World Health Organization site
Skip Navigation Links

Main
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: 12 December 2020
Main ID:  NCT00053989
Date of registration: 05/02/2003
Prospective Registration: No
Primary sponsor: Roswell Park Cancer Institute
Public title: NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
Scientific title: Non-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Disorders
Date of first enrolment: January 29, 2002
Target sample size: 41
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT00053989
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:     Philip L. McCarthy, MD
Address: 
Telephone:
Email:
Affiliation:  Roswell Park Cancer Institute
Key inclusion & exclusion criteria

DISEASE CHARACTERISTICS:

- Diagnosis of aplastic anemia

- Severe disease

- Failed at least 1 course of standard immunosuppressive regimen with cyclosporine
and anti-thymocyte globulin OR

- Histologically confirmed hematologic malignancy including the following:

- Acute leukemia

- Any of the following types:

- Acute myeloid leukemia (AML) with antecedent myelodysplastic syndromes

- Secondary AML

- AML with high-risk cytogenetic abnormalities

- Acute lymphoblastic leukemia with high-risk cytogenetic abnormalities

- Resistant or recurrent disease after combination chemotherapy with at least
1 standard regimen OR

- In first remission at high risk of relapse

- Chronic myelogenous leukemia

- Chronic phase meeting at least 1 of the following criteria:

- Failed imatinib mesylate

- Failed interferon after at least 6 months of treatment with minimum of
21 million units of interferon per week

- Unable to tolerate interferon

- Accelerated phase (blasts less than 20%)

- Myeloproliferative and myelodysplastic syndromes

- Myelofibrosis (after splenectomy)

- Refractory anemia

- Refractory anemia with excess blasts

- Chronic myelomonocytic leukemia

- Lymphoproliferative disease

- Chronic lymphocytic leukemia

- Symptomatic disease after first-line chemotherapy

- Low-grade non-Hodgkin's lymphoma (recurrent or persistent)

- Symptomatic disease after first-line chemotherapy

- Multiple myeloma

- Progressive disease after autologous stem cell transplantation

- Waldenstrom's macroglobulinemia

- Failed 1 standard regimen

- Non-Hodgkin's lymphoma meeting the following criteria:

- Intermediate or high grade

- Controlled and chemosensitive disease

- First remission lymphoblastic or small non-cleaved cell lymphoma at high
risk of relapse

- Hodgkin's lymphoma

- Relapsed and chemosensitive disease

- Not eligible for standard myeloablative allogeneic stem cell transplantation

- Availability of any of the following donor types:

- Related donor matched at 5 or 6 HLA antigens (A, B, DR)

- Unrelated donor fully matched by molecular analysis at A, B, DRB1, and DQB1 loci

- Single antigen mismatch at C allowed

- Cord blood that is 4, 5, or 6 match with recipient HLA antigens (A, B, DR) NOTE:
No syngeneic donors permitted

- No uncontrolled CNS disease (for hematologic malignancies) NOTE: A new classification
scheme for adult non-Hodgkin's lymphoma has been adopted by PDQ. The terminology of
"indolent" or "aggressive" lymphoma will replace the former terminology of "low",
"intermediate", or "high" grade lymphoma. However, this protocol uses the former
terminology.

PATIENT CHARACTERISTICS:

Age

- 4 to 75 (if related or unrelated donor peripheral blood or marrow transplantation)

- 4 to 60 (if unrelated cord blood transplantation)

Performance status

- Karnofsky > 50%

Life expectancy

- Not specified

Hematopoietic

- Not specified

Hepatic

- Bilirubin less than 3 times normal

- Alkaline phosphatase less than 3 times normal

- AST/ALT less than 3 times normal

- No Child's class B or C liver failure

Renal

- Creatinine clearance greater than 40 mL/min

Cardiovascular

- Cardiac ventricular ejection fraction at least 35% by MUGA

- No cardiovascular disease

Pulmonary

- DLCO at least 40% of predicted, corrected for hemoglobin and/or alveolar ventilation

Other

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- HIV antibody negative

- No uncontrolled diabetes mellitus

- No active serious infection

- No other disease that would preclude study therapy

- No other concurrent malignancy except non-melanoma skin cancer

- No concurrent serious psychiatric illness

PRIOR CONCURRENT THERAPY:

Biologic therapy

- See Disease Characteristics

- patients may have received a prior autologous blood or marrow transplantation (BMT)

- At least 6 months since prior allogeneic BMT

Chemotherapy

- See Disease Characteristics

- At least 2 weeks since prior chemotherapy, radiation or surgery

Endocrine therapy

- Not specified

Radiotherapy

- At least 2 weeks since prior radiotherapy

Surgery

- At least 2 weeks since prior surgery



Age minimum: 4 Years
Age maximum: 75 Years
Gender: All
Health Condition(s) or Problem(s) studied
Aplastic Anemia
Chronic Myeloproliferative Disorders
Fanconi Anemia
Leukemia
Lymphoma
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
Intervention(s)
Biological: anti-thymocyte globulin
Biological: graft-versus-tumor induction therapy
Biological: sargramostim
Biological: therapeutic allogeneic lymphocytes
Drug: cyclophosphamide
Drug: fludarabine phosphate
Drug: methylprednisolone
Drug: mycophenolate mofetil
Drug: tacrolimus
Procedure: allogeneic bone marrow transplantation
Procedure: peripheral blood stem cell transplantation
Procedure: umbilical cord blood transplantation
Primary Outcome(s)
Day 100 Best Response [Time Frame: from start of conditioning on day -6 or -5 through day +100 after HSC infusion]
Day 100 TRM [Time Frame: from start or conditioning (day -6 or -5) through day +100 after HSC infusion]
Secondary Outcome(s)
Acute GvHD [Time Frame: Day +100]
OS [Time Frame: 1 year]
PFS [Time Frame: 1 year]
Secondary ID(s)
CDR0000269673
RP01-05
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available: Yes
Date Posted: 10/02/2020
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT00053989
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history