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

• ClinicalTrials.gov Identifier: NCT04421339
• Recruitment Status: Completed
• First Posted: June 9, 2020
• Last Update Posted: May 15, 2023
• Sponsor: The University of Texas Health Science Center, Houston
• Collaborator: Huntington's Disease Society of America
• Information provided by (Responsible Party): Erin Furr Stimming, The University of Texas Health Science Center, Houston

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Huntington Disease	Dietary Supplement: Melatonin; Other: Placebo	Not Applicable

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 15 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Double (Participant, Care Provider)
• Primary Purpose: Treatment
• Official Title: Melatonin for Huntington's Disease (HD) Gene Carriers With HD Related Sleep Disturbance - a Pilot Study
• Actual Study Start Date: January 22, 2021
• Actual Primary Completion Date: September 16, 2022
• Actual Study Completion Date: September 16, 2022

Arms and Interventions

Arm	Intervention/treatment
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.
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.

Outcome Measures

Primary Outcome Measures :

1. Change in sleep quality, as assessed by the Pittsburgh Sleep Quality Index (PSQI) [ Time Frame: Baseline, week 5, week 9 ]

   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.

Secondary Outcome Measures :

1. Improvement in sleep quality, as assessed by the Huntington's disease (HD) sleep questionnaire [ Time Frame: Baseline, week 5, week 9 ]

   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.

2. Improvement in daytime somnolence, as evaluated by the Epworth Sleepiness Scale (ESS) [ Time Frame: Baseline, week 5, week 9 ]

   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.

3. Improvement in self-perceived cognitive function, as assessed by the Quality of Life in Neurological Disorders questionnaire (NeuroQOLv2.0) Cognition Function - Short Form [ Time Frame: Baseline, week 5, week 9 ]

   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.

4. Improvement in global cognitive functioning, as assessed by the Montreal cognitive assessment (MoCA) [ Time Frame: Baseline, week 5, week 9 ]

   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.

5. Improvement in quality of life, as evaluated by the Huntington's Disease Quality of Life Questionnaire (HDQoL) [ Time Frame: Baseline, week 5, week 9 ]
   It is a disease-specific scale containing 40 questions, with answers including different types of frequency from "never" to "all of the time".

Eligibility Criteria

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

Criteria

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.

Contacts and Locations

Locations

United States, Texas

The University of Texas Health Science Center at Houston

Houston, Texas, United States, 77030

Sponsors and Collaborators

The University of Texas Health Science Center, Houston

Huntington's Disease Society of America

Investigators

• Principal Investigator: Erin Furr Stimming, MD; The University of Texas Health Science Center, Houston

More Information

• Responsible Party: Erin Furr Stimming, Associate Professor, The University of Texas Health Science Center, Houston
• ClinicalTrials.gov Identifier: NCT04421339
• Other Study ID Numbers: HSC-MS-19-1111
• First Posted: June 9, 2020
• Last Update Posted: May 15, 2023
• Last Verified: May 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Erin Furr Stimming, The University of Texas Health Science Center, Houston:

Huntington's Disease; Melatonin

Additional relevant MeSH terms:

Dyssomnias; Parasomnias; Huntington Disease; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Cognition Disorders; Neurocognitive Disorders; Melatonin; Antioxidants; Molecular Mechanisms of Pharmacological Action; Protective Agents; Physiological Effects of Drugs; Central Nervous System Depressants