An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

NCT05367960 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: BPN14770-CNS-302
• Study Type: interventional
• Target: 314
• Locations: 1 country
• Sites: 17

Brief Summary

This is a 4-year, open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in adult males) and Study BPN14770-CNS-204 (in adolescent males).

Conditions

Fragile X Syndrome

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of BPN14770

  Long-term safety and tolerability of BPN14770 in these subjects with fragile X syndrome (FXS) who were treated in one of those parent clinical trials. Safety/tolerability endpoint: Treatment Emergent Adverse Events (TEAEs), which will be coded using the Medical Dictionary for Regulatory Activities. Subject incidence of each system organ class and unique preferred term will be tabulated, including all TEAEs, at least possibly related TEAEs, TEAEs resulting discontinuation of study treatment, TEAEs by intensity, and treatment-emergent SAEs. The safety analysis will be descriptive in nature.

  24 months

Secondary Outcomes (8)

• National Institute of Health (NIH)- Toolbox Cognitive Battery (TCB)

  24 Months

• Numerical rating scale scores (NRS) scores

  24 Months

• Caregiver Global Impression of Improvement (CaGI-I) assessments:

  24 Months

• Clinical Global Impression Severity (CGI-S) assessments:

  24 Months

• Vineland-3 Adaptive Behavior Scale (Vineland-3)

  24 Months

Study Arms (1)

Study Drug (BPN14770)

OTHER

25mg BID Study Drug BPN14770 (Adults) or 15mg BID Study Drug BPN14770 (Adolescents with body weight \<43 kg)

Drug: Zatolmilast/ BPN14770

Interventions

Zatolmilast/ BPN14770 DRUG

25mg zatolmilast/BPN14770 (Adults) or 15 mg zatolmilast/BPN14770 (Adolescents \<43 kg)

Study Drug (BPN14770)

Eligibility Criteria

• Age: 9 Years - 45 Years
• Gender Eligibility Details: Male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subject has completed the Week 13 visit, whether on treatment or discontinued from treatment, from one of two parent clinical trials with
• BPN14770:
• Study 204
• Study 301 Subjects who discontinued study treatment early due to AEs deemed related to study treatment by the investigator in one of the parent studies will NOT be eligible, regardless of whether the Week 13 visit was completed.
• Subjects with a history of seizure disorder who are currently receiving treatment with antiepileptics must have remained seizure-free during the parent study. Any subject experiencing a seizure during the parent study is not eligible to continue into this long-term safety study.
• Subject must be willing to practice barrier methods of contraception while on study, if sexually active. Abstinence is also considered a reasonable form of birth control in this study population.
• Subject has a parent, legal authorized guardian, or consistent caregiver.
• Subject and caregiver are able to attend the clinic regularly and reliably.
• If subject is his own legal guardian, he is able to understand and sign an informed consent form to participate in the study.
• For subjects who are not their own legal guardian, subject's parent/legally authorized guardian is able to understand and sign an informed consent form for their child to participate in the study.
• If subject is not his own legal guardian, subject must provide assent for participation in the study if he has the cognitive ability to do so.

You may not qualify if:

• \. History of, or current cardiovascular, renal, hepatic, respiratory, gastrointestinal, psychiatric, neurologic, cerebrovascular, or other systemic disease that would place the subject at risk or potentially interfere with the interpretation of the safety, tolerability, or efficacy of the study treatment. Common conditions such as mild hypertension, etc. are allowed per the principal investigator's (PI) judgement as long as they are stable and controlled by medical therapy that is constant for at least 4 weeks before randomization.
• \. Renal impairment, defined as serum creatinine \>1.25×ULN per the latest available laboratory results from the parent study.
• \. Cirrhosis, unstable chronic liver disease or acute liver disease. Hepatic impairment, defined as ALT or AST elevation \> 2 × ULN per the latest available laboratory results from the parent study. Subjects with stable chronic hepatitis B on oral anti-viral therapy and subjects cured of chronic hepatitis C are allowed. Subjects with Gilbert syndrome are allowed.
• \. Clinically significant abnormalities, in the investigator's judgement, in safety laboratory tests, vital signs, or ECG, as measured at the final visit of the parent study.
• \. Substance abuse documented during the parent study.
• \. Significant hearing or visual impairment that may affect the subject's ability to complete the test procedures.
• \. Concurrent major psychiatric condition (eg, major depressive disorder, schizophrenia, or bipolar disorder) as confirmed by the investigator. Subjects with additional diagnosis of autism spectrum disorder or anxiety disorder will be allowed.
• \. Subject has active diseases that would interfere with participation, such as acquired immunodeficiency disorder, hepatitis C, hepatitis B, or tuberculosis.

Sponsors & Collaborators

• Tetra Discovery Partners: lead

Study Sites (17)

Amnova Clinical Research

Irvine, California, 92604, United States

Location

Thompson Autism & Neurodevelopment Center - CHOC

Orange, California, 92828, United States

Location

MIND Institute UC Davis Medical Center

Sacramento, California, 95817, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

U Mass

Worcester, Massachusetts, 01655, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Suburban Research Associates

Media, Pennsylvania, 19063, United States

Location

Clinic for Special Children

Strasburg, Pennsylvania, 17579, United States

Location

Greenwood Genetic Center

Greenville, South Carolina, 29605, United States

Location

University of Utah and Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome

Interventions

BPN14770

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Study Officials

• Elizabeth Berry-Kravis, MD

  Rush University Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 29, 2022
• First Posted: May 10, 2022
• Study Start: November 1, 2022
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: October 3, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (17)