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NCT05358886 | Recruiting | Fragile X Syndrome

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

Tetra Discovery Partners

Brief Summary:

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

Condition or disease

Fragile X Syndrome

Intervention/treatment

BPN14770/ damping

Placebo

Phase

Phase 3

Study Type : Interventional
Estimated Enrollment : 150 participants
Masking : Quadruple
Primary Purpose : Treatment
Official Title : A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome
Actual Study Start Date : November 1, 2022
Estimated Primary Completion Date : February 2024
Estimated Study Completion Date : July 2024
Arm Intervention/treatment

Active Comparator: Study Drug

25mg BID BPN14770

Other: BPN14770/ buffering

Placebo Comparator: Placebo

Placebo

Drug: Placebo
Ages Eligible for Study: 18 Years to 45 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No
Criteria
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.
  • Behavioral and other non-pharmacological treatments/interventions must be stable for 4 weeks prior to screening and must remain stable during the period between screening and first dose of study treatment and throughout the study. Minor changes in hours or times of therapy that are not considered clinically significant will not be exclusionary. Changes in therapies provided through a program (eg, due to a vacation) are allowed.
  • 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.
  • Subject has a parent, legal authorized guardian, or consistent caregiver.
  • Subject and caregiver are able to attend the clinic regularly and reliably.
  • If subject is his own legal guardian, he is able to understand and sign informed consent to participate in the study.
  • 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.
  • 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.
Exclusion Criteria
  • 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.
  • a. Common conditions such as mild hypertension, etc. are allowed per the principal investigator's judgement as long as they are stable and controlled by medical therapy that is constant for at least 4 weeks before randomization.
  • Renal impairment, defined as serum creatinine > 1.25 × ULN at screening
  • Hepatic impairment, defined as ALT or AST elevation > 2 × ULN at screening. Note: LFTs may be repeated after 1 week to evaluate return to acceptable limits; if LFTs remain elevated, the subject is ineligible to participate.
  • Clinically significant abnormalities, in the investigator's judgement, in safety laboratory tests, vital signs, or ECG, as measured during screening.
  • History of substance abuse within the past year, according to investigator assessment.
  • Positive COVID-19 test during screening.
  • Significant hearing or visual impairment that may affect the subject's ability to complete the test procedures.
  • Concurrent major psychiatric condition (eg, major depressive disorder, schizophrenia, or bipolar disorder) as diagnosed by the investigator. Subjects with the additional diagnosis of autism spectrum disorder or anxiety disorder will be allowed.
  • Subject has active diseases that would interfere with participation, such as acquired immunodeficiency disorder, hepatitis C, hepatitis B, or tuberculosis.
  • Subject is planning to commence psychotherapy or cognitive behavior therapy during the period of the study or had begun psychotherapy or cognitive behavior therapy within 4 weeks prior to screening.
  • Subject is an immediate family member of anyone employed by the sponsor, investigator, or study staff.
  • Subject has a body mass index of less than 18 kg/m2 or greater than 36 kg/m2.
  • Subject has participated in another clinical trial within the 30 days preceding Screening
A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

Location Details

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

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Recruiting

United States, California

Amnova Clinical Research

Irvine, California, United States, 92604

Recruiting

United States, California

CHOC Thompson Autism Center

Orange, California, United States, 92868

Recruiting

United States, California

UC Davis Health System

Sacramento, California, United States, 95817

Recruiting

United States, Colorado

Children's Hospital Colorado

Denver, Colorado, United States, 80045

Not yet recruiting

United States, Georgia

Emory University School of Medicine

Atlanta, Georgia, United States, 30307

Recruiting

United States, Illinois

Rush University Medical Center

Chicago, Illinois, United States, 60612

Not yet recruiting

United States, Kansas

University of Kansas Medical Center

Kansas City, Kansas, United States, 66160

Recruiting

United States, Maryland

Kennedy Krieger Institute

Baltimore, Maryland, United States, 21205

Recruiting

United States, Massachusetts

University of Massachusetts Medical School

Worcester, Massachusetts, United States, 01655

Recruiting

United States, New York

Icahn School of Medicine at Mount Sinai Hospital

New York, New York, United States, 10029

Recruiting

United States, Ohio

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Recruiting

United States, Pennsylvania

Suburban Research Associates

Media, Pennsylvania, United States, 19063

Not yet recruiting

United States, Pennsylvania

Clinic for Special Children

Strasburg, Pennsylvania, United States, 17579