Prader-Willi SyndromeNCT04102839
PWS Outcomes Assessment Study
An observational study of Prader-Willi Syndrome, run by Soleno Therapeutics, Inc. at 1 site in United States.
Trial summary
This is a longitudinal study during which qualitative interviews will be conducted with caregivers of Soleno C601/C602 study subjects. There is an additional option for caregivers to collect video data of PWS patients doing specific activities of daily life. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Interview
- How many people
- 59 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jul 2019to 8 Jun 2021
- Phase
- Observational
- 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 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Caregivers of PWS patients who are participating in Soleno C601 and C602 Clinical Studies.
Who may be able to join
- Eligible caregivers must:
- Be willing and able to provide informed consent in English
- Care for a C601/C602 trial subject or potential trial subject
- Have access to a smartphone or the internet
- Be able to converse in English
- Eligible PWS participants for the optional daily life video capture must:
Show all 11Show fewer
- Be willing and able to provide informed consent or assent.
- Be enrolled in the C601/C602 trial
- Be able to converse in English
- Have a caregiver who has access to an Apple or Android smartphone or has internet access and is willing to use a wi-fi-only device to record and upload videos
- Have a caregiver who is willing and able to record activity videos
Who cannot join
- n/a
Trial locations and contacts
1 site in 1 country.
About this study
Official title: PWS Outcome Assessment Study
This is a longitudinal study during which qualitative interviews will be conducted with caregivers of Soleno C601/C602 study subjects. There is an additional option for caregivers to collect video data of PWS patients doing specific activities of daily life.
The purpose of this study is to understand the real-world and nuanced impact of a potential therapeutic on individual PWS patients. The results of this study will complement the outcomes being captured during the Soleno C601/C602 clinical studies.
There is no treatment or intervention associated with this study.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Interview
- Keywords
- Prader-Willi Syndrome, PWS
- 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.
InterviewOther
Qualitative Interview
What is being measured
The primary outcome is the main question the study is designed to answer.
Meaningful Change in Symptom Categories
The percentage of caregivers reporting meaningful change in each of the symptom categories in the Study C601 treated arm versus the Study C601 placebo arm as measured by the five-point quantification scale anchored in the qualitative questions (Scale: 1=very meaningful negative change, 2=slightly meaningful negative change, 3=no meaningful change, 4=slightly meaningful change, 5=very meaningful positive change)
Measured: From baseline for interview #2 (Week 13-16), From baseline for interview #3 (Week 26-28), From baseline for interview #4 (Week 65-67)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2019
- Study start15 Jul 2019
- Primary completion8 Jun 2021
- Study completion8 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Family based
- Time perspective
- Other
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Soleno Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Casimir Trials
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04102839
- Sponsor's protocol ID
- CAS-SOL001-01
- Status last verified
- Oct 2024
- First submitted
- 23 Aug 2019
- Last updated
- 4 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PWS Outcome Assessment Study
- ClinicalTrials.gov ID
- NCT04102839
- Acronym / sponsor ID
- CAS-SOL001-01
- Conditions
- Prader-Willi Syndrome
- Keywords
- Prader-Willi Syndrome, PWS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 59 participants (actual)
- Lead sponsor
- Soleno Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Casimir Trials
- Study start
- 15 Jul 2019 (actual)
- Primary completion
- 8 Jun 2021 (actual)
- Study completion
- 8 Jun 2021 (actual)
- First posted
- 25 Sep 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a longitudinal study during which qualitative interviews will be conducted with caregivers of Soleno C601/C602 study subjects. There is an additional option for caregivers to collect video data of PWS patients doing specific activities of daily life.
The purpose of this study is to understand the real-world and nuanced impact of a potential therapeutic on individual PWS patients. The results of this study will complement the outcomes being captured during the Soleno C601/C602 clinical studies.
There is no treatment or intervention associated with this study.
Study design
- Observational model
- Family based
- Time perspective
- Other
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Caregivers of PWS patients who are participating in Soleno C601 and C602 Clinical Studies.
Key inclusion criteria (11 listed)
- Be willing and able to provide informed consent in English
- Care for a C601/C602 trial subject or potential trial subject
- Have access to a smartphone or the internet
- Be able to converse in English
- Be willing and able to provide informed consent or assent.
Key exclusion criteria (1 listed)
- n/a
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 |
|---|---|---|
| InterviewQualitative Interview | Other | – |
Primary outcome
Meaningful Change in Symptom Categories
Time frame: From baseline for interview #2 (Week 13-16), From baseline for interview #3 (Week 26-28), From baseline for interview #4 (Week 65-67)
The percentage of caregivers reporting meaningful change in each of the symptom categories in the Study C601 treated arm versus the Study C601 placebo arm as measured by the five-point quantification scale anchored in the qualitative questions (Scale: 1=very meaningful negative change, 2=slightly meaningful negative change, 3=no meaningful change, 4=slightly meaningful change, 5=very meaningful positive change)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2024.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Prader-Willi Syndrome, run by Soleno Therapeutics, Inc. at 1 site in United States.
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 11 inclusion and 1 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 4 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
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?
Meaningful Change in Symptom Categories (time frame: From baseline for interview #2 (Week 13-16), From baseline for interview #3 (Week 26-28), From baseline for interview #4 (Week 65-67)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 4 Nov 2024.