LeukemiaNCT00301860
Donor Stem Cell Transplant Followed By Donor White Blood Cell Infusions in Treating Young Patients With Hematologic Cancer
A study testing anti-thymocyte globulin, filgrastim and therapeutic allogeneic lymphocytes in people with Leukemia and Lymphoma, run by University of California, San Francisco at 1 site in United States.
Trial summary
RATIONALE: Giving chemotherapy before a donor peripheral blood stem cell transplant helps stop both the growth of cancer cells and the patient's immune system from rejecting the donor's stem cells. Read more about this study
- Who is it for?
- Ages Up to 21 years
- What is being tested?
- anti-thymocyte globulin, filgrastim and therapeutic allogeneic lymphocytes
- Type of treatment
- Surgery or procedure
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2003to Nov 2007
- Phase
- Interventional
- 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
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of one of the following hematopoietic malignancies:
- Acute lymphoblastic leukemia or myeloid leukemia with < 30% blasts in the bone marrow
- Juvenile myelomonocytic leukemia
- Chronic myelogenous leukemia in chronic or accelerated phase
- Relapsed non-Hodgkin's or Hodgkin's lymphoma in at least partial remission
- Considered at high risk (> 30%) of toxic death with standard hematopoietic stem cell transplantation (HSCT), as indicated by at least one of the following:
- Creatinine > 1.5 times normal OR creatinine clearance < 70 mL/min OR tubular damage that is not corrected by cessation of chemotherapy
- DLCO < 60% of predicted OR history of prior intubation due to lung disease (intubation for surgery excluded)
- Shortening fraction < 30%
- History of disseminated fungal infection during chemotherapy OR currently receiving antifungal agents OR history of ≥ 2 septic episodes (confirmed by cultures) that required ICU support
- Patients with improving fungal or other infections eligible
- Improving infection is defined as confirmed negative cultures on 2 separate occasions, at least 1 week apart, and/or stable or improving imaging studies (e.g., CT scan) of the infected site
- Two imaging studies taken at least 2 weeks apart must show stable or improved disease
- History of stroke or abnormal MRI/MRA OR leukoencephalopathy OR seizures that are not fully controlled with anticonvulsants (> 2 episodes of seizures in the preceding year or 1 episode of status epilepticus in a patient who is receiving anticonvulsant therapy)
- History of prior significant bleeding (e.g., pulmonary, CNS, or gastrointestinal) OR history of a clotting disorder as manifested by prior significant thromboses (e.g., superior vena cava, inferior vena cava, or femoral vein)
- Failed conventional therapies and not eligible for myeloablative protocols
- May have failed prior conventional HSCT
- No active CNS leukemia
- Unrelated or related donor available, meeting the following criteria:
- Matched for at least 7/8 loci by high-resolution typing
- One mismatch at A, B, or C loci allowed
- Fully matched at DRB1 locus
PATIENT CHARACTERISTICS:
- ECOG performance status (PS) 0-2 OR Karnofsky PS 60-100%
- No active/progressing viral, bacterial, protozoal, or fungal infection
- Transaminases ≤ 5 times normal (except in the presence of autoimmune liver disease)
- Shortening fraction ≥ 25%
- DLCO ≥ 40% OR pulse oximetry ≥ 85% on room air
- Glomerular filtration rate ≥ 40 mL/min
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
- Prior prolonged intensive chemotherapy (> 3 years of therapy or ≥ 3 different chemotherapeutic protocols) allowed
Trial locations and contacts
1 site in 1 country.
United States1 site
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115
About this study
Official title: Feasibility of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation Followed by Donor Lymphocyte Infusions for Children at High Risk for Complications With Conventional Transplantation
RATIONALE: Giving chemotherapy before a donor peripheral blood stem cell transplant helps stop both the growth of cancer cells and the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving chemotherapy, such as fludarabine and melphalan, and antithymocyte globulin before transplant and cyclosporine and methotrexate after transplant may stop this from happening.
