Prader-Willi SyndromePhase 3NCT03440814

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

A Phase 3 study testing DCCR in people with Prader-Willi Syndrome, run by Soleno Therapeutics, Inc. at 29 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

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. Read more about this study

Who is it for?: Ages 4 years and older
Conditions the study is for: Prader-Willi Syndrome
What is being tested?: DCCR
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 127 participants
Where: 29 sitesin 2 countries
Study dates: 9 May 2018to 1 May 2020
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 4 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

29 sites in 2 countries.

Country

City, postcode or hospital

29 sites in 2 countries

United States20 sites

Emory Children's Center
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
National Institutes of Health Hatfield Clinical Research Center
Bethesda, Maryland, 20892
Boston Children's Hospital
Boston, Massachusetts, 02115
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, 43205
Research Institute of Dallas
Dallas, Texas, 75231
University of Florida Gainesville
Gainesville, Florida, 32608
Indiana University School of Medicine
Indianapolis, Indiana, 46202
Kansas University Medical Center
Kansas City, Kansas, 66160
Sparrow Clinical Research Institute
Lansing, Michigan, 48912
NYU Winthrop Hospital
Mineola, New York, 11501
Vanderbilt University
Nashville, Tennessee, 37232
University of California, Irvine
Orange, California, 92868
Stanford University
Palo Alto, California, 94305
St. Joseph's University Medical Center
Paterson, New Jersey, 07503
Children's Minnesota
Saint Paul, Minnesota, 55102
University of Utah
Salt Lake City, Utah, 84113
Rady Children's Hospital San Diego
San Diego, California, 92123
Seattle Children's Hospital
Seattle, Washington, 98105

Other trials in United States

United Kingdom9 sites

Birmingham Women's and Children's Hospital
Birmingham, B4 6NH
Fulbourn Hospital
Cambridge, CB21 5ER
The Queen Elizabeth University
Glasgow, Scottland, G51 4TF
Hull and East Yorkshire Hospitals NHS Trust
Hull, Yorkshire, HU3 2JZ
Alder Hey Children's Hospital NHS Foundation Trust
Liverpool, L12 2AP
Aintree University Hospital NHS Foundation Trust
Liverpool, L9 7AL
Royal London Hospital
London, E1 1BB
Chelsea and Westminster Hospital
London, SW10 9NH
Hammersmith Hospital
London, W12 OHS

Other trials in United Kingdom

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) 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.

Conditions: Prader-Willi Syndrome
Being tested: DCCR, Placebo for DCCR
Keywords: PWS, Prader-Willi Syndrome
Medical subject headings: Prader-Willi Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

DCCRDrug
Once daily oral administration
Placebo for DCCRDrug
Once daily oral administration

Study groups in NCT03440814
Study group	Role	What they receive
DCCR	Experimental	75 - 450 mg DCCR Receives: Drug: DCCR
Placebo	Placebo comparator	75 - 450 mg placebo for DCCR Receives: Drug: Placebo for DCCR

What is being measured

The primary outcome is the main question the study is designed to answer.

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

Secondary outcomes (3)

Clinical Global Impression of Improvement (CGI-I) 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.
Measured: at Visit 7 (Week 13)
Caregiver Global Impression of Change (GI-C) 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.
Measured: at Visit 7 (Week 13)
Change in Fat Mass (kg) From Baseline at 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).
Measured: Baseline to Visit 7 (Week 13)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

9 May 20181 May 2020

First posted22 Feb 2018
Study start9 May 2018
Primary completion1 May 2020
Study completion1 May 2020

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 127 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03440814
Sponsor's protocol ID: C601
Status last verified: Jun 2023
First submitted: 13 Feb 2018
Last updated: 21 Sep 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Strong TV, Miller JL, McCandless SE, Gevers E, Yanovski JA, Matesevac L, Bohonowych J, Ballal S, Yen K, Hirano P, Cowen NM, Bhatnagar A. Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. J Neurodev Disord. 2024 Apr 26;16(1):22. doi: 10.1186/s11689-024-09536-x.
PubMed 38671361
Miller JL, Gevers E, Bridges N, Yanovski JA, Salehi P, Obrynba KS, Felner EI, Bird LM, Shoemaker AH, Angulo M, Butler MG, Stevenson D, Goldstone AP, Wilding J, Lah M, Shaikh MG, Littlejohn E, Abuzzahab MJ, Fleischman A, Hirano P, Yen K, Cowen NM, Bhatnagar A; C601/C602 Investigators. Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study. Obesity (Silver Spring). 2024 Feb;32(2):252-261. doi: 10.1002/oby.23928. Epub 2023 Nov 2.
PubMed 37919617
Miller JL, Gevers E, Bridges N, Yanovski JA, Salehi P, Obrynba KS, Felner EI, Bird LM, Shoemaker AH, Angulo M, Butler MG, Stevenson D, Abuzzahab J, Barrett T, Lah M, Littlejohn E, Mathew V, Cowen NM, Bhatnagar A; DESTINY PWS Investigators. Diazoxide Choline Extended-Release Tablet in People With Prader-Willi Syndrome: A Double-Blind, Placebo-Controlled Trial. J Clin Endocrinol Metab. 2023 Jun 16;108(7):1676-1685. doi: 10.1210/clinem/dgad014.
PubMed 36639249

