot join
- Patients who have insufficient mental and/or motor capacity to complete testing measures.
- Patients less than 6 years or older than 16 years of age.
- Patients receiving CoQ supplementation within one month prior to the study.
- Patients with evidence of disease which may adversely affect CoQ absorption, e.g. chronic diarrhea or inflammatory bowel disease.
- Patients participating in other research studies or having exposure to investigational drugs within one month prior to this study.
- Females, capable of bearing children, who are unsure of their pregnancy status or not practicing adequate birth control methods.
Show all 9Show fewer
- Females who are pregnant.
- Patients with a known allergy to CoQ.
- Patients receiving drug treatment which is know to affect CoQ, e.g. cholesterol-lowering drugs such as "statins".
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Evaluation of Coenzyme q10 (Liq-nol®) Efficacy in Pediatric Patients With Down Syndrome
The purpose of this study is to measure the effects of LiQ-NOL supplementation on language production using the Clinical Evaluation of Language Fundamentals test, language sampling using the mean length of utterance test, and speech articulation using the Goldman-Fristoe Test of Articulation.
Read the detailed description
This is a randomized, double-blind, placebo-controlled clinical study of CoQ efficacy in a crossover design. Patients will be screened, enrolled, and studied in the outpatient clinic of the Thomas Center for Down Syndrome at CCHMC. All patients will be randomly assigned to either a liquid product (LiQ-NOL®) and an identical placebo liquid. Study drug and placebo will be administered twice a day (morning and evening).
- Conditions
- Down Syndrome
- Being tested
- Ubiquinol-10 Syrup, syrup (placebo)
- Keywords
- Down syndrome, coenzyme Q10, language function, behavior
- Medical subject headings
- Down Syndrome, Language, Behavior
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ubiquinol-10 SyrupDietary supplement
Also called LiQ-NOL
10 mg/kg/day in 2 divided doses for 3 months or Placebo Syrup for 3 months
syrup (placebo)Dietary supplement
syrup looks exactly like the Ubiquinol-10 Syrup but has no active ingredients or supplementation
| Study group | Role | What they receive |
|---|---|---|
| Syrup | Placebo comparator | identical placebo formulation to be administered twice a day. Receives: Dietary Supplement: syrup (placebo) |
| Ubiquinol-10 Syrup | Active comparator | CoQ (LiQ-NOL®) 10.0 mg/kg/d to be administered twice a day Receives: Dietary Supplement: Ubiquinol-10 Syrup |
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure the effects of LiQ-NOL on language skills, expressive language ability, and speech articulation.
Measured: beginning and end of 3 month treatment period
Secondary outcomes (1)
To evaluate the effects of LiQ-NOL on child behavior.
Measured: beginning and end of 3 month treatment period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2009 (estimated)
- Study startJan 2005
- Primary completionNov 2012
- Study completionDec 2012
Why it stopped: no funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Down SyndromeStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael V Miles, Pharm.D., Principal investigator, Childrens Hospital Medical Center, CincinnatiFrancis Hickey, M.D., Principal investigator, Childrens Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00891917
- Sponsor's protocol ID
- 2008-0489
- Status last verified
- Jan 2014
- First submitted
- 29 Apr 2009
- Last updated
- 9 Sep 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Miles MV, Patterson BJ, Chalfonte-Evans ML, Horn PS, Hickey FJ, Schapiro MB, Steele PE, Tang PH, Hotze SL. Coenzyme Q10 (ubiquinol-10) supplementation improves oxidative imbalance in children with trisomy 21. Pediatr Neurol. 2007 Dec;37(6):398-403. doi: 10.1016/j.pediatrneurol.2007.08.003.
PubMed 18021919Miles MV, Patterson BJ, Schapiro MB, Hickey FJ, Chalfonte-Evans M, Horn PS, Hotze SL. Coenzyme Q10 absorption and tolerance in children with Down syndrome: a dose-ranging trial. Pediatr Neurol. 2006 Jul;35(1):30-7. doi: 10.1016/j.pediatrneurol.2005.11.004.
