Evaluating VNX001 for bladder pain relief in patients with interstitial cystitis

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Multi-Center Single Dose Study to Evaluate the Safety and Effectiveness of VNX001 Compared to Placebo, and the Individual Components of Lidocaine, and Heparin in Subjects With Interstitial Cystitis/Bladder Pain Syndrome Who Have an Episode of Acute Bladder Pain of Moderate to Severe Intensity; The Engage 2024 Study

This study is testing if a new treatment called VNX001 can help relieve bladder pain in people with interstitial cystitis better than its individual ingredients or a placebo.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 120 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Vaneltix Pharma, Inc. (Industry-sponsored)
• Drugs / interventions: chemotherapy, radiation, cyclophosphamide
• Locations: 14 sites (Escondido, California and 13 other locations)
• Trial ID: NCT05737121 on ClinicalTrials.gov

What this trial studies

This Phase 2 clinical trial is designed to assess the safety and efficacy of VNX001, a combination product, compared to its individual components (lidocaine and heparin) and a placebo in patients suffering from interstitial cystitis/bladder pain syndrome (IC/BPS). The study will enroll approximately 120 to 180 participants across 12 sites in the United States, who will receive a single dose of either VNX001, placebo, alkalinized lidocaine, or alkalinized heparin. Participants will be randomly assigned to one of the treatment groups and will be monitored for pain reduction 24-48 hours post-dose. The trial aims to provide insights into the effectiveness of VNX001 in alleviating acute bladder pain of moderate to severe intensity.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a history of interstitial cystitis/bladder pain syndrome and experiencing acute bladder pain of moderate to severe intensity.

Not a fit: Patients who are pregnant, lactating, or have not previously received therapeutic intravesicular anesthetic treatment may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could offer a new effective treatment option for patients suffering from bladder pain due to interstitial cystitis.

How similar studies have performed: Other studies have explored similar approaches, but the specific combination of VNX001 is novel and has not been previously tested.

Eligibility criteria

Inclusion Criteria:

* Be able and willing to give a signed informed consent and to follow study instructions
* Be male or female, ≥ 18 years of age
* Have a history of IC/BPS for at least 9 months prior to the study
* Have a score of ≥ 16 and ≤ 30 on the Pelvic Pain and Urgency/Frequency (PUF) questionnaire, completed at screening
* Have an episode of acute bladder pain of moderate to severe intensity with a minimum score of 5 on the 11-point bladder pain NRS at time of screening and 15 minutes post void immediately prior to study drug administration.
* Have previously received a therapeutic intravesicular anesthetic treatment according to medication history

Exclusion Criteria:

