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

NCT05737121 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: VNX001-111; Engage 2024
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 14

Brief 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), Who Have an Episode of Acute Bladder Pain of Moderate to Severe Intensity.

Conditions

Interstitial Cystitis; Bladder Pain Syndrome

Keywords

interstitial cystitis (IC); bladder pain syndrome (BPS); alkalinized lidocaine; heparin; intravesical; VNX001

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• 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

• Change in bladder pain from baseline to 1, 4, 8, 12, and 24 hours post-dose

  1, 4, 8, 12, and 24 hours

• Change in Question 3 of the Patient Overall Rating of Improvement of Symptoms (PORIS) questionnaire

  1, 10, and 24 hours

• Use of optional open-label intravesical administration of VNX001

  48 hours

• Adverse events (AEs)

  72 hours

Study Arms (4)

VNX001

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

Alkalinized buffer, administered as a single dose via intravesical instillation; n=15 (anticipated)

Drug: Placebo

Lidocaine

EXPERIMENTAL

Lidocaine HCl (200 mg) in alkalinized buffer, administered as a single dose via intravesical instillation; n=45 (anticipated)

Drug: Lidocaine

Heparin

EXPERIMENTAL

Heparin sodium (50,000 USPU) in alkalinized buffer, administered as a single dose via intravesical instillation; n=15 (anticipated)

Drug: Heparin

Interventions

VNX001 DRUG

VNX001 (alkalinized lidocaine HCl and heparin sodium)

Also known as: Alkalinized lidocaine HCl and heparin sodium

VNX001

Placebo DRUG

Inactive placebo for VNX001

Also known as: Placebo (for VNX001)

Placebo

Lidocaine DRUG

Alkalinized lidocaine hydrochloride

Also known as: Alkalinized lidocaine HCl

Lidocaine

Heparin DRUG

Alkalinized heparin sodium

Also known as: Alkalinized heparin sodium

Heparin

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Vaneltix Pharma, Inc.: lead
• Prevail Infoworks: collaborator

Study Sites (14)

IC Study LLC

Escondido, California, 92025, United States

SUSPENDED

University of California Los Angeles Center for Women's Pelvic Health

Los Angeles, California, 90095, United States

COMPLETED

The Clark Center for Urogynecology

Newport Beach, California, 92663, United States

RECRUITING

The Continence Center Medical Group, Inc dba Southern California Continence Center

Newport Beach, California, 92663, United States

COMPLETED

University of California San Diego Medical Center

San Diego, California, 92103, United States

RECRUITING

Prestige Medical Group

Tustin, California, 92780, United States

RECRUITING

United Research Institute

Hialeah, Florida, 33012, United States

RECRUITING

Florida Urology Partners

Tampa, Florida, 33615, United States

RECRUITING

Georgia Urology

Cartersville, Georgia, 30120, United States

RECRUITING

Southern Clinical Research Associates LLC

Metairie, Louisiana, 70001, United States

RECRUITING

Bay State Clinical Trials

Watertown, Massachusetts, 02472, United States

RECRUITING

Sheldon Freedman MD LTD

Las Vegas, Nevada, 89144, United States

RECRUITING

Northwell Health

Lake Success, New York, 11042, United States

RECRUITING

The Wake Forest Institute of Regenerative Medicine

Winston-Salem, North Carolina, 27101, United States

RECRUITING

Related Links

• ENGAGE2024 Study Website

MeSH Terms

Conditions

Cystitis, Interstitial

Interventions

Heparin; Lidocaine

Condition Hierarchy (Ancestors)

Cystitis; Urinary Bladder Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Glycosaminoglycans; Polysaccharides; Carbohydrates; Acetanilides; Anilides; Amides; Organic Chemicals; Aniline Compounds; Amines

Central Study Contacts

Vaneltix Pharma, Inc.

CONTACT

732-354-3217; info@vaneltix.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: February 10, 2023
• First Posted: February 21, 2023
• Study Start: May 22, 2023
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: January 22, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (14)