Ignite Creation Date:
2025-12-24 @ 9:37 PM
Ignite Modification Date:
2026-01-05 @ 1:45 AM
Study NCT ID:
NCT04271332
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-30
First Post:
2020-02-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion
Sponsor:
Clinical Research Associates, LLC
Organization:
Study Overview
Official Title:
An Exploratory, Randomized, Double-Blind, Placebo-Controlled and Open-label Extension Study of the Safety, Tolerability, and Efficacy of Arbaclofen in Subjects With 16p11.2 Deletion
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 receive either placebo or arbaclofen in a double-blind study design. If the subject completes all study requirements through Visit 4 (Close-out Visit), he/she may be eligible for an optional open-label study with arbaclofen.
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 study staff will be blinded to treatment assignment throughout the study. Both placebo and arbaclofen will be administered as orally disintegrating tablets. Dosing will be flexible, with subjects titrating up to the highest tolerated dose, with maximum permissible dose dependent on age. The treatment period is 16 weeks, after which subjects will taper off of study drug. Subjects will be required to attend multiple study visits, and to communicate with the study staff by phone multiple times throughout the study.
The primary efficacy assessment focuses on speech. Secondary and exploratory endpoints assess motor, memory, general cognitive, and other neuropsychological abilities, and brain electrophysiology. The safety evaluations include blood tests, physical exam, and assessment of adverse events.
Subjects who complete study participation through the end of the Withdrawal Period may be eligible to enroll in a subsequent open-label study. Dosing will be flexible, with subjects titrating up to the highest tolerated dose, with maximum permissible dose dependent on age. The treatment period is 16 weeks, after which subjects will taper off arbaclofen.
The primary efficacy assessment focuses on speech. Secondary and exploratory endpoints assess motor, memory, general cognitive, and other neuropsychological abilities, and brain electrophysiology. The safety evaluations include blood tests, physical exam, and assessment of adverse events.
Subjects who complete study participation through the end of the Withdrawal Period may be eligible to enroll in a subsequent open-label study. Dosing will be flexible, with subjects titrating up to the highest tolerated dose, with maximum permissible dose dependent on age. The treatment period is 16 weeks, after which subjects will taper off arbaclofen.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: