Acute LeukemiasPhase 4NCT01339988
Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide for Hematological Malignancies Hematocrit (HCT)
A Phase 4 study testing Busulfan/Cyclophosphamide in people with Acute Leukemias and Chronic Leukemias, run by Tel-Aviv Sourasky Medical Center.
Trial summary
Long-term follow-up studies have demonstrated significant late toxicities of total body irradiation (TBI), which are most marked in children radiated at a young age. Growth failure, decline in cognitive function, and endocrine abnormalities have all been described. Read more about this study
- Who is it for?
- Ages Up to 21 years, men only
- Conditions the study is for
- Acute Leukemias, Chronic Leukemias, Myelodysplastic Syndrome
- What is being tested?
- Busulfan/Cyclophosphamide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- No sites listed yet
- Study dates
- Jun 2011to Jun 2019
- Phase
- Phase 4
- 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 21 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Suitable cord blood from matched unrelated or related donor.
- Cardiac (Echo/EKG): shortening fraction ≥ 27%
- Electrolytes within normal CCHMC limits.
- Pulmonary function tests: DLCO ≥ 50%
- Renal: creatinine clearance/GFR ≥ 60 ml/minute/1.73m2
- Lumbar puncture: no leukemic infiltrate.
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- CBC: ANC ≥ 1000ml and unsupported platelet count of ≥ 50,000/ml
- Documented HSV and CMV titers, Hepatitis B surface antigen, Hepatitis C by serology, HIV by serology: all negative.
- Hepatic transaminases < 2.5x normal; Total bilirubin < 2 mg/dl Patients who do not meet above organ function criteria (liver, cardiac, renal), due to the presence of a tumor compromising these organs, may have exception made for these criteria and remain eligible for treatment plan after consultation with Program Director of Blood and Marrow Transplant
Who cannot join
- Patients with neoplastic or non-neoplastic disease of any major organ system that would compromise their ability to withstand the pre-transplant conditioning regimen.
- Patients with uncontrolled (culture or biopsy positive) infections requiring intravenous antivirals, antibiotics, or antifungals. Patients on prolonged antifungal therapy with a history of fungal infection should be considered for a non-myeloablative protocol.
- Patients who are pregnant or lactating. Patients of childbearing potential must practice an effective method of birth control while participating on this treatment plan.
- HIV seropositive patients
About this study
Official title: Treatment Plan for Hematologic Malignancies Using Intravenous Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide to Examine Results, Success and Side Effects of Treatment With Chemotherapy Only, as a Preparative Therapy for Patients With Cord Blood Transplants
Long-term follow-up studies have demonstrated significant late toxicities of total body irradiation (TBI), which are most marked in children radiated at a young age. Growth failure, decline in cognitive function, and endocrine abnormalities have all been described. Good outcomes can be achieved with alkylating agents only as a preparative regimen. This plan will use a combination of busulfan and cyclophosphamide (Bu/Cy) with or without antithymocyte globulin (ATG) to reduce the late toxicities of therapy that includes TBI.
- Conditions
- Acute Leukemias, Chronic Leukemias, Myelodysplastic Syndrome, Juvenile Myelomonocytic Leukemia
- Being tested
- Busulfan/Cyclophosphamide
- Keywords
- Stem-cells, Transplantation, Busulfex, Cyclophosphamide, Total-Body-Ittadiation
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Juvenile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Busulfan/CyclophosphamideDrug
Also called Cytoxan
Conditioning regimen:
Cyclophosphamide 50mg/kg/day for 4 days + Busulfan 0.8-1.0mg/kg/day for 4 days.
| Study group | Role | What they receive |
|---|---|---|
| Busulfan/Cyclophosphamide | Experimental | Not described Receives: Drug: Busulfan/Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Measured: Five years
Secondary outcomes (1)
Disease free survival
Measured: Five years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2011 (estimated)
- Study startJun 2011
- Primary completionJun 2014 (estimated)
- Study completionJun 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute LeukemiasStudies of the same condition, open and under way.
- More trials run by Tel-Aviv Sourasky Medical 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tel-Aviv Sourasky Medical Center
- Sponsor type
- Other government
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01339988
- Sponsor's protocol ID
- TASMC-11-MB-442-CTIL
- Status last verified
- Apr 2011
- First submitted
- 20 Apr 2011
- Last updated
- 21 Apr 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment Plan for Hematologic Malignancies Using Intravenous Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide to Examine Results, Success and Side Effects of Treatment With Chemotherapy Only, as a Preparative Therapy for Patients With Cord Blood Transplants
- ClinicalTrials.gov ID
- NCT01339988
- Acronym / sponsor ID
- TASMC-11-MB-442-CTIL
- Conditions
- Acute Leukemias, Chronic Leukemias, Myelodysplastic Syndrome, Juvenile Myelomonocytic Leukemia
- Keywords
- Stem-cells, Transplantation, Busulfex, Cyclophosphamide, Total-Body-Ittadiation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Tel-Aviv Sourasky Medical CenterOther government
- Study start
- Jun 2011
- Primary completion
- Jun 2014 (estimated)
- Study completion
- Jun 2019 (estimated)
- First posted
- 21 Apr 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.
Long-term follow-up studies have demonstrated significant late toxicities of total body irradiation (TBI), which are most marked in children radiated at a young age. Growth failure, decline in cognitive function, and endocrine abnormalities have all been described. Good outcomes can be achieved with alkylating agents only as a preparative regimen. This plan will use a combination of busulfan and cyclophosphamide (Bu/Cy) with or without antithymocyte globulin (ATG) to reduce the late toxicities of therapy that includes TBI.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Busulfan/Cyclophosphamide | Experimental | Drug: Busulfan/Cyclophosphamide |
Who may qualify
- Ages
- Up to 21 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Suitable cord blood from matched unrelated or related donor.
- Cardiac (Echo/EKG): shortening fraction ≥ 27%
- Electrolytes within normal CCHMC limits.
- Pulmonary function tests: DLCO ≥ 50%
- Renal: creatinine clearance/GFR ≥ 60 ml/minute/1.73m2
Key exclusion criteria (4 listed)
- Patients with neoplastic or non-neoplastic disease of any major organ system that would compromise their ability to withstand the pre-transplant conditioning regimen.
- Patients with uncontrolled (culture or biopsy positive) infections requiring intravenous antivirals, antibiotics, or antifungals. Patients on prolonged antifungal therapy with a history of fungal infection should be considered for a non-myeloablative protocol.
- Patients who are pregnant or lactating. Patients of childbearing potential must practice an effective method of birth control while participating on this treatment plan.
- HIV seropositive patients
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 |
|---|---|---|
| Busulfan/CyclophosphamideAlso: CytoxanConditioning regimen: Cyclophosphamide 50mg/kg/day for 4 days + Busulfan 0.8-1.0mg/kg/day for 4 days. | Drug | Busulfan/Cyclophosphamide |
Primary outcome
Overall survival
Time frame: Five years
1 secondary outcome
Disease free survival
Time frame: Five years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2011.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Busulfan/Cyclophosphamide in people with Acute Leukemias and Chronic Leukemias, run by Tel-Aviv Sourasky Medical Center.
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 9 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: status not confirmed, as of the sponsor's last update on 21 Apr 2011. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Overall survival (time frame: Five years). 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 Apr 2011.