An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

• Conditions: Pompe disease (acid alpha-glucosidase deficiency); MedDRA version: 17.1Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2015-000584-14-Outside-EU/EEA
• Lead Sponsor: Genzyme Corporation Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-04-15
• Last Update Posted: 10 July 2015

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 4

Inclusion Criteria

•The patient's legal guardian(s) must have provided written informed consent prior to any study-related procedures being performed
•The patient must have had a clinical diagnosis of Pompe disease as defined by documented acid alpha-glucosidase (GAA) deficiency (deficient endogenous GAA activity) in skin fibroblasts, muscle, or blood, or 2 GAA mutations. Consent was also sought from the biological parent(s) for parental GAA mutational analysis, but was not a requirement for study eligibility
•The patient must have not received Myozyme® or any rhGAA therapies prior to enrollment in the study
•The patient must be CRIM negative via Western Blot analysis performed on skin fibroblasts or via 2 known CRIM negative mutations (in which case CRIM status was to be confirmed by Western Blot analysis after enrollment)
•The patient's legal guardian(s) must have the ability to comply with the clinical protocol

Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•The patient had any medical condition that, in the opinion of the Investigator, could be exacerbated/precipitated by or interfere with the study regimen or assessments; such conditions may include but were not limited to human immunodeficiency virus, cancer, Hepatitis B, Hepatitis C, Cytomegalovirus, Herpes Simplex, John Cunningham (JC) virus, Parvovirus, or Epstein Barr virus or tuberculosis
•The patient had used any investigational product within 30 days prior to study enrollment
•The patient had or was required to have any live vaccination within 1 month prior to enrollment

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified