VNX001 for Interstitial Cystitis

Recruiting at 13 trial locations

Overseen By Vaneltix Pharma, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Vaneltix Pharma, Inc.

Must not be taking: Antidepressants, Narcotics, Marijuana, others

Disqualifiers: Pregnancy, Neurogenic bladder, CNS disorders, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 7

Duration: Single dose

120 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests VNX001, a combination of heparin and lidocaine, to help patients with chronic bladder pain from IC/BPS. Heparin repairs the bladder lining, and lidocaine numbs the bladder to reduce pain. These treatments are commonly used to manage IC/BPS symptoms.

Will I have to stop taking my current medications?

The trial requires that you stop using certain medications before participating. You must not use local anesthetics within 48 hours, lidocaine patches within 14 days, pain medications within 6 hours, narcotics or medical marijuana within 3 weeks, and certain other drugs like tricyclic antidepressants unless on a stable dose. Check with the study team for specific guidance on your medications.

What data supports the effectiveness of the drug VNX001 for interstitial cystitis?

Research shows that a combination of heparin and alkalinized lidocaine can provide immediate relief from pain and urgency in patients with interstitial cystitis. In one case, a patient experienced significant improvement in symptoms and bladder capacity after receiving this treatment. ^{1 2 3 4 5}

Is the combination of heparin and alkalinized lidocaine safe for humans?

The combination of heparin and alkalinized lidocaine has been used in studies for interstitial cystitis and has shown to provide symptom relief without reported safety concerns in the provided research. ^{1 2 3 4 5}

How is the VNX001 treatment for interstitial cystitis different from other treatments?

VNX001 is unique because it combines heparin and alkalinized lidocaine, which are administered directly into the bladder (intravesical instillation) to provide immediate relief from pain and urgency associated with interstitial cystitis, unlike other treatments that may not offer such rapid symptom relief. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for adults over 18 with moderate-to-severe bladder pain due to interstitial cystitis/bladder pain syndrome, lasting at least 9 months. Participants must have responded positively to previous anesthetic bladder treatments and not be allergic to heparin or lidocaine. Pregnant women, individuals with certain medical conditions, those on prohibited medications or who've had recent bladder procedures are excluded.

Inclusion Criteria

I may have had a bladder examination for my bladder pain.

My PUF questionnaire score is between 16 and 30.

I have had bladder pain for at least 9 months.

Exclusion Criteria

You have used ketamine in the past.

I have had bladder tumors before, whether cancerous or not.

I have heart issues or other conditions that could affect the study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive a single dose of VNX001, placebo, alkalinized lidocaine, or alkalinized heparin via intravesical instillation

Single dose

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

72 hours

Follow-up assessments at 6, 10, 12, 24, and 48 hours post-dose

Open-label extension (optional)

Participants may opt into receiving an additional single dose of VNX001

24-48 hours post-dose

Treatment Details

Interventions

• Lidocaine and Heparin
• Placebo
• VNX001

Trial Overview The study tests VNX001's effectiveness in reducing bladder pain against its components (heparin and lidocaine) and a placebo. It's a Phase 2 trial where participants are randomly assigned to receive one of the treatments in a blinded manner, meaning neither they nor the researchers know which treatment they're getting.

Participant Groups

Group I: VNX001 Experimental Treatment 1 Intervention

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)

Group II: Lidocaine Experimental Treatment 1 Intervention

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

Group III: Heparin Experimental Treatment 1 Intervention

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

Group IV: Placebo Placebo Group 1 Intervention

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

Lidocaine and Heparin is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Lidocaine Hydrochloride for:

• Local anesthesia
• Arrhythmias
• Pain relief

Approved in United States as Heparin Sodium for:

• Anticoagulation
• Thrombosis prevention

Approved in European Union as Lidocaine Hydrochloride for:

• Local anesthesia
• Arrhythmias
• Pain relief

Approved in European Union as Heparin Sodium for:

• Anticoagulation
• Thrombosis prevention

Approved in Canada as Lidocaine Hydrochloride for:

• Local anesthesia
• Arrhythmias
• Pain relief

Approved in Canada as Heparin Sodium for:

• Anticoagulation
• Thrombosis prevention

Who Is Running the Clinical Trial?

