A study to compare alglucosidase alfa manufactured at the 160 L scale and the 4000 L scale in patients with infantile-onset Pompe disease who have never been treated.

Phase 1

• 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: EUCTR2011-005595-42-DE
• Lead Sponsor: Genzyme Corporation, a Sanofi company

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-11-30
• Study Completion: 2014-12-01
• Last Update Posted: 4 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 4

Inclusion Criteria

1. The patient’s parent(s)/legal guardian(s) is willing and able to provide signed informed consent.
2. The patient must be less than or equal to 12 months of age at the time of the first study infusion.
3. The patient must have a documented GAA enzyme deficiency from blood, skin, or muscle tissue.
4. The patient must be naïve to treatment with alglucosidase alfa.
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
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

1. The patient is cross-reactive immunologic material (CRIM) negative.
2. The patient requires invasive ventilatory support at the time of enrollment.
3. The patient has decompensated clinical heart failure.
4. The patient has a major congenital abnormality, excluding cardiac hypertrophy.
5. The patient has a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
6. The patient is currently receiving any investigational product.
7. The patient is currently participating in another clinical study.
8. The patient and/or the patient’s parent(s)/legal guardian(s) is unable to adhere to the requirements of the study.

Study & Design

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