Key result
Intramuscular diaphragm delivery of rAAV1-CMV-GAA gene vector is the subject of a proposed phase I/II open-label trial in patients with Pompe disease, with no results reported in the abstract.
Why the study?
Does rAAV1-CMV-GAA gene vector improve safety and muscle function in human subjects with Pompe disease?
Does rAAV1-CMV-GAA gene vector improve safety and muscle function in human subjects with Pompe disease?
This proposed phase I/II trial aims to evaluate the safety of diaphragm delivery of a recombinant AAV gene vector in patients with Pompe disease.
Proposed AAV1-CMV-GAA diaphragm delivery trial in Pompe; leaves open human safety and efficacy.
A recombinant virus vector constructed from adeno-asso-ciated virus (AAV) has been engineered to carry the humanacid alpha-glucosidase (hGAA) gene expressed from thecytomegalovirus (CMV) enhancer/promoter. The constructhas been shown to produce hGAA in animal models ofPompe disease, which directly matches the proposed humanstudies. The proposed clinical trial is an open label, phase I/II study administering rAAV1-CMV-GAAgene vector in-tramuscularly into the diaphragms of human subjects withPompe disease. Safety parameters will be measurement ofchange in: serum chemistries and hematology, urinalysis,muscle function testing, whole blood assay for vector ge-nomes, immunologic response to GAA and AAV, as well asreported subject symptom history.
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Byrne et al. (2014) studied Pompe disease. rAAV1-CMV-GAA gene vector was evaluated on Safety parameters including serum chemistries, hematology, urinalysis, muscle function testing, whole blood assay for vector genomes, immunologic response to GAA and AAV, and subject symptom history. Intramuscular diaphragm delivery of rAAV1-CMV-GAA gene vector is the subject of a proposed phase I/II open-label trial in patients with Pompe disease, with no results reported in the abstract.
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