Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): A Phase 2 Trial of Intracoronary Gene Therapy of Sarcoplasmic Reticulum Ca 2+ -ATPase in Patients With Advanced Heart Failure
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Background—: Adeno-associated virus type 1/sarcoplasmic reticulum Ca 2+ -ATPase was assessed in a randomized, double-blind, placebo-controlled, phase 2 study in patients with advanced heart failure. Methods and Results—: Thirty-nine patients received intracoronary adeno-associated virus type 1/sarcoplasmic reticulum Ca 2+ -ATPase or placebo. Seven efficacy parameters were assessed in 4 domains: symptoms (New York Heart Association class, Minnesota Living With Heart Failure Questionnaire), functional status (6-minute walk test, peak maximum oxygen consumption), biomarker (N-terminal prohormone brain natriuretic peptide), and left ventricular function/remodeling (left ventricular ejection fraction, left ventricular end-systolic volume), plus clinical outcomes. The primary end point success criteria were prospectively defined as achieving efficacy at 6 months in the group-level (concordant improvement in 7 efficacy parameters and no clinically significant worsening in any parameter), individual-level (total score for predefined clinically meaningful changes in 7 efficacy parameters), or outcome end points (cardiovascular hospitalizations and time to terminal events). Efficacy in 1 analysis had to be associated with at least a positive trend in the other 2 analyses. This combination of requirements resulted in a probability of success by chance alone of 2.7%. The high-dose group versus placebo met the prespecified criteria for success at the group-level, individual-level, and outcome analyses (cardiovascular hospitalizations) at 6 months (confirmed at 12 months) and demonstrated improvement or stabilization in New York Heart Association class, Minnesota Living With Heart Failure Questionnaire, 6-minute walk test, peak maximum oxygen consumption, N-terminal prohormone brain natriuretic peptide levels, and left ventricular end-systolic volume. Significant increases in time to clinical events and decreased frequency of cardiovascular events were observed at 12 months (hazard ratio=0.12; P =0.003), and mean duration of cardiovascular hospitalizations over 12 months was substantially decreased (0.4 versus 4.5 days; P =0.05) on high-dose treatment versus placebo. There were no untoward safety findings. Conclusions—: The Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID) study demonstrated safety and suggested benefit of adeno-associated virus type 1/sarcoplasmic reticulum Ca 2+ -ATPase in advanced heart failure, supporting larger confirmatory trials. Clinical Trial Registration—: http://www.clinicaltrials.gov . Unique identifier: NCT00454818.
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Recent hemophilia clinical trials excluded patients whose neutralizing antibodies were above 1:4.
“Recent clinical trials for hemophilia have used even more stringent criteria for patient exclusion, removing patients whose neutralizing antibodies were above 1:4.31,43”
From Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates (2015) · cites this paper as [43]
The Discussion reports that the CUPID heart-failure trial screened patients by neutralizing-antibody titer and later excluded all antibody-positive patients. It does not report an exclusion threshold above 1:4 or an exclusion policy for hemophilia trials; the qualifying titer it gives for CUPID is below 1:2. (LLM, conf=0.86)
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Gene therapy for the treatment of heart failure: promise postponed
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Self-citationStrategy to detect pre-existing immunity to AAV gene therapy
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Self-citation
- Authors
- Jessup, M., Greenberg, B., Mancini, D., Cappola, T., Pauly, D. F., Jaski, B., Yaroshinsky, A., Zsebo, K. M., Dittrich, H., & Hajjar, R. J.
- Affiliations
- UF Health Shands Hospital (US)
- ORCID
- Venue
- Circulation
- Published
- 2011-07-19
- Volume
- 124
- Issue
- 3
- Pages
- 304-313
- ISSN
- 0009-7322 (ISSN-L)
- 1524-4539
- PMC
- PMC5843948
- OpenAlex
- W2123490348
- Cited by
- 760
- References
- 74
- Citations by year
- 2026: 31 · 2025: 29 · 2024: 20 · 2023: 29 · 2022: 31 · 2021: 33 · 2020: 37 · 2019: 47 · 2018: 37 · 2017: 55 · 2016: 57 · 2015: 75 · 2014: 94 · 2013: 89 · 2012: 80
- FWCI
- 55.48
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Circulation (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Heart, Lung, and Blood Institute (P50HL112324, R01HL080498, R01HL078691, R01HL078731, R01HL083156)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Heart Failure Treatment and Management
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Cardiac electrophysiology and arrhythmias
- Keywords
- adeno-associated virus type 1, sarcoplasmic reticulum Ca2+-ATPase, gene therapy, advanced heart failure, calcium handling, left ventricular remodeling, N-terminal proBNP, cardiovascular hospitalization, NYHA functional class, 6-minute walk test, peak oxygen uptake, left ventricular ejection fraction, left ventricular end-systolic volume
- MeSH terms
- Adenoviridae, Adult, Aged, Calcium, Double-Blind Method, Exercise Test, Female, Heart Diseases, Heart Failure, Humans, Injections, Intra-Arterial, Male, Middle Aged, Oxygen Consumption, Severity of Illness Index, Genetic Therapy, Up-Regulation, Treatment Outcome, Disease Progression, Sarcoplasmic Reticulum Calcium-Transporting ATPases
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