A Phase 2 Randomized, Double-blind, Placebo-controlled, Study of Zatolmilast (BPN14770) in Subjects With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome)
| Registry | NCT06717438 |
|---|---|
| Status | Active not recruiting |
| Phase | Phase 2 |
| Study type | Interventional; randomized, parallel, masking: triple, treatment |
| Enrollment | 30 (Actual) |
| Sponsor | Shionogi (Industry) |
| Conditions | Jordan's Syndrome, PPP2R5D Neurodevelopmental Disorder |
| Interventions | BPN14770, Placebo (Drug) |
| Dates | start 2025-05-13, primary completion 2027-12-31 (estimated), completion 2027-12-31 (estimated), first posted 2024-12-05, last updated 2026-06-09 |
| Locations | 3 sites in United States |
Related Papers Related Patents Related Grants Related Experts
All trials for Jordan's Syndrome All trials for PPP2R5D Neurodevelopmental Disorder All trials of BPN14770 All trials of Placebo
The primary objective of this study is to assess the efficacy, safety, and tolerability of BPN14770 in participants aged 9 to 45 years with JS.
| Arm | Type | Interventions | Description |
|---|---|---|---|
| BPN14770 | Experimental | Drug: BPN14770 | Participants will receive a weight adjusted dose of BPN14770 twice daily (BID) during the 24-week double-blind period. Participants who complete the double-blind period will have the opportunity to continue in the open-label extension (OLE) period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week OLE period. Eligible participants who complete the double-blind period and OLE period may be offered the opportunity to participate in a 48-week extended open-label extension (Ext OLE) period. Adult participants (≥18 years of age at the start of Ext OLE) will receive an age-adjusted dose of BPN14770 BID during the Ext OLE period, while participants younger than 18 years at the start of Ext OLE will receive a weight-adjusted dose of BPN14770 BID. |
| Placebo | Placebo comparator | Drug: BPN14770; Drug: Placebo | Participants will receive BPN14770-matching placebo BID during the 24-week double-blind period. Participants who complete the double-blind period will have the opportunity to continue in the OLE period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week OLE period. Eligible participants who complete the double-blind period and OLE period may be offered the opportunity to participate in a 48-week Ext OLE period. Adult participants (≥18 years of age at the start of Ext OLE) will receive an age-adjusted dose of BPN14770 BID during the Ext OLE period, while participants younger than 18 years at the start of Ext OLE will receive a weight-adjusted dose of BPN14770 BID. |
Sex: All, ages: 9 Years to 45 Years
Inclusion Criteria: 1. Participant is aged 9 to 45 years, inclusive. 2. Participant has confirmed/documented history of PPP2R5D Neurodevelopmental Disorder. 3. Current treatment with no more than 3 prescribed psychotropic medications. 4. Participant has a parent, legal authorized guardian or consistent caregiver. Exclusion Criteria: 1. Participant has body weight less than 25 kilograms (kg). 2. Clinically significant abnormalities, in the investigator's judgment, in safety laboratory tests, vital signs, or electrocardiogram (ECG), as measured during Screening. 3. Concurrent major psychiatric condition (e.g., Major Depressive Disorder, Schizophrenia or Bipolar Disorder) as diagnosed by the investigator. Participants with additional diagnosis of Autism Spectrum Disorder or Anxiety Disorder will be allowed. 4. Participant is planning to commence psychotherapy or cognitive behavior therapy (CBT) during the period of the study or had begun psychotherapy or CBT within 4 weeks prior to Screening.
| Facility | City | Country | Status |
|---|---|---|---|
| Rush University Medical Center | Chicago, Illinois | United States | |
| Boston Children's Hospital | Boston, Massachusetts | United States | |
| Seattle Children's Hospital | Seattle, Washington | United States |