Zatolmilast for PPP2R5D Syndrome
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you can be on up to 3 prescribed psychotropic medications.
How does the drug BPN14770 differ from other treatments for PPP2R5D Syndrome?
BPN14770, also known as Zatolmilast, is unique because it targets the underlying molecular mechanisms of PPP2R5D Syndrome by modulating the activity of protein phosphatase 2A (PP2A), which is involved in key neuronal processes. This approach is novel as there are currently no standard treatments specifically addressing the genetic and molecular basis of this condition.12345
What is the purpose of this trial?
The primary objective of this study is to assess the safety and tolerability of BPN14770 in participants aged 9 to 45 years with JS.
Eligibility Criteria
This trial is for individuals aged 9 to 45 with Jordan's Syndrome (JS), a neurodevelopmental disorder. It includes those with related conditions like Klinefelter and Triple X Syndromes, but the full eligibility criteria are not provided.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Double-blind Treatment
Participants receive either BPN14770 or placebo BID for 24 weeks
Open-label Extension
Participants receive a weight adjusted dose of BPN14770 BID for 24 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- BPN14770
Find a Clinic Near You
Who Is Running the Clinical Trial?
Shionogi
Lead Sponsor
Dr. Isao Teshirogi
Shionogi
Chief Executive Officer since 2008
PhD in Pharmaceutical Sciences from the University of Tokyo
Dr. Takuko Sawada
Shionogi
Chief Medical Officer since 2022
MD from a recognized institution (specific details not found)