Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial), a First-in-Human Phase 1/2 Clinical Trial
Open accessAbstract
Background SERCA2a deficiency is commonly seen in advanced heart failure (HF). This study is designed to investigate safety and biological effects of enzyme replacement using gene transfer in patients with advanced HF.Methods and results A total of 9 patients with advanced HF (New York Heart Association [NYHA] Class III/IV, ejection fraction [EF] < or = 30%, maximal oxygen uptake [VO2 max] <16 mL.kg.min, with maximal pharmacological and device therapy) received a single intracoronary infusion of AAV1/SERCA2a in the open-label portion of this ongoing study. Doses administered ranged from 1.4 x 10(11) to 3 x 10(12) DNase resistant particles per patient. We present 6- to 12-month follow-up data for these patients. AAV1/SERCA2a demonstrated an acceptable safety profile in this advanced HF population. Of the 9 patients treated, several demonstrated improvements from baseline to month 6 across a number of parameters important in HF, including symptomatic (NYHA and Minnesota Living with Heart Failure Questionnaire, 5 patients), functional (6-minute walk test and VO2 max, 4 patients), biomarker (NT-ProBNP, 2 patients), and LV function/remodeling (EF and end-systolic volume, 5 patients). Of note, 2 patients who failed to improve had preexisting anti-AAV1 neutralizing antibodies.Conclusions Quantitative evidence of biological activity across a number of parameters important for assessing HF status could be detected in several patients without preexisting neutralizing antibodies in this open-label study, although the number of patients in each cohort is too small to conduct statistical analyses. These findings support the initiation of the Phase 2 double-blind, placebo-controlled portion of this study.
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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 [31]
The Discussion (page unavailable) says the CUPID heart-failure trial screened for low or absent neutralizing antibodies and later excluded all antibody-positive patients. It does not describe a hemophilia trial or an exclusion threshold of 1:4; the titers it reports are 1:2 and <1:2. (LLM, conf=0.78)
Experimental — accuracy not yet measuredPre-existing antibodies correlated with decreased efficacy of gene therapy in clinical trials where administration exposes vector to serum.
“the presence of pre-existing antibodies has been correlated with decreased efficacy of gene therapy in clinical trials where the route of administration exposes vector to serum.26,30,31”
From Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates (2015) · cites this paper as [31]
The Discussion reports a potential difference in heart-failure progression among the first nine CUPID patients: both patients with pre-existing neutralizing antibodies worsened, and vector sequences were undetectable in both. But it calls the findings too preliminary to draw conclusions. This supports a possible association in one trial, not an established correlation across clinical trials. (LLM, conf=0.85)
Experimental — accuracy not yet measuredPre-existing antibodies inhibit AAV transduction broadly documented following mouse, rabbit, NHP, and human gene therapy administrations.
“The observation that pre-existing antibodies inhibit AAV transduction has been broadly documented following mouse, rabbit, non-human primate, and human gene therapy administrations.31,57–59”
From Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates (2015) · cites this paper as [31]
The Discussion (page not provided) says preexisting neutralizing antibodies can block viral-vector entry, and the paper reports suggestive findings in two antibody-positive CUPID patients. Its References include a mouse transduction study, but our copy does not document the claimed rabbit or non-human-primate findings; the Discussion also says its human data are too preliminary for conclusions. (LLM, conf=0.84)
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Self-citation
- Authors
- Jaski, B. E., Jessup, M. L., Mancini, D. M., Cappola, T. P., Pauly, D. F., Greenberg, B., Borow, K., Dittrich, H., Zsebo, K. M., & Hajjar, R. J.
- Affiliations
- San Diego Cardiac Center (US)
- Hospital of the University of Pennsylvania (US)
- NewYork–Presbyterian Hospital (US)
- UF Health Shands Hospital (US)
- University of California San Diego (US)
- Celladon Corporation (United States) (US)
- Icahn School of Medicine at Mount Sinai (US)
- Corresponding author
- Jaski, Brian E.
- ORCID
- Venue
- Journal of Cardiac Failure
- Publisher
- Elsevier BV
- Published
- 2009-04
- Volume
- 15
- Issue
- 3
- Pages
- 171-181
- ISSN
- 1071-9164 (ISSN-L)
- PMC
- PMC2752875
- OpenAlex
- W2010344791
- Cited by
- 457
- References
- 49
- Citations by year
- 2026: 18 · 2025: 19 · 2024: 15 · 2023: 15 · 2022: 13 · 2021: 24 · 2020: 14 · 2019: 20 · 2018: 18 · 2017: 25 · 2016: 23 · 2015: 36 · 2014: 38 · 2013: 40 · 2012: 45
- FWCI
- 26.91
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Journal of Cardiac Failure (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- University of California, San Diego
- National Heart, Lung, and Blood Institute (HL 78731, HL-080498, R01HL080498, R01HL078691, HL 083156, R01HL078731, R01HL083156, R01HL088434)
- 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
- Physical Sciences › Engineering › Biomedical Engineering › Mechanical Circulatory Support Devices
- Keywords
- SERCA2a, advanced heart failure, gene therapy, calcium handling, enzyme replacement therapy, left ventricular remodeling, NT-proBNP, treatment efficacy and safety
- MeSH terms
- Exercise Test, Female, Heart Failure, Humans, Infusions, Intra-Arterial, Male, Middle Aged, Oxygen Consumption, Peptide Fragments, Stroke Volume, Systole, Genetic Therapy, Up-Regulation, Natriuretic Peptide, Brain, Ventricular Remodeling, Sarcoplasmic Reticulum Calcium-Transporting ATPases
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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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