Status:
COMPLETED
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Lead Sponsor:
University of Florida
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Pompe Disease
Eligibility:
All Genders
2-18 years
Phase:
PHASE1
PHASE2
Brief Summary
Pompe disease is an inherited condition of acid alpha-glucosidase (GAA) deficiency resulting in lysosomal accumulation of glycogen in all tissues. Glycogen accumulation leads to muscle dysfunction and...
Detailed Description
The goal of the current study is to evaluate an experimental gene transfer procedure in which normal copies of the GAA gene are inserted into cells. In this study, a modified virus, adeno-associated v...
Eligibility Criteria
Inclusion
- Male or female subjects 2-18 years of age.
- Have a diagnosis of Pompe, as defined by protein assay, DNA sequence of the acid alpha-glucosidase gene and clinical symptoms of the disease.
- Using assisted ventilation at baseline. Mechanical Ventilation is defined as any use of ventilation support, (including but not limited to BiPAP, CPAP), a minimum of 1 hours per day.
- Willing to discontinue aspirin, aspirin-containing products and other drugs that may alter platelet function, 7 days prior to dosing, resuming 24 hours after the dose has been administered.
Exclusion
- The subject must not:
- Have required acute, as distinguished from long-term, maintenance or chronic suppressive, oral or intravenous antibiotic therapy for a respiratory infection within 15 days prior to baseline screening.
- Have required oral or systemic corticosteroids within the last 15 days prior to baseline screening.
- Have a platelet count less than 75,000/ cu mm.
- Have an INR greater than 1.3.
- Serological evidence of hepatitis B, hepatitis C, or HIV positive.
- Be currently or within the past 30 days participating in any other research protocol involving investigational agents or therapies.
- Have received gene transfer agents within the past 6 months.
- Have history of platelet dysfunction, evidence of abnormal platelet function at screening or history of recent use of drugs that may alter platelet function which the subject is unable/unwilling to discontinue for study agent administration.
- Have any other concurrent condition which, in the opinion of the investigator, would make the subject unsuitable for the study.
Key Trial Info
Start Date :
September 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2015
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT00976352
Start Date
September 1 2010
End Date
December 1 2015
Last Update
September 14 2018
Active Locations (1)
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1
Shands at the University of Florida
Gainesville, Florida, United States, 32610