Trial reportNeurourology and urodynamics2020
Evaluating the safety and potential activity of URO-902 (hMaxi-K) gene transfer by intravesical instillation or direct injection into the bladder wall in female participants with idiopathic (non-neurogenic) overactive bladder syndrome and detrusor overactivity from two double-blind, imbalanced, placebo-controlled randomized phase 1 trials.
Trial report in Neurourology and urodynamics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
17 citing papers in PubMed, 1 synthesis or guideline pooled it, 33 citations in OpenAlex.
- A systematic review and in silico study of potential genetic markers implicated in cases of overactive bladder.American journal of obstetrics and gynecology · 2023Pooled it
- Gene Therapy With URO-902 (pVAX/The Journal of urology · 2025Trial
- Trial
- Ion Channels as Therapeutic Targets in Overactive Bladder: A Narrative Review of Mechanisms, Translational Evidence, and Current Gaps.Lower urinary tract symptoms · 2026Review
- Recent Technological Advances in Monitoring and Neuromodulation of Non-Malignant and Neurogenic Bladder Conditions.Advanced healthcare materials · 2026Review
- CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trials.EClinicalMedicine · 2025Article
- Promising Experimental Treatment in Animal Models and Human Studies of Interstitial Cystitis/Bladder Pain Syndrome.International journal of molecular sciences · 2024Review
- Non-pharmacological and drug treatment of autonomic dysfunction in multiple system atrophy: current status and future directions.Journal of neurology · 2023Review
- Stress Urinary Incontinence: An Unsolved Clinical Challenge.Biomedicines · 2023Review
- Role of hyperpolarization-activated cyclic nucleotide-gated channels in aging bladder phenotype.Life sciences · 2022Article
- Drugs Currently Undergoing Preclinical or Clinical Trials for the Treatment of Overactive Bladder: A Review.Current therapeutic research, clinical and experimental · 2022Review
- Gene Therapy for Overactive Bladder: A Review of BK-Channel α-Subunit Gene Transfer.Therapeutics and clinical risk management · 2021Review
- Pharmacological Management of Urinary Incontinence: Current and Emerging Treatment.Clinical pharmacology : advances and applications · 2021Review
- Hyperglycemic memory in the rat bladder detrusor is associated with a persistent hypomethylated state.Physiological reports · 2020Article
- Urinary bladder smooth muscle ion channels: expression, function, and regulation in health and disease.American journal of physiology. Renal physiology · 2020Review
- Current pharmacotherapy of overactive bladder.International braz j urol : official journal of the Brazilian Society of UrologyReview
- Pharmacology of the lower urinary tract: update on LUTS treatment.Therapeutic advances in urologyReview
Corrections and comments
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Authors and funding
10 authors at 9 institutions in 2 countries.
Funding
Abstract
aimsTwo phase 1 trials were performed in healthy women with the overactive bladder (OAB) syndrome and urodynamically demonstrated detrusor overactivity (DO), with the aim to demonstrate the safety and potential efficacy of URO-902, which comprises a gene therapy plasmid vector expressing the human big potassium channel α subunit.
methodsION-02 (intravesical instillation) and ION-03 (direct injection) were double-blind, placebo-controlled, multicenter studies without overlap in enrollment between studies. Active doses were administered and evaluated sequentially (lowest dose first) for safety. ION-02 participants received either 5000 µg or 10 000 µg URO-902, or placebo. ION-03 participants received either 16 000 or 24 000 µg URO-902, or placebo, injected directly into the bladder wall using cystoscopy. Primary outcome variables were safety parameters occurring subsequent to URO-902 administration; secondary efficacy variables also were evaluated.
resultsAmong the safety outcomes, there were no dose-limiting toxicities or significant adverse events (AEs) preventing dose escalation during either trial, and no participants withdrew due to AEs. For efficacy, in ION-02 (N = 21), involuntary detrusor contractions on urodynamics at 24 weeks in patients receiving URO-902 (P < .0508 vs placebo) and mean urgency incontinence episodes in the 5000 µg group (P = .0812 vs placebo) each showed a downward trend. In ION-03 (N = 13), significant reduction versus placebo in urgency episodes (16 000 µg, P = .036; 24 000 µg, P = .046) and number of voids (16 000 µg, -2.16, P = .044; 24 000 µg, -2.73, P = .047) were observed 1 week after injection.
conclusionPromising safety and efficacy results in these preliminary phase 1 studies suggest gene transfer may be a promising therapy for OAB/DO, warranting further investigation.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.