Study of Zatolmilast (BPN14770) in Participants With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome [JS])

Phase 2

Not yet recruiting

• Conditions: Jordan's Syndrome; PPP2R5D Neurodevelopmental Disorder
• Interventions: Drug: BPN14770; Drug: Placebo

• Registration Number: NCT06717438
• Lead Sponsor: Shionogi

Brief Summary

The primary objective of this study is to assess the safety and tolerability of BPN14770 in participants aged 9 to 45 years with JS.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-12-02
• Study First Submitted QC: 2024-12-02
• Study First Posted: 2024-12-05
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-10
• Last Update Posted: 2025-03-11
• Study Start: 2027-03-01
• Primary Completion: 2030-01-31
• Study Completion: 2030-01-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
BPN14770	BPN14770	Participants will receive a weight adjusted dose of BPN14770 twice daily (BID) during the 24-week Double-blind Period. Participants in the BPN14770 arm who complete the Double-blind Period will have the opportunity to continue in the Open-label Extension Period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week Open-label Extension Period.
Placebo	Placebo	Participants will receive BPN14770 matching placebo BID during the 24-week Double-blind Period. Participants in the placebo arm who complete the Double-blind Period will have the opportunity to continue in the Open-label Extension Period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week Open-label Extension Period.
Placebo	BPN14770	Participants will receive BPN14770 matching placebo BID during the 24-week Double-blind Period. Participants in the placebo arm who complete the Double-blind Period will have the opportunity to continue in the Open-label Extension Period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week Open-label Extension Period.

Primary Outcome Measures

Name	Time	Method
Number of Participants at Suicidality Risk	Up to Week 48
Number of Participants with Treatment-emergent Adverse Events (TEAEs)	Day 1 up to Week 50
Discontinuation-emergent Signs and Symptoms (DESS) Questionnaire Score	Week 50
Number of Participants with Seizures	Up to Week 50

Trial Locations

Locations (3)

Rush University Medical Center; 🇺🇸 Chicago, Illinois, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States