LeukemiaPhase 1/2NCT00112567
Total-Body Irradiation, Thiotepa, and Fludarabine in Treating Young Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer
A Phase 1/2 study testing fludarabine phosphate, thiotepa and peripheral blood stem cell transplantation in people with Leukemia and Myelodysplastic Syndromes, run by Fred Hutchinson Cancer Center at 2 sites in United States.
Trial summary
RATIONALE: Chemotherapy, such as fludarabine and thiotepa, and radiation therapy may destroy cancerous blood-forming cells (stem cells) in the blood and bone marrow. Giving healthy stem cells from a donor whose blood closely resembles the patient's blood will help the patient's bone marrow make new stem cells that… Read more about this study
- Who is it for?
- Ages Up to 20 years
- Conditions the study is for
- Leukemia, Myelodysplastic Syndromes
- What is being tested?
- fludarabine phosphate, thiotepa and peripheral blood stem cell transplantation
- Type of treatment
- Surgery or procedure
- How many people
- 20 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 2003to Jul 2007
- Phase
- Phase 1/2
- 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 Up to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of a life-threatening hematologic malignancy, including any of the following:
- Acute leukemia advanced beyond first remission
- Acute leukemia in first remission* with very high-risk prognostic features, including any of the following:
- Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL)
- ALL or acute myeloid leukemia (AML) with 11q23 chromosomal abnormality
- Hypodiploid ALL
- Failed to achieve first remission within 1 month after induction therapy
- Secondary AML
- Myelodysplastic syndromes with International Prognostic Index score > 1
- Chronic myelogenous leukemia in accelerated or blast phase NOTE: *Must be approved by PCC
- Haploidentical family donor available
- No suitable HLA-matched related or unrelated donor available
- No related donor mismatched for a single HLA-A, -B, -C, -DRB1, or -DQB1 antigen available
PATIENT CHARACTERISTICS:
Age
- Under 21
Performance status
- Not specified
Life expectancy
- At least 6 months
Hematopoietic
- Not specified
Hepatic
- SGPT and SGOT < 2 times upper limit of normal (ULN)*
- Bilirubin < 2 times ULN* NOTE: *Unless due to malignancy
Renal
- Not specified
Cardiovascular
- Ejection fraction ≥ 45%
Pulmonary
- DLCO ≥ 60% of predicted
Other
- Not pregnant or nursing
- Fertile patients must use effective contraception
- HIV negative
PRIOR CONCURRENT THERAPY:
Biologic therapy
- No second bone marrow transplantation, after a first regimen containing total body irradiation
- No concurrent growth factors until day 21 post-transplantation
Chemotherapy
- Not specified
Endocrine therapy
- Not specified
Radiotherapy
- See Biologic therapy
Surgery
- Not specified
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98104
Seattle Cancer Care Alliance
Seattle, Washington, 98109-1023
About this study
Official title: A Phase I/II Study of Total Body Irradiation, Thiotepa, and Fludarabine as Conditioning for Haploidentical CD34+ Purified Peripheral Blood Stem Cell Transplants
RATIONALE: Chemotherapy, such as fludarabine and thiotepa, and radiation therapy may destroy cancerous blood-forming cells (stem cells) in the blood and bone marrow. Giving healthy stem cells from a donor whose blood closely resembles the patient's blood will help the patient's bone marrow make new stem cells that become red blood cells, white blood cells, and platelets.
PURPOSE: This phase I/II trial is studying the side effects of total-body irradiation, fludarabine, and thiotepa and to see how well they work in treating young patients who are undergoing a donor stem cell transplant for hematologic cancer.
Read the detailed description
OBJECTIVES:
Primary
- Determine the safety of a conditioning regimen without anti-thymocyte globulin comprising total body irradiation, thiotepa, and fludarabine followed by CD34-positive-selected haploidentical allogeneic peripheral blood stem cell transplantation in young patients with life-threatening hematologic malignancies.
Secondary
- Determine the risk for severe graft-vs-host disease in patients treated with this regimen.
- Determine the kinetics of immune reconstitution in patients treated with this regimen.
- Determine the risk for life-threatening infections in patients treated with this regimen.
OUTLINE:
- Conditioning regimen: Patients 7 years of age and under undergo total body irradiation twice daily on days -9 to -7. Patients over 7 years of age undergo total body irradiation once on day -7. All patients receive fludarabine IV once daily on days -6 to -2 and thiotepa IV over 2 hours twice on day -5.
- CD34-positive (CD34+)-selected haploidentical allogeneic peripheral blood stem cell transplantation (PBSCT): Patients undergo CD34+-selected allogeneic PBSCT on days 0 and 2.
Patients with acute lymphoblastic leukemia or CNS disease also receive methotrexate intrathecally twice before transplantation and 4 times after day 35 post-transplantation. Male patients with lymphoid malignancies undergo additional radiotherapy to the testes.
After completion of study treatment, patients are followed for at least 100 days, at 1 year, and then periodically thereafter.
PROJECTED ACCRUAL: A total of 20 patients (10 patients ≤ 7 years of age and 10 patients > 7 years of age) will be accrued for this study within 3 years.
