Fragile X Syndrome Clinical Trial
Official title:
A Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study of BPN14770 in Adult Males With Fragile X Syndrome
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.
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. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT05418049 -
Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge
|
Phase 2 | |
Enrolling by invitation |
NCT01364818 -
Brain Connectivity in Neurodevelopmental Disorders in Response to Treatment
|
N/A | |
Completed |
NCT01120626 -
Randomized Controlled Study of Donepezil in Fragile X Syndrome
|
Phase 2 | |
Completed |
NCT00965432 -
A Single-Dose Study in Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107
|
Phase 1 | |
Completed |
NCT01204151 -
Teaching Math Skills to Individuals With Fragile X Syndrome
|
N/A | |
Active, not recruiting |
NCT00334971 -
Aromatase Activity and Ovarian Growth Factors in African-American Versus Caucasian Women
|
N/A | |
Enrolling by invitation |
NCT06139172 -
Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities
|
N/A | |
Recruiting |
NCT04977986 -
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome
|
Phase 3 | |
Active, not recruiting |
NCT04698551 -
NIPD on cffDNA for Triplet Repeat Diseases
|
||
Completed |
NCT03722290 -
Metformin in Children and Adults With Fragile X Syndrome
|
Phase 2 | |
Completed |
NCT05030129 -
Single Blind Study of Ergoloid Mesylates, 5-HTP and the Combination in Adult Males With Fragile X Syndrome
|
Phase 2 | |
Recruiting |
NCT05957549 -
Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG
|
N/A | |
Recruiting |
NCT04141163 -
Metformin in Patients With Fragile X
|
Phase 1/Phase 2 | |
Not yet recruiting |
NCT06081348 -
Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders
|
Phase 2 | |
Completed |
NCT00858689 -
Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
|
N/A | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
Enrolling by invitation |
NCT03802799 -
Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS
|
Phase 2/Phase 3 | |
Recruiting |
NCT05295277 -
Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort
|
||
Enrolling by invitation |
NCT03836300 -
Parent and Infant Inter(X)Action Intervention (PIXI)
|
N/A | |
Completed |
NCT01725152 -
Ganaxolone Treatment in Children With Fragile X Syndrome
|
Phase 2 |