PURPOSE: This clinical trial is studying how well donor stem cell transplant, using low-dose chemotherapy and antithymocyte globulin, followed by donor white blood cell infusions work in treating young patients with hematologic cancer.
Read the detailed description
OBJECTIVES:
- Determine the feasibility of allogeneic hematopoietic stem cell transplantation using a reduced-intensity conditioning regimen, in terms of whole blood engraftment rate at 100 days post transplant, in pediatric patients with hematopoietic malignancies who are at high risk for complications with conventional transplantation.
- Determine the feasibility of donor lymphocyte infusions (DLIs), in terms of number of patients who receive at least one DLI by 12 months post transplant, in patients treated with this regimen.
- Determine the toxicities of the conditioning regimen, in terms of 100-day post transplant nonrelapse-related death rate, in these patients.
- Determine the toxicity of DLI, in terms of acute and chronic graft-vs-host disease rate and 12-month post transplant nonrelapse-related death rate, in these patients.
OUTLINE: This is a pilot study.
- Reduced-intensity conditioning regimen: Patients receive fludarabine IV on days -6 to -2; antithymocyte globulin IV on days -5 to -2; and melphalan IV on days -3 and -2.
- Transplantation: Patients undergo allogeneic peripheral blood stem cell transplantation on day 0. Patients also receive filgrastim (G-CSF) IV beginning on day 5 and continuing until blood counts recover.
- Graft-vs-host disease (GVHD) prophylaxis: Patients receive cyclosporine IV or orally beginning on day -1 and continuing until at least day 28 and methotrexate IV on days 1, 3, and 6.
- Donor lymphocyte infusion (DLI): Patients with mixed chimerism, no acute GVHD requiring therapy, and no relapse/progression post transplant at day 90 may receive DLI. At least 30 days after discontinuation of immunosuppression, patients may receive up to 2 DLIs at least 8-12 weeks apart in the absence of GVHD.
At the completion of study treatment, patients are followed periodically for 2 years.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.
- Being tested
- anti-thymocyte globulin, filgrastim, therapeutic allogeneic lymphocytes, cyclosporine, fludarabine phosphate
- Keywords
- childhood acute lymphoblastic leukemia in remission, childhood acute myeloid leukemia in remission, recurrent childhood acute lymphoblastic leukemia, recurrent childhood acute myeloid leukemia, recurrent childhood small noncleaved cell lymphoma, juvenile myelomonocytic leukemia, chronic phase chronic myelogenous leukemia, accelerated phase chronic myelogenous leukemia, relapsing chronic myelogenous leukemia, recurrent childhood large cell lymphoma, recurrent childhood lymphoblastic lymphoma, recurrent/refractory childhood Hodgkin lymphoma, childhood chronic myelogenous leukemia
- Medical subject headings
- Leukemia, Lymphoma, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Burkitt Lymphoma, Leukemia, Myelomonocytic, Juvenile, Leukemia, Myeloid, Chronic-Phase, Leukemia, Myeloid, Accelerated Phase, Dendritic Cell Sarcoma, Interdigitating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
filgrastimBiological
therapeutic allogeneic lymphocytesBiological
cyclosporineDrug
melphalanDrug
methotrexateDrug
allogeneic hematopoietic stem cell transplantationProcedure or surgery
peripheral blood stem cell transplantationProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2006 (estimated)
- Study startJan 2003
- Primary completionMar 2007
- Study completionNov 2007
Why it stopped: lack of efficacy
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 University of California, San FranciscoThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Biljana Horn, MD, Study chair, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00301860
- Sponsor's protocol ID
- CDR0000462439
- Other IDs
- UCSF-02164, UCSF-H10216-21819-03
- Status last verified
- Jul 2013
- First submitted
- 9 Mar 2006
- Last updated
- 15 Jul 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation Followed by Donor Lymphocyte Infusions for Children at High Risk for Complications With Conventional Transplantation
- ClinicalTrials.gov ID
- NCT00301860
- Acronym / sponsor ID
- CDR0000462439
- Keywords
- childhood acute lymphoblastic leukemia in remission, childhood acute myeloid leukemia in remission, recurrent childhood acute lymphoblastic leukemia, recurrent childhood acute myeloid leukemia, recurrent childhood small noncleaved cell lymphoma, juvenile myelomonocytic leukemia, chronic phase chronic myelogenous leukemia, accelerated phase chronic myelogenous leukemia
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: lack of efficacy
- Enrolment
- 8 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jan 2003
- Primary completion
- Mar 2007 (actual)
- Study completion
- Nov 2007 (actual)
- First posted
- 13 Mar 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 chemotherapy before a donor peripheral blood stem cell transplant helps stop both the growth of cancer cells and the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving chemotherapy, such as fludarabine and melphalan, and antithymocyte globulin before transplant and cyclosporine and methotrexate after transplant may stop this from happening.
