Melatonin for Huntington's Disease (HD) Gene Carriers With HD Related Sleep Disturbance - a Pilot Study

The purpose of the study is to evaluate the efficacy of exogenous melatonin in improving sleep quality in HD gene carriers.

Study Overview

• Status: Completed
• Conditions: Huntington Disease
• Intervention / Treatment: Dietary supplement: Melatonin; Other: Placebo

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Texas
    • Houston, Texas, United States, 77030
      • The University of Texas Health Science Center at Houston

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Verified HD mutation carriers.
• Patients who report sleep disturbance as measured by a score ≥5 on the Pittsburgh Sleep Quality Index (PSQI);
• Stable concomitant medication (no change of medication during last 30 days prior to inclusion);
• Written informed consent by prospective study participant before conduct of any trial-related procedure;
• Participant must be able to make an informed decision of whether or not to participate in the study.

Exclusion Criteria:

• Pregnant or nursing women;
• Woman of childbearing potential, not using highly effective methods of contraception such as oral, topical or injected contraception, intrauterine device (IUD), contraceptive vaginal ring, or double barrier method such as diaphragm and condom with spermicide) or not surgically sterile (via hysterectomy, ovariectomy or bilateral tubal ligation) or not at least one year post menopausal;
• Presence of any medically not controllable disease (e.g. uncontrolled arterial hypertension or diabetes mellitus);
• Use of benzodiazepines, sedating antidepressants (mirtazapine or trazodone) or sedating antipsychotics (olanzapine and quetiapine) in the previous four weeks;
• Severe cognitive disorders defined as a score < 18 on the MOCA;
• Participation in another investigative drug trial within 2 months;
• Subjects who are unlikely to be compliant and attend scheduled clinic visits as required as determined by the Investigator.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Other: Placebo; Participants will receive placebo once a day (30 min prior to bedtime) for four weeks, followed by one-week washout before crossing-over.
Experimental: Melatonin	Dietary supplement: Melatonin; Participants will receive melatonin 5 mg once a day (30 min prior to bedtime) for four weeks, followed by one-week washout before crossing-over.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in sleep quality, as assessed by the Pittsburgh Sleep Quality Index (PSQI)	The PSQI is a 9-question instrument used to measure the quality and patterns of sleep in adults. Scoring of the answers is based on a 0 to 3 scale, whereby 3 reflects the negative extreme on the Likert Scale. A global sum of "5"or greater indicates a "poor" sleeper.	Baseline, week 5, week 9

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Improvement in sleep quality, as assessed by the Huntington's disease (HD) sleep questionnaire	This questionnaire contained 45 questions that focused on different sleep-related issues such as duration, quality of sleep, abnormal nocturnal behavior and quality of life. The questionnaire has a total score of 19 points, scores of (0 - 3) represent the 'normal' range whilst, scores of (4 - 6) reflect 'mild' and scores of 7 and above indicate a 'significant' sleep disturbance.	Baseline, week 5, week 9
Improvement in daytime somnolence, as evaluated by the Epworth Sleepiness Scale (ESS)	The ESS is a questionnaire designed to measure the subject's general level of daytime sleepiness. This scale scores from 0-24, with higher scores showing severe excessive daytime sleepiness.	Baseline, week 5, week 9
Improvement in self-perceived cognitive function, as assessed by the Quality of Life in Neurological Disorders questionnaire (NeuroQOLv2.0) Cognition Function - Short Form	It is an 8-question instrument designed and validated to evaluate perceived difficulties in cognitive abilities (e.g., memory, attention, and decision making, or in the application of such abilities to everyday tasks (e.g., planning, organizing, calculating, remembering and learning). The score ranges from 0-100, with higher scores meaning a better or worse outcome according to the domain evaluated.	Baseline, week 5, week 9
Improvement in global cognitive functioning, as assessed by the Montreal cognitive assessment (MoCA)	The MoCA was designed as a rapid screening instrument for mild cognitive dysfunction. Scores on the MoCA range from zero to 30, with a score of 26 and higher generally considered normal.	Baseline, week 5, week 9
Improvement in quality of life, as evaluated by the Huntington's Disease Quality of Life Questionnaire (HDQoL)	It is a disease-specific scale containing 40 questions, with answers including different types of frequency from "never" to "all of the time".	Baseline, week 5, week 9

Collaborators and Investigators

• Sponsor: The University of Texas Health Science Center, Houston
• Collaborators: Huntington's Disease Society of America
• Investigators: Principal Investigator: Erin Furr Stimming, MD, The University of Texas Health Science Center, Houston

Study record dates

Study Major Dates

• Study Start (Actual): January 22, 2021
• Primary Completion (Actual): September 16, 2022
• Study Completion (Actual): September 16, 2022

Study Registration Dates

• First Submitted: June 4, 2020
• First Submitted That Met QC Criteria: June 5, 2020
• First Posted (Actual): June 9, 2020

Study Record Updates

• Last Update Posted (Actual): May 15, 2023
• Last Update Submitted That Met QC Criteria: May 12, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Melatonin; Huntington's Disease
• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Sleep Wake Disorders; Neurocognitive Disorders; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Dyskinesias; Heredodegenerative Disorders, Nervous System; Dementia; Cognition Disorders; Chorea; Dyssomnias; Parasomnias; Huntington Disease; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Protective Agents; Antioxidants; Melatonin
• Other Study ID Numbers: HSC-MS-19-1111

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No