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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT00976352 |
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Date of registration:
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13/07/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
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Scientific title:
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Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients With Pompe Disease |
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Date of first enrolment:
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September 2010 |
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Target sample size:
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9 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00976352 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Barry J Byrne, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Florida |
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Name:
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Shelley Collins, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Florida |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- 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 Criteria:
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.
Age minimum:
2 Years
Age maximum:
18 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pompe Disease
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Intervention(s)
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Drug: rAAV1-CMV-GAA (study agent) Administration
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Other: RMST
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Primary Outcome(s)
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Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration.
[Time Frame: Change from baseline to 365 post study agent administration.]
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Secondary Outcome(s)
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Maximal Inspiratory Pressure
[Time Frame: Baseline and 365 post study agent administration]
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Evaluation of Ventilatory Performance Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training (RMST) Compared to RMST Alone.
[Time Frame: Screening, Baseline, and 365 post study agent administration.]
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Evaluation of Tidal Volume Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training, Compared to Respiratory Muscle Strength Training Alone.
[Time Frame: Screening, Baseline, and Day 365 post study agent administration]
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Secondary ID(s)
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PGTC PD-AAV004-N
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P01HL059412-06
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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