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Recruiting NCT04808505

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

Phase III Interventional Glycogen Storage Disease Type II Infantile Onset

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Cipaglucosidase alfa, Miglustat.
Who it may be relevant to
Registry conditions: Glycogen Storage Disease Type II Infantile Onset. Basic parameters: up to 17 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Germany, Italy, Netherlands, Taiwan +1
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

Interventions

  • Biological Cipaglucosidase alfa
    Sterile lyophilized powder intravenous (IV) infusion
  • Drug Miglustat
    65 mg oral capsules

Primary outcome measures

  • Proportion of subjects with infusion-associated reactions (IARs) [Time frame: 104 weeks]

Eligibility criteria

Inclusion criteria

Cohort 1:

  • Male or female subjects who are aged 6 months to < 18 years on Day 1
  • Subject must have documentation of IOPD genotype
  • Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  • 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
  • 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
  • Subjects must have experienced a clinical decline on their current rhGAA dose and frequency

Cohort 2:

  • Male or female subjects who are aged 0 to <6 months at Day 1
  • Subject must have documentation of IOPD genotype
  • Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
  • 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

  • Subject requires invasive ventilation (eg, tracheostomy)
  • 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)
  • 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
  • Subject has prior history of illness or condition known to affect motor function
  • Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 6 centers
  • University of Florida Clinical Research Center — Gainesville
  • The Emory Clinic — Atlanta
  • Duke University Early Phase Research Unit — Durham
  • Cincinnati Children's Hospital Medical Center — Cincinnati
  • UPMC Hospital of Pittsburgh — Pittsburgh
  • University of Utah, Clinical and Translational Sciences Institute — Salt Lake City
Germany · 4 centers
  • Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizi — Giessen
  • Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum — Heidelberg
  • SphinCS GmbH — Höchheim
  • Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus — Münster
Italy · 1 center
  • AOU Federico II — Naples
Netherlands · 1 center
  • Erasmus MC, Sophia Kinderziekenhuis — Rotterdam
Taiwan · 1 center
  • National Taiwan University Hospital — Taipei
United Kingdom · 1 center
  • Great Ormond Street Hospital for Children NHS Foundation Trust — London

Identifiers

NCT: NCT04808505 · ATB200-08

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