Ignite Creation Date:
2025-12-25 @ 3:16 AM
Ignite Modification Date:
2025-12-26 @ 1:55 AM
Study NCT ID:
NCT04808505
Status:
RECRUITING
Last Update Posted:
2025-11-17
First Post:
2021-03-10
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['France']}, 'interventionBrowseModule': {'meshes': [{'id': 'C059896', 'term': 'miglustat'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 36}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-07-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'nctId': 'NCT04327973', 'statusForNctId': 'AVAILABLE', 'hasExpandedAccess': True}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2027-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-14', 'studyFirstSubmitDate': '2021-03-10', 'studyFirstSubmitQcDate': '2021-03-17', 'lastUpdatePostDateStruct': {'date': '2025-11-17', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2021-03-22', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of subjects with infusion-associated reactions (IARs)', 'timeFrame': '104 weeks'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Glycogen Storage Disease Type II Infantile Onset']}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nCohort 1:\n\n1. Male or female subjects who are aged 6 months to \\< 18 years on Day 1\n2. Subject must have documentation of IOPD genotype\n3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis\n4. 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\n5. 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\n6. Subjects must have experienced a clinical decline on their current rhGAA dose and frequency\n\nCohort 2:\n\n1. Male or female subjects who are aged 0 to \\<6 months at Day 1\n2. Subject must have documentation of IOPD genotype\n3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis\n4. Subject is ERT-naïve\n\nLong-term Extension (Cohort 1 or Cohort 2):\n\n1\\. 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.\n\nExclusion Criteria:\n\nCohort 1 and Cohort 2, unless specified\n\n1. Subject requires invasive ventilation (eg, tracheostomy)\n2. 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)\n3. 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\n4. Subject has prior history of illness or condition known to affect motor function\n5. Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)'}, 'identificationModule': {'nctId': 'NCT04808505', 'acronym': 'ROSSELLA', 'briefTitle': 'A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18', 'organization': {'class': 'INDUSTRY', 'fullName': 'Amicus Therapeutics'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'ATB200-08'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects', 'description': 'Pediatric IOPD subjects 6 months to \\<18 years experiencing clinical decline', 'interventionNames': ['Biological: Cipaglucosidase alfa', 'Drug: Miglustat']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects', 'description': 'Pediatric IOPD subjects \\<6 months', 'interventionNames': ['Biological: Cipaglucosidase alfa', 'Drug: Miglustat']}], 'interventions': [{'name': 'Cipaglucosidase alfa', 'type': 'BIOLOGICAL', 'otherNames': ['ATB200'], 'description': 'Sterile lyophilized powder intravenous (IV) infusion', 'armGroupLabels': ['Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects', 'Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects']}, {'name': 'Miglustat', 'type': 'DRUG', 'otherNames': ['AT2221'], 'description': '65 mg oral capsules', 'armGroupLabels': ['Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects', 'Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of Florida Clinical Research Center', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'The Emory Clinic', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Duke University Early Phase Research Unit', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '54229', 'city': 'Cincinnati', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'UPMC Hospital of Pittsburgh', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of Utah, Clinical and Translational Sciences Institute', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '35392', 'city': 'Giessen', 'status': 'RECRUITING', 'country': 'Germany', 'facility': 'Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie', 'geoPoint': {'lat': 50.58727, 'lon': 8.67554}}, {'zip': '69120', 'city': 'Heidelberg', 'status': 'RECRUITING', 'country': 'Germany', 'facility': 'Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)', 'geoPoint': {'lat': 49.40768, 'lon': 8.69079}}, {'zip': '65239', 'city': 'Höchheim', 'status': 'RECRUITING', 'country': 'Germany', 'facility': 'SphinCS GmbH', 'geoPoint': {'lat': 50.36667, 'lon': 10.45}}, {'zip': '48149', 'city': 'Münster', 'status': 'RECRUITING', 'country': 'Germany', 'facility': 'Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1', 'geoPoint': {'lat': 51.96236, 'lon': 7.62571}}, {'zip': '80131', 'city': 'Naples', 'status': 'RECRUITING', 'country': 'Italy', 'facility': 'AOU Federico II', 'geoPoint': {'lat': 40.85216, 'lon': 14.26811}}, {'zip': '3015 GD', 'city': 'Rotterdam', 'status': 'RECRUITING', 'country': 'Netherlands', 'facility': 'Erasmus MC, Sophia Kinderziekenhuis', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'zip': '100', 'city': 'Taipei', 'status': 'WITHDRAWN', 'country': 'Taiwan', 'facility': 'National Taiwan University Hospital', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'zip': 'WC1N3JH', 'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital for Children NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'centralContacts': [{'name': 'For Site', 'role': 'CONTACT', 'email': 'patientadvocacy@amicusrx.com', 'phone': '609-662-2000'}, {'name': 'For Patient', 'role': 'CONTACT', 'email': 'patientadvocacy@amicusrx.com', 'phone': '609-662-2000'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Amicus Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}