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Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

NCT01898364 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2023-03-23

No results posted yet for this study

Summary

Primary Objective:

To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

Secondary Objective:

To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

Conditions

  • Pompe Disease
  • Glycogen Storage Disease Type II (GSD II)
  • Acid Maltase Deficiency

Interventions

DRUG

GZ402666

Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-08-19
Primary Completion
2015-02-25
Completion
2015-02-25

Countries

  • United States
  • Belgium
  • Denmark
  • France
  • Germany
  • Netherlands
  • United Kingdom

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 NCT01898364 on ClinicalTrials.gov