A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
ROSSELLA
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
1 other identifier
interventional
36
6 countries
14
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jul 2023
Typical duration for phase_3
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 10, 2021
CompletedFirst Posted
Study publicly available on registry
March 22, 2021
CompletedStudy Start
First participant enrolled
July 18, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 1, 2027
January 22, 2026
January 1, 2026
4 years
March 10, 2021
January 20, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Proportion of subjects with infusion-associated reactions (IARs)
104 weeks
Study Arms (2)
Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects
EXPERIMENTALPediatric IOPD subjects 6 months to \<18 years experiencing clinical decline
Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
EXPERIMENTALPediatric IOPD subjects \<6 months
Interventions
Sterile lyophilized powder intravenous (IV) infusion
65 mg oral capsules
Eligibility Criteria
You may qualify if:
- Cohort 1:
- Male or female subjects who are aged 6 months to \< 18 years on Day 1
- Subject must have documentation of IOPD genotype
- Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- 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
- 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
- Subjects must have experienced a clinical decline on their current rhGAA dose and frequency
- Cohort 2:
- Male or female subjects who are aged 0 to \<6 months at Day 1
- Subject must have documentation of IOPD genotype
- Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- Subject is ERT-naïve
- Long-term Extension (Cohort 1 or Cohort 2):
- \. Subject must have, in the opinion of the investigator, benefited from therapy with cipaglucosidase alfa/miglustat during the 104-week primary treatment period with no significant safety concerns.
You may not qualify if:
- Cohort 1 and Cohort 2, unless specified
- Subject requires invasive ventilation (eg, tracheostomy)
- 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)
- Subject has a history of life-threatening IARs/hypersensitivity (eg, anaphylaxis and severe cutaneous reactions) to ERT (eg, alglucosidase alfa, cipaglucosidase alfa, miglustat) or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful
- Subject has prior history of illness or condition known to affect motor function
- Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (14)
University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
The Emory Clinic
Atlanta, Georgia, 30322, United States
Duke University Early Phase Research Unit
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 54229, United States
UPMC Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, 84108, United States
Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie
Giessen, 35392, Germany
Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)
Heidelberg, 69120, Germany
SphinCS GmbH
Höchheim, 65239, Germany
Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1
Münster, 48149, Germany
AOU Federico II
Naples, 80131, Italy
Erasmus MC, Sophia Kinderziekenhuis
Rotterdam, 3015 GD, Netherlands
National Taiwan University Hospital
Taipei, 100, Taiwan
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N3JH, United Kingdom
MeSH Terms
Interventions
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
- Expanded Access
- Yes
Study Record Dates
First Submitted
March 10, 2021
First Posted
March 22, 2021
Study Start
July 18, 2023
Primary Completion (Estimated)
July 1, 2027
Study Completion (Estimated)
July 1, 2027
Last Updated
January 22, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will not share