Treatment of bladder pain using VNX001

A Phase 2b, Open Label, Single-Arm, Multi-Center, Multiple Dose, 14-Day Study to Evaluate the Safety, Efficacy, and Frequency of Intravesical Administration of VNX001 in Acute Treatment of Subjects With Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) Who Have an Episode of Acute Bladder Pain of Moderate to Severe Intensity

This study is testing a new treatment called VNX001 to see if it can help people with moderate to severe bladder pain feel better.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Vaneltix Pharma, Inc. (Industry-sponsored)
• Drugs / interventions: chemotherapy, radiation, cyclophosphamide
• Locations: 5 sites (Tucson, Arizona and 4 other locations)
• Trial ID: NCT06394830 on ClinicalTrials.gov

What this trial studies

This open-label study aims to evaluate the safety and efficacy of VNX001 in treating acute instances of moderate to severe bladder pain in patients with Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS). Participants will receive up to six intravesical doses of the study drug over a 14-day period, with assessments of pain levels before and after dosing. The study will also monitor the frequency of doses administered and the overall tolerability of VNX001. Participants will be required to attend multiple clinic visits and maintain a daily diary to track their symptoms and any side effects.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a history of IC/BPS for at least 9 months and experiencing acute bladder pain.

Not a fit: Patients who have not previously completed the VNX001-111 study or those with less severe bladder pain may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce bladder pain and improve the quality of life for patients suffering from IC/BPS.

How similar studies have performed: While this approach is being tested in this specific context, similar studies have shown promise in treating bladder pain with intravesical therapies.

Eligibility criteria

Inclusion Criteria:

1. Enrolled and completed Study VNX001-111 (i.e., at any site that is concurrently conducting the VNX001-111 study, a subject must have previously enrolled and completed Study VNX001-111 to be eligible for entry into the VNX001-110 study) and meet all other inclusion/exclusion criteria.
2. Be able and willing to give a signed informed consent and to follow study instructions.
3. Be male or female, ≥ 18 years of age.
4. Have a history of IC/BPS for at least 9 months prior to the study, either treated (e.g. Elmiron®, RIMSO-50®), or untreated.
5. Have received a cystoscopy in association with their diagnosis of IC/BPS within 1 year of screening.
6. Have a score of ≥ 14 and ≤ 30 on the PUF questionnaire, completed at screening.
7. Have an episode of acute bladder pain of moderate to severe intensity with a minimum score of 4 on the 11-point bladder pain NRS at time of screening and 15 minutes post void immediately prior to study drug administration.

Exclusion Criteria:

1. For females, have a positive pregnancy test at screening or be pregnant or lactating.

   Note: Females considered to be of child-bearing potential must commit to using a consistent and medically acceptable method of birth control for the duration of the study: hormonal (i.e., oral, transdermal patch, implant, or injection) on a stable dose for at least 3 months prior to screening; double barrier (i.e., condom with spermicide or diaphragm with spermicide) consistently for at least 2 weeks prior to screening; and intrauterine device for at least 3 months prior to screening; or only have a partner who has been vasectomized for at least 6 months prior to screening or exclusively has same-sex partners. Female participants who are abstinent from heterosexual intercourse as part of their usual lifestyle will also be eligible for participation. Females considered to be of non-childbearing potential include: At least 1 year postmenopausal; surgically sterile (tubal ligation, bilateral oophorectomy, salpingectomy, of hysterectomy); congenitally sterile; diagnosed as infertile and not undergoing treatment to reverse infertility.
2. 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 study.
3. Postmenopausal women who, if taking hormone replacement therapy, have not been stabilized on a regimen of hormone replacement therapy within 3 months of screening.
4. Have a known hypersensitivity to heparin or lidocaine.
5. Have used any local anesthetic by any route (other than intravesical instillation) 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.
6. 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.
7. 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.
8. Have a known abnormal laboratory test value that, in the investigator's judgement, is clinically significant.
9. 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).
10. 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.
11. 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 CNS disorder.
12. 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.
13. Had bladder instillation therapy within 14 days prior to study entry.
14. Had an in-office cystoscopy within 7 days prior to study drug administration.
15. Had dilatation (hydrodistension) of bladder within 3 months of study entry.
16. Evidence or suspected presence of cancer detected during cystoscopy.
17. Has received any investigational drug or device within 30 days prior to screening.
18. Is currently enrolled in another investigational drug or device study.
19. Is unwilling or unable to abide by the requirements of the study.
20. Have an actively bleeding lesion or area in the bladder as detected by dipstick urinalysis and investigator assessment, immediately prior to randomization (Section 9.5.1.6). If the investigator determines that active bleeding is not occurring and that it is safe for the subject, the subject may continue in the study.
21. Have a history of coagulopathy or taking anticoagulants.
22. Are taking any of the following medications:

    * Phenytoin
    * Carbamazepine
    * St. John's Wort
    * Phenobarbital
    * Rifampin
23. 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

Tucson, Arizona and 4 other locations

• Arizona Urology Specialists — Tucson, Arizona, United States (Completed)
• Valley Urology, Inc. — Fresno, California, United States (Suspended)
• Prestige Medical Group — Tustin, California, United States (Suspended)
• Georgia Urology — Cartersville, Georgia, United States (Recruiting)
• Oregon Urology Institute — Springfield, Oregon, 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