A Phase 3 interventional study of DCCR and Placebo for DCCR in Prader-Willi Syndrome, sponsored by Soleno Therapeutics, Inc.. Completed at 29 sites in 2 countries. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2023-09-21.
Sponsored by Soleno Therapeutics, Inc. (part of Neurocrine Biosciences) · Phase 3, Interventional, and Treatment
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.
144 studies on the registry are indexed under Prader-Willi Syndrome; 23 are open to participants now.
This study's enrollment of 127 is above the median of 30 across 100 interventional studies indexed under Prader-Willi Syndrome.
Browse Prader-Willi Syndrome studies →Soleno Therapeutics, Inc. is the lead sponsor of 7 studies on the registry; 1 is open to participants now.
First submitted before Soleno Therapeutics, Inc. became part of Neurocrine Biosciences.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
75 - 450 mg DCCR
Drug: DCCR
75 - 450 mg placebo for DCCR
Drug: Placebo for DCCR
Once daily oral administration
Once daily oral administration
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.
Time frame: Baseline to Visit 7 (Week 13)
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.
Time frame: 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.
Time frame: 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).
Time frame: Baseline to Visit 7 (Week 13)
People with Prader-Willi syndrome (PWS) were recruited for this study. Those who met the eligibility criteria were enrolled / randomized. Caregivers of subjects were responsible for completing the protocol-specified caregiver questionnaires and were not enrolled in this study. The first site in US started recruiting subjects on 17May2018; the first site in UK started recruiting subjects on 26Jun2019; all sites were either hospitals or academic medical centers.
| Milestone | DCCR | Placebo for DCCR |
|---|---|---|
| Started | 84 | 42 |
| Randomized (may or may not have taken study drug) | 85 | 42 |
| Intent-to-treat (itt) population | 82 | 42 |
| Completed | 79 | 41 |
| Not completed | 5 | 1 |
| Withdrew: Adverse event | 2 | 1 |
| Withdrew: Lost to follow-up | 2 | 0 |
| Withdrew: Consent withdrawn by parent/legal guardian | 1 | 0 |
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.
| score on a scale | DCCR | Placebo for DCCR |
|---|---|---|
| Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) | -5.94 ± 0.879 | -4.27 ± 1.145 |
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.
| Participants | DCCR | Placebo for DCCR |
|---|---|---|
| 1 = Very Much Improved | 0 | 0 |
| 2 = Much improved | 5 | 0 |
| 3 = Minimally improved | 25 | 2 |
| 4 = No change | 41 | 37 |
| 5 = Minimally worse | 11 | 3 |
| 6 = Much worse | 0 | 0 |
| 7 = Very much worse | 0 | 0 |
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.
| Participants | DCCR | Placebo for DCCR |
|---|---|---|
| 1 = Very Much Improved | 4 | 1 |
| 2 = Much improved | 6 | 3 |
| 3 = Minimally improved | 22 | 8 |
| 4 = No change | 39 | 20 |
| 5 = Minimally worse | 6 | 5 |
| 6 = Much worse | 4 | 3 |
| 7 = Very much worse | 1 | 2 |
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).
| kg | DCCR | Placebo for DCCR |
|---|---|---|
| Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13) | -0.80 ± 0.356 | 0.25 ± 0.444 |
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.
| score on a scale | DCCR | Placebo for DCCR |
|---|---|---|
| Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) - Pre-COVID Analysis | -6.64 ± 1.001 | -3.51 ± 1.278 |
Collected over Baseline to Visit 7 (Week 13). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| DCCR | 0/84 (0%) | 6/84 (7.1%) | 69/84 (82.1%) |
| Placebo for DCCR | 0/42 (0%) | 0/42 (0%) | 31/42 (73.8%) |
| Event | DCCR | Placebo for DCCR |
|---|---|---|
| PneumoniaInfections and infestations | 2/84 | 0/42 |
| Lower respiratory tract infectionInfections and infestations | 1/84 | 0/42 |
| Staphylococcal infectionInfections and infestations | 1/84 | 0/42 |
| Abnormal behaviourPsychiatric disorders | 1/84 | 0/42 |
| AggressionPsychiatric disorders | 1/84 | 0/42 |
| Pulmonary oedemaRespiratory, thoracic and mediastinal disorders | 1/84 | 0/42 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 1/84 | 0/42 |
| Oedema peripheralGeneral disorders | 1/84 | 0/42 |
| Erythema multiformeSkin and subcutaneous tissue disorders | 1/84 | 0/42 |
| Event | DCCR | Placebo for DCCR |
|---|---|---|
| HypertrichosisSkin and subcutaneous tissue disorders | 30/84 | 6/42 |
| Oedema peripheralGeneral disorders | 16/84 | 4/42 |
| HeadacheNervous system disorders | 5/84 | 6/42 |
| Upper respiratory tract infectionInfections and infestations | 9/84 | 5/42 |
| HyperglycaemiaMetabolism and nutrition disorders | 10/84 | 0/42 |
| DiarrhoeaGastrointestinal disorders | 1/84 | 4/42 |
| HirsutismSkin and subcutaneous tissue disorders | 6/84 | 3/42 |
| Abdominal painGastrointestinal disorders | 1/84 | 3/42 |
| PyrexiaGeneral disorders | 5/84 | 0/42 |
| Blood glucose increasedInvestigations | 5/84 | 2/42 |
Safety population includes all randomized subjects who receive any amount of study drug.
| Age, Continuous(years) | DCCR | Placebo for DCCR | Total |
|---|---|---|---|
| Mean | 13.2 (4 to 40) | 13.6 (5 to 44) | 13.4 (4 to 44) |
| Age, Customized(Participants) | DCCR | Placebo for DCCR | Total |
|---|---|---|---|
| <8 years | 18 | 7 | 25 |
| 8 to <12 years | 21 | 14 | 35 |
| 12 to <18 years | 30 | 11 | 41 |
| 18 to <65 years | 15 | 10 | 25 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | DCCR | Placebo for DCCR | Total |
|---|---|---|---|
| Female | 47 | 23 | 70 |
| Male | 37 | 19 | 56 |
| Ethnicity (NIH/OMB)(Participants) | DCCR | Placebo for DCCR | Total |
|---|---|---|---|
| Hispanic or Latino | 6 | 7 | 13 |
| Not Hispanic or Latino | 77 | 34 | 111 |
| Unknown or Not Reported | 1 | 1 | 2 |
| Race/Ethnicity, Customized(Participants) | DCCR | Placebo for DCCR | Total |
|---|---|---|---|
| American Indian or Alaska Native | 1 | 0 | 1 |
| Asian | 0 | 1 | 1 |
| Native Hawaiian or Pacific Islander | 1 | 0 | 1 |
| Black or African American | 4 | 2 | 6 |
| White | 72 | 34 | 106 |
| Multiple | 5 | 3 | 8 |
| Other | 1 | 2 | 3 |
| HQ-CT Total Score(score on a scale) | DCCR | Placebo for DCCR | Total |
|---|---|---|---|
| Mean | 23.0 ± 6.01 | 21.9 ± 5.08 | 22.7 ± 5.72 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from May 2018 to primary completion in May 2020, 23 months.
Results were first posted in Sep 2023, 3 years 4 months after primary completion.
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Studies of the drug tested here: Diazoxide choline