VNX001 for Interstitial Cystitis

Recruiting at 14 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, VNX001, to reduce bladder pain in individuals with interstitial cystitis (IC) or bladder pain syndrome (BPS). Researchers compare VNX001, a combination of lidocaine and heparin, to each component separately and to a placebo (a harmless substance with no active ingredients). Individuals who have had IC/BPS for at least nine months and are experiencing moderate to severe bladder pain may be suitable for this study. The goal is to determine the effectiveness and safety of VNX001 in relieving bladder pain. As a Phase 2 trial, this study measures how well VNX001 works in an initial, smaller group, offering participants a chance to contribute to important research on bladder pain relief.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that the treatments in this study, including VNX001, lidocaine, and heparin, have generally been well-tolerated. When used for bladder pain, studies indicate that lidocaine is safe and effective, with few side effects. Specifically, when applied in the bladder, lidocaine is absorbed predictably, and no serious side effects have been reported.

Heparin, often used with lidocaine, has undergone testing in various studies. These studies found that heparin relieved symptoms of interstitial cystitis, a painful bladder condition, without causing severe side effects. The combination of lidocaine and heparin, as in VNX001, provided pain relief in previous research, with only minor side effects reported.

Overall, earlier trials have shown that the treatments under study are safe, suggesting they are well-tolerated for bladder pain. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for interstitial cystitis, which often involve oral medications, VNX001 is unique because it combines two active ingredients, lidocaine and heparin, in a single intravesical instillation. This direct delivery to the bladder is designed to provide targeted relief faster than traditional oral methods. Researchers are excited about VNX001 because it could offer a more effective and quicker way to alleviate the painful symptoms associated with interstitial cystitis, potentially transforming the treatment landscape for this chronic condition.

What evidence suggests that this trial's treatments could be effective for interstitial cystitis?

Research has shown that a mix of heparin and alkalinized lidocaine, as in VNX001, can significantly ease bladder pain and urgency in people with interstitial cystitis (IC). This trial will compare VNX001, which combines lidocaine and heparin, with treatments of lidocaine alone and heparin alone. Studies have found that the combination relieves symptoms more effectively than lidocaine alone. In one study, patients experienced immediate pain relief and improved bladder control. Lidocaine alone can also help by increasing bladder capacity, reducing pain. Heparin has provided relief from IC symptoms for up to 12 hours. This treatment calms the bladder nerves that cause pain and urgency. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

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 heart issues or other conditions that could affect the study.

I had a bladder examination with a scope within the last week.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Industry Sponsor

Prevail Infoworks, Inc

Collaborator

Trials

4

Recruited

1,000+

Published Research Related to This Trial

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]

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]

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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3859274/

Intravesical Heparin: Interstitial Cystitis (Painful Bladder ... The authors concluded that alkalinized lidocaine and heparin provide up to 12 hours of relief from urgency and pain associated with IC. It should be noted that ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0090429504010532

Successful downregulation of bladder sensory nerves with ... In group 2, 50% of the subjects experienced at least 4 hours of symptom relief from the single instillation, and 16 (80%) of 20 reported significant sustained ...

3. canjurol.com canjurol.com/html/subscriber/Spdf/V22I02/V22I2_14_DrParsons.pdf

Heparin and alkalinized lidocaine versus ... Conclusions: In this pilot study the heparin-lidocaine combination results in significantly better relief of IC symptoms compared to alkalinized lidocaine and ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4708535/

Review of intravesical therapies for bladder pain syndrome ... Successful downregulation of bladder sensory nerves with combination of heparin and alkalinized lidocaine in patients with interstitial cystitis. Urology ...

5. ajog.org ajog.org/article/S0002-9378(24)00609-4/fulltext

Bladder instillations vs onabotulinumtoxinA injection for ... Successful downregulation of bladder sensory nerves with combination of heparin and alkalinized lidocaine in patients with interstitial cystitis.

6. auanet.org auanet.org/guidelines-and-quality/guidelines/diagnosis-and-treatment-interstitial-of-cystitis/bladder-pain-syndrome-(2022)

Diagnosis and Treatment of Interstitial Cystitis/Bladder Pain ... An update to the literature in 2022 further showed the efficacy of heparin and lidocaine combined for relieving pain and urgency symptoms associated with IC/BPS ...

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1743609515337103

Alkalinized Lidocaine and Heparin Provide Immediate ... The combination of alkalinized lidocaine and heparin provides up to 12 hours of relief from urgency and pain associated with IC.

8. tau.amegroups.org tau.amegroups.org/article/view/8189/html

Review of intravesical therapies for bladder pain syndrome ... Parsons et al. reported on 48 patients undergoing instillation with 10,000 units of heparin 3 times a week for 3 months. Fifty-six percent attained “good ...