- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04808505
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (ROSSELLA)
March 21, 2024 updated by: Amicus Therapeutics
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
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.
Study Overview
Status
Recruiting
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
36
Phase
- Phase 3
Expanded Access
Available outside the clinical trial.
See expanded access record.
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: For Site
- Phone Number: 215-921-7600
- Email: PompeSiteInfo@amicusrx.com
Study Contact Backup
- Name: For Patient
- Phone Number: 215-921-7600
- Email: patientadvocacy@amicusrx.com
Study Locations
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Garches, France, 92380
- Not yet recruiting
- Hôpital Raymond Poincaré, Neurologie et réanimation pédiatriques
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Gießen, Germany, 35392
- Not yet recruiting
- Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie
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Hochheim, Germany, 65239
- Not yet recruiting
- SphinCS GmbH
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Münster, Germany, 48149
- Not yet recruiting
- Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1
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Naples, Italy, 80131
- Not yet recruiting
- AOU Federico II
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Padova, Italy, 35128
- Not yet recruiting
- Azienda Ospedale Inherited Metabolic Disease Department
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Taipei, Taiwan, 100
- Recruiting
- National Taiwan University Hospital
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London, United Kingdom, WC1N3JH
- Not yet recruiting
- Great Ormond Street Hospital For Children NHS Foundation Trust
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Florida
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Gainesville, Florida, United States, 32610
- Recruiting
- University of Florida Clinical Research Center
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Georgia
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Atlanta, Georgia, United States, 30322
- Recruiting
- The Emory Clinic
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North Carolina
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Durham, North Carolina, United States, 27710
- Recruiting
- Duke University Early Phase Research Unit
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 17 years (Child)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
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):
1. 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.
Exclusion Criteria:
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)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects
Pediatric IOPD subjects 6 months to <18 years experiencing clinical decline
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Sterile lyophilized powder intravenous (IV) infusion
Other Names:
65 mg oral capsules
Other Names:
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Experimental: Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
Pediatric IOPD subjects <6 months
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Sterile lyophilized powder intravenous (IV) infusion
Other Names:
65 mg oral capsules
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Proportion of subjects with infusion-associated reactions (IARs)
Time Frame: 104 weeks
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104 weeks
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
July 18, 2023
Primary Completion (Estimated)
April 1, 2027
Study Completion (Estimated)
April 1, 2027
Study Registration Dates
First Submitted
March 10, 2021
First Submitted That Met QC Criteria
March 17, 2021
First Posted (Actual)
March 22, 2021
Study Record Updates
Last Update Posted (Actual)
March 25, 2024
Last Update Submitted That Met QC Criteria
March 21, 2024
Last Verified
August 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Carbohydrate Metabolism, Inborn Errors
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Lysosomal Storage Diseases, Nervous System
- Glycogen Storage Disease Type II
- Glycogen Storage Disease
- Hypoglycemic Agents
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Antiviral Agents
- Enzyme Inhibitors
- Anti-HIV Agents
- Anti-Retroviral Agents
- Glycoside Hydrolase Inhibitors
- Miglustat
Other Study ID Numbers
- ATB200-08
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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