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Safety and Efficacy Study of VNX001 Compared to Its Individual Components (Lidocaine and Heparin) or Placebo in Subjects With IC/BPS

V

Vaneltix Pharma

Status and phase

Enrolling
Phase 2

Conditions

Interstitial Cystitis
Bladder Pain Syndrome

Treatments

Drug: Placebo
Drug: Heparin
Drug: Lidocaine
Drug: VNX001

Study type

Interventional

Funder types

Industry
Other

Identifiers

NCT05737121
VNX001-111 Engage 2024;

Details and patient eligibility

About

This is a Phase 2, prospective, randomized, double-blind, placebo-controlled, multi-center, single-dose, pharmacodynamic study designed to evaluate the efficacy and safety of the combination product (VNX001) versus placebo and its individual components (heparin sodium and lidocaine hydrochloride (HCl)) for the reduction of bladder pain in patients with interstitial cystitis (IC) / bladder pain syndrome (BPS), Who Have an Episode of Acute Bladder Pain of Moderate to Severe Intensity.

Full description

This is a Phase 2, prospective, randomized, double-blind, placebo-controlled, multi-center, single-dose, pharmacodynamic study designed to evaluate the efficacy and safety of the combination product (VNX001) versus placebo and its individual components (heparin sodium and lidocaine hydrochloride) for the reduction of bladder pain in patients with IC/BPS, Who Have an Episode of Acute Bladder Pain of Moderate to Severe Intensity. The study will enroll a target of 120 subjects, with a maximum of 180 subjects, across approximately 12 sites in the United States. Each study subject will receive a single dose of VNX001, placebo (alkalinized phosphate buffer), alkalinized lidocaine, or alkalinized heparin by random assignment. The randomization ratio will be 3:1:3:1, respectively. At 24-48-hours post-dose, all subjects will be given the option of requesting a single dose of VNX001.

Enrollment

120 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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 received a cystoscopy in association with their diagnosis of IC/BPS within 1 year of screening.
  • 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 with positive response, therapeutic intravesicular anesthetic treatments 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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Double Blind

120 participants in 4 patient groups, including a placebo group

VNX001
Experimental group
Description:
VNX001 (lidocaine HCl \[200 mg\] and heparin sodium \[50,000 USPU\] in alkalinized buffer), administered as a single dose via intravesical instillation; n=45 (anticipated)
Treatment:
Drug: VNX001
Placebo
Placebo Comparator group
Description:
Alkalinized buffer, administered as a single dose via intravesical instillation; n=15 (anticipated)
Treatment:
Drug: Placebo
Lidocaine
Experimental group
Description:
Lidocaine HCl (200 mg) in alkalinized buffer, administered as a single dose via intravesical instillation; n=45 (anticipated)
Treatment:
Drug: Lidocaine
Heparin
Experimental group
Description:
Heparin sodium (50,000 USPU) in alkalinized buffer, administered as a single dose via intravesical instillation; n=15 (anticipated)
Treatment:
Drug: Heparin

Trial contacts and locations

11

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Central trial contact

Vaneltix Pharma, Inc.

Data sourced from clinicaltrials.gov

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