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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00250939
Other study ID # AGLU02804
Secondary ID
Status Completed
Phase Phase 2
First received November 8, 2005
Last updated February 4, 2014
Start date February 2005
Est. completion date November 2006

Study information

Verified date February 2014
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority European Union: European Medicines Agency
Study type Interventional

Clinical Trial Summary

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective is to evaluate the safety, pharmacokinetics (PK) and efficacy of Myozyme treatment.


Recruitment information / eligibility

Status Completed
Enrollment 5
Est. completion date November 2006
Est. primary completion date July 2006
Accepts healthy volunteers No
Gender Both
Age group 5 Years to 18 Years
Eligibility Inclusion Criteria:

- patient's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related procedures; patient's signature required if patient understands informed consent

- patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity or GAA gene mutations

- patient must have demonstrable muscle weakness

- patient must be greater than or equal to five years of age and younger than eighteen years of age

- patient must be able to provide 3 reproducible FVC tests in sitting position during screening

- patient must perform muscle function testing

- patient must ambulate 10 meters (assistive devices permitted)

- patient and legal guardian must comply with the clinical protocol

Exclusion Criteria:

- patient requires the use of invasive ventilatory support

- patient requires the use of noninvasive ventilatory support while awake and in an upright position

- patient has received enzyme replacement therapy with GAA from any source

- patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another clinical or observational study

- patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities

- Female patients pregnant, lactating or unwilling to practice birth control methods during study

- Male patients unwilling to use barrier contraceptives during study

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Intervention

Biological:
Myozyme
20 mg/kg qow

Locations

Country Name City State
Netherlands Sophia Kinderziekenhuis, Erasmus MC Rotterdam

Sponsors (1)

Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

Netherlands, 

Outcome

Type Measure Description Time frame Safety issue
Primary safety and PK profile rhGAA 74 weeks No
Primary FVC 74 weeks No
Primary MMT 74 weeks No
Primary Effect of treatment on muscle function 74 weeks No
See also
  Status Clinical Trial Phase
Completed NCT00158600 - A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease Phase 3
Recruiting NCT04174105 - Gene Transfer Study in Patients With Late Onset Pompe Disease Phase 1/Phase 2
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT03285126 - Pompe Gene Therapy- Screening for Eligibility
Recruiting NCT05083806 - MSOT in Pompe Disease N/A
Recruiting NCT05272969 - Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
Active, not recruiting NCT03694561 - Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Completed NCT00455195 - Late-Onset Treatment Study Extension Protocol Phase 4
Completed NCT00731081 - Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme® N/A
Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Completed NCT01288027 - Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa Phase 4
Recruiting NCT00567073 - Pompe Pregnancy Sub-Registry
Completed NCT03893240 - Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease N/A
Withdrawn NCT04094948 - Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Phase 2
Recruiting NCT03911505 - ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Phase 3
Recruiting NCT06150820 - A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease N/A
Completed NCT03729362 - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Phase 3
Not yet recruiting NCT06178432 - Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) Early Phase 1
Completed NCT00268944 - Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support Phase 3
Active, not recruiting NCT04138277 - A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD Phase 3