VNX001 for Painful Bladder Syndrome

Recruiting at 2 trial locations

Overseen By Vaneltix Pharma, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Vaneltix Pharma, Inc.

Must not be taking: Antidepressants, Anticonvulsants, St. John's Wort

Disqualifiers: Pregnancy, Neurogenic bladder, CNS disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: 8

Duration: 14 days

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment option, VNX001, for individuals with Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS), which causes painful bladder symptoms. The study evaluates how effectively VNX001 relieves sudden, intense bladder pain when taken as needed over a two-week period. Participants will provide insights into the required dosage and its impact on pain levels. Candidates may be suitable for this trial if they have experienced IC/BPS for at least nine months and suffer from moderate to severe bladder pain. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to the development of a potentially effective new therapy.

Do I need to stop taking my current medications for the trial?

The trial does not specify if you need to stop all current medications, but certain medications are prohibited. You cannot take phenytoin, carbamazepine, St. John's Wort, phenobarbital, or rifampin. If you are on a tricyclic antidepressant or a GABA analogue like gabapentin or pregabalin, you must be on a stable dose for at least 3 weeks.

Is there any evidence suggesting that VNX001 is likely to be safe for humans?

Research shows that VNX001, a mix of lidocaine and heparin, may help with bladder pain. Past studies have used these ingredients, and one study found that using both together eased bladder pain more than lidocaine alone.

Lidocaine is a local anesthetic that numbs tissue, and heparin helps prevent blood clots. Both are well-known in medicine, but their combined use for bladder pain remains under study. So far, research indicates that when this combination is put directly into the bladder, it seems to relieve pain and symptoms.

Regarding safety, earlier trials tested VNX001, and participants generally handled the treatment well. No major safety issues were reported. However, since the current trial is in phase 2, researchers continue to monitor safety and side effects. This phase aims to confirm that the treatment is safe and effective in a larger group, so more safety information may emerge after this study. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Painful Bladder Syndrome, which often include oral medications or procedures like bladder instillations, VNX001 offers a unique approach with its intravesical administration. This method involves directly delivering the treatment into the bladder, potentially providing faster and more targeted relief. VNX001 combines buffered lidocaine HCl and sodium heparin, which may offer an effective dose to alleviate pain more efficiently. Researchers are excited because this targeted delivery could potentially reduce systemic side effects and improve patient comfort compared to traditional therapies.

What evidence suggests that VNX001 might be an effective treatment for Painful Bladder Syndrome?

Studies have shown that a combination of heparin and alkalinized lidocaine, as in VNX001, can significantly reduce bladder pain and urgency in people with Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS). Specifically, this mixture reduced bladder pain by 38%, compared to 13% with other treatments. It also lowered urgency by 42%, versus 8% in similar comparisons. This effect occurs because the treatment targets and calms the bladder's sensory nerves. Overall, initial results suggest VNX001 might effectively manage pain for those suffering from IC/BPS. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals who have completed Study VNX001-111 and suffer from Interstitial Cystitis / Bladder Pain Syndrome (IC/BPS). It's designed to help those with moderate to severe bladder pain by testing a new treatment called VNX001.

Inclusion Criteria

I completed Study VNX001-111 and meet all other requirements.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1 day

1 visit (in-person)

Treatment

Participants receive up to 6 intravesical doses of VNX001 over 14 days to treat acute bladder pain

14 days

Up to 6 visits (in-person) or 1 combined visit (in-person) and 5 telephone visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 days

1 follow-up call

What Are the Treatments Tested in This Trial?

Interventions

• VNX001

Trial Overview The study tests the safety and effectiveness of up to six doses of VNX001, administered directly into the bladder over two weeks. Participants will track their pain levels before and after each dose, as well as any side effects.

How Is the Trial Designed?

Group I: Open Label Arm Experimental Treatment 1 Intervention

VNX001 intravesical administration PRN up to 6 doses over 14 days. The VNX001 effective dose is 15 mL of buffered lidocaine HCl (200 mg) and sodium heparin (50,000 USPU).

Who Is Running the Clinical Trial?

Vaneltix Pharma, Inc.

Lead Sponsor

Trials

2

Recruited

170+

Prevail Infoworks, Inc

Collaborator

Trials

4

Recruited

1,000+

Prevail Infoworks

Industry Sponsor

Published Research Related to This Trial

The review analyzed 100 studies on various therapeutic approaches for interstitial cystitis/bladder pain syndrome (IC/BPS), highlighting that most research focuses on agents recommended by the American Urological Association and those approved for other diseases.

Emerging treatments, including stem cells and plant-derived agents, show promise, but the overall methodological quality of animal studies needs improvement to enhance the translation of findings to clinical practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Systematic Review of Therapeutic Approaches Used in Experimental Models of Interstitial Cystitis/Bladder Pain Syndrome. Kuret, T., Peskar, D., Erman, A., et al.[2021]

Intravesical administration of resiniferatoxin (RTX) during spinal shock in rats significantly improved bladder function, reducing pressures and contraction amplitudes, indicating its efficacy in modulating bladder activity.

RTX works by desensitizing TRPV1-expressing bladder afferents, suggesting that early treatment could prevent the development of neurogenic detrusor overactivity (NDO) and improve the quality of life for patients with spinal cord injuries.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of early intravesical administration of resiniferatoxin to spinal cord-injured rats in neurogenic detrusor overactivity. Oliveira, R., Coelho, A., Franquinho, F., et al.[2020]

In a randomized, double-blind, placebo-controlled trial involving 96 patients with interstitial cystitis/bladder pain syndrome, KRP-116D significantly improved symptoms compared to placebo, with a mean reduction of 5.2 points in the symptom index score versus 3.4 points for placebo.