PURPOSE: This clinical trial is studying how well donor stem cell transplant, using low-dose chemotherapy and antithymocyte globulin, followed by donor white blood cell infusions work in treating young patients with hematologic cancer.
Detailed description
OBJECTIVES:
- Determine the feasibility of allogeneic hematopoietic stem cell transplantation using a reduced-intensity conditioning regimen, in terms of whole blood engraftment rate at 100 days post transplant, in pediatric patients with hematopoietic malignancies who are at high risk for complications with conventional transplantation.
- Determine the feasibility of donor lymphocyte infusions (DLIs), in terms of number of patients who receive at least one DLI by 12 months post transplant, in patients treated with this regimen.
- Determine the toxicities of the conditioning regimen, in terms of 100-day post transplant nonrelapse-related death rate, in these patients.
- Determine the toxicity of DLI, in terms of acute and chronic graft-vs-host disease rate and 12-month post transplant nonrelapse-related death rate, in these patients.
OUTLINE: This is a pilot study.
- Reduced-intensity conditioning regimen: Patients receive fludarabine IV on days -6 to -2; antithymocyte globulin IV on days -5 to -2; and melphalan IV on days -3 and -2.
- Transplantation: Patients undergo allogeneic peripheral blood stem cell transplantation on day 0. Patients also receive filgrastim (G-CSF) IV beginning on day 5 and continuing until blood counts recover.
- Graft-vs-host disease (GVHD) prophylaxis: Patients receive cyclosporine IV or orally beginning on day -1 and continuing until at least day 28 and methotrexate IV on days 1, 3, and 6.
- Donor lymphocyte infusion (DLI): Patients with mixed chimerism, no acute GVHD requiring therapy, and no relapse/progression post transplant at day 90 may receive DLI. At least 30 days after discontinuation of immunosuppression, patients may receive up to 2 DLIs at least 8-12 weeks apart in the absence of GVHD.
At the completion of study treatment, patients are followed periodically for 2 years.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 21 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 |
|---|---|---|
| anti-thymocyte globulin | Biological | – |
| filgrastim | Biological | – |
| therapeutic allogeneic lymphocytes | Biological | – |
| cyclosporine | Drug | – |
| fludarabine phosphate | Drug | – |
| melphalan | Drug | – |
| methotrexate | Drug | – |
| allogeneic hematopoietic stem cell transplantation | Procedure or surgery | – |
| peripheral blood stem cell transplantation | Procedure or surgery | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early (reason given: lack of efficacy). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jul 2013.
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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.
Biljana Horn, MD study chair
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing anti-thymocyte globulin, filgrastim and therapeutic allogeneic lymphocytes in people with Leukemia and Lymphoma, run by University of California, San Francisco 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?
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: stopped early, as of the sponsor's last update on 15 Jul 2013. 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.
Are results available for this study?
The study was stopped early (reason given: lack of efficacy). 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.
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 15 Jul 2013; AzTrials last refreshed its copy on 7 Nov 2026.