A Randomized Study of BPN14770 in Male Adolescents (Aged 12 to < 18 Years) With Fragile X Syndrome
Sponsored by Tetra Discovery Partners
About this trial
Last updated 2 years ago
Study ID
Status
Type
Phase
Placebo
Accepting
Not accepting
Trial Timing
Ended a year ago
What is this trial about?
What are the participation requirements?
Inclusion Criteria
- 1. Patient is male adolescent aged 12 to < 18 years. 2. Patient has FXS with a molecular genetic confirmation of the full fragile X mental retardation-1 (FMR1) mutation (≥ 200 CGG repetitions).
Exclusion Criteria
1. Patient is male adolescent aged 12 to < 18 years.
2. Patient has FXS with a molecular genetic confirmation of the full fragile X mental retardation-1 (FMR1) mutation (≥ 200 CGG repetitions).
3. Current treatment with ≤ 3 prescribed psychotropic medications. Anti-epileptic medications are permitted and are not counted as psychotropic medications if they are used for treatment of seizures. Anti-epileptics for other indications, such as the treatment of mood disorders, count towards the limit of permitted medications.
4. Permitted concomitant psychotropic medications must be at a stable dose and dosing regimen for at least 4 weeks before screening and must remain stable during the period between screening and the commencement of study drug.
5. Anti-epileptic medications must be at a stable dose and dosing regimen for 12 weeks before screening and must remain stable during the period between screening and the commencement of study drug.
6. Patients with a history of seizure disorder who are currently receiving treatment with anti-epileptics must have been seizure-free for 3 months before screening or must be seizure-free for 2 years if not currently receiving anti-epileptics.
7. Behavioral and other non-pharmacological treatments/interventions must be stable for 4 weeks before screening and must remain stable during the period between screening and the commencement of study drug, 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 school program, due to school vacations, are allowed.
8. Patient must be willing to practice barrier methods of contraception while on study, if sexually active. Abstinence is also considered a reasonable form of birth control in this study population.
9. Patient has a parent(s), legal authorized guardian(s), or consistent caregiver(s).
10. Patient and caregiver are able to attend the clinic regularly and reliably.
11. Patient's parent(s), legal authorized guardian(s), or consistent caregiver(s) is able to understand and sign an informed consent form to participate in the study.
12. Patient must provide assent for participation in the study if the patient has the cognitive ability to provide assent.
13. To participate in the Part 1 PK only: patients must be able to swallow capsules. Patient Exclusion Criteria
10. Patient has active diseases that would interfere with participation, such as acquired immunodeficiency disorder, hepatitis C, hepatitis B, or tuberculosis.
11. Patient is an immediate family member of anyone employed by the sponsor, PI, or study staff.
12. Patient has weight < 60 lbs (27.2 kg) or a BMI greater than the 97th percentile for his age according to the Centers for Disease Control and Prevention (refer to Appendix 1). To participate in the Part 1 cohort receiving 50 mg dose, patient must weigh ≥ 75 lbs (34 kg).
13. Patient has participated in another clinical trial within the 30 days before screening. -