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

27 Participants Needed

Trial Summary

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.

What data supports the effectiveness of the drug Cidofovir for treating hemorrhagic cystitis?

Research shows that Cidofovir has been effective in treating hemorrhagic cystitis caused by viruses like BK polyomavirus, especially when other treatments have failed. Patients have experienced significant improvement and reduced viral loads with Cidofovir, and it has been well tolerated with minimal side effects in several cases. ^{1 2 3 4 5}

Is cidofovir safe for treating hemorrhagic cystitis?

Cidofovir has been used to treat hemorrhagic cystitis in patients, and it was generally well tolerated with no side effects in some cases. However, there have been reports of kidney problems and low white blood cell counts (neutropenia) in some patients, indicating potential risks. ^{1 2 5 6 7}

How does the drug cidofovir differ from other treatments for hemorrhagic cystitis?

Cidofovir is unique because it is a nucleotide analog that targets DNA viruses and can be administered directly into the bladder (intravesical instillation) for treating virus-associated hemorrhagic cystitis, unlike other treatments that are primarily supportive, such as hydration and transfusions. It has shown success in cases where standard treatments are not established, particularly in patients with viral infections like BK polyomavirus and adenovirus. ^{1 2 4 5 8}

What is the purpose of this trial?

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 virus as compared to standard of care alone. The safety of cidofovir will also be studied.

Research Team

Borje S. Andersson, MD,PHD

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

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

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)

Treatment Details

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.

Participant Groups

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+

References

1. Denmark pubmed.ncbi.nlm.nih.gov

Neutropenia and renal dysfunction due to intravesical cidofovir for virus-associated hemorrhagic cystitis after kidney and allogenic hematopoietic stem cell transplantations. [2020]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Hemorrhagic cystitis: A successful outcome for a challenging complication in stem cell transplant. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Cidofovir bladder instillation for the treatment of BK hemorrhagic cystitis after allogeneic stem cell transplantation. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Intravesicular cidofovir for the treatment of polyomavirus-associated hemorrhagic cystitis. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Treatment of BK virus-associated hemorrhagic cystitis and simultaneous CMV reactivation with cidofovir. [2018]

6. United States pubmed.ncbi.nlm.nih.gov

Cidofovir in the Treatment of BK Virus-Associated Hemorrhagic Cystitis after Allogeneic Hematopoietic Stem Cell Transplantation. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Efficacy of Cidofovir in Treatment of BK Virus-Induced Hemorrhagic Cystitis in Allogeneic Hematopoietic Cell Transplant Recipients. [2019]

8. United States pubmed.ncbi.nlm.nih.gov

Intravesical instillation of cidofovir in the treatment of hemorrhagic cystitis caused by adenovirus type 11 in a bone marrow transplant recipient. [2018]