Cidofovir for Hemorrhagic Cystitis

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Must not be taking: Foscarnet, Liposomal amphotericin B, Aminoglycosides

Disqualifiers: Low creatinine clearance, 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: 4

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether adding cidofovir to usual care can help people with hemorrhagic cystitis, a painful bladder condition caused by the BK virus. Researchers aim to determine if cidofovir reduces symptoms more effectively than standard treatments alone and will also assess its safety. It is suitable for patients who have undergone a stem cell transplant and are experiencing symptoms like blood in the urine due to this condition. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important medical advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are using foscarnet, liposomal amphotericin B, or aminoglycoside.

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

Research has shown that cidofovir has been tested for treating bladder inflammation caused by the BK virus. In earlier studies, patients experienced noticeable improvement after taking cidofovir. However, monitoring kidney health during treatment is crucial to prevent possible side effects. While cidofovir can be effective, careful monitoring ensures its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Cidofovir is unique because it introduces an antiviral approach to treating hemorrhagic cystitis, a condition often managed with pain relief and bladder irrigation. Unlike standard treatments that focus on symptom management, Cidofovir directly targets viral causes that can contribute to the condition, potentially addressing the underlying issue rather than just alleviating symptoms. Researchers are excited about Cidofovir's potential to offer a more targeted and effective therapy, possibly leading to better outcomes for patients who struggle with the limitations of current standard care options.

What evidence suggests that cidofovir might be an effective treatment for hemorrhagic cystitis?

Research has shown that cidofovir, a drug used to fight viruses, may help treat hemorrhagic cystitis, a condition characterized by blood in the urine caused by the BK virus. In this trial, some participants will receive Standard of Care plus Cidofovir. Studies have found that many patients experienced a significant reduction in symptoms after taking cidofovir. Specifically, one study found that 45% of patients saw their severe symptoms completely disappear. Another review indicated that most patients experienced at least some improvement after using cidofovir. These results suggest that cidofovir might effectively reduce symptoms of hemorrhagic cystitis caused by the BK virus. ^{3 4 6 7 8}

Who Is on the Research Team?

Borje S. Andersson, MD,PHD

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for individuals aged 6 or older who have a bladder infection known as hemorrhagic cystitis, caused by the BK virus. Participants must show symptoms and have a certain level of BK virus in their urine. They cannot join if they've used cidofovir before, had specific treatments like formalin, have low kidney function, or are using certain other medications.

Inclusion Criteria

Patient must sign the informed consent document.

I am 6 years old or older.

I have mild bladder symptoms from a stem cell transplant and tested positive for BK virus.

Exclusion Criteria

My kidney function, measured by creatine clearance, is below 55 ml/min.

I am currently taking foscarnet, liposomal amphotericin B, or an aminoglycoside.

I am using cidofovir for bladder treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either cidofovir and standard of care or standard of care alone for hemorrhagic cystitis

4 weeks

3 visits per week (in-person)

End-of-Treatment

Urine collected to test BK virus level and complete urinary problems questionnaire

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Cidofovir

Trial Overview The study aims to see if adding cidofovir to standard care helps with symptoms of hemorrhagic cystitis better than standard care alone. It's also looking at how safe cidofovir is when used this way.

How Is the Trial Designed?

Group I: Standard of Care + Cidofovir Experimental Treatment 1 Intervention

Cidofovir 0.5 mg/kg IV 3 x week for 4 weeks

Group II: No Cidofovir Active Control 1 Intervention

Standard of Care: Pharmacologic management of pain, spasms, and urinary urgency with medications, hyper-hydration, or continuous bladder irrigation.

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/29679772/

Efficacy of Cidofovir in Treatment of BK Virus-Induced ... Cidofovir is an antiviral agent with growing evidence as a therapeutic intervention. ... The primary outcome measure was a reduction in BK-HC severity (graded ...

2. astctjournal.org astctjournal.org/article/S1083-8791(18)30190-3/fulltext

Efficacy of Cidofovir in Treatment of BK Virus–Induced ... Twelve transplant recipients received cidofovir for BK virus hemorrhagic cystitis. · Mean hemorrhagic cystitis severity decreased significantly after cidofovir.

3. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/7293/528164/Role-of-Cidofovir-in-the-Treatment-of-BK-Virus

Role of Cidofovir in the Treatment of BK Virus Haemorrhagic ... 10/22 (45%) of the patients had a complete resolution of Grade2+HC symptoms with cidofovir, although the difference was not statistically ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S0006497119859955

Efficacy of Cidofovir in Treatment of BK Virus Induced ... In our retrospective institutional review, a majority of patients achieved at least partial remission after cidofovir treatment with similar ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT01295645?term=Cidofovir&viewType=Table&rank=1

Cidofovir Versus Best Supportive Care for Hemorrhagic ... The goal of this clinical research study is to learn if adding cidofovir to the standard of care can improve symptoms of hemorrhagic cystitis caused by the BK ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/19522651/

Cidofovir for BK Virus-Associated Hemorrhagic Cystitis 03). After a median follow-up of 287 days, overall survival and total treatment-related mortality rates were 63% and 40% for patients achieving ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/26718666/

Cidofovir in the Treatment of BK Virus-Associated ... Cidofovir is an effective therapy for BKV-HC but requires very precise renal function management to avoid toxicity.

8. clinicaltrials.gov clinicaltrials.gov/study/NCT01816646

Intravesical Cidofovir for Hemorrhagic Cystitis The goal of this clinical research study is to learn how the drug cidofovir given as 1 dose directly into the bladder is absorbed by the body.