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Completed

NCT Number: NCT03440814

A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight

Age range

4 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

The Queen Elizabeth University, Glasgow, Scottland, United Kingdom

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (patients, as appropriate)
  • Genetically-confirmed Prader-Willi syndrome and hyperphagic
  • In a stable care setting for at least 6 months prior to Visit 1
  • Caregiver must have been caring for the patient for at least 6 months prior to Visit 1

Exclusion criteria

  • Have participated in an interventional clinical study (i.e., investigational drug or device, approved drugs or device evaluated for unapproved use) within prior 3 months
  • Positive urine pregnancy test (in females of child-bearing potential) or females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation
  • Any other known disease and/or condition, which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol

Treatment and study plan

DCCR

Drug

Once daily oral administration

Placebo for DCCR

Drug

Once daily oral administration

Primary outcomes

  1. Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)

    Time frame: Baseline to Visit 7 (Week 13)

    Hyperphagia-related behaviors were assessed by the validated hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors that was completed by the caregiver. The HQ-CT consists of nine items with responses ranging from 0-4 units each (possible total score range: 0-36). The HQ-CT was assessed at Screening, Baseline (Visit 2), and approximately every 4 weeks post-dose at Week 4, Week 8, and Week 13. A decrease in score from baseline represented improvement.

Secondary outcomes

  1. Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13)

    Time frame: at Visit 7 (Week 13)

    The Clinical Global Impression of Improvement (CGI-I) is a single statement designed to assess the Investigator's overall perception of change in the subject's condition across the course of the clinical trial. The Investigator provided a response to "Compared to the subject's condition at enrollment, the subject's condition is:" by rating the subject's behavior using a 7-point response scale: Very much improved, Much improved, Minimally improved, No change, Minimally worse, Much worse, and Very much worse. The Investigator only took into account the subject's PWS condition.

  2. Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13)

    Time frame: at Visit 7 (Week 13)

    The Caregiver Global Impression of Change (GI-C) is a single statement designed to assess the caregiver's overall perception of change in the subject across the course of the clinical trial. The caregiver provided a response to "Please choose the response below that best describes the overall change in the person's PWS since they started taking the study medication" using a 7-point graded response scale: Very much better, Moderately better, A little better, No change, A little worse, Moderately worse, and Very much worse.

  3. Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)

    Time frame: Baseline to Visit 7 (Week 13)

    Whole body scans were performed. Reports included a breakdown of the following regions: left arm, right arm, trunk, left leg, right leg, and head. Each region was evaluated for body fat mass (g).

Sponsors and collaborators

Lead sponsor

Soleno Therapeutics, Inc.

Industry

Registry information

Official study title

A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Feb 22, 2018
Registry last updated
Sep 21, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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