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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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04808505
Recruitment Status : Recruiting
First Posted : March 22, 2021
Last Update Posted : January 19, 2023
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

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 ...

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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
Estimated Study Start Date : April 2023
Estimated Primary Completion Date : April 2027
Estimated Study Completion Date : April 2027


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




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


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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. Prior to initiation of study drug subject must have documentation of IOPD genotype, CRIM status, and no or low measurable GAA enzyme activity
  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. Prior to initiation of study drug subject must have documentation of IOPD genotype, CRIM status, and no or low measurable GAA enzyme activity
  3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  4. Subject is ERT-naïve

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 hypersensitivity to any of the excipients in cipaglucosidase alfa, approved rhGAA, or miglustat
  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)

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: January 19, 2023
Last Verified: January 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