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 | Terminated Phase 4 Results DMC

• 2 other identifiers: AGLU075102011-005595-42
• Study Type: interventional
• Target: 4
• Locations: 3 countries
• Sites: 17

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Cardiac Function at Week 52

  Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.

  Baseline, Week 52

Secondary Outcomes (3)

• Percentage of Participants With Estimated Probability of Survival

  Up to Week 52

• Number of Participants With Invasive Ventilator-Free Survival

  Up to Week 52

• Change From Baseline in Motor Development Status at Week 52

  Baseline, Week 52

Study Arms (2)

Alglucosidase Alfa 4000 L material (Non-US participants)

EXPERIMENTAL

Alglucosidase alfa 4000 L material for 52 weeks.

Biological: alglucosidase alfa

Alglucosidase Alfa 160 L material (US participants)

ACTIVE COMPARATOR

Alglucosidase alfa 160 L material for 52 weeks.

Biological: alglucosidase alfa

Interventions

alglucosidase alfa BIOLOGICAL

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

Also known as: Lumizyme

Alglucosidase Alfa 4000 L material (Non-US participants)

Eligibility Criteria

• Age: Up to 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (17)

Unknown Facility

Little Rock, Arkansas, United States

Location

Unknown Facility

Oakland, California, United States

Location

Unknown Facility

Gainsville, Florida, United States

Location

Unknown Facility

Decatur, Georgia, United States

Location

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

Cambridge, Massachusetts, United States

Location

Unknown Facility

Detroit, Michigan, United States

Location

Unknown Facility

Las Vegas, Nevada, United States

Location

Unknown Facility

New Brunswick, New Jersey, United States

Location

Unknown Facility

New York, New York, United States

Location

Unknown Facility

Durham, North Carolina, United States

Location

Unknown Facility

Cincinnati, Ohio, United States

Location

Unknown Facility

Fort Worth, Texas, United States

Location

Unknown Facility

Seattle, Washington, United States

Location

Unknown Facility

Geiben, Germany

Location

Unknown Facility

Mainz, Germany

Location

Unknown Facility

Taipei, Taiwan

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi

Contact -US@sanofi.com

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2012
• First Posted: May 14, 2012
• Study Start: August 1, 2012
• Primary Completion: December 1, 2014
• Study Completion: December 1, 2014
• Last Updated: January 18, 2016
• Results First Posted: January 18, 2016

Record last verified: 2015-12

Locations

TW (1); US (14); DE (2)