A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects with Deletion- or Nondeletion-type Angelman Syndrome

Condition studied

Angelman Syndrome

Investigational medicinal product(s)

Eligibility

Primary endpoint

All Subprotocols - Primary Safety Endpoint: TEAEs and SAEs, frequency, severity, and relationship to investigational product, procedure, and premedication throughout the study., Subprotocol GTX-102-CL210A - Primary Efficacy Endpoint: Bayley-4 Cognitive raw score without caregiver input, change from Baseline at Day 338., Subprotocol GTX-102-CL210B - Primary Efficacy Endpoint: MDRI Net response at Day 338, with the following domains - assessments: Cognition - Bayley-4 Cognitive raw score, Communication - Bayley-4 Receptive Communication raw score, Behavior - ABC-C Hyperactivity/Noncompliance, Sleep - ASA Sleep rating, Motor Function - ASA Gross Motor rating., Subprotocol GTX-102-CL210C - Primary Efficacy Endpoint: MDRI Net response at Day 338, with the following domains - assessments: Expressive Communication - Vineland-3 Expressive Communication raw score, Receptive Communication – Vineland-3 Receptive Communication raw score, Behavior - ABC-C Irritability subscale score, Motor Function - ASA Gross Motor rating., Subprotocol GTX-102-CL210D - Primary Efficacy Endpoint: MDRI Net response at TxD 338, with the following domains - assessments: Cognition - Bayley-4 Cognitive raw score, Communication - Bayley-4 Receptive Communication raw score, Behavior - ABC-C Hyperactivity/Noncompliance, Sleep - ASA Sleep rating, Motor Function - ASA Gross Motor rating.

Endpoint detail

Subprotocol GTX-102-CL210A - Change from Baseline at Day 338 in: Bayley-4 Receptive Communication raw score, Bayley-4 Gross Motor raw score, Subprotocol GTX-102-CL210B - Change from Baseline at Day 338 in: Bayley-4 Cognitive raw score, Bayley-4 Receptive Communication raw score, Vineland-3 Receptive Communication raw score, Vineland-3 Expressive Communication raw score, ABC-C Hyperactivity/Noncompliance subscale score, ASA Sleep rating, ASA Gross Motor rating, Bayley-4 Gross Motor raw score., Subprotocol GTX-102-CL210C - Change from Baseline at Day 338 in: Vineland-3 Receptive Communication raw score, Vineland-3 Expressive Communication raw score, ABC-C Irritability subscale score, ASA Gross Motor rating., Subprotocol GTX-102-CL210D - Change from Baseline (or pretreatment) at TxD 338: Bayley-4 Cognitive raw score, Bayley-4 Receptive Communication raw score, Vineland-3 Receptive Communication raw score, Vineland-3 Expressive Communication raw score, ABC-C Hyperactivity/Noncompliance subscale score, ASA Sleep rating, ASA Gross Motor rating, Bayley-4 Gross Motor raw score.