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Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

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ClinicalTrials.gov Identifier: NCT04808505
Recruitment Status : Recruiting
First Posted : March 22, 2021
Last Update Posted : August 12, 2021
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Brief Summary:
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with classic IOPD.

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type II Infantile Onset Biological: Cipaglucosidase alfa (ATB200) Drug: Miglustat (AT2221) Phase 3

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 22 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, Pharmacokinetics, Efficacy, 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
Estimated Study Start Date : August 2021
Estimated Primary Completion Date : February 2025
Estimated Study Completion Date : February 2025


Arm Intervention/treatment
Experimental: Cipaglucosidase Alfa/Miglustat -treated ERT-experienced subjects experiencing clinical decline Biological: Cipaglucosidase alfa (ATB200)
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules

Drug: Miglustat (AT2221)
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules

Experimental: Cipaglucosidase Alfa/Miglustat treated ERT-naïve subjects Biological: Cipaglucosidase alfa (ATB200)
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules

Drug: Miglustat (AT2221)
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules

No Intervention: Approved rhGAA-treated ERT-naive subjects



Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (TEAEs) from baseline [ Time Frame: 52 weeks ]
  2. Proportion of subjects with the composite endpoint at the end of Stage 1 for Cohort 2 and Cohort 3 [ Time Frame: 52 weeks ]


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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:

  1. Male or female subjects who are aged < 18 years at screening (For Cohort 1), 0 to <6 month (For Cohort 2 &3)
  2. Confirmed diagnosis of classic IOPD
  3. Subjects must have experienced a clinical decline based on their current approved rhGAA dose and frequency (For Cohort 1)

Exclusion Criteria:

  1. Subject received any investigational drug or any investigational biologic for Pompe disease within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening.
  2. Subject requires invasive ventilation (eg, tracheostomy) or respiratory assistance as defined in study protocol
  3. Subject has received any gene therapy at any time.
  4. Subject who is CRIM-negative and has not received prophylactic immunomodulation (For Cohort 1)
  5. Subject with high and sustained antibody titers as defined in study protocol (For Cohort 1)
  6. Subject has any prior history of certain condition or any intercurrent illness as defined in study protocol
  7. Subject has a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat
  8. Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (For Cohort 1)

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
Sponsors and Collaborators
Amicus Therapeutics
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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: August 12, 2021
Last Verified: August 2021

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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