PubMed 16814082
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Coenzyme q10 (Liq-nol®) Efficacy in Pediatric Patients With Down Syndrome
- ClinicalTrials.gov ID
- NCT00891917
- Acronym / sponsor ID
- 2008-0489
- Conditions
- Down Syndrome
- Keywords
- Down syndrome, coenzyme Q10, language function, behavior
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: no funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- Jan 2005
- Primary completion
- Nov 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 1 May 2009 (estimated)
- 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.
The purpose of this study is to measure the effects of LiQ-NOL supplementation on language production using the Clinical Evaluation of Language Fundamentals test, language sampling using the mean length of utterance test, and speech articulation using the Goldman-Fristoe Test of Articulation.
Detailed description
This is a randomized, double-blind, placebo-controlled clinical study of CoQ efficacy in a crossover design. Patients will be screened, enrolled, and studied in the outpatient clinic of the Thomas Center for Down Syndrome at CCHMC. All patients will be randomly assigned to either a liquid product (LiQ-NOL®) and an identical placebo liquid. Study drug and placebo will be administered twice a day (morning and evening).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Syrup | Placebo comparator | Dietary Supplement: syrup (placebo)identical placebo formulation to be administered twice a day. |
| Ubiquinol-10 Syrup | Active comparator | Dietary Supplement: Ubiquinol-10 SyrupCoQ (LiQ-NOL®) 10.0 mg/kg/d to be administered twice a day |
Who may qualify
- Ages
- 6 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Patients ranging from 6.0 years to 16 years of age.
- Patients will have proven Trisomy 21.
- Females, incapable of bearing children or capable of practicing adequate birth control methods. Abstinence will be acceptable.
- Written informed consent will be obtained from parents of all subjects prior to enrollment. Verbal assent will be obtained from all patients with DS who have sufficient decision making ability and are at least 11 years old.
Key exclusion criteria (9 listed)
- Patients who have insufficient mental and/or motor capacity to complete testing measures.
- Patients less than 6 years or older than 16 years of age.
- Patients receiving CoQ supplementation within one month prior to the study.
- Patients with evidence of disease which may adversely affect CoQ absorption, e.g. chronic diarrhea or inflammatory bowel disease.
- Patients participating in other research studies or having exposure to investigational drugs within one month prior to this study.
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 |
|---|---|---|
| Ubiquinol-10 SyrupAlso: LiQ-NOL10 mg/kg/day in 2 divided doses for 3 months or Placebo Syrup for 3 months | Dietary supplement | Ubiquinol-10 Syrup |
| syrup (placebo)syrup looks exactly like the Ubiquinol-10 Syrup but has no active ingredients or supplementation | Dietary supplement | Syrup |
Primary outcome
To measure the effects of LiQ-NOL on language skills, expressive language ability, and speech articulation.
Time frame: beginning and end of 3 month treatment period
1 secondary outcome
To evaluate the effects of LiQ-NOL on child behavior.
Time frame: beginning and end of 3 month treatment period
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2020.
- Background: PubMed 18021919Miles MV, Patterson BJ, Chalfonte-Evans ML, Horn PS, Hickey FJ, Schapiro MB, Steele PE, Tang PH, Hotze SL. Coenzyme Q10 (ubiquinol-10) supplementation improves oxidative imbalance in children with trisomy 21. Pediatr Neurol. 2007 Dec;37(6):398-403. doi: 10.1016/j.pediatrneurol.2007.08.003.
- Background: PubMed 16814082Miles MV, Patterson BJ, Schapiro MB, Hickey FJ, Chalfonte-Evans M, Horn PS, Hotze SL. Coenzyme Q10 absorption and tolerance in children with Down syndrome: a dose-ranging trial. Pediatr Neurol. 2006 Jul;35(1):30-7. doi: 10.1016/j.pediatrneurol.2005.11.004.
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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.
Michael V Miles, Pharm.D. principal investigator
Childrens Hospital Medical Center, Cincinnati
Francis Hickey, M.D. principal investigator
Childrens Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ubiquinol-10 Syrup in people with Down Syndrome, run by Children's Hospital Medical Center, Cincinnati 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 4 inclusion and 9 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: withdrawn, as of the sponsor's last update on 9 Sep 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 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
To measure the effects of LiQ-NOL on language skills, expressive language ability, and speech articulation. (time frame: beginning and end of 3 month treatment period). 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 NCT00891917, which also lists 2 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 9 Sep 2020.