DCCR for Prader-Willi Syndrome

Not currently recruiting at 22 trial locations

Overseen By C614 Project Manager

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Soleno Therapeutics, Inc.

Disqualifiers: Pregnancy, Breastfeeding, Recent investigational study

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the long-term safety of DCCR tablets for individuals with Prader-Willi syndrome, a genetic condition affecting appetite, growth, and overall development. The study includes participants from a previous related study who meet specific conditions from that study. Participants must have completed or participated in the previous study phases. This trial excludes those who are pregnant or involved in other drug studies. As a Phase 3 trial, it represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that DCCR is likely to be safe for humans?

Research has shown that DCCR, also known as diazoxide choline, is generally safe for people with Prader-Willi syndrome. Studies have found that it can help improve various symptoms of the condition. In one study, most participants did not experience major problems with the treatment. A safety board reviewed data from more than half of the participants in an ongoing study and recommended that the trial continue, indicating no major safety concerns.

While some side effects might occur, as with any treatment, evidence so far suggests that DCCR is a safe option for many patients with Prader-Willi syndrome. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Prader-Willi syndrome?

DCCR is unique because it targets Prader-Willi Syndrome in a novel way by modulating specific receptors involved in appetite and metabolism. Unlike standard treatments, which primarily focus on managing symptoms through behavioral approaches and hormone therapy, DCCR offers a direct biochemical intervention. Researchers are excited about DCCR because it has the potential to reduce hyperphagia (uncontrolled eating) and improve metabolic health, which could significantly improve the quality of life for individuals with Prader-Willi Syndrome.

What evidence suggests that DCCR might be an effective treatment for Prader-Willi syndrome?

Research has shown that DCCR (diazoxide choline), the treatment under study in this trial, may benefit people with Prader-Willi syndrome. One study found that it improved various symptoms. Participants who took DCCR had healthier levels of muscle and fat. Doctors also observed less aggressive behavior, a common issue for those with Prader-Willi syndrome. Most participants tolerated the treatment well, experiencing no serious side effects. Overall, these findings suggest DCCR could be a promising option for managing symptoms of Prader-Willi syndrome. ^{1 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with Prader-Willi Syndrome who have previously participated in a specific study (C602) and completed it, or discontinued from it at least 16 weeks prior. They must provide consent to participate.

Inclusion Criteria

Participant must have participated in and completed the Study C602 Randomized Withdrawal (RW) Period

I was in Study C602 but stopped participating at least 16 weeks ago.

Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate)

Exclusion Criteria

Positive urine pregnancy test (in females of child-bearing potential)

Participation in a clinical study of an investigational drug (including approved drugs for unapproved uses), investigational device, or therapeutic intervention subsequent to the C602 Open-Label Extension End of Treatment Visit.

I am not pregnant, breastfeeding, nor planning to become pregnant within 30 days after the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive DCCR (diazoxide choline) extended-release tablets to evaluate long-term safety

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• DCCR

Trial Overview The trial is testing the long-term safety of DCCR tablets for patients with Prader-Willi Syndrome. It's an open-label extension, meaning everyone knows they're getting the actual drug, not a placebo.

How Is the Trial Designed?

Group I: DCCR Experimental Treatment 1 Intervention

75 - 525 mg DCCR

Who Is Running the Clinical Trial?

Soleno Therapeutics, Inc.

Lead Sponsor

Trials

6

Recruited

410+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37919617/

Diazoxide choline extended-release tablet in people with ... Conclusions: DCCR administration to people with PWS was well tolerated and associated with broad-ranging improvements in the syndrome. Sustained ...

2. investors.soleno.life investors.soleno.life/static-files/20d6b0c7-5576-4a89-8f3c-e580caddc15f

Long-term Efficacy Results of Diazoxide Choline Prader-Willi syndrome – Clinical genetics, diagnosis and treatment approaches: An update. Curr Pediatr 2019; 15(4):207-244. 2. Miller Rev JL, Lynn CH, Driscoll ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03440814

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

4. academic.oup.com academic.oup.com/jcem/article/108/7/1676/6987298

Diazoxide Choline Extended-Release Tablet in People With ... DCCR treatment was associated with significant improvements in body composition and clinician-reported outcomes. Prader-Willi syndrome, hyperphagia, DCCR. Issue ...

5. journals.plos.org journals.plos.org/plosone/article?id=10.1371/journal.pone.0221615

A randomized pilot efficacy and safety trial of diazoxide ... DCCR treatment resulted in a reduction in the number of subjects displaying aggressive behaviors (-57.1%, n = 10, p = 0.01), clinically-relevant ...

6. fpwr.org fpwr.org/blog/dccr-clinical-trial-should-continue-says-data-safety-monitoring-board

DCCR Clinical Trial Should Continue, Says Data Safety ... Based on the review of data from more than 50% of patients enrolled, the Data Safety Monitoring Board has recommended continuing a phase III ...

7. pwsausa.org pwsausa.org/fda-extends-review-period-for-dccr-what-it-means-for-the-pws-community/

FDA Extends Review Period for DCCR: What It Means ... Soleno submitted its NDA for DCCR in June 2024, and the FDA granted Priority Review in August. Priority Review is reserved for drugs that could ...

8. investors.soleno.life investors.soleno.life/node/8661/pdf

September 15, 2017 Soleno Therapeutics Presents Updated Safety and Efficacy Data from Clinical Trial of DCCR for. Treatment of Prader-Willi Syndrome. September ...