* For females, have a positive pregnancy test at screening or be pregnant or lactating
* Males who are sexually active with females and are not willing to commit to an acceptable method of birth control for the duration of the
* Postmenopausal women who, if taking hormone replacement therapy, have not been stabilized on a regimen of hormone replacement therapy within 3 months of screening
* Have a known hypersensitivity to heparin or lidocaine
* Have used any local anesthetic by any route within 48-hours prior to study drug administration, or used a lidocaine patch or lidocaine containing topical compounds within 14 days prior to study drug administration
* Have used a tricyclic antidepressant, or a gamma-aminobutyric acid (GABA) analogue (gabapentin or pregabalin), unless taking a stable dose of the medication for ≥ 3 weeks. The stable dose of gabapentin may not exceed 1,200 mg per day, and the stable dose of pregabalin may not exceed 150 mg per day
* Have used any pain medication within 6 hours prior to study drug administration
* Have used narcotics or medical marijuana ≤ 3 weeks prior to study entry
* Have used prohibited drugs as determined by self-report, positive urine drug screen, or in the opinion of the investigator be under the influence of drugs affecting mentation precluding their ability to follow the study protocol or bias study results
* Have a known abnormal laboratory test value that, in the investigator's judgement, is clinically significant.
* Have a neurogenic bladder or other disorder that, in the opinion of the investigator, may cause neurogenic bladder (including Parkinson's disease, multiple sclerosis, epilepsy, myasthenia gravis, movement disorders, spinal cord damage)
* Have pain or a pain disorder that, in the opinion of the investigator, would make it difficult to discriminate pelvic pain of bladder origin from the other pain
* Have any of the following central nervous system (CNS) conditions that in the opinion of the investigator would impact the subject's study participation due to their ability to follow the study protocol or bias study results, severe diagnosed: major depressive disorder, bipolar disorder, schizophrenia, general anxiety disorder, attention deficit disorder, obsessive compulsive disorder, or other major central nervous system disorder
* Have history of arrhythmias, conduction disturbances, or cardiac disease, or any coexisting medical condition that, in the opinion of the investigator, may be significant or interfere with study procedures or interpretation of study results
* Had anesthetic bladder instillation therapy within 14 days prior to study entry
* Had an in-office cystoscopy within 7 days of study drug administration
* Had dilatation (hydrodistension) of bladder within 3 months of study entry
* Evidence or suspected presence of cancer detected during cystoscopy 7 days prior to or at time of initial screening
* Has received any investigational drug or device within 30 days prior to screening
* Is currently enrolled in another investigational drug or device study
* Is unwilling or unable to abide by the requirements of the study
* Have an actively bleeding lesion or area in the bladder as detected by dipstick urinalysis and investigator assessment, immediately prior to randomization
* Have a history of coagulopathy or taking anticoagulants.
* Are taking any of the following medications, which are inducers of CYP1A2 and/or CYP3A4: Phenytoin, Carbamazepine, St. John's Wort, Phenobarbital, Rifampin
* Have had any of the following:

  * Bacterial cystitis within 30 days as demonstrated by a positive urine culture (≥ 105 bacteria per mL)
  * History of pelvic irradiation or radiation cystitis
  * History or presence of uterine, cervical, pelvic, rectal, ovarian, or vaginal cancer
  * History of benign or malignant bladder tumors
  * Current chemotherapy
  * History or presence of tuberculous cystitis
  * History or presence of chemical cystitis, including that due to cyclophosphamide
  * History or presence of urinary schistosomiasis
  * Bladder or ureteral calculi
  * Clinically significant infectious vaginitis
  * Currently uncontrolled genital herpes
  * History or presence of urethral diverticulum
  * Presence of bladder fistulae
  * History of ketamine use

Where this trial is running

Escondido, California and 13 other locations

• IC Study LLC — Escondido, California, United States (Suspended)
• University of California Los Angeles Center for Women's Pelvic Health — Los Angeles, California, United States (Completed)
• The Clark Center for Urogynecology — Newport Beach, California, United States (Recruiting)
• The Continence Center Medical Group, Inc dba Southern California Continence Center — Newport Beach, California, United States (Completed)
• University of California San Diego Medical Center — San Diego, California, United States (Recruiting)
• Prestige Medical Group — Tustin, California, United States (Recruiting)
• United Research Institute — Hialeah, Florida, United States (Recruiting)
• Florida Urology Partners — Tampa, Florida, United States (Recruiting)
• Georgia Urology — Cartersville, Georgia, United States (Recruiting)
• Southern Clinical Research Associates LLC — Metairie, Louisiana, United States (Recruiting)
• Bay State Clinical Trials — Watertown, Massachusetts, United States (Recruiting)
• Sheldon Freedman MD LTD — Las Vegas, Nevada, United States (Recruiting)
• Northwell Health — Lake Success, New York, United States (Recruiting)
• The Wake Forest Institute of Regenerative Medicine — Winston-Salem, North Carolina, United States (Recruiting)

Study contacts

• Study coordinator: Vaneltix Pharma, Inc.

Conditions Interstitial Cystitis; Bladder Pain Syndrome; interstitial cystitis; bladder pain syndrome; alkalinized lidocaine; heparin; intravesical; VNX001