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A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

NCT01597596 · Status: TERMINATED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2016-01-18

Study results available
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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.

Conditions

  • Pompe Disease (Infantile-Onset)
  • Glycogen Storage Disease Type II (GSD II)
  • Glycogenosis 2
  • Acid Maltase Deficiency

Interventions

BIOLOGICAL

alglucosidase alfa

Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW)

BIOLOGICAL

alglucosidase alfa

IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW.

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-08-31
Primary Completion
2014-12-31
Completion
2014-12-31

Countries

  • United States
  • Germany
  • Taiwan

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01597596 on ClinicalTrials.gov