A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
NCT ID: NCT04808505
Last Updated: 2026-01-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
36 participants
INTERVENTIONAL
2023-07-18
2027-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects
Pediatric IOPD subjects 6 months to \<18 years experiencing clinical decline
Cipaglucosidase alfa
Sterile lyophilized powder intravenous (IV) infusion
Miglustat
65 mg oral capsules
Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
Pediatric IOPD subjects \<6 months
Cipaglucosidase alfa
Sterile lyophilized powder intravenous (IV) infusion
Miglustat
65 mg oral capsules
Interventions
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Cipaglucosidase alfa
Sterile lyophilized powder intravenous (IV) infusion
Miglustat
65 mg oral capsules
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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)
17 Years
ALL
No
Sponsors
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Amicus Therapeutics
INDUSTRY
Responsible Party
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Locations
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University of Florida Clinical Research Center
Gainesville, Florida, United States
The Emory Clinic
Atlanta, Georgia, United States
Duke University Early Phase Research Unit
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
UPMC Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, United States
Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie
Giessen, , Germany
Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)
Heidelberg, , Germany
SphinCS GmbH
Höchheim, , Germany
Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1
Münster, , Germany
AOU Federico II
Naples, , Italy
Erasmus MC, Sophia Kinderziekenhuis
Rotterdam, , Netherlands
National Taiwan University Hospital
Taipei, , Taiwan
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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ATB200-08
Identifier Type: -
Identifier Source: org_study_id
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