Transplantation InfectionPhase 2NCT01295645
Cidofovir Versus Best Supportive Care for Hemorrhagic Cystitis
A Phase 2 study testing Cidofovir and No Cidofovir in people with Transplantation Infection, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Transplantation Infection
- What is being tested?
- Cidofovir and No Cidofovir
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Mar 2011to 15 Mar 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- HSCT patients with symptomatic hemorrhagic cystitis (minimal Grade 1 symptoms of HC per NCI criteria) and positive BKV in urine >1x103 DNA copies/ml
- Age >/= 6 years
- Patient must sign the informed consent document.
Who cannot join
- Creatine clearance < 55 ml/min, calculated using ideal body weight (IBW) using Cockcroft-Gault equation
- Concomitant use of foscarnet, liposomal amphotericin B or aminoglycoside
- Use of cidofovir for bladder instillation
- Use of formalin or hyperbaric oxygen treatment
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: A Phase II Randomized Study of Cidofovir Versus Best Supportive Care for Polyomavirus Hominis Type I (BK) Virus Related Hemorrhagic Cystitis After Stem Cell Transplant
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.
Read the detailed description
The Study Drug:
Cidofovir is an anti-viral drug that is designed to treat or prevent infections caused by certain kinds of viruses.
Study Groups:
If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 study groups and you will have an equal chance of being in either group:
- If you are in Group 1, you will receive standard of care drugs and cidofovir. The doctor will tell you more about the standard of care drugs that you will take and the risks for them.
- If you are in Group 2, you will only receive standard of care drugs.
Study Drug Administration:
If you are in Group 1, you will receive cidofovir by vein over about 1 hour 3 times per week. You will also receive standard of care, which may include oral pain drugs taken every 4 to 6 hours as needed, oral drugs for urinary urgency taken 2 times daily, and fluids given by vein to increase your urination rate.
If you are in Group 2, you will only receive the standard of care, as described above.
Study Visits:
Each week:
- Blood (about 2 tablespoons) will be drawn for routine tests.
- You will have a physical exam, including measurement of your vital signs.
Every 2 weeks, you will complete the questionnaire about urinary problems.
Length of Study:
If you are in Group 1, you may continue taking the study drug for up to 4 weeks. Group 2 will take the standard of care for 4 weeks. If the doctor thinks it is needed Groups 1 and 2 can continue to receive standard of care after this study is over. If your symptoms get worse during the study and you are not receiving cidofovir, you may be eligible for further treatments, which may include cidofovir, after the study ends. You will no longer be able to take the study drug if the disease gets worse or intolerable side effects occur.
Your participation on the study will be over once you have completed the end-of-treatment visit and the follow-up visit.
End-of-Treatment Visit:
After you finish treatment:
- Urine will be collected to test the level of BK virus.
- You will complete the questionnaire about urinary problems.
Follow-up Visit:
Four (4) weeks after treatment ends:
- You will have a physical exam, including measurement of your vital signs.
- Urine will be collected to test the level of BK virus.
This is an investigational study. Cidofovir is FDA approved and commercially available for the treatment of several viral infections. Its use in patients with the BK virus after a stem cell transplant is investigational.
Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Transplantation Infection
- Being tested
- Cidofovir, No Cidofovir
- Keywords
- Stem Cell Transplant, Hemorrhagic Cystitis, Bladder inflammation, Frequent urination, Pain, Polyomavirus hominis type I, BK virus, BKV, Cidofovir, Vistide
- Medical subject headings
- Cystitis, Hemorrhagic, Cystitis, Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CidofovirDrug
Also called Vistide
0.5 mg/kg by vein (IV) 3 times a week for 4 weeks.
No CidofovirOther
Standard of Care: Pharmacologic management of pain, spasms, and urinary urgency with medications, hyper-hydration, or continuous bladder irrigation.