- Conditions
- Leukemia, Myelodysplastic Syndromes
- Being tested
- fludarabine phosphate, thiotepa, peripheral blood stem cell transplantation, radiation therapy
- Keywords
- recurrent childhood acute lymphoblastic leukemia, childhood myelodysplastic syndromes, childhood acute lymphoblastic leukemia in remission, recurrent childhood acute myeloid leukemia, childhood acute myeloid leukemia in remission, secondary acute myeloid leukemia, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, childhood chronic myelogenous leukemia, relapsing chronic myelogenous leukemia, de novo myelodysplastic syndromes, secondary myelodysplastic syndromes, previously treated myelodysplastic syndromes
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Accelerated Phase, Blast Crisis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
thiotepaDrug
peripheral blood stem cell transplantationProcedure or surgery
radiation therapyRadiation
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
Secondary outcomes (3)
Risk of severe graft-versus-host disease
Kinetics of immune reconstitution
Risk of life-threatening infections
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2005 (estimated)
- Study startApr 2003
- Study completionJul 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies 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 1/2
- Primary purpose
- Treatment
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Ann E. Woolfrey, MD, Principal investigator, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00112567
- Sponsor's protocol ID
- 1629.00
- Other IDs
- FHCRC-1629.00, CDR0000430650
- Status last verified
- Sep 2010
- First submitted
- 2 Jun 2005
- Last updated
- 21 Sep 2010
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of Total Body Irradiation, Thiotepa, and Fludarabine as Conditioning for Haploidentical CD34+ Purified Peripheral Blood Stem Cell Transplants
- ClinicalTrials.gov ID
- NCT00112567
- Acronym / sponsor ID
- 1629.00
- Conditions
- Leukemia, Myelodysplastic Syndromes
- Keywords
- recurrent childhood acute lymphoblastic leukemia, childhood myelodysplastic syndromes, childhood acute lymphoblastic leukemia in remission, recurrent childhood acute myeloid leukemia, childhood acute myeloid leukemia in remission, secondary acute myeloid leukemia, accelerated phase chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Apr 2003
- Study completion
- Jul 2007
- First posted
- 3 Jun 2005 (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: Chemotherapy, such as fludarabine and thiotepa, and radiation therapy may destroy cancerous blood-forming cells (stem cells) in the blood and bone marrow. Giving healthy stem cells from a donor whose blood closely resembles the patient's blood will help the patient's bone marrow make new stem cells that become red blood cells, white blood cells, and platelets.
PURPOSE: This phase I/II trial is studying the side effects of total-body irradiation, fludarabine, and thiotepa and to see how well they work in treating young patients who are undergoing a donor stem cell transplant for hematologic cancer.
Detailed description
OBJECTIVES:
Primary
- Determine the safety of a conditioning regimen without anti-thymocyte globulin comprising total body irradiation, thiotepa, and fludarabine followed by CD34-positive-selected haploidentical allogeneic peripheral blood stem cell transplantation in young patients with life-threatening hematologic malignancies.
Secondary
- Determine the risk for severe graft-vs-host disease in patients treated with this regimen.
- Determine the kinetics of immune reconstitution in patients treated with this regimen.
- Determine the risk for life-threatening infections in patients treated with this regimen.
OUTLINE:
- Conditioning regimen: Patients 7 years of age and under undergo total body irradiation twice daily on days -9 to -7. Patients over 7 years of age undergo total body irradiation once on day -7. All patients receive fludarabine IV once daily on days -6 to -2 and thiotepa IV over 2 hours twice on day -5.
- CD34-positive (CD34+)-selected haploidentical allogeneic peripheral blood stem cell transplantation (PBSCT): Patients undergo CD34+-selected allogeneic PBSCT on days 0 and 2.
Patients with acute lymphoblastic leukemia or CNS disease also receive methotrexate intrathecally twice before transplantation and 4 times after day 35 post-transplantation. Male patients with lymphoid malignancies undergo additional radiotherapy to the testes.
After completion of study treatment, patients are followed for at least 100 days, at 1 year, and then periodically thereafter.
PROJECTED ACCRUAL: A total of 20 patients (10 patients ≤ 7 years of age and 10 patients > 7 years of age) will be accrued for this study within 3 years.
Study design
- Primary purpose
- Treatment
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, 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 |
|---|---|---|
| fludarabine phosphate | Drug | – |
| thiotepa | Drug | – |
| peripheral blood stem cell transplantation | Procedure or surgery | – |
| radiation therapy | Radiation | – |
Primary outcome
Safety
3 secondary outcomes
Risk of severe graft-versus-host disease
Kinetics of immune reconstitution
Risk of life-threatening infections
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 21 Sep 2010.
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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.
Ann E. Woolfrey, MD principal investigator
Fred Hutchinson Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing fludarabine phosphate, thiotepa and peripheral blood stem cell transplantation in people with Leukemia and Myelodysplastic Syndromes, run by Fred Hutchinson Cancer Center at 2 sites 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?
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: finished, as of the sponsor's last update on 21 Sep 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 combined 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.
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?
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?
Safety. 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 21 Sep 2010.