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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 : May 6, 2024
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Study Description
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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.

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type II Infantile Onset Biological: Cipaglucosidase alfa Drug: Miglustat Phase 3

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : July 18, 2023
Estimated Primary Completion Date : April 2027
Estimated Study Completion Date : April 2027

Resource links provided by the National Library of Medicine

Drug Information available for: Miglustat

Arms and Interventions
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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
 Biological: Cipaglucosidase alfa
Sterile lyophilized powder intravenous (IV) infusion
Other Name: ATB200

Drug: Miglustat
65 mg oral capsules
Other Name: AT2221
Experimental: Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
Pediatric IOPD subjects <6 months
 Biological: Cipaglucosidase alfa
Sterile lyophilized powder intravenous (IV) infusion
Other Name: ATB200

Drug: Miglustat
65 mg oral capsules
Other Name: AT2221


Outcome Measures
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Primary Outcome Measures :
  1. Proportion of subjects with infusion-associated reactions (IARs) [ Time Frame: 104 weeks ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Cohort 1:

  1. Male or female subjects who are aged 6 months to < 18 years on Day 1
  2. Subject must have documentation of IOPD genotype
  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. Subject must have documentation of IOPD genotype
  3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  4. 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

  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 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
  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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04808505


Contacts
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Contact: For Site 215-921-7600 PompeSiteInfo@amicusrx.com
Contact: For Patient 215-921-7600 patientadvocacy@amicusrx.com

Locations
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United States, Florida
University of Florida Clinical Research Center Recruiting
Gainesville, Florida, United States, 32610
United States, Georgia
The Emory Clinic Recruiting
Atlanta, Georgia, United States, 30322
United States, North Carolina
Duke University Early Phase Research Unit Recruiting
Durham, North Carolina, United States, 27710
United States, Utah
University of Utah, Clinical and Translational Sciences Institute Recruiting
Salt Lake City, Utah, United States, 84108
France
Hôpital Raymond Poincaré, Neurologie et réanimation pédiatriques Not yet recruiting
Garches, France, 92380
Germany
Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie Not yet recruiting
Gießen, Germany, 35392
SphinCS GmbH Not yet recruiting
Hochheim, Germany, 65239
Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1 Not yet recruiting
Münster, Germany, 48149
Italy
AOU Federico II Not yet recruiting
Naples, Italy, 80131
Azienda Ospedale Inherited Metabolic Disease Department Not yet recruiting
Padova, Italy, 35128
Taiwan
National Taiwan University Hospital Withdrawn
Taipei, Taiwan, 100
United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust Not yet recruiting
London, United Kingdom, WC1N3JH
Sponsors and Collaborators
Amicus Therapeutics
More Information
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Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT04808505    
Other Study ID Numbers: ATB200-08
First Posted: March 22, 2021    Key Record Dates
Last Update Posted: May 6, 2024
Last Verified: August 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Lysosomal Storage Diseases
Miglustat
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Glycoside Hydrolase Inhibitors
Hypoglycemic Agents
Physiological Effects of Drugs