NCT03569631

A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome

A Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study of BPN14770 in Adult Males With Fragile X Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Tetra Discovery Partners · Industry
• Sex: Male
• Age: 18 Years – 45 Years
• Healthy volunteers: Not accepted

Summary

This is a single-center, randomized, double-blind, 2-period crossover study to explore the effects of BPN14770 on cognitive function and behavior in subjects with Fragile X Syndrome. Subjects will receive both active treatment with BPN14770 capsules and matching placebo capsules in the course of the study. One treatment will be administered during each of the 12-week study periods.

Detailed description

A total of 30 subjects will be enrolled. The study consists of a screening period of up to 28 days prior to initial treatment, followed by two double-blind treatment periods, each 12 weeks long. A final follow-up visit or phone contact for safety is planned one week after the conclusion of Period 2. Eligible subjects will be randomized in a blinded, balanced (1:1) fashion to receive either 25 mg BPN14770 capsules or matching placebo capsules during Period 1, followed by the opposite treatment during Period 2. One capsule will be taken twice daily during both double-blind periods. Subjects will return to the clinic at the end of Weeks 2, 6, and 12 of each study period. Cognitive and behavioral evaluations will be repeated at Weeks 6 and 12 of each Period. Additionally, patients will be monitored for adverse events via a telephone call at the end of Week 1 of each Period, and one week following completion of Period 2 or following early discontinuation. During clinic visits, adverse effects will be assessed, and laboratory measures, vital signs, and ECGs will be performed. Suicidality risk will also be evaluated at each clinic visit; if a concern is detected, the subject will be referred for further evaluation and treatment. Cognitive and behavioral assessments will be performed during each clinic visit. Pharmacodynamic measures of CNS function will be obtained to evaluated effects of the drug in the brain. Pharmacokinetic samples will be collected to confirm that study drug is present and to estimate plasma exposure at Week 12 of each Period.

Conditions

• Fragile X Syndrome
• FXS
• Fra(X) Syndrome

Interventions

Type	Name	Description
DRUG	BPN14770	25 mg BPN14770 capsules
DRUG	Placebo	Placebo capsules to mimic 25 mg BPN14770 capsules

Timeline

• Start date: 2018-07-09
• Primary completion: 2020-07-31
• Completion: 2020-07-31
• First posted: 2018-06-26
• Last updated: 2024-12-19
• Results posted: 2024-12-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study