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

NCT06717438 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: BPN14770-CNS-205
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 3

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.

Conditions

PPP2R5D Neurodevelopmental Disorder; Jordan's Syndrome

Keywords

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

Outcome Measures

Primary Outcomes (5)

• 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

Study Arms (2)

BPN14770

EXPERIMENTAL

Participants will receive a weight adjusted dose of BPN14770 twice daily (BID) during the 24-week double-blind period. Participants who complete the double-blind period will have the opportunity to continue in the open-label extension (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 extended open-label extension (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.

Drug: BPN14770

Placebo

PLACEBO COMPARATOR

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.

Drug: BPN14770; Drug: Placebo

Interventions

BPN14770 DRUG

Capsules for oral administration

Also known as: Zatolmilast

BPN14770; Placebo

Placebo DRUG

Capsules for oral administration

Placebo

Eligibility Criteria

• Age: 9 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shionogi: lead

Study Sites (3)

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98101, United States

Location

MeSH Terms

Interventions

BPN14770

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2024
• First Posted: December 5, 2024
• Study Start: May 13, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: June 9, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)