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

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ClinicalTrials.gov Identifier: NCT04808505
Recruitment Status : Recruiting
First Posted : March 22, 2021
Last Update Posted : January 19, 2023
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Tracking Information
First Submitted Date  ICMJE March 10, 2021
First Posted Date  ICMJE March 22, 2021
Last Update Posted Date January 19, 2023
Estimated Study Start Date  ICMJE April 2023
Estimated Primary Completion Date April 2027   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 22, 2022)		 Proportion of subjects with infusion-associated reactions (IARs) [ Time Frame: 104 weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: March 17, 2021)
  • Incidence of treatment-emergent adverse events (TEAEs) from baseline [ Time Frame: 52 weeks ]
  • Proportion of subjects with the composite endpoint at the end of Stage 1 for Cohort 2 and Cohort 3 [ Time Frame: 52 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Official Title  ICMJE An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Glycogen Storage Disease Type II Infantile Onset
Intervention  ICMJE
  • Biological: Cipaglucosidase alfa
    Sterile lyophilized powder intravenous (IV) infusion
    Other Name: ATB200
  • Drug: Miglustat
    65 mg oral capsules
    Other Name: AT2221
Study Arms  ICMJE
  • Experimental: Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects
    Pediatric IOPD subjects 6 months to <18 years experiencing clinical decline
    Interventions:
    • Biological: Cipaglucosidase alfa
    • Drug: Miglustat
  • Experimental: Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
    Pediatric IOPD subjects <6 months
    Interventions:
    • Biological: Cipaglucosidase alfa
    • Drug: Miglustat
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: November 22, 2022)		 36
Original Estimated Enrollment  ICMJE
 (submitted: March 17, 2021)		 22
Estimated Study Completion Date  ICMJE April 2027
Estimated Primary Completion Date April 2027   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Cohort 1:

  1. Male or female subjects who are aged 6 months to < 18 years on Day 1
  2. Prior to initiation of study drug subject must have documentation of IOPD genotype, CRIM status, and no or low measurable GAA enzyme activity
  3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  4. Subject must have received ERT for at least 6 months immediately before enrollment. For subjects whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months before enrollment
  5. Subjects aged ≥ 12 to < 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged ≥ 5 to < 12 years must perform one valid 6MWT (≥ 40 meters) at screening; Subjects aged 18 months to < 5 years must be ambulatory and assessed to be likely to be able to perform 6MWT (≥ 40 meters) when they turn 5 years old
  6. Subjects must have experienced a clinical decline on their current rhGAA dose and frequency

Cohort 2:

  1. Male or female subjects who are aged 0 to <6 months at Day 1
  2. Prior to initiation of study drug subject must have documentation of IOPD genotype, CRIM status, and no or low measurable GAA enzyme activity
  3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  4. Subject is ERT-naïve

Exclusion Criteria:

Cohort 1 and Cohort 2, unless specified

  1. Subject requires invasive ventilation (eg, tracheostomy)
  2. Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving prophylactic immunomodulation (Cohort 2)
  3. Subject has a hypersensitivity to any of the excipients in cipaglucosidase alfa, approved rhGAA, or miglustat
  4. Subject has prior history of illness or condition known to affect motor function
  5. Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: For Site 215-921-7600 PompeSiteInfo@amicusrx.com
Contact: For Patient 215-921-7600 patientadvocacy@amicusrx.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04808505
Other Study ID Numbers  ICMJE ATB200-08
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Amicus Therapeutics
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Amicus Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Amicus Therapeutics
Verification Date January 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP