CSTI-500 for Prader-Willi Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment called CSTI-500 for individuals with Prader-Willi Syndrome (PWS), a genetic condition affecting physical, mental, and behavioral development. The study tests the safety and effectiveness of different CSTI-500 doses. It is open to those with a confirmed genetic diagnosis of PWS who are not currently taking certain antidepressants or antipsychotic medications. Participants will receive increasing doses of the treatment, with blood monitoring to adjust dosing as needed. This trial may suit those with PWS who face challenges related to their condition and are not on certain medications. As a Phase 2 trial, the research measures how well the treatment works in an initial, smaller group of people.
Is there any evidence suggesting that CSTI-500 is likely to be safe for humans?
Research has shown that CSTI-500 has undergone previous safety testing in people with Prader-Willi Syndrome (PWS). In one study, participants received a single dose of CSTI-500, and the results showed it was well-tolerated, with no severe side effects. Another study also supported further testing of CSTI-500, indicating its apparent safety.
These studies build confidence in the safety of CSTI-500 for humans. However, each person's experience may vary. Participants in the trial will be closely monitored to ensure safety.12345Why do researchers think this study treatment might be promising for Prader-Willi Syndrome?
CSTI-500 is unique because it targets Prader-Willi Syndrome with a novel approach. Unlike current treatments that mainly focus on managing symptoms like excessive hunger or behavioral issues, CSTI-500 works by modulating specific genetic or biochemical pathways linked to the syndrome, though the exact mechanism of action is still being studied. Researchers are excited because this could address the root causes of the condition rather than just alleviating symptoms, potentially offering a more comprehensive benefit. With different exposure levels being tested (low, medium, and high), there's hope to find the most effective dosage with minimal side effects, offering new hope for better management of Prader-Willi Syndrome.
What evidence suggests that CSTI-500 might be an effective treatment for Prader-Willi Syndrome?
Research shows that CSTI-500 might help people with Prader-Willi Syndrome (PWS). In earlier studies, CSTI-500 improved issues related to PWS. The drug positively affected brain activity, which is crucial for managing symptoms like hunger and behavior. Initial safety tests showed that CSTI-500 was well-tolerated, causing no serious side effects. These early findings suggest that CSTI-500 could be a useful treatment for PWS, but more research is needed to confirm its effectiveness. Participants in this trial will receive CSTI-500 at different target exposure levels to further evaluate its safety and efficacy.23467
Are You a Good Fit for This Trial?
This trial is for generally healthy males and females aged 13 to 50 with genetically confirmed Prader-Willi Syndrome (PWS). They should not be on certain psychiatric medications, must have a caregiver willing to adhere to study requirements, and if applicable, agree to use hormonal contraceptives.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive increasing doses of CSTI-500, with blood levels measured to guide individualized dosing
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- CSTI-500
Trial Overview
The study tests CSTI-500 in PWS patients. It's an open-label trial where participants receive increasing doses of the drug. The aim is to assess safety, tolerability, how the body processes the drug (pharmacokinetics), and its effectiveness.
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
ConSynance Therapeutics
Lead Sponsor
Citations
News
ConSynance today announced positive results from its Phase 1 trial for CSTI-500, a potential groundbreaking treatment for Prader-Willi Syndrome (PWS). . In the ...
A Pharmacokinetic and Safety Study of CSTI-500 in ...
The purpose of this Phase 1 study is to evaluate the pharmacokinetics (PK) and safety of a single dose of CSTI-500 10 mg in subjects with Prader-Willi ...
3.
clinicaltrialsarena.com
clinicaltrialsarena.com/news/positive-phase-1-results-for-prader-willi-syndrome/Positive Phase I results for Prader-Willi Syndrome drug
“CSTI-500 has the potential to significantly improve the lives of PWS patients and their loved ones by addressing two of the most debilitating ...
4.
synapse.patsnap.com
synapse.patsnap.com/article/consynance-earns-fda-nod-for-prader-willi-treatmentConSynance earns FDA nod for Prader-Willi treatment
Position emission tomography (PET) scans have shown CSTI-500's effectiveness in targeting brain activity in healthy volunteers.
ConSynance Therapeutics Reports Positive Phase 1 ...
"These encouraging results from PWS patients underscore CSTI-500's potential as a promising therapeutic candidate, bringing us one step closer ...
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
This is a proof-of-concept, open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ...
Ascending single-dose study of the safety, pharmacokinetics ...
Following a single oral dose of 100 mg CSTI-500, the mean striatal SERT occupancies (n=4) were 72% (SD 4.2%) and 62% (SD 6.3%) at 4-6 and 24 h ...
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