| Study group | Role | What they receive |
|---|---|---|
| Standard of Care + Cidofovir | Experimental | Cidofovir 0.5 mg/kg IV 3 x week for 4 weeks Receives: Drug: Cidofovir |
| No Cidofovir | Active comparator | Standard of Care: Pharmacologic management of pain, spasms, and urinary urgency with medications, hyper-hydration, or continuous bladder irrigation. Receives: Other: No Cidofovir |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients with Microbiologic Response
Response defined as improvement in symptoms based on questionnaire, reduction or stability in grade of hemorrhagic cystitis, and level of virus must be a 1 log reduction from baseline when the BKV was first diagnosed.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2011 (estimated)
- Study start17 Mar 2011
- Primary completion15 Mar 2028 (estimated)
- Study completion15 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transplantation InfectionStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Borje S. Andersson, MD,PHD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01295645
- Sponsor's protocol ID
- 2010-0518
- Other IDs
- NCI-2011-00250
- Status last verified
- Sep 2026
- First submitted
- 10 Feb 2011
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Randomized Study of Cidofovir Versus Best Supportive Care for Polyomavirus Hominis Type I (BK) Virus Related Hemorrhagic Cystitis After Stem Cell Transplant
- ClinicalTrials.gov ID
- NCT01295645
- Acronym / sponsor ID
- 2010-0518
- Conditions
- Transplantation Infection
- Keywords
- Stem Cell Transplant, Hemorrhagic Cystitis, Bladder inflammation, Frequent urination, Pain, Polyomavirus hominis type I, BK virus, BKV
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 27 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 17 Mar 2011 (actual)
- Primary completion
- 15 Mar 2028 (estimated)
- Study completion
- 15 Mar 2028 (estimated)
- First posted
- 14 Feb 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The Study Drug:
Cidofovir is an anti-viral drug that is designed to treat or prevent infections caused by certain kinds of viruses.
Study Groups:
If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 study groups and you will have an equal chance of being in either group:
- If you are in Group 1, you will receive standard of care drugs and cidofovir. The doctor will tell you more about the standard of care drugs that you will take and the risks for them.
- If you are in Group 2, you will only receive standard of care drugs.
Study Drug Administration:
If you are in Group 1, you will receive cidofovir by vein over about 1 hour 3 times per week. You will also receive standard of care, which may include oral pain drugs taken every 4 to 6 hours as needed, oral drugs for urinary urgency taken 2 times daily, and fluids given by vein to increase your urination rate.
If you are in Group 2, you will only receive the standard of care, as described above.
Study Visits:
Each week:
- Blood (about 2 tablespoons) will be drawn for routine tests.
- You will have a physical exam, including measurement of your vital signs.
Every 2 weeks, you will complete the questionnaire about urinary problems.
Length of Study:
If you are in Group 1, you may continue taking the study drug for up to 4 weeks. Group 2 will take the standard of care for 4 weeks. If the doctor thinks it is needed Groups 1 and 2 can continue to receive standard of care after this study is over. If your symptoms get worse during the study and you are not receiving cidofovir, you may be eligible for further treatments, which may include cidofovir, after the study ends. You will no longer be able to take the study drug if the disease gets worse or intolerable side effects occur.
Your participation on the study will be over once you have completed the end-of-treatment visit and the follow-up visit.
End-of-Treatment Visit:
After you finish treatment:
- Urine will be collected to test the level of BK virus.
- You will complete the questionnaire about urinary problems.
Follow-up Visit:
Four (4) weeks after treatment ends:
- You will have a physical exam, including measurement of your vital signs.
- Urine will be collected to test the level of BK virus.
This is an investigational study. Cidofovir is FDA approved and commercially available for the treatment of several viral infections. Its use in patients with the BK virus after a stem cell transplant is investigational.
Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard of Care + Cidofovir | Experimental | Drug: CidofovirCidofovir 0.5 mg/kg IV 3 x week for 4 weeks |
| No Cidofovir | Active comparator | Other: No CidofovirStandard of Care: Pharmacologic management of pain, spasms, and urinary urgency with medications, hyper-hydration, or continuous bladder irrigation. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- HSCT patients with symptomatic hemorrhagic cystitis (minimal Grade 1 symptoms of HC per NCI criteria) and positive BKV in urine >1x103 DNA copies/ml
- Age >/= 6 years
- Patient must sign the informed consent document.
Key exclusion criteria (4 listed)
- Creatine clearance < 55 ml/min, calculated using ideal body weight (IBW) using Cockcroft-Gault equation
- Concomitant use of foscarnet, liposomal amphotericin B or aminoglycoside
- Use of cidofovir for bladder instillation
- Use of formalin or hyperbaric oxygen treatment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CidofovirAlso: Vistide0.5 mg/kg by vein (IV) 3 times a week for 4 weeks. | Drug | Standard of Care + Cidofovir |
| No CidofovirStandard of Care: Pharmacologic management of pain, spasms, and urinary urgency with medications, hyper-hydration, or continuous bladder irrigation. | Other | No Cidofovir |
Primary outcome
Number of Patients with Microbiologic Response
Time frame: 8 weeks
Response defined as improvement in symptoms based on questionnaire, reduction or stability in grade of hemorrhagic cystitis, and level of virus must be a 1 log reduction from baseline when the BKV was first diagnosed.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Borje S. Andersson, MD,PHD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cidofovir and No Cidofovir in people with Transplantation Infection, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients with Microbiologic Response (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026; AzTrials last refreshed its copy on 26 Oct 2026.