KRP-116D was well-tolerated, with only mild to moderate adverse reactions, indicating a favorable safety profile for patients undergoing treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of intravesical instillation of KRP-116D (50% dimethyl sulfoxide solution) for interstitial cystitis/bladder pain syndrome in Japanese patients: A multicenter, randomized, double-blind, placebo-controlled, clinical study. Yoshimura, N., Homma, Y., Tomoe, H., et al.[2021]

In a study involving 39 spinal cord injured adults, both capsaicin (CAP) and resiniferatoxin (RTX) showed similar efficacy in improving detrusor hyperreflexia, with 78% of CAP patients and 80% of RTX patients experiencing clinical improvement after 30 days.

There were no significant differences in side effects between the two treatments, indicating that both CAP and RTX are well-tolerated options for patients with detrusor hyperreflexia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravesical capsaicin versus resiniferatoxin for the treatment of detrusor hyperreflexia in spinal cord injured patients: a double-blind, randomized, controlled study. de Sèze, M., Wiart, L., de Sèze, MP., et al.[2013]

Capsaicin and resiniferatoxin (RTX) show potential as treatments for neurogenic bladder, but more extensive studies are necessary to confirm their effectiveness and safety.

Currently, vanilloid receptor agonists are not recommended for routine use in overactive bladder or interstitial cystitis due to insufficient evidence of efficacy and concerns about the risks associated with catheterization.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Use of vanilloids in urologic disorders. Foster, HE., Lake, AG.[2019]

In a study using TRPV1 knock-out and wild-type mice, it was found that TRPV1 is crucial for the development of bladder mechanical hyperreactivity associated with cystitis, indicating its role in visceral pain.

While TRPV1 is important for the sensation of pain related to bladder inflammation, it does not affect the overall inflammatory response or sensitivity to thermal stimuli, suggesting that other pathways may be involved in these processes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lack of TRPV1 inhibits cystitis-induced increased mechanical sensitivity in mice. Wang, ZY., Wang, P., Merriam, FV., et al.[2021]

In women with sensory urgency (SU), there is a significant increase in TRPV1 mRNA expression in the trigonal mucosa of the bladder, suggesting a potential role of TRPV1 in bladder hypersensitivity.

In contrast, patients with idiopathic detrusor overactivity (IDO) showed no differences in TRPV1 expression between trigonal and body mucosa, indicating that TRPV1 may not be involved in IDO-related symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The molecular basis of urgency: regional difference of vanilloid receptor expression in the human urinary bladder. Liu, L., Mansfield, KJ., Kristiana, I., et al.[2007]

The vanilloid receptor type 1 (VR-1) is found in sensory neurons of the urinary bladder, and its activation by substances like capsaicin or resiniferatoxin (RTX) can lead to a temporary desensitization of these neurons, reducing their reactivity to stimuli.

Intravesical administration of capsaicin or RTX has shown potential therapeutic benefits for patients with bladder conditions, such as detrusor hyperreflexia and idiopathic detrusor instability, by targeting the abundant VR-1 fibers in the bladder.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vanilloid receptor and detrusor instability. Cruz, F.[2019]

The study found that prolonged stimulation of alpha 1A adrenoceptors with phenylephrine (PHE) induces chronic visceral pain (CVP) in female Wistar rats, which can be reversed by silodosin, indicating a potential therapeutic target for managing CVP.

The mechanism appears to involve the interaction between alpha 1A adrenoceptors and TRPV1 nociceptors, as TRPV1 knockout mice did not exhibit pain from PHE, and PHE also enhanced ATP release from human urothelial cells, suggesting a role in bladder hyperactivity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Bladder pain induced by prolonged peripheral alpha 1A adrenoceptor stimulation involves the enhancement of transient receptor potential vanilloid 1 activity and an increase of urothelial adenosine triphosphate release. Matos, R., Cordeiro, JM., Coelho, A., et al.[2018]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05737121

Safety and Efficacy Study of VNX001 Compared to Its ... This is a Phase 2, prospective, randomized, double-blind, placebo-controlled, multi-center, single-dose, pharmacodynamic study designed to evaluate the efficacy ...

2. engage-24.com engage-24.com/safety-and-efficacy-study-of-vnx001-compared-to-its-individual-components-lidocaine-and-heparin-or-placebo-in-subjects-with-ic-bps/

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

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7306012/

The role of bladder instillation in the treatment of bladder pain ... Successful downregulation of bladder sensory nerves with combination of heparin and alkalinized lidocaine in patients with interstitial cystitis ...

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

Heparin and alkalinized lidocaine versus ... The heparin-lidocaine combination significantly reduced the % of bladder pain (38% versus 13%, p = 0.029) and urgency (42% versus 8% p = 0.003) compared to ...

5. isrctn.com isrctn.com/ISRCTN56132730

Phase II, multicentre, randomised, double blind, placebo ... Intravesical alkalised lidocaine may offer a therapeutic approach to treat the pain and bladder inflammation ... Interstitial cystitis/painful bladder syndrome.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11973989/

2025 Canadian Urological Association Guideline In 2015, a pilot crossover study in 14 patients compared 50 000 units of heparin with 200 mg of lidocaine to 200 mg of lidocaine alone and found a significant ...

7. go.drugbank.com go.drugbank.com/drugs/DB01109/clinical_trials?conditions=DBCOND0028936%2CDBCOND0037842&phase=2&purpose=treatment&status=recruiting

Heparin Recruiting Phase 2 Trials for Interstitial Cystitis ... Safety and Efficacy Study of VNX001 Compared to Its Individual Components (Lidocaine and Heparin) or Placebo in Subjects With IC/BPS.