Graft vs Host DiseasePhase 1NCT00001748
HLA-Mismatched Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Hematological Malignancies
A Phase 1 study testing Peripheral blood progenitor cell transplant in people with Graft vs Host Disease and Hematologic Neoplasms, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Many patients with hematological malignancies potentially curable by bone marrow transplantation are not considered for transplantation because an HLA identical family or unrelated donor is unavailable. For these patients the only curative option is a transplant from a partially matched family donor. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Graft vs Host Disease, Hematologic Neoplasms, Lymphoma
- What is being tested?
- Peripheral blood progenitor cell transplant
- Type of treatment
- Surgery or procedure
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 1998to May 2000
- Phase
- Phase 1
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
Patient:
Ages 10-45 years.
Chronic myelogenous leukemia, any of these categories: accelerated phase or blast transformation.
Acute lymphoblastic leukemia, any of these categories: Adults (greater than 18 years) in any remission with high-risk features (presenting leukocyte count greater than 100,000/cu mm, Karyotypes t9; 22, t4, t19, t11, biphenotypic leukemia). All second remissions, primary induction failure including partial remission, partially responding or untreated relapse.
Acute myelogenous leukemia (AML): All AML in second or subsequent remission, primary induction failure or partial remission and resistant relapse.
Myelodysplastic syndromes, any of these categories: refractory anemia with excess of blasts, transformation to acute leukemia, chronic myelomonocytic leukemia.
Myeloproliferative disorders undergoing transformation to terminal stages.
Chronic lymphocytic leukemia (CLL) in Richter transformation.
High-grade lymphoma, refractory to standard treatment approaches, mantle cell lymphoma.
No major organ dysfunction precluding transplantation.
DLCO greater than 65% predicted.
Left ventricular ejection fraction: greater than 40% predicted.
ECOG performance status of 0 or 1.
Informed consent given. Informed consent from parents for minors.
Women of childbearing age with a negative pregnancy test may participate.
Donor:
Partially HLA matched family donor (3-5/6 matches).
Fit to receive G-CSF and give peripheral blood stem cells (normal blood count, normotensive, and no history of stroke).
Informed consent given.
Patients or donors must not be pregnant or nursing.
Must not have ECOG performance status of 2 or more.
No severe psychiatric illness in patient or donor: Mental deficiency sufficiently severe as to make compliance with the BMT treatment unlikely and making informed consent impossible.
No major anticipated illness or organ failure incompatible with survival from BMT.
DLCO must not be less than 65% predicted.
No left ventricular ejection fraction: less than 40% predicted.
Must not have serum creatinine greater than 3 mg/dl.
Must not have serum bilirubin greater than 4 mg/dl, Transaminases greater than 3 times the upper limit of normal.
Donor or patient must not be HIV positive.
Must not have history of other malignancies except basal cell or squamous carcinoma of the skin, positive PAP smear and subsequent negative follow up.
Donor must be fit to receive G-CSF and undergo apheresis.
Must not fail to mobilize adequate numbers of CD34+ cells after two cycles of G-CSF.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Heart, Lung and Blood Institute (NHLBI)
Bethesda, Maryland, 20892
About this study
Many patients with hematological malignancies potentially curable by bone marrow transplantation are not considered for transplantation because an HLA identical family or unrelated donor is unavailable. For these patients the only curative option is a transplant from a partially matched family donor. Such transplants are feasible but are less successful than matched sibling donor transplants. The main problems with mismatched transplants are graft rejection, graft-vs-host disease, and regimen-related mortality. This restricts the use of mismatched transplants to patients less than 45 years at high risk of dying from the hematological malignancy.
This protocol evaluates a new preparative regimen designed to ensure stem cell engraftment by increased immunosuppression, followed by a G-CSF mobilized T cell depleted, stem cell rich, peripheral blood progenitor cell (PBPC) transplant from a mismatched related donor in patients with high risk hematological malignancies.
Read more
This phase I study evaluates engraftment and GVHD following T cell depleted, HLA-mismatched PBPC transplants. Stopping rules will be used to make modifications to the protocol in the event of graft failure.
The end points of the study are graft take, acute and chronic GVHD, leukemic relapse, transplant-related mortality, death and leukemia-free survival. Patients will be followed up for 5 years. It is planned to treat up to 35 patients aged between 10 and 45 years.
Read the detailed description
Many patients with hematological malignancies potentially curable by bone marrow transplantation are not considered for transplantation because an HLA identical family or unrelated donor is unavailable. For these patients the only curative option is a transplant from a partially matched family donor. Such transplants are feasible but are less successful than matched sibling donor transplants. The main problems with mismatched transplants are graft rejection, graft-vs-host disease, and regimen-related mortality. This restricts the use of mismatched transplants to patients less than 45 years at high risk of dying from the hematological malignancy.
This protocol evaluates a new preparative regimen designed to ensure stem cell engraftment by increased immunosuppression, followed by a G-CSF mobilized T cell depleted, stem cell rich, peripheral blood progenitor cell (PBPC) transplant from a mismatched related donor in patients with high risk hematological malignancies.
This phase I study evaluates engraftment and GVHD following T cell depleted, HLA-mismatched PBPC transplants. Stopping rules will be used to make modifications to the protocol in the event of graft failure.
The end points of the study are graft take, acute and chronic GVHD, leukemic relapse, transplant-related mortality, death and leukemia-free survival. Patients will be followed up for 5 years. It is planned to treat up to 35 patients aged between 10 and 45 years.
- Conditions
- Graft vs Host Disease, Hematologic Neoplasms, Lymphoma, Myelodysplastic Syndromes, Myeloid Leukemia
- Being tested
- Peripheral blood progenitor cell transplant
- Keywords
- Cyclophosphamide, Donor Apheresis, Graft vs. Host Disease, Graft-Versus-Leukemia, Leukemic Relapse, Myeloma, Peripheral Blood Stem Cells, Whole Body Irradiation, Acute Lymphoblastic Leukemia (ALL), Chronic Lymphocytic Leukemia (CLL), Chronic Myelogenous Leukemia (CML), Hematological Malignancies, Myelodysplastic Syndrome, Myeloproliferative Disorders
- Medical subject headings
- Graft vs Host Disease, Hematologic Neoplasms, Lymphoma, Myelodysplastic Syndromes, Leukemia, Myeloid, Neoplasms, Plasma Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Lymphocytic, Chronic, B-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Peripheral blood progenitor cell transplantProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2002 (estimated)
- Study startJun 1998
- Study completionMay 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Graft vs Host DiseaseStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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
- Enrolment
- 35 participants
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001748
- Sponsor's protocol ID
- 980122
- Other IDs
- 98-H-0122
- Status last verified
- Aug 1999
- First submitted
- 3 Nov 1999
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Lane TA, Law P, Maruyama M, Young D, Burgess J, Mullen M, Mealiffe M, Terstappen LW, Hardwick A, Moubayed M, et al. Harvesting and enrichment of hematopoietic progenitor cells mobilized into the peripheral blood of normal donors by granulocyte-macrophage colony-stimulating factor (GM-CSF) or G-CSF: potential role in allogeneic marrow transplantation. Blood. 1995 Jan 1;85(1):275-82.
PubMed 7528570