Fanconi AnemiaPhase 1NCT00317876
Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia
A Phase 1 study testing cyclophosphamide, cyclosporine and methotrexate in people with Fanconi Anemia, run by Fred Hutchinson Cancer Center at 3 sites in 2 countries.
Trial summary
RATIONALE: Giving low doses of chemotherapy, such as cyclophosphamide, before a donor bone marrow transplant helps stop the growth of abnormal cells. It also stops the patient's immune system from rejecting the donor's bone marrow. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fanconi Anemia
- What is being tested?
- cyclophosphamide, cyclosporine and methotrexate
- Type of treatment
- Surgery or procedure
- How many people
- 25 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Jun 1998to Jul 2003
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
DISEASE CHARACTERISTICS:
- Diagnosis of Fanconi's anemia by chromosome fragility with a diepoxybutane (DEB) or mitomycin C test
- Hemoglobin ≤ 8.0 g/dL, absolute granulocyte count ≤ 1,000/mm^3, or platelet count ≤ 50,000/mm^3
- No refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute leukemia
- HLA-identical related donor available
PATIENT CHARACTERISTICS:
- Glomerular filtration rate ≥ 30% predicted for age
- No liver disease (e.g., active hepatitis or moderate to severe portal fibrosis/cirrhosis by biopsy)
- No symptomatic cardiac insufficiency or symptomatic arrhythmia
- No other diseases that would severely limit the probability of survival
- No HIV seropositivity
- Not pregnant or nursing
- Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
- Not specified
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
Seattle Cancer Care Alliance
Seattle, Washington, 98109-1023
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109-1024
About this study
Official title: Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia
RATIONALE: Giving low doses of chemotherapy, such as cyclophosphamide, before a donor bone marrow transplant helps stop the growth of abnormal cells. It also stops the patient's immune system from rejecting the donor's bone marrow. The donated bone marrow stem cells may replace the patient's immune system and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and methotrexate before or after transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects and best dose of cyclophosphamide in treating patients who are undergoing a donor bone marrow transplant for Fanconi's anemia.
Read the detailed description
OBJECTIVES:
- Decrease the conditioning-related toxicity of cyclophosphamide without decreasing the engraftment rate to < 90% in patients undergoing allogeneic bone marrow transplantation for Fanconi's anemia.
OUTLINE: This is a multicenter, dose-finding study of cyclophosphamide.
- Nonmyeloablative conditioning regimen: Patients receive cyclophosphamide IV on days -5 to -2.
Cohorts of 5-10 patients receive decreasing doses of cyclophosphamide until the optimal dose (OD) is determined. The OD is defined as the dose at which ≥ 4 of 5 patients achieve engraftment and < 1 of 10 patients experiences dose-limiting toxicity.
- Allogeneic bone marrow transplantation (BMT): Patients undergo allogeneic BMT on day 0.
- Graft-vs-host-disease (GVHD) prophylaxis: Patients receive cyclosporine orally or IV twice daily beginning on day -1 and continuing until day 49, followed by a taper on days 50-180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11.
After completion of study treatment, patients are followed periodically for 5 years.
PROJECTED ACCRUAL: A total of 27 patients will be accrued for this study.
- Conditions
- Fanconi Anemia
- Being tested
- cyclophosphamide, cyclosporine, methotrexate, allogeneic bone marrow transplantation, nonmyeloablative allogeneic hematopoietic stem cell transplantation
- Keywords
- Fanconi anemia
- Medical subject headings
- Fanconi Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cyclophosphamideDrug
cyclosporineDrug
methotrexateDrug
allogeneic bone marrow transplantationProcedure or surgery
nonmyeloablative allogeneic hematopoietic stem cell transplantationProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
Conditioning-related toxicity
Measured: 100 days post-transplant
Graft rejection
Measured: 100 days post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2006 (estimated)
- Study startJun 1998
- Primary completionJul 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fanconi AnemiaStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson 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 1
- Primary purpose
- Treatment
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Hans-Peter Kiem, MD, Principal investigator, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00317876
- Sponsor's protocol ID
- 1288.00
- Other IDs
- FHCRC-1288.00, CDR0000481264
- Status last verified
- Apr 2012
- First submitted
- 24 Apr 2006
- Last updated
- 20 Apr 2012
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia
- ClinicalTrials.gov ID
- NCT00317876
- Acronym / sponsor ID
- 1288.00
- Conditions
- Fanconi Anemia
- Keywords
- Fanconi anemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jun 1998
- Primary completion
- Jul 2003 (actual)
- First posted
- 25 Apr 2006 (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.
RATIONALE: Giving low doses of chemotherapy, such as cyclophosphamide, before a donor bone marrow transplant helps stop the growth of abnormal cells. It also stops the patient's immune system from rejecting the donor's bone marrow. The donated bone marrow stem cells may replace the patient's immune system and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and methotrexate before or after transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects and best dose of cyclophosphamide in treating patients who are undergoing a donor bone marrow transplant for Fanconi's anemia.
Detailed description
OBJECTIVES:
- Decrease the conditioning-related toxicity of cyclophosphamide without decreasing the engraftment rate to < 90% in patients undergoing allogeneic bone marrow transplantation for Fanconi's anemia.
OUTLINE: This is a multicenter, dose-finding study of cyclophosphamide.
- Nonmyeloablative conditioning regimen: Patients receive cyclophosphamide IV on days -5 to -2.
Cohorts of 5-10 patients receive decreasing doses of cyclophosphamide until the optimal dose (OD) is determined. The OD is defined as the dose at which ≥ 4 of 5 patients achieve engraftment and < 1 of 10 patients experiences dose-limiting toxicity.
- Allogeneic bone marrow transplantation (BMT): Patients undergo allogeneic BMT on day 0.
- Graft-vs-host-disease (GVHD) prophylaxis: Patients receive cyclosporine orally or IV twice daily beginning on day -1 and continuing until day 49, followed by a taper on days 50-180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11.
After completion of study treatment, patients are followed periodically for 5 years.
PROJECTED ACCRUAL: A total of 27 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Masking
- None (open label)
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| cyclophosphamide | Drug | – |
| cyclosporine | Drug | – |
| methotrexate | Drug | – |
| allogeneic bone marrow transplantation | Procedure or surgery | – |
| nonmyeloablative allogeneic hematopoietic stem cell transplantation | Procedure or surgery | – |
Primary outcome
Conditioning-related toxicity
Time frame: 100 days post-transplant
Graft rejection
Time frame: 100 days post-transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2012.
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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.
Hans-Peter Kiem, MD principal investigator
Fred Hutchinson Cancer Center
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing cyclophosphamide, cyclosporine and methotrexate in people with Fanconi Anemia, run by Fred Hutchinson Cancer Center at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Apr 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Brazil. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Conditioning-related toxicity (time frame: 100 days post-transplant). The study lists 2 primary outcomes in all. 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 20 Apr 2012.