Main
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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:
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ClinicalTrials.gov |
Last refreshed on:
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18 October 2021 |
Main ID: |
NCT00630253 |
Date of registration:
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05/03/2008 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
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Scientific title:
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A Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi Anemia |
Date of first enrolment:
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February 17, 2000 |
Target sample size:
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31 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/show/NCT00630253 |
Study type:
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Interventional |
Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Margaret L. MacMillan, MD |
Address:
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Telephone:
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Email:
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Affiliation:
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Masonic Cancer Center, University of Minnesota |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Patients must be <60 years of age with a diagnosis of Fanconi Anemia (FA).
- Patients must have an HLA-A, B, DRB1 identical sibling donor. Patients and donors will
be typed for HLA-A and B using serological or molecular techniques and for DRB1 using
high resolution molecular typing.
- Patients with FA must have moderately severe aplastic anemia (AA), early
myelodysplastic syndrome (MDS) with no excess blasts with or without chromosomal
abnormalities.
- In patients <18 years of age, moderately severe aplastic anemia is defined as
having at least one of the following:
- platelet count <40 x 10^9/L
- absolute neutrophil count (ANC) <10 x 10^8/L
- Hgb <9 g/dL
- In patients 18-60 years of age, moderately severe aplastic anemia is defined as
having at least one of the following:
- platelet count <20 x 10^9/L
- absolute neutrophil count ANC <5 x 10^8/L
- Hgb <8 g/dL
- Early myelodysplastic syndrome, with multilineage dysplasia with < 5% blasts,
with or without chromosomal anomalies.
- Adequate major organ function including:
- Cardiac: ejection fraction >45%
- Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
- Karnofsky performance status >70% or Lansky >50%
- Women of child bearing age must be using adequate birth control and have a negative
pregnancy test.
Exclusion Criteria:
- Active bacterial infection within one week of hematopoietic cell transplant (HCT)
- Active fungal infection at time of HCT.
- Late MDS with greater than 5% blasts in bone marrow.
- Acute myelogenous leukemia (AML) or history of AML
- Malignant solid tumor (e.g. squamous cell carcinoma of the head/neck/cervix) within 2
years of HCT.
- Pregnant or lactating female.
Age minimum:
N/A
Age maximum:
59 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Fanconi Anemia
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Intervention(s)
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Drug: Methylprednisolone
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Drug: Cyclophosphamide
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Procedure: Hematopoietic Stem Cell Transplantation
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Drug: Fludarabine
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Drug: Filgrastim
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Biological: Anti-Thymocyte Globulin
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Drug: Cyclosporine
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Drug: Mycophenolate Mofetil
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Primary Outcome(s)
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Number of Participants Experiencing Graft Failure
[Time Frame: From Day 1 to event, assessed up to100 days]
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Secondary Outcome(s)
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Number of Participants Experiencing Overall Survival
[Time Frame: 1 Year]
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Number of Participants With Acute Graft-Versus-Host Disease (GVHD)
[Time Frame: Day 42]
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Number of Participants With Chronic Graft-Versus-Host Disease (GVHD)
[Time Frame: 1 Year]
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Number of Participants With Transplant Related Deaths
[Time Frame: Day 100]
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Secondary ID(s)
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0001M34441
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MT2000-09
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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