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Liq-NOL Efficacy in Pediatric Patients With Down Syndrome

Primary Purpose

Down Syndrome

Status

Withdrawn

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Ubiquinol-10 Syrup

syrup (placebo)

About this trial

This is an interventional treatment trial for Down Syndrome focused on measuring Down syndrome, coenzyme Q10, language function, behavior

Eligibility Criteria

6 Years - 16 Years (Child)All SexesDoes not accept healthy volunteers

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".

Sites / Locations

Cincinnati Children's Hospital Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Active Comparator

Arm Label

Syrup

Ubiquinol-10 Syrup

Arm Description

identical placebo formulation to be administered twice a day.

CoQ (LiQ-NOL®) 10.0 mg/kg/d to be administered twice a day

Outcomes

Primary Outcome Measures

To measure the effects of LiQ-NOL on language skills, expressive language ability, and speech articulation.

Secondary Outcome Measures

To evaluate the effects of LiQ-NOL on child behavior.

Full Information

NCT ID

NCT00891917

First Posted

April 29, 2009

Last Updated

September 4, 2020

Sponsor

Children's Hospital Medical Center, Cincinnati

1. Study Identification

Unique Protocol Identification Number

NCT00891917

Brief Title

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

2. Study Status

Record Verification Date

January 2014

Overall Recruitment Status

Withdrawn

Why Stopped

no funding

Study Start Date

January 2005 (undefined)

Primary Completion Date

November 2012 (Actual)

Study Completion Date

December 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Children's Hospital Medical Center, Cincinnati

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Down Syndrome

Keywords

Down syndrome, coenzyme Q10, language function, behavior

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderOutcomes Assessor

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Syrup

Arm Type

Placebo Comparator

Arm Description

identical placebo formulation to be administered twice a day.

Arm Title

Ubiquinol-10 Syrup

Arm Type

Active Comparator

Arm Description

CoQ (LiQ-NOL®) 10.0 mg/kg/d to be administered twice a day

Intervention Type

Dietary Supplement

Intervention Name(s)

Ubiquinol-10 Syrup

Other Intervention Name(s)

LiQ-NOL

Intervention Description

10 mg/kg/day in 2 divided doses for 3 months or Placebo Syrup for 3 months

Intervention Type

Dietary Supplement

Intervention Name(s)

syrup (placebo)

Intervention Description

syrup looks exactly like the Ubiquinol-10 Syrup but has no active ingredients or supplementation

Primary Outcome Measure Information:

Title

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

Secondary Outcome Measure Information:

Title

To evaluate the effects of LiQ-NOL on child behavior.

Time Frame

beginning and end of 3 month treatment period

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

16 Years

Accepts Healthy Volunteers

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".

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Michael V Miles, Pharm.D.

Organizational Affiliation

Childrens Hospital Medical Center, Cincinnati

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Francis Hickey, M.D.

Organizational Affiliation

Childrens Hospital Medical Center, Cincinnati

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cincinnati Children's Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

18021919

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.

Results Reference

background

PubMed Identifier

16814082

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.

Results Reference

background

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Liq-NOL Efficacy in Pediatric Patients With Down Syndrome

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