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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A Study of CSTI-500 in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Recruiting
Cerebellar TMS and Satiety in Prader-Willi Syndrome
Prader-Willi Syndrome
Enrolling by invitationObservational study
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Active, not recruitingPhase 1/2
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
Hypothalamic Obesity, Prader-Willi Syndrome
Active, not recruiting
Impact of Bright Light Therapy on Prader-Willi Syndrome
Prader-Willi Syndrome, Excessive Daytime Sleepiness

All Prader-Willi Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 21 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
ClinicalTrials.gov ID: NCT03440814
Acronym / sponsor ID: C601
Conditions: Prader-Willi Syndrome
Keywords: PWS, Prader-Willi Syndrome
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 127 participants (actual)
Lead sponsor: Soleno Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: 9 May 2018 (actual)
Primary completion: 1 May 2020 (actual)
Study completion: 1 May 2020 (actual)
First posted: 22 Feb 2018 (actual)
Results: Posted 21 Sep 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 2

Arm	Type	Receives
DCCR	Experimental	Drug: DCCR75 - 450 mg DCCR
Placebo	Placebo comparator	Drug: Placebo for DCCR75 - 450 mg placebo for DCCR

Who may qualify

Ages: 4 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (4 listed)

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

Key exclusion criteria (3 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
DCCROnce daily oral administration	Drug	DCCR
Placebo for DCCROnce daily oral administration	Drug	Placebo

Primary outcome

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.

3 secondary outcomes

Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13)
Time frame: at Visit 7 (Week 13)
Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13)
Time frame: at Visit 7 (Week 13)
Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)
Time frame: Baseline to Visit 7 (Week 13)

Results and safety

Results posted to the registry on 21 Sep 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
DCCR	84	79
Placebo for DCCR	42	41

Primary outcome: Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)

Time frame: Baseline to Visit 7 (Week 13). Least squares mean (Standard Error). Unit: score on a scale

Measure	DCCR n = 82	Placebo for DCCR n = 42
Value	-5.94 (0.879)	-4.27 (1.145)

DCCR vs Placebo for DCCR: Mean Difference (Final Values): -1.67 (95% CI -4.24 to 0.89), p = 0.1983, Mixed Models Analysis

Population analysed: Intent to treat (ITT) population includes all randomized subjects who had a Baseline HQ-CT value and at least one post-Baseline HQ-CT value.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Baseline to Visit 7 (Week 13). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
DCCR	6 of 84 (7.1%)	69 of 84 (82.1%)	0 of 84
Placebo for DCCR	0 of 42 (0%)	31 of 42 (73.8%)	0 of 42

Limitations and caveats (from the sponsor): All subjects were enrolled and had baseline visits prior to the COVID-19 pandemic; however, there was a subset of subjects who needed to complete some of their visits after the onset of the pandemic. The pandemic forced numerous restrictions on normal activities and involved significant disruptions, which had a substantially adverse but inconsistent effect on the study conduct and results. For this reason, the ITT population - Pre-COVID analysis includes all data through March 1, 2020.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 38671361Strong TV, Miller JL, McCandless SE, Gevers E, Yanovski JA, Matesevac L, Bohonowych J, Ballal S, Yen K, Hirano P, Cowen NM, Bhatnagar A. Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. J Neurodev Disord. 2024 Apr 26;16(1):22. doi: 10.1186/s11689-024-09536-x.
Derived from this study: PubMed 37919617Miller JL, Gevers E, Bridges N, Yanovski JA, Salehi P, Obrynba KS, Felner EI, Bird LM, Shoemaker AH, Angulo M, Butler MG, Stevenson D, Goldstone AP, Wilding J, Lah M, Shaikh MG, Littlejohn E, Abuzzahab MJ, Fleischman A, Hirano P, Yen K, Cowen NM, Bhatnagar A; C601/C602 Investigators. Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study. Obesity (Silver Spring). 2024 Feb;32(2):252-261. doi: 10.1002/oby.23928. Epub 2023 Nov 2.
Derived from this study: PubMed 36639249Miller JL, Gevers E, Bridges N, Yanovski JA, Salehi P, Obrynba KS, Felner EI, Bird LM, Shoemaker AH, Angulo M, Butler MG, Stevenson D, Abuzzahab J, Barrett T, Lah M, Littlejohn E, Mathew V, Cowen NM, Bhatnagar A; DESTINY PWS Investigators. Diazoxide Choline Extended-Release Tablet in People With Prader-Willi Syndrome: A Double-Blind, Placebo-Controlled Trial. J Clin Endocrinol Metab. 2023 Jun 16;108(7):1676-1685. doi: 10.1210/clinem/dgad014.

Related trials for Prader-Willi Syndrome

RecruitingPhase 2
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Recruiting
Cerebellar TMS and Satiety in Prader-Willi Syndrome
Prader-Willi Syndrome
Enrolling by invitationObservational study
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Active, not recruitingPhase 1/2
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
Hypothalamic Obesity, Prader-Willi Syndrome
Active, not recruiting
Impact of Bright Light Therapy on Prader-Willi Syndrome
Prader-Willi Syndrome, Excessive Daytime Sleepiness

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Evelien Gevers, MD, PhD principal investigator

Queen Mary University of London, Barts Health NHS Trust

Jennifer L. Miller, MD principal investigator

University of Florida

29 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing DCCR in people with Prader-Willi Syndrome, run by Soleno Therapeutics, Inc. at 29 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 21 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 29 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 21 Sep 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) (time frame: Baseline to Visit 7 (Week 13)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03440814, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Sep 2023.