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

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06717438

Enrollment

Registered

2024-12-05

Start date

2025-05-13

Completion date

2027-12-31

Last updated

2026-06-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Jordan's Syndrome, PPP2R5D Neurodevelopmental Disorder

Keywords

Jordan's Syndrome, JS, PPP2R5D Neurodevelopmental Disorder, Zatolmilast, BPN14770

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.

Interventions

DRUGBPN14770

Capsules for oral administration

DRUGPlacebo

Capsules for oral administration

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

9 Years to 45 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame
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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jun 10, 2026