BPN14770 for Fragile X Syndrome

Recruiting at 23 trial locations

Overseen By CEO

Age: < 65

Sex: Male

Trial Phase: Phase 3

Sponsor: Tetra Discovery Partners

Must be taking: Antiepileptics

Disqualifiers: Cardiovascular, Renal, Hepatic, Psychiatric, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

300 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a drug called BPN14770 to see if it is safe for extended use in males with fragile X syndrome. The study includes those who have already participated in earlier trials of the drug. BPN14770 aims to help by improving brain function related to the condition. It has been previously studied for its potential to improve cognitive function and behavioral outcomes in patients with fragile X syndrome.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you have a seizure disorder, you must have been seizure-free during the parent study while on antiepileptic treatment.

How does the drug BPN14770 differ from other treatments for Fragile X Syndrome?

BPN14770 is unique because it works by inhibiting phosphodiesterase-4D (PDE4D), which is different from other treatments that often target different pathways, such as mGluR5. This drug has shown significant improvements in cognitive function and daily activities in adults with Fragile X Syndrome, making it a novel option compared to existing treatments. ^{1 2 3 4 5}

Research Team

Elizabeth Berry-Kravis, MD, PhD

Principal Investigator

Rush University Medical Center

Eligibility Criteria

This trial is for males with Fragile X Syndrome who completed a previous BPN14770 study. They must be able to consent or provide assent, use contraception if sexually active, have a caregiver, and attend regular clinic visits. Excluded are those with substance abuse, renal/hepatic impairment, significant lab/ECG abnormalities from the parent study, major psychiatric conditions (except autism or anxiety), certain diseases like AIDS or hepatitis, or participation in other trials within 30 days.

Inclusion Criteria

Subject has a parent, legal authorized guardian, or consistent caregiver

Subject has completed the Week 13 visit from one of two parent clinical trials with BPN14770: Study 204 or Study 301

Subject must be willing to practice barrier methods of contraception while on study, if sexually active

Exclusion Criteria

I have cirrhosis or unstable liver disease.

I do not have any major health issues that could affect my participation in the study.

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants continue receiving BPN14770 to assess long-term safety and tolerability

4 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months

Treatment Details

Interventions

• Zatolmilast/ BPN14770

Trial Overview The trial tests Zatolmilast/BPN14770 as an ongoing treatment for subjects who've finished prior studies on it. It's an open-label extension meaning everyone gets the drug and there's no placebo group; participants know what they're taking.

Participant Groups

Group I: Study Drug (BPN14770) Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Tetra Discovery Partners

Lead Sponsor

Trials

9

Recruited

1,000+

Findings from Research

In two phase 2b clinical trials involving 175 adults and 139 adolescents with Fragile X syndrome, the mGluR5 antagonist mavoglurant did not show significant improvement in behavioral symptoms after 12 weeks of treatment, failing to confirm previous findings.

The safety profile of mavoglurant was consistent with earlier studies, showing few adverse events, but the trials indicated that the methylation status of the FMR1 gene did not predict treatment efficacy, suggesting a need for further research in younger populations and with different assessment markers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Berry-Kravis, E., Des Portes, V., Hagerman, R., et al.[2016]

The 2020 Special Issue on fragile X syndrome (FXS) presents nine new studies that provide updated insights into treatment options for this condition.

These manuscripts collectively contribute to the understanding of FXS and may inform future therapeutic strategies, although specific details on the findings were not provided in the abstract.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fragile X Syndrome: Recent Research Updates toward Capturing Treatments' Improvement in Clinical Trials. Budimirovic, DB., Protic, DD.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Inhibition of phosphodiesterase-4D in adults with fragile X syndrome: a randomized, placebo-controlled, phase 2 clinical trial. [2022]

2. Korea (South) pubmed.ncbi.nlm.nih.gov

Fragile site X chromosomes in mentally retarded boys. [2015]

3. United States pubmed.ncbi.nlm.nih.gov

Dysregulated NMDA-Receptor Signaling Inhibits Long-Term Depression in a Mouse Model of Fragile X Syndrome. [2020]

4. United States pubmed.ncbi.nlm.nih.gov

Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. [2016]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Fragile X Syndrome: Recent Research Updates toward Capturing Treatments' Improvement in Clinical Trials. [2022]