Liq-NOL Efficacy in Pediatric Patients With Down Syndrome

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.

Study Overview

• Status: Withdrawn
• Conditions: Down Syndrome
• Intervention / Treatment: Dietary supplement: syrup (placebo); Dietary supplement: Ubiquinol-10 Syrup

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 Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 16 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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".

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Investigators: Principal Investigator: Michael V Miles, Pharm.D., Childrens Hospital Medical Center, Cincinnati; Principal Investigator: Francis Hickey, M.D., Childrens Hospital Medical Center, Cincinnati

Publications and helpful links

General 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.
• 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.

Study record dates

Study Major Dates

• Study Start: January 1, 2005
• Primary Completion (Actual): November 1, 2012
• Study Completion (Actual): December 1, 2012

Study Registration Dates

• First Submitted: April 29, 2009
• First Submitted That Met QC Criteria: April 30, 2009
• First Posted (Estimate): May 1, 2009

Study Record Updates

• Last Update Posted (Actual): September 9, 2020
• Last Update Submitted That Met QC Criteria: September 4, 2020
• Last Verified: January 1, 2014

More Information

Terms related to this study

• Keywords: behavior; Down syndrome; coenzyme Q10; language function
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Syndrome; Down Syndrome
• Other Study ID Numbers: 2008-0489

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No