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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 22 February 2021
Main ID:  NCT01384513
Date of registration: 27/06/2011
Prospective Registration: Yes
Primary sponsor: Sidney Kimmel Cancer Center at Thomas Jefferson University
Public title: A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies
Scientific title: A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies
Date of first enrolment: August 4, 2011
Target sample size: 40
Recruitment status: Active, not recruiting
URL:  https://clinicaltrials.gov/show/NCT01384513
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Dolores Grosso, DNP, CRNP
Address: 
Telephone:
Email:
Affiliation:  Sidney Kimmel Cancer Center at Thomas Jefferson University
Name:     Neal Flomenberg, MD
Address: 
Telephone:
Email:
Affiliation:  Sidney Kimmel Cancer Center at Thomas Jefferson University
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Any patient with hematologic or oncologic diagnosis in which allogeneic HSCT is
thought to be beneficial, and in whom front-line therapy has already been applied.
Patients treated on this protocol will be without morphological evidence of disease
(complete remission or "CR"), or if the patient has evidence of disease, the patient
must have had at least a good partial response (PR) to the most recent therapy and the
disease must be chemoresponsive.

2. Patients treated on this study will have:

- Acute leukemia in 1st or 2nd CR

- MDS (myelodysplastic syndrome), specific subtypes of RA (refractory anemia) or
RARS (refractory anemia with ringed sideroblasts) subtypes.

- Hodgkin or Indolent Non-Hodgkin's lymphoma with chemosensitive disease

- Myeloma without morphological evidence of disease, or a deep PR to the most
recent therapy

- Myeloproliferative disorders with at least a PR to current therapy

- Aplastic Anemia

- A hematological or oncological disease (not listed) that meets the criteria
reviewed above (in CR or with a good PR).

3. Patients must have a related donor who is HLA mismatched at 2, 3, or 4 antigens at the
HLA-A; B; C; DR loci in the GVHD direction. (Patients with related donors who are HLA
identical or are a 1-antigen mismatch may be treated on this therapeutic approach, but
will have their outcomes will not be part of the statistical aims of the study (see
Summary section).

4. Patients must adequate organ function:

- LVEF (Left ventricular end diastolic function) of >50%

- DLCO (Diffusing Capacity of the Lung for Carbon Monoxide ) =50% of predicted
corrected for hemoglobin

- Adequate liver function as defined by a serum bilirubin <1.8, AST or ALT < 2.5X
upper limit of normal

- Creatinine Clearance of = 60 mL/min

5. Performance status = 80% (TJU Karnofsky) for patients = 60 years old or =70% for
patients < 60 years old.

6. HCT-CI Score = 4 points for patients = 60 years old or = 5 points for patients < 60
years old.

7. Patients must be willing to use contraception if they have childbearing potential

8. Able to give informed consent

Exclusion Criteria:

1. Performance status < 80% (TJU Karnofsky) for patients = 60 years old or <70% for
patients < 60.

2. Hematopoietic Cell Transplant-Comorbidity Index (HCT-CI) Score > 4 points for patients
= 60 years old or > 5 points for patients < 60.

3. HIV positive

4. Active involvement of the central nervous system with malignancy

5. Inability to obtain informed consent

6. Pregnancy

7. Patients with life expectancy of < 6 months for reasons other than their underlying
hematologic/oncologic disorder

8. Patients who have received alemtuzumab within 8 weeks of the transplant admission, or
who have recently received horse or rabbit anti-thymocyte globulin and have an
anti-thymocyte globulin level of > 2 ugm/ml

9. Patients with evidence of another malignancy, exclusive of a skin cancer that requires
only local treatment, should not be enrolled on this protocol



Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Adult Acute Lymphoblastic Leukemia in Remission
Adult Acute Myeloid Leukemia in Remission
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Adult Acute Myeloid Leukemia With Del(5q)
Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)
Adult Nasal Type Extranodal NK/T-cell Lymphoma
Aplastic Anemia
Childhood Acute Lymphoblastic Leukemia in Remission
Childhood Acute Myeloid Leukemia in Remission
Childhood Myelodysplastic Syndromes
Chronic Eosinophilic Leukemia
Chronic Myelomonocytic Leukemia
Chronic Neutrophilic Leukemia
Essential Thrombocythemia
Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue
Juvenile Myelomonocytic Leukemia
Mastocytosis
Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable
Nodal Marginal Zone B-cell Lymphoma
Polycythemia Vera
Previously Treated Myelodysplastic Syndromes
Primary Myelofibrosis
Recurrent Adult Acute Lymphoblastic Leukemia
Recurrent Adult Acute Myeloid Leukemia
Recurrent Adult Diffuse Small Cleaved Cell Lymphoma
Recurrent Adult Grade III Lymphomatoid Granulomatosis
Recurrent Adult Hodgkin Lymphoma
Recurrent Childhood Acute Lymphoblastic Leukemia
Recurrent Childhood Acute Myeloid Leukemia
Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma
Recurrent Grade 1 Follicular Lymphoma
Recurrent Grade 2 Follicular Lymphoma
Recurrent Marginal Zone Lymphoma
Recurrent Mycosis Fungoides/Sezary Syndrome
Recurrent Small Lymphocytic Lymphoma
Recurrent/Refractory Childhood Hodgkin Lymphoma
Refractory Anemia
Refractory Anemia With Ringed Sideroblasts
Refractory Hairy Cell Leukemia
Refractory Multiple Myeloma
Secondary Myelodysplastic Syndromes
Splenic Marginal Zone Lymphoma
T-cell Large Granular Lymphocyte Leukemia
Waldenström Macroglobulinemia
Intervention(s)
Biological: Donor Lymphocyte Infusion (DLI)
Device: Allogeneic hematopoietic stem cell transplantation
Drug: Busulfan
Drug: Cyclophosphamide (CY)
Drug: Fludarabine
Drug: Mycophenolate mofetil
Drug: Tacrolimus
Procedure: Peripheral blood stem cell transplantation (PBSCT)
Radiation: Total Body Irradiation (TBI)
Primary Outcome(s)
Overall Survival (OS) in patients with haploidentical family donors with hematological malignancies in morphological or radiographic remission or with chemosensitive, indolent diseases [Time Frame: At 2 years]
Secondary Outcome(s)
Engraftment Rates [Time Frame: Assessed up to 2 years]
Incidence and severity of GVHD, graded according to standard criteria [Time Frame: Assessed up to 2 years]
Incidence of Treatment Related Mortality (TRM) [Time Frame: At 100 days]
Lymphoid Reconstitution [Time Frame: 100 days post-transplant]
Relapse Rate [Time Frame: At 2 years]
Relapse Related Mortality [Time Frame: At 2 years]
Treatment Related Mortality (TRM) [Time Frame: At 2 years]
Secondary ID(s)
11D.247
2011-31
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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