Immune Tolerance Induction Study

• 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: EUCTR2015-000583-34-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: 9

Inclusion Criteria

•The patient (and/or patient's legal guardian if patient is < 18years) must provide written informed consent prior to any study-related procedures that are performed;
•The patient must have a confirmed diagnosis of Pompe disease defined as a documented acid a-glucosidase (GAA) enzyme deficiency from any tissue source or 2 GAA gene mutations;
•The patient (and/or legal guardian) must have ability to comply with clinical protocol;
•If the patient is Cross-reacting immunologic material (CRIM)-positive, he/she must have received at least 6 consecutive months of Myozyme infusions (20mg/kg qow)
•If the patient is CRIM-negative, he/she must have received at least 1 Myozyme infusion prior to enrollment
•Regimen A only: The patient exhibits clinical decline; The patient has persistent high anti-recombinant human acid a-glucosidase (anti-rhGAA) antibody titers and/or tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme;
•Regimen B only: The patient is CRIM-negative AND The patient does not exhibit clinical decline; OR ALL OF THE FOLLOWING: The patient is CRIM-negative AND The patient exhibits clinical decline AND The patient does NOT exhibit high anti-rhGAA antibody titers and has NOT tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme.

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 has a clinical condition unrelated to Pompe disease that would interfere with program assessments;
•The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C;
•The patient is at risk of reactivation or has positive serology suggestive of active infection for cytomegalovirus, Herpes simplex, JC virus, Parvovirus or Epstein Barr virus;
•The patient is at risk of reactivation of tuberculosis or has regular contact with individuals who are being actively treated for tuberculosis;
•The patient has low serum albumin;
•The patient has a major congenital abnormality;
•The patient has used any investigational product (other than alglucosidase alfa) within 30 days prior to study enrollment;
•The patient is pregnant or lactating;
•The patient has had or is required to have any live vaccination within one month prior to enrollment.

Study & Design

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