A Phase 2 Randomized, Double-blind, Placebo-controlled, Study of Zatolmilast (BPN14770) in Subjects With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome)
Trial Snapshot
- Phase
- Phase 2
- Status
- Active, not recruiting
- Sponsor
- Enrollment
- 30
- Locations
- 6
- Primary Endpoint
- Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 9 Years to 45 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Participant is aged 9 to 45 years, inclusive.
- •Participant has confirmed/documented history of PPP2R5D Neurodevelopmental Disorder.
- •Current treatment with no more than 3 prescribed psychotropic medications.
- •Participant has a parent, legal authorized guardian or consistent caregiver.
Exclusion Criteria
- •Participant has body weight less than 25 kilograms (kg).
- •Clinically significant abnormalities, in the investigator's judgment, in safety laboratory tests, vital signs, or electrocardiogram (ECG), as measured during Screening.
- •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.
- •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.
Arms & Interventions
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.
Intervention: BPN14770 (Drug)
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.
Intervention: 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.
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Time Frame: Day 1 up to Week 50
Discontinuation-emergent Signs and Symptoms (DESS) Questionnaire Score
Time Frame: Week 50
Number of Participants with Seizures
Time Frame: Up to Week 50
Number of Participants at Suicidality Risk
Time Frame: Up to Week 48
Secondary Outcomes
No secondary outcomes reported