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Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03714373
Recruitment Status : Active, not recruiting
First Posted : October 22, 2018
Last Update Posted : November 23, 2022
Sponsor:
Information provided by (Responsible Party):
Soleno Therapeutics, Inc.

Study Description
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Brief Summary:
This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: DCCR Drug: Placebo for DCCR Phase 3

Detailed Description:
115 PWS participants who completed clinical study C601 will be enrolled into the OLE Period. All participants in the Open Label Extension (OLE) Period will receive open-label DCCR. The actual number of participants eligible to enroll in the double-blind, placebo-controlled, randomized withdrawal (RW) period will be limited to those participants taking DCCR in the OLE Period at the time of the RW Period Visit 1 (Baseline/Randomization Visit).The treatment groups in the C602 RW Period are those participants randomized to receive DCCR and those participants randomized to receive Placebo. Participants will be randomized in a 1:1 ratio (DCCR:Placebo).
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 115 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Extended-Release Tablets in Participants With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
Actual Study Start Date : October 1, 2018
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : June 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: OLE DCCR
75 - 525 mg DCCR
 Drug: DCCR
Once daily oral administration of open-label DCCR tablet(s) during the OLE Period
Experimental: RW DCCR
75 - 525 mg DCCR
 Drug: DCCR
Once daily oral administration of double-blind (DCCR) tablet(s) during the RW Period
Placebo Comparator: RW Placebo
75 - 525 mg Placebo for DCCR
 Drug: Placebo for DCCR
Once daily oral administration of double-blind (placebo for DCCR) tablet(s) during the RW Period


Outcome Measures
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Primary Outcome Measures :
  1. Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported [ Time Frame: Baseline to end of OLE (up to 4 years) ]
    Safety analyses will be conducted in all participants who receive at least one dose of DCCR. Adverse events will be described by type and level of severity.

  2. Change from RW Period Baseline in HQ-CT Total Score [ Time Frame: RW Period Baseline to Week 16 ]
    Hyperphagia-related behaviors will be assessed by the hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors. The HQ-CT consists of nine items with responses ranging from 0-4 (best to worst). Scores from 9 items will be summed for a possible total score range of 0-36.


Secondary Outcome Measures :
  1. Change from Baseline in HQ-CT Total Score [ Time Frame: Baseline to end of OLE (up to 4 years) ]
    Hyperphagia-related behaviors will be assessed by the hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors. The HQ-CT consists of nine items with responses ranging from 0-4 (best to worst). Scores from 9 items will be summed for a possible total score range of 0-36.

  2. Change in Body Fat Mass [ Time Frame: Baseline to end of OLE (up to 4 years) ]
    Change in Body Fat Mass (kg) using DXA

  3. Clinical Global Impression of Improvement (CGI-I) [ Time Frame: RW Period Week 16 ]
    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 provides 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 (best to worst).

  4. Clinical Global Impression of Severity (CGI-S) [ Time Frame: RW Period Week 16 ]
    The CGI-S is a single statement designed to assess the Investigator's overall perception of the severity of the participant's illness across the course of the clinical trial. The Investigator provides a response to "Considering your total clinical experience with this particular population, how ill is this participant at this time:" by rating the subject's behavior using a 7-point response scale (best to worst).

  5. Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported [ Time Frame: through the end of the RW Period, 16 weeks ]
    Safety analyses will be conducted in all participants who receive at least one dose of randomized withdrawal study drug. Adverse events will be described by type and level of severity.


Eligibility Criteria
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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

OLE Period Key Inclusion Criteria:

  • Successful completion of clinical study C601
  • Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate)

OLE Period Key Exclusion Criteria:

  • 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 90 days after study participation
  • Any new disease, condition, or circumstance which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol (e.g., an anticipated change of care setting)

RW Period Key Inclusion Criteria:

  • Provide voluntary, written informed consent (parent[s] / legal guardian[s] of participant); provide voluntary, written assent (participants, as appropriate); this includes consent for randomization and potential treatment with placebo for up to 16 weeks
  • Currently participating in clinical study C602 and complete the OLE End of Treatment Visit procedures

RW Period Key Exclusion Criteria:

  • Positive urine pregnancy test (in females of child-bearing potential)
  • Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03714373


Locations
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Sponsors and Collaborators
Soleno Therapeutics, Inc.
More Information
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Responsible Party: Soleno Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03714373    
Other Study ID Numbers: C602
First Posted: October 22, 2018    Key Record Dates
Last Update Posted: November 23, 2022
Last Verified: November 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Soleno Therapeutics, Inc.:
PWS
Prader-Willi Syndrome
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
 Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overweight
Overnutrition
Nutrition Disorders