Abstract
Background aims
Interest in cell-based therapy using extracellular vesicles (EVs) is intensifying,
building upon promising preclinical research and a handful of published clinical studies.
Registered clinical trials remain small, heterogeneous in design and underpowered
to determine safety and efficacy on their own. A scoping review of registered studies
can identify opportunities to pool data and perform meta-analysis.
Methods
Registered trials were identified by searching clinical trial databases (Clinicaltrials.gov,
the World Health Organization International Clinical Trials Registry Platform and
the Chinese Clinical Trial Registry) on June 10, 2022.
Results
Seventy-three trials were identified and included for analysis. Mesenchymal stromal
cells (MSCs) were the most common cell type from which EVs were derived (49 studies,
67%). Among the 49 identified MSC-EV studies, 25 were controlled trials (51%) with
a combined total of 3094 participants anticipated to receive MSC-derived EVs (2225
in controlled studies). Although EVs are being administered to treat a broad range
of conditions, trials treating patients with coronavirus disease-2019 and/or acute
respiratory distress syndrome were observed most commonly. Despite heterogeneity between
studies, we anticipate that at least some of the studies could be combined in meaningful
meta-analysis and that a combined sample size of 1000 patients would provide the ability
to detect a ≥5% difference in mortality with MSC-EVs compared to controls and could
be achieved by December 2023.
Conclusions
This scoping review identifies potential barriers that may stall clinical translation
of EV-based treatment, and our analysis calls for more standardized product characterization,
use of quantifiable product quality attributes and consistent outcome reporting in
future clinical trials.
Key Words
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Article info
Publication history
Published online: May 14, 2023
Accepted:
April 12,
2023
Received:
October 12,
2022
Publication stage
In Press Corrected ProofIdentification
Copyright
© 2023 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.
