A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects with Angelman Syndrome

Conditions

Angelman Syndrome

Brief summary

Change from Baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338

Detailed description

Net Response in MDRI at Day 338, Change from Baseline in ABC-C Hyperactivity/Noncompliance Subscale Score at Day 338, Change from Baseline at Day 338 in Bayley-4 Receptive Communication raw score, Change from Baseline in ASA Sleep Rating Raw Score at Day 338, Number of Participants with Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs), Severity of AEs and Relationship to Investigational Drug, Procedure, and Premedication, Changes from Baseline in Vineland Adaptive Behavior Scales-3 (Vineland-3) Receptive Communication raw score at Day 338, Changes from Baseline in Vineland-3 Expressive Communication raw score at Day 338, Changes from Baseline in Bayley-4 Gross Motor raw score at Day 338, Changes from Baseline in ASA Gross Motor rating at day 338

Related papers

No related papers found yet.

Interventions

DRUGGTX-102

DRUGGTX/UX Diluent and Flush Solution

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Change from Baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338	—

Secondary

Measure	Time frame
Net Response in MDRI at Day 338, Change from Baseline in ABC-C Hyperactivity/Noncompliance Subscale Score at Day 338, Change from Baseline at Day 338 in Bayley-4 Receptive Communication raw score, Change from Baseline in ASA Sleep Rating Raw Score at Day 338, Number of Participants with Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs), Severity of AEs and Relationship to Investigational Drug, Procedure, and Premedication, Changes from Baseline in Vineland Adaptive Behavior Scales-3 (Vineland-3) Receptive Communication raw score at Day 338, Changes from Baseline in Vineland-3 Expressive Communication raw score at Day 338, Changes from Baseline in Bayley-4 Gross Motor raw score at Day 338, Changes from Baseline in ASA Gross Motor rating at day 338	—

Measure

Time frame

Net Response in MDRI at Day 338, Change from Baseline in ABC-C Hyperactivity/Noncompliance Subscale Score at Day 338, Change from Baseline at Day 338 in Bayley-4 Receptive Communication raw score, Change from Baseline in ASA Sleep Rating Raw Score at Day 338, Number of Participants with Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs), Severity of AEs and Relationship to Investigational Drug, Procedure, and Premedication, Changes from Baseline in Vineland Adaptive Behavior Scales-3 (Vineland-3) Receptive Communication raw score at Day 338, Changes from Baseline in Vineland-3 Expressive Communication raw score at Day 338, Changes from Baseline in Bayley-4 Gross Motor raw score at Day 338, Changes from Baseline in ASA Gross Motor rating at day 338

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Countries

Germany, Netherlands, Poland, Spain

Outcome results

None listed