Vaneltix Pharma, Inc.

Lead Sponsor

Trials

2

Recruited

170+

Prevail Infoworks, Inc

Collaborator

Trials

4

Recruited

1,000+

Findings from Research

The combination of heparin and alkalinized lidocaine significantly reduced bladder pain and urgency symptoms in patients with interstitial cystitis compared to alkalinized lidocaine alone, with pain reduction of 38% versus 13% and urgency reduction of 42% versus 8%.

This combination therapy also resulted in higher absorption of lidocaine, with serum levels of 0.45 µg/mL for the heparin-lidocaine combination compared to 0.20 µg/mL for standard lidocaine, indicating improved efficacy in symptom relief.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Heparin and alkalinized lidocaine versus alkalinized lidocaine for treatment of interstitial cystitis symptoms. Parsons, CL., Koziol, JA., Proctor, JG., et al.[2015]

In a multicenter, double-blind, placebo-controlled trial involving 18 patients, the combination of alkalinized lidocaine and heparin significantly reduced pain associated with interstitial cystitis (IC) by 42% compared to a 21% reduction with the placebo.

The treatment also provided significant relief from urinary urgency, with a 35% reduction in urgency scores for the active drug versus 13% for the control, demonstrating its efficacy in managing IC symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Alkalinized lidocaine and heparin provide immediate relief of pain and urgency in patients with interstitial cystitis. Parsons, CL., Zupkas, P., Proctor, J., et al.[2022]

A 64-year-old woman with interstitial cystitis (IC) showed significant improvement in symptoms after receiving intravesical treatment with a combination of heparin and alkalinized lidocaine, administered twice a week for 12 months.

Her symptom scores decreased notably, and bladder capacity increased from 90 ml to 300 ml, suggesting that this treatment may be an effective option for patients with IC who do not respond to conventional therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Intravesical therapy of heparin and lidocaine for interstitial cystitis : a case report]. Matsuo, T., Shida, Y., Hayashida, Y., et al.[2013]

The combination of intravesical instillation of heparin and alkalized lidocaine, along with hydrodistension and transurethral fulguration, significantly improved symptoms of interstitial cystitis in female patients, as evidenced by reductions in symptom scores and increases in bladder capacity over a 12-month follow-up period.

This treatment approach was found to be effective and may reduce the economic burden on patients, suggesting it could be a preferred option for managing interstitial cystitis, especially when combined with hydrodistension and fulguration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Treatment of intravesical instillation with fulguration-hydrodistention on female interstitial cystitis]. Xin, P., Zhang, H., Jiang, ZM.[2023]

A 12-week treatment of intravesical instillations combining heparin and alkalized lidocaine was found to be effective in 76.7% of patients with interstitial cystitis by the end of the treatment, with significant symptom improvement noted as early as the fourth instillation.

The treatment was safe, with no severe adverse events reported, and its effects were maintained for up to 6 months after the last instillation, although efficacy gradually diminished over time.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

On- and post-treatment symptom relief by repeated instillations of heparin and alkalized lidocaine in interstitial cystitis. Nomiya, A., Naruse, T., Niimi, A., et al.[2022]

References

1. Canada pubmed.ncbi.nlm.nih.gov

Heparin and alkalinized lidocaine versus alkalinized lidocaine for treatment of interstitial cystitis symptoms. [2015]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Alkalinized lidocaine and heparin provide immediate relief of pain and urgency in patients with interstitial cystitis. [2022]

3. Japan pubmed.ncbi.nlm.nih.gov

[Intravesical therapy of heparin and lidocaine for interstitial cystitis : a case report]. [2013]

4. China pubmed.ncbi.nlm.nih.gov

[Treatment of intravesical instillation with fulguration-hydrodistention on female interstitial cystitis]. [2023]

5. Australia pubmed.ncbi.nlm.nih.gov

On- and post-treatment symptom relief by repeated instillations of heparin and alkalized lidocaine in interstitial cystitis. [2022]