A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Conditions

• Glycogen Storage Disease Type II Infantile Onset

Eligibility Criteria

Sex

ALL

Age

N/A - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Cipaglucosidase alfa — BIOLOGICAL
  Sterile lyophilized powder intravenous (IV) infusion
• Miglustat — DRUG
  65 mg oral capsules

Study Details

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.

Key Dates

Start date

Jul 18, 2023

Status verified

Jan 2026

Primary completion

Jul 31, 2027

Completion

Jul 31, 2027

Study Design

Enrollment

36 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects
  Pediatric IOPD subjects 6 months to \<18 years experiencing clinical decline
• Experimental: Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
  Pediatric IOPD subjects \<6 months

Primary Outcome Measure

Proportion of subjects with infusion-associated reactions (IARs) [ Time Frame: 104 weeks ]

Central Contacts

• For Site
  609-662-2000
  [email protected]
• For Patient
  609-662-2000
  [email protected]

Locations (6)

Find similar trials in Gainesville, FL