Active, Not Recruiting

NCT Number: NCT06717438

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Jordan's Syndrome PPP2R5D Neurodevelopmental Disorder

Age range

9 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Rush University Medical Center, Chicago, Illinois, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

BPN14770

Drug

Capsules for oral administration

Other names: Zatolmilast

Placebo

Drug

Capsules for oral administration

Primary outcomes

Change From Baseline at Week 24 in Numerical Rating Scale Within the Domain of Language/Communication

Time frame: Baseline, Week 24
Number of Participants with Treatment-emergent Adverse Events (TEAEs)

Time frame: Day 1 up to Week 96
Discontinuation-emergent Signs and Symptoms (DESS) Questionnaire Score

Time frame: Week 98
Number of Participants with Seizures

Time frame: Up to Week 96
Number of Participants at Suicidality Risk

Time frame: Up to Week 96

Sponsors and collaborators

Lead sponsor

Shionogi

Industry

Registry information

Official study title

A Phase 2 Randomized, Double-blind, Placebo-controlled, Study of Zatolmilast (BPN14770) in Subjects With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome)

Important dates

Study start: 2025
Primary completion: 2027
Study completion: 2027
First posted: Dec 5, 2024
Registry last updated: Jun 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.