Clinical trial NCT00891917: Liq-NOL Efficacy in Pediatric Patients With Down Syndrome
Official title: Evaluation of Coenzyme q10 (Liq-nol®) Efficacy in Pediatric Patients With Down Syndrome
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Down Syndrome
Keywords: Down syndrome
coenzyme Q10
language function
behavior
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2009-05-01
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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.

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).

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.
* 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".

Primary outcomes:
measure: To measure the effects of LiQ-NOL on language skills, expressive language ability, and speech articulation.
timeFrame: beginning and end of 3 month treatment period
Secondary outcomes:
measure: To evaluate the effects of LiQ-NOL on child behavior.
timeFrame: beginning and end of 3 month treatment period
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Childrens Hospital Medical Center, Cincinnati
name: Michael V Miles, Pharm.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Childrens Hospital Medical Center, Cincinnati
name: Francis Hickey, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: Miles 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.