Liq-NOL Efficacy in Pediatric Patients With Down Syndrome

Study Overview
=================
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).

Official Title
-----------------
Evaluation of Coenzyme q10 (Liq-nol®) Efficacy in Pediatric Patients With Down Syndrome

Conditions
-----------------
Down Syndrome

Intervention / Treatment
-----------------
* Dietary Supplement: Ubiquinol-10 Syrup
* Dietary Supplement: syrup (placebo)


Participation Criteria
=================
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.

Ages Eligible for Study
-----------------
Minimum Age: 6 Years
Maximum Age: 16 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Syrup<br>identical placebo formulation to be administered twice a day. | Dietary Supplement: syrup (placebo)<br>* syrup looks exactly like the Ubiquinol-10 Syrup but has no active ingredients or supplementation<br>|
| Active Comparator: Ubiquinol-10 Syrup<br>CoQ (LiQ-NOL®) 10.0 mg/kg/d to be administered twice a day | Dietary Supplement: Ubiquinol-10 Syrup<br>* 10 mg/kg/day in 2 divided doses for 3 months or Placebo Syrup for 3 months<br>* Other names: LiQ-NOL;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To measure the effects of LiQ-NOL on language skills, expressive language ability, and speech articulation. |  | beginning and end of 3 month treatment period | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the effects of LiQ-NOL on child behavior. |  | beginning and end of 3 month treatment period | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Down syndrome, coenzyme Q10, language function, behavior