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

Interstitial Cystitis Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05737121
• Other study ID #: VNX001-111
• Status: Recruiting
• Phase: Phase 2
• Start date: May 22, 2023
• Est. completion date: May 22, 2024

Study information

• Verified date: May 2023
• Source: Vaneltix Pharma, Inc.
• Contact: Vaneltix Pharma, Inc.
• Phone: 732-354-3217
• Email: info[@]vaneltix.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: May 22, 2024
• Est. primary completion date: May 22, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Bladder Pain Syndrome
• Cystitis
• Cystitis, Interstitial
• Interstitial Cystitis
• Syndrome

Intervention

Drug:
• VNX001
VNX001 (alkalinized lidocaine HCl and heparin sodium)
• Placebo
Inactive placebo for VNX001
• Lidocaine
Alkalinized lidocaine hydrochloride
• Heparin
Alkalinized heparin sodium

Locations

Country	Name	City	State
United States	Georgia Urology	Cartersville	Georgia
United States	IC Study LLC	Escondido	California
United States	Northwell Health	Lake Success	New York
United States	University of California Los Angeles Center for Women's Pelvic Health	Los Angeles	California
United States	Southern Clinical Research Associates LLC	Metairie	Louisiana
United States	The Continence Center Medical Group, Inc dba Southern California Continence Center	Newport Beach	California
United States	University of California San Diego Medical Center	San Diego	California
United States	Prestige Medical Group	Santa Ana	California

Sponsors (2)

Lead Sponsor	Collaborator
Vaneltix Pharma, Inc.	Prevail Infoworks, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Sum of bladder pain intensity differences from baseline to 12 hours post-dose (SPID-12)	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.	12 hours
Secondary	Sum of bladder pain intensity differences from baseline to 6, 10, and 24 hours post-dose (SPID-6, SPID-10, and SPID-24, respectively)	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.	6, 10, or 24 hours
Secondary	Change in bladder pain from baseline to 1, 4, 8, 12, and 24 hours post-dose	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.	1, 4, 8, 12, and 24 hours
Secondary	Change in Question 3 of the Patient Overall Rating of Improvement of Symptoms (PORIS) questionnaire	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).	1, 10, and 24 hours
Secondary	Use of optional open-label intravesical administration of VNX001	Number of patients requesting the optional open-label intravesical administration of VNX001 at 24-48 hours after randomized study drug administration	48 hours
Secondary	Adverse events (AEs)	Incidence of treatment-emergent adverse events (TEAEs), drug-related adverse events (AEs), and discontinuations due to AEs	72 hours