An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease
Phase 2
Completed
- Conditions
- Pompe disease10021605
- Registration Number
- NL-OMON55395
- Lead Sponsor
- Genzyme Europe BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
Inclusion Criteria
Patients with Pompe disease who previously completed a neoGAA study.
Exclusion Criteria
* The patient is concurrently participating in another clinical study using
investigational treatment.
* The patient, in the opinion of the Investigator, is unable to adhere to the
requirements of the study.
* The patient has clinically significant organic disease (with the exception of
symptoms relating to Pompe disease), including clinically significant
cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other
medical condition, serious intercurrent illness, or extenuating circumstance
that, in the opinion of the Investigator, precludes participation in the study
or potentially decreases survival.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>To eveluate the long term safety of neoGAA in Pompe disease patients</p><br>
- Secondary Outcome Measures
Name Time Method <p>To evaluate ECG, Immunogenicity assessments, PK, skeletal muscle glycogen<br /><br>content, skeletal muscle glycogen content, urinary Hex4, plasma analysis of<br /><br>circulating mRNA and micro RNA, serum analyses of skeletal muscle RNA<br /><br>expression</p><br>