Basic Information

Gene symbol ATP2A2 Synonyms ATP2B, DAR, DD, SERCA2 Type of gene protein-coding
Description ATPase sarcoplasmic/endoplasmic reticulum Ca2+ transporting 2

GTO ID GTC0187
Trial ID NCT01966887
Disease Heart Failure
Altered gene SERCA2
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment AAV1-CMV-Serca2a
PhasePhase2
Recruitment statusTerminated
TitlePhase 2 Study of SERCA2a Gene Transfer in Patients With Severe Heart Failure
Year2013
CountryFrance
Company sponsorAssistance Publique - Hôpitaux de Paris
Other ID(s)P081252
Vector information
Vectoradeno-associated virus
ConstructAAV1-CMV-SERCA2a
Vector typeadeno-associated virus serotype 1 (AAV1) vector
Transgene/Inserted genesarcoplasmic reticulum calcium ATPase gene
Regulatory elementa cytomegalovirus (CMV) promoter

Clinical Result

Cohort 1
Administration route intracoronary injection
Dosage 1E13 DRP (DNase-resistant particles)
Pts 5
Age Adult, Older_Adult
Outcome At 6 months, LVESV was increased compared with baseline: median (interquartile range) in AAV1/SERCA2a = 13 (13; 14) mL, P = 0.74
References PMID: 28393439

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