NCT05163808

A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

A Randomized, Double-blind, Placebo-controlled, Two-Part Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 163 (actual)
• Sponsor: Tetra Discovery Partners · Industry
• Sex: Male
• Age: 9 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

This is a 2-part study, with each part having a unique set of objectives for male adolescents aged 9 to \< 18 years with fragile X syndrome (FXS). Part 1 is an open-label, single-dose, pharmacokinetics (PK) assessment of BPN14770 25 mg and 50 mg, while Part 2 is double-blind (DB) and randomized between two treatment groups (Study Drug and Placebo).

Detailed description

In amendment 4 the enrollment age range what changed to allow enrollment in participants as young as 9 years of age. This only affects Part 2 as Part 1 was completed prior to this amendment.

Conditions

• Fragile X Syndrome

Interventions

Type	Name	Description
DRUG	zatolmilast	Subjects will receive a 15 mg or 25 mg dose of zatolmilast (BPN14770) or placebo
DRUG	Placebo	Placebo

Timeline

• Start date: 2022-03-29
• Primary completion: 2025-09-02
• Completion: 2025-09-02
• First posted: 2021-12-20
• Last updated: 2026-02-04

Locations

17 sites across 1 country: United States

Regulatory

• FDA-regulated drug study