Clinical trial NCT05367960: An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome
Official title: An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Fragile X Syndrome
Lead sponsor: Tetra Discovery Partners
Sponsor class: INDUSTRY
First posted: 2022-05-10
Enrollment: 300
Sex: MALE
Minimum age (years): 9
Maximum age (years): 45
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a 4-year, open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in adult males) and Study BPN14770-CNS-204 (in adolescent males).

Detailed description: This is a 4-year open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in adult males) and Study BPN14770-CNS-204 (in adolescent males). The primary objective of this OLE is to assess the long-term safety and tolerability of BPN14770 in these subjects with fragile X syndrome (FXS) who were treated in one of those parent clinical trials.

Eligibility criteria: Inclusion Criteria:

1. Subject has completed the Week 13 visit, whether on treatment or discontinued from treatment, from one of two parent clinical trials with

   BPN14770:
   1. Study 204
   2. Study 301 Subjects who discontinued study treatment early due to AEs deemed related to study treatment by the investigator in one of the parent studies will NOT be eligible, regardless of whether the Week 13 visit was completed.
2. Subjects with a history of seizure disorder who are currently receiving treatment with antiepileptics must have remained seizure-free during the parent study. Any subject experiencing a seizure during the parent study is not eligible to continue into this long-term safety study.
3. Subject 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.
4. Subject has a parent, legal authorized guardian, or consistent caregiver.
5. Subject and caregiver are able to attend the clinic regularly and reliably.
6. If subject is his own legal guardian, he is able to understand and sign an informed consent form to participate in the study.
7. 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.
8. 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:

* 1. 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. Common conditions such as mild hypertension, etc. are allowed per the principal investigator's (PI) judgement as long as they are stable and controlled by medical therapy that is constant for at least 4 weeks before randomization.

  2. Renal impairment, defined as serum creatinine \>1.25×ULN per the latest available laboratory results from the parent study.

  3. Cirrhosis, unstable chronic liver disease or acute liver disease. Hepatic impairment, defined as ALT or AST elevation \> 2 × ULN per the latest available laboratory results from the parent study. Subjects with stable chronic hepatitis B on oral anti-viral therapy and subjects cured of chronic hepatitis C are allowed. Subjects with Gilbert syndrome are allowed.

  4. Clinically significant abnormalities, in the investigator's judgement, in safety laboratory tests, vital signs, or ECG, as measured at the final visit of the parent study.

  5. Substance abuse documented during the parent study.

  6. Significant hearing or visual impairment that may affect the subject's ability to complete the test procedures.

  7. Concurrent major psychiatric condition (eg, major depressive disorder, schizophrenia, or bipolar disorder) as confirmed by the investigator. Subjects with additional diagnosis of autism spectrum disorder or anxiety disorder will be allowed.

  8. Subject has active diseases that would interfere with participation, such as acquired immunodeficiency disorder, hepatitis C, hepatitis B, or tuberculosis.

  9. Subject has participated in another clinical trial within the 30 days preceding screening OTHER THAN one of the two studies required per Inclusion Criteria 1.

Primary outcomes:
measure: Safety and tolerability of BPN14770
timeFrame: 24 months
Secondary outcomes:
measure: National Institute of Health (NIH)- Toolbox Cognitive Battery (TCB)
timeFrame: 24 Months
measure: Numerical rating scale scores (NRS) scores
timeFrame: 24 Months
measure: Caregiver Global Impression of Improvement (CaGI-I) assessments:
timeFrame: 24 Months
measure: Clinical Global Impression Severity (CGI-S) assessments:
timeFrame: 24 Months
measure: Vineland-3 Adaptive Behavior Scale (Vineland-3)
timeFrame: 24 Months
measure: Verbal Knowledge Test from the Stanford-Binet (ed 5) IQ assessment
timeFrame: 24 Months
measure: Aberrant Behavior Checklist (ABC) scores
timeFrame: 24 Months
measure: Anxiety, Depression, and Mood Scale (ADAMS) scores
timeFrame: 24 Months
Other outcomes:
Locations:
city: Irvine
country: United States
facility: Amnova Clinical Research
state: California
city: Orange
country: United States
facility: Thompson Autism & Neurodevelopment Center - CHOC
state: California
city: Sacramento
country: United States
facility: MIND Institute UC Davis Medical Center
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Worcester
country: United States
facility: U Mass
state: Massachusetts
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Media
country: United States
facility: Suburban Research Associates
state: Pennsylvania
city: Strasburg
country: United States
facility: Clinic for Special Children
state: Pennsylvania
city: Greenville
country: United States
facility: Greenwood Genetic Center
state: South Carolina
city: Salt Lake City
country: United States
facility: University of Utah and Primary Children's Hospital
state: Utah
Study officials:
affiliation: Rush University Medical Center
name: Elizabeth Berry-Kravis, MD
role: PRINCIPAL_INVESTIGATOR
References: