Clinical trial NCT01597596: A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Official title: A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Pompe Disease (Infantile-Onset)
Glycogen Storage Disease Type II (GSD II)
Glycogenosis 2
Acid Maltase Deficiency
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2012-05-14
Enrollment: 4
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.


Eligibility criteria: Inclusion Criteria:

* The participant's parent/legal guardian was willing and able to provide signed informed consent.
* The participant might be less than or equal to 12 months of age.
* The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue.
* The participant might be naïve to treatment with alglucosidase alfa.

Exclusion Criteria:

* The participant was cross-reactive immunologic material negative.
* The participant required invasive ventilator support at the time of enrollment.
* The participant had decompensated clinical heart failure.
* The participant had a major congenital abnormality, excluding cardiac hypertrophy.
* The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
* The participant was currently receiving any investigational product.
* The participant was participating in another clinical study.
* The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.

Primary outcomes:
measure: Change From Baseline in Cardiac Function at Week 52
timeFrame: Baseline, Week 52
Secondary outcomes:
measure: Percentage of Participants With Estimated Probability of Survival
timeFrame: Up to Week 52
measure: Number of Participants With Invasive Ventilator-Free Survival
timeFrame: Up to Week 52
measure: Change From Baseline in Motor Development Status at Week 52
timeFrame: Baseline, Week 52
Other outcomes:
Locations:
city: Little Rock
country: United States
state: Arkansas
city: Oakland
country: United States
state: California
city: Gainsville
country: United States
state: Florida
city: Decatur
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Cambridge
country: United States
state: Massachusetts
city: Detroit
country: United States
state: Michigan
city: Las Vegas
country: United States
state: Nevada
city: New Brunswick
country: United States
state: New Jersey
city: New York
country: United States
state: New York
city: Durham
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Fort Worth
country: United States
state: Texas
city: Seattle
country: United States
state: Washington
city: Geiben
country: Germany
city: Mainz
country: Germany
city: Taipei
country: Taiwan
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References: