CompletedPhase 3

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

United States171 participantsStarted 2022-11-01

Plain-language summary

A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) with Fragile X Syndrome

Who can participate

Age range18 Years – 45 Years

SexMALE

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Inclusion criteria

✓. Male subject aged 18 to 45 years at screening visit.
✓. Subject has FXS with a molecular genetic confirmation of the full fragile X mental retardation-1 (FMR1)mutation (≥200 CGG repetitions).
✓. Subject is able to swallow capsules.
✓. Current treatment with ≤3 prescribed psychotropic medications. Anti-epileptic medications are permitted and are not counted as psychotropic medications if they are used for the treatment of seizures. Anti-epileptics for other indications, such as the treatment of mood disorders, count towards the limit of permitted medications.
✓. Permitted concomitant psychotropic medications must be at a stable dose and dosing regimen for at least 4 weeks prior to screening and must remain stable during the period between screening and the commencement of the study treatment.
✓. Anti-epileptic medications must be at a stable dose and dosing regimen for 12 weeks prior to screening and must remain stable during the period between screening and commencement of the study treatment.
✓. Subjects with a history of seizure disorder who are currently receiving treatment with anti-epileptics must have been seizure free for 3 months preceding screening or must be seizure free for 2 years if not currently receiving anti-epileptics.

Exclusion criteria

✕. Subject must be willing to practice barrier methods of contraception while on the study if sexually active. Abstinence is also considered a reasonable form of birth control in this study population.
✕0. Subject has a parent, legal authorized guardian, or consistent caregiver.
✕1. Subject and caregiver are able to attend the clinic regularly and reliably.
✕2. If subject is his own legal guardian, he is able to understand and sign informed consent to participate in the study.
✕3. For subjects who are not their own legal guardian, subject's parent/legally authorized guardian is able to understand and sign an informed consent form for their child to participate in the study.
✕4. If subject is not his own legal guardian, subject must provide assent for participation in the study if he has the cognitive ability to do so.
✕. Inability to successfully complete the NIH-TCB picture vocabulary and oral reading assessments at screening and baseline. The ability to complete the NIH-TBC oral reading and picture vocabulary subtest at baseline is defined as the ability to complete both subtests, with (1) confirmation from the clinician administering that the test administrations are valid (noted on the administration form) and (2) generation of valid test scores for each test.
✕. History of or current cardiovascular, renal, hepatic, respiratory, gastrointestinal, psychiatric, neurologic, cerebrovascular, or other systemic disease that would place the subject at risk or potentially interfere with the interpretation of the safety, tolerability, or efficacy of the study treatment.

What they're measuring

National Institutes of Health Toolbox Cognitive Battery cognition crystallized composite score (NIH-TCB CCC)

Timeframe: 13 Weeks

Trial details

NCT IDNCT05358886

SponsorTetra Discovery Partners

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-07-18

View on ClinicalTrials.gov More Fragile X Syndrome trials