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

Sponsor

Vaneltix Pharma, Inc. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05737121

Collaborator

Prevail Infoworks, Inc (Other)

120

Enrollment

Locations

Arms

11.8

Anticipated Duration (Months)

Patients Per Site

1.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Condition or Disease	Intervention/Treatment	Phase
Interstitial Cystitis Bladder Pain Syndrome	Drug: VNX001 Drug: Placebo Drug: Lidocaine Drug: Heparin	Phase 2

Detailed 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. 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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

120 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

IC/BPS patients will be randomly assigned to receive a single dose of one of the following treatments in a ratio of 3:1:3:1: VNX001 (n=45): alkalinized buffered lidocaine HCl (200 mg) and heparin sodium (50,000 USPU), administered as an intravesical instillation Placebo (n=15): alkalinized buffer, administered as an intravesical instillation Lidocaine (n=45): alkalinized buffered lidocaine HCl (200 mg), administered as an intravesical instillation Heparin (n=15): alkalinized buffered heparin sodium (50,000 USPU), administered as an intravesical instillation At 24-48-hours post-dose, all subjects will be given the option of requesting a single open-label dose of VNX001.IC/BPS patients will be randomly assigned to receive a single dose of one of the following treatments in a ratio of 3:1:3:1:VNX001 (n=45): alkalinized buffered lidocaine HCl (200 mg) and heparin sodium (50,000 USPU), administered as an intravesical instillation Placebo (n=15): alkalinized buffer, administered as an intravesical instillation Lidocaine (n=45): alkalinized buffered lidocaine HCl (200 mg), administered as an intravesical instillation Heparin (n=15): alkalinized buffered heparin sodium (50,000 USPU), administered as an intravesical instillation At 24-48-hours post-dose, all subjects will be given the option of requesting a single open-label dose of VNX001.

Masking:

Double (Participant, Investigator)

Masking Description:

Double-blind

Primary Purpose:

Treatment

Official Title:

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Multi-Center Single Dose Study to Evaluate the Safety and Effectiveness of VNX001 Compared to Placebo, the Individual Components of Lidocaine, and Heparin in Subjects With Interstitial Cystitis/Bladder Pain Syndrome

Anticipated Study Start Date :

Mar 7, 2023

Anticipated Primary Completion Date :

Mar 1, 2024

Anticipated Study Completion Date :

Mar 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: VNX001 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)	Drug: VNX001 VNX001 (alkalinized lidocaine HCl and heparin sodium) Other Names: Alkalinized lidocaine HCl and heparin sodium
Placebo Comparator: Placebo Alkalinized buffer, administered as a single dose via intravesical instillation; n=15 (anticipated)	Drug: Placebo Inactive placebo for VNX001 Other Names: Placebo (for VNX001)
Experimental: Lidocaine Lidocaine HCl (200 mg) in alkalinized buffer, administered as a single dose via intravesical instillation; n=45 (anticipated)	Drug: Lidocaine Alkalinized lidocaine hydrochloride Other Names: Alkalinized lidocaine HCl
Experimental: Heparin Heparin sodium (50,000 USPU) in alkalinized buffer, administered as a single dose via intravesical instillation; n=15 (anticipated)	Drug: Heparin Alkalinized heparin sodium Other Names: Alkalinized heparin sodium

Outcome Measures

Primary Outcome Measures

Sum of bladder pain intensity differences from baseline to 12 hours post-dose (SPID-12) [12 hours]
A measure of the sum of bladder pain intensity differences from baseline to 12 hours post-dose (SPID-12), as determined using an 11-point numerical rating scale (NRS) for bladder pain. The 11-point NRS for bladder pain is a scale from 0 to 10, with 0 indicating no bladder pain and 10 indicating the worst imaginable bladder pain.

Secondary Outcome Measures

Sum of bladder pain intensity differences from baseline to 6, 10, and 24 hours post-dose (SPID-6, SPID-10, and SPID-24, respectively) [6, 10, or 24 hours]
A measure of the sum of bladder pain intensity differences from baseline to 6, 10, and 24 hours post-dose (SPID-6, SPID-10, and SPID-24, respectively), as determined using an 11-point numerical rating scale (NRS) for bladder pain. The 11-point NRS for bladder pain is a scale from 0 to 10, with 0 indicating no bladder pain and 10 indicating the worst imaginable bladder pain.
Change in bladder pain from baseline to 1, 4, 8, 12, and 24 hours post-dose [1, 4, 8, 12, and 24 hours]
Average absolute change and average percentage change in bladder pain from baseline to 1, 4, 8, 12, and 24 hours post-dose, as determined using an 11-point numerical rating scale (NRS) for bladder pain. The 11-point NRS for bladder pain is a scale from 0 to 10, with 0 indicating no bladder pain and 10 indicating the worst imaginable bladder pain.
Change in Question 3 of the Patient Overall Rating of Improvement of Symptoms (PORIS) questionnaire [1, 10, and 24 hours]
Percentage of subjects achieving ≥ 50% improvement in Question 3 of the PORIS questionnaire at 1, 10, and 24 hours post-dose. The PORIS questionnaire is an assessment of the subject's condition after treatment compared to before treatment. In particular, Question 3 of the PORIS questionnaire asks subjects to select the category that best describes the overall change in their condition compared to before receiving study medication; the choices are: worse, no better (0% improvement), slightly improved (25% improvement), moderately improved (50% improvement), greatly improved (75% improvement), or symptoms gone (100% improvement).
Use of optional open-label intravesical administration of VNX001 [48 hours]
Number of patients requesting the optional open-label intravesical administration of VNX001 at 24-48 hours after randomized study drug administration
Adverse events (AEs) [72 hours]
Incidence of treatment-emergent adverse events (TEAEs), drug-related adverse events (AEs), and discontinuations due to AEs

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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 moderate-to-severe symptoms of bladder pain of bladder origin for at least 9 months prior to the study
May or may not have received a cystoscopy in association with their diagnosis of interstitial cystitis/bladder pain syndrome prior to or at time of screening
Have a score of ≥ 16 and ≤ 30 on the Pelvic Pain and Urgency/Frequency (PUF) questionnaire, completed at screening
Have moderate to severe pain: a minimum score of 5 is required 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 bladder instillation therapy within 7 days prior to study entry or had a prior bladder instillation with heparin and lidocaine and did not respond
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 (cystoscopy is not required for entry into the study)
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
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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	IC Study LLC	Escondido	California	United States	92025
2	University of California Los Angeles	Los Angeles	California	United States	90095
3	University of California San Diego	San Diego	California	United States	92103
4	Prestige Medical Group	Santa Ana	California	United States	92705
5	Georgia Urology	Cartersville	Georgia	United States	30120
6	Southern Clinical Research Associates LLC	Metairie	Louisiana	United States	70001
7	Boston Urogynecology Associates	Cambridge	Massachusetts	United States	02138
8	Northwell Health	Lake Success	New York	United States	11042