Status:
ACTIVE_NOT_RECRUITING
Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion
Lead Sponsor:
Clinical Research Associates, LLC
Conditions:
16P11.2 Deletion Syndrome
Eligibility:
All Genders
5-17 years
Phase:
PHASE2
Brief Summary
This Phase 2 study examines the safety, tolerability, and efficacy of arbaclofen in pediatric subjects with 16p11.2 deletion. Male or female subjects aged 5 to 17 years of age will be randomized to re...
Detailed Description
This study will enroll male or female subjects, aged 5 to 17 years, who have the 16p11.2 bp4-bp5 deletion. Subjects will be randomized to treatment with either placebo or arbaclofen. Subjects and stud...
Eligibility Criteria
Inclusion
- To be eligible to participate in this study, all of the following criteria must be met:
- Diagnosis of 16p11.2 BP4-BP5 deletion.
- Male or female subjects, 5 through 17 years of age, at Screening.
- Neurodevelopmental disability requiring current educational or other therapeutic support.
- Any educational, behavioral, other therapeutic, and/or dietary interventions must have been continuous during the 2 months prior to Screening (with the exception of the anti-epileptic drug (AED) 6-month requirement in Inclusion Criterion #5). Subjects and their parent/caregiver/legally authorized representative (LAR) may not electively initiate new, or modify ongoing, interventions for the duration of the study. Typical school vacations are not considered modifications of stable programming.
- Subjects with a history of seizure disorder must have been seizure-free and on a stable antiepileptic therapy regimen for 6 months OR must have been seizure-free for the 3 years prior to Screening if not currently receiving antiepileptics. If currently receiving treatment with antiepileptics that are typically monitored by serum concentration, serum concentrations of the antiepileptic drugs must be tested and confirmed to be within the therapeutic range at Screening.
- All medication regimens must be stable for 30 days prior to Screening.
- Prior to the conduct of any study-specific procedures, the subject must provide assent to participate in the study (if developmentally appropriate), and the parent/caregiver/LAR must provide written informed consent. If the caregiver attending the clinic visits is not the parent or LAR, written consent must also be obtained from the parent or LAR for the subject's participation in the study.
- The subject's parent/caregiver/LAR must be able to speak and understand English sufficiently to understand the nature of the study and to allow for the completion of all study assessments. The parent/caregiver/LAR should be capable of providing reliable information about the subject's condition, agree to oversee the administration of the study drug, and accompany the subject to all clinic visits. The same parent/caregiver/LAR should accompany the subject to each visit.
- Negative pregnancy test for females who are 9 years of age or older. Both male and female subjects who are sexually active must agree to consistently use an accepted form of contraception (i.e., surgical sterilization, intrauterine contraceptive device, subcutaneous implant, oral contraceptive, or diaphragm or condom in combination with contraceptive cream/jelly, or abstinence) throughout the trial and for 30 days (females) or 90 days (males) following last study drug administration.
Exclusion
- Subjects are not permitted to enroll in the study if any of the following criteria are met:
- Subjects with additional known genetic disorder besides 16p11.2 BP4-BP5 deletion.
- Subjects receiving remote or hybrid schooling at Screening.
- Subjects with an additional neurologic or psychiatric condition that might confound performance on assessments measures, e.g., significant impairment in hearing or vision, severe motor impairment (e.g., non-ambulatory) from cerebral palsy, birth injury, or other injury, or cleft lip or palate (including submucous cleft).
- Subjects with any seizures within the previous 6 months; subjects who are not currently receiving antiepileptics AND have had one or more seizures during the 3 years prior to Screening; and subjects who are shown to have non-therapeutic AED levels at Screening.
- Subjects with any medical or psychiatric condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. This includes, but is not limited to substance use disorders, impairment of renal function, evidence or history of malignancy or any significant hematological, endocrine, cardiovascular, respiratory, hepatic, or gastrointestinal disease.
- Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
- Subjects who are currently treated or have been treated with racemic baclofen in the last 30 days.
- Subjects currently treated with antipsychotic medication(s).
- Subjects currently treated with more than 2 psychoactive medications, including antiepileptics used as an anti-seizure treatment, but not including sleep aids used on an as-needed basis.
- Subjects currently treated with drugs having anxiolytic properties, including but not limited to: buspirone and beta-blockers. Benzodiazepines administered on a regular schedule (more than 3x/week) are not permitted. Use of antidepressants may be permitted with approval of the Medical Monitor. Other prohibited and restricted medications are shown in Appendix A.
- Subjects currently treated with vigabatrin, tiagabine, or riluzole.
- Subjects taking another investigational drug currently or within the last 30 days.
- Subjects who are not able or willing to take oral disintegrating tablets.
- Subjects who have a history of hypersensitivity to racemic baclofen.
- Subjects who, in the Investigator's opinion, might not be suitable for the study.
Key Trial Info
Start Date :
September 1 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 23 2025
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT04271332
Start Date
September 1 2022
End Date
March 23 2025
Last Update
May 30 2024
Active Locations (4)
Enter a location and click search to find clinical trials sorted by distance.
1
Boston Children's Hospital
Boston, Massachusetts, United States, 15031
2
New York State Psychiatric Institute (NYSPI)
New York, New York, United States, 10032
3
Texas Children's Hospital
Houston, Texas, United States, 77054
4
University of Washington
Seattle, Washington, United States, 98915