BPN14770 for Fragile X Syndrome

Recruiting in Palo Alto (17 mi)

+21 other locations

Overseen byElizabeth Berry-Kravis, MD, PhD

Age: < 65

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Tetra Discovery Partners

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

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.

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

+5 more

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

+6 more

Participant Groups

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.

1Treatment groups

Experimental Treatment

Group I: Study Drug (BPN14770)Experimental Treatment1 Intervention

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