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

NCT ID: NCT06717438

Last Updated: 2026-01-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-13

Study Completion Date

2030-01-31

Brief Summary

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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

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Conditions

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Jordan's Syndrome PPP2R5D Neurodevelopmental Disorder

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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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.

Group Type EXPERIMENTAL

BPN14770

Intervention Type DRUG

Capsules for oral administration

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.

Group Type PLACEBO_COMPARATOR

BPN14770

Intervention Type DRUG

Capsules for oral administration

Placebo

Intervention Type DRUG

Capsules for oral administration

Interventions

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BPN14770

Capsules for oral administration

Intervention Type DRUG

Placebo

Capsules for oral administration

Intervention Type DRUG

Other Intervention Names

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Zatolmilast

Eligibility Criteria

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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.
Minimum Eligible Age

9 Years

Maximum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shionogi

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Rush University Medical Center

Chicago, Illinois, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Countries

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United States

Other Identifiers

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BPN14770-CNS-205

Identifier Type: -

Identifier Source: org_study_id

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