Myelofibrosis Clinical Trial
Official title:
Pilot Safety and Feasibility Trial of Mycophenolate and Sirolimus for Prevention of GVHD in Mismatched Unrelated and Related Donor Hematopoietic Stem Cell Transplantation for Hematologic Malignancies
Verified date | August 2018 |
Source | Stanford University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This pilot phase I/II trial studies the side effects and how well sirolimus and mycophenolate mofetil work in preventing graft versus host disease (GvHD) in patients with hematologic malignancies undergoing hematopoietic stem cell transplant (HSCT). Biological therapies, such as sirolimus and mycophenolate mofetil, use substances made from living organisms that may stimulate or suppress the immune system in different ways and stop tumor cells from growing. Giving sirolimus and mycophenolate mofetil after hematopoietic stem cell transplant may be better in preventing graft-versus-host disease.
Status | Terminated |
Enrollment | 1 |
Est. completion date | July 2018 |
Est. primary completion date | April 2017 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 3 Years to 30 Years |
Eligibility |
Inclusion Criteria: - Subjects must have one of the following disease categories: - Acute myeloid leukemia (AML) beyond 2nd remission or relapsed/refractory disease - Acute lymphoblastic leukemia (ALL) beyond 2nd remission or relapsed/refractory disease - Chronic myeloid leukemia (CML) beyond 2nd chronic phase or in blast crises - Myelodysplastic syndrome (MDS) - Myeloproliferative disorders including myeloid metaplasia and myelofibrosis - High risk non-Hodgkin's lymphoma (NHL) in first remission - Relapsed or refractory NHL - Hodgkin's lymphoma (HL) beyond first remission - Performance status by Karnofsky of >= 70% or Lansky > 70% for patients < 16 years of age - Human leukocyte antigen (HLA) mismatched related or unrelated donor identified 8/10 or 9/10 - Willingness to take oral medications during the transplantation period - Willingness and ability to sign a written informed consent (assent if applicable) Exclusion Criteria: - Prior myeloablative allogeneic or autologous HSCT - Human immunodeficiency virus (HIV) infection - Pregnant or lactating females - Evidence of uncontrolled active infection - Down syndrome - Serum creatinine (CR) < 1.5mg/dl or 24 hour CR clearance < 50 ml/min - Direct bilirubin > 2 x upper limit of normal (ULN) - Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 x ULN - Carbon monoxide diffusing capability test (DLCO) > 60% predicted and in children- room air oxygen saturation > 92% - Left ventricular ejection fraction < 45% and in children-shortening fraction < 26% - Fasting cholesterol > 300 mg/dl or triglycerides > 300 while on lipid lowering agents - Patients who have received an investigational drug within 30 days of enrollment in study - Patients with prior malignancies except basal cell carcinoma or treated carcinoma in-situ; cancer treated with curative intent > 5 years will be allowed; cancer treatment with curative intent =< 5 years will not be allowed |
Country | Name | City | State |
---|---|---|---|
United States | Stanford University, School of Medicine | Palo Alto | California |
Lead Sponsor | Collaborator |
---|---|
Stanford University |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Incidence of acute GvHD grade 3 & 4, according to the Glucksberg staging criteria | Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum. | Up to 60 days post-transplant | |
Primary | Incidence of acute GvHD grade 3 & 4, according to the Glucksberg staging criteria | Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum. | Up to 100 days post-transplant | |
Secondary | Incidence of thrombotic microangiopathy defined according to the bone marrow transplant clinical trials network toxicity committee | Defined as: red blood cell fragmentation and at least two schistocytes per high-power field on peripheral smear; concurrent increased serum lactate dehydrogenase measurement above institutional baseline; concurrent doubling of serum creatinine or 50% increase in creatinine clearance from baseline and/or neurological dysfunction without other explanations; and negative direct and indirect Coombs. Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum. | Up to 100 days | |
Secondary | Incidence of venous-occlusive disease (VOD) using Modified Seattle Criteria | Severe VOD will be considered a dose limiting toxicity. Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum. | Up to 100 days | |
Secondary | Severity of mucositis determined by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.03 | Grade III and IV will be considered dose limiting toxicities. Statistical analysis results will be reported using summary tables, figures, and data listings. Categorical variables will be summarized by numbers and percentages of subjects in corresponding categories. | Up to 100 days | |
Secondary | Time to neutrophil engraftment defined as first of 3 consecutive days with the absolute neutrophil count is > 500/ul in the peripheral blood | Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum. | Baseline to up to 100 days | |
Secondary | Time to platelet engraftment defined as the first day of a minimum of three consecutive measurements on different days when platelet count > 50,000/mm^3 and patient is transfusion-independent for a minimum of 7 days | Statistical analysis results will be reported using summary tables, figures, and data listings. Continuous variables will be summarized using the mean, standard deviation, median, minimum, and maximum. | Baseline to up to 100 days |
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