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Active, not recruiting Phase 2

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

NCT06717438 · tracked via the Priya Life Science USA tracker
Sponsor
Phase
Phase 2
Started
2025-05-13
Last updated
2026-06-09

Condition(s) studied

Jordan's SyndromePPP2R5D Neurodevelopmental Disorder

Investigational drug(s) / intervention(s)

BPN14770Placebo

BPN14770: Capsules for oral administration

Placebo: Capsules for oral administration

Study 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.

Eligibility

Sex
ALL
Min age
9 Years
Max age
45 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Change From Baseline at Week 24 in Numerical Rating Scale Within the Domain of Language/Communication — Baseline, Week 24
  • Number of Participants with Treatment-emergent Adverse Events (TEAEs) — Day 1 up to Week 96
  • Discontinuation-emergent Signs and Symptoms (DESS) Questionnaire Score — Week 98
  • Number of Participants with Seizures — Up to Week 96
  • Number of Participants at Suicidality Risk — Up to Week 96

Trial sites (3)

FacilityCityRegionStatus
Rush University Medical Center Chicago Illinois
Boston Children's Hospital Boston Massachusetts
Seattle Children's Hospital Seattle Washington
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06717438 on ClinicalTrials.gov ↗ ← All trials in the USA