Safety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion
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|ClinicalTrials.gov Identifier: NCT04271332|
Recruitment Status : Suspended (Covid 19 Restrictions at sites)
First Posted : February 17, 2020
Last Update Posted : August 3, 2021
|Condition or disease||Intervention/treatment||Phase|
|16P11.2 Deletion Syndrome||Drug: Arbaclofen Drug: Placebo oral tablet||Phase 2|
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 12 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||An Exploratory, Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Tolerability, and Efficacy of Arbaclofen in Subjects With 16p11.2 Deletion|
|Estimated Study Start Date :||September 2021|
|Estimated Primary Completion Date :||March 2023|
|Estimated Study Completion Date :||May 2023|
Arbaclofen will be dosed flexibly, with maximum permissible dose depending on age.
Placebo Comparator: Placebo
The placebo tablet is manufactured to match arbaclofen in shape, size, color, and taste, and will be administered in the same manner as arbaclofen.
Drug: Placebo oral tablet
Manufactured to match Arbaclofen in size, shape, color and taste
Other Name: Sugar pill
- Goldman-Fristoe Test of Articulation, 3rd edition (GFTA-3), Sounds-in-Words [ Time Frame: 12 weeks ]The GFTA-3 is the most widely used, standardized test of articulation for children and adolescents.
- Wide Range Assessment of Memory and Learning - 2nd edition (WRAML2) [ Time Frame: 12 weeks ]The WRAML2 is a standardized test that measures memory functioning.
- Bruininks-Oseretsky Test - 2nd edition (BOT-2), Fine Motor Control and Body Coordination subtests [ Time Frame: 12 weeks ]The BOT-2 is an individually administered, comprehensive measure of gross and fine motor skills.
- Differential Ability Scale, 2nd edition (DAS-II) [ Time Frame: 12 weeks ]The DAS-II is an individually-administered clinical instrument for assessing the cognitive abilities that are important to learning.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04271332
|United States, Massachusetts|
|Boston Children's Hospital|
|Boston, Massachusetts, United States, 15031|
|United States, Texas|
|Texas Children's Hospital|
|Houston, Texas, United States, 77054|
|United States, Washington|
|University of Washington|
|Seattle, Washington, United States, 98915|
|Study Director:||Paul Wang, MD||Clinical Research Associates, LLC|