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Prader-Willi SyndromePhase 3NCT03714373

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

A Phase 3 study testing DCCR in people with Prader-Willi Syndrome, run by Soleno Therapeutics, Inc. at 28 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

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. 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
115 participants
Where
28 sitesin 2 countries
Study dates
1 Oct 2018to 17 Aug 2023
Phase
Phase 3
Are there results?
None posted in the registry

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

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

Trial locations and contacts

28 sites in 2 countries.

28 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, 37212

  • 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, 84108

  • Rady Children's Hospital San Diego

    San Diego, California, 92123

  • Seattle Children's Hospital

    Seattle, Washington, 98105

Other trials in United States

United Kingdom8 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

  • 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: 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

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.

Read the 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).

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 of open-label DCCR tablet(s) during the OLE Period

  • DCCRDrug

    Once daily oral administration of double-blind (DCCR) tablet(s) during the RW Period

  • Placebo for DCCRDrug

    Once daily oral administration of double-blind (placebo for DCCR) tablet(s) during the RW Period

Study groups in NCT03714373
Study groupRoleWhat they receive
OLE DCCRExperimental75 - 525 mg DCCR
Receives: Drug: DCCR
RW DCCRExperimental75 - 525 mg DCCR
Receives: Drug: DCCR
RW PlaceboPlacebo comparator75 - 525 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.

  1. Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported

    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.

    Measured: Baseline to end of OLE (up to 4 years)

  2. Change from RW Period Baseline in HQ-CT Total Score

    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.

    Measured: RW Period Baseline to Week 16

Secondary outcomes (5)

  1. Change from Baseline in HQ-CT Total Score

    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.

    Measured: Baseline to end of OLE (up to 4 years)

  2. Change in Body Fat Mass

    Change in Body Fat Mass (kg) using DXA

    Measured: Baseline to end of OLE (up to 4 years)

  3. Clinical Global Impression of Improvement (CGI-I)

    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).

    Measured: RW Period Week 16

  4. Clinical Global Impression of Severity (CGI-S)

    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).

    Measured: RW Period Week 16

  5. Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported

    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.

    Measured: through the end of the RW Period, 16 weeks

Timeline

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

  1. First posted22 Oct 2018
  2. Study start1 Oct 2018
  3. Primary completion17 Aug 2023
  4. Study completion17 Aug 2023

Resources

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
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
115 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03714373
Sponsor's protocol ID
C602
Status last verified
Apr 2024
First submitted
27 Sep 2018
Last updated
19 Apr 2024
Results posted
No
Data monitoring committee
No
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

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 28 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?

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 19 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

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)). The study lists 2 primary outcomes in all. 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 NCT03714373, which also lists 2 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 19 Apr 2024.

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