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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. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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20 March 2023 |
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Main ID: |
NCT02224872 |
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Date of registration:
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18/08/2014 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia
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Scientific title:
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A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes |
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Date of first enrolment:
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August 2014 |
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Target sample size:
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18 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02224872 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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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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Amy DeZern, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Patients with relapsed or refractory SAA or very SAA defined:
- Bone marrow (< 25% cellular)
- Peripheral cytopenias (at least 2 of 3)
- ANC < 500 per ml
- Platelets < 20,000 per ml
- Absolute retic < 60,000 or corrected retic < 1%
- Very severe: as above, but ANC < 200
- Disease may be designated as acquired or inherited if previous counts known
(these other bone marrow failure disorders that are characterized by aplastic
anemia may go by additional names such as dyskeratosis congenita or PNH)
- Failed at least one course of immunosuppressive therapy (if presumed acquired
disease). Patients with inherited disease will be characterized as refractory and
do not require immunosuppressive first.
- Age 0- upper age limit as determined by current institutional standards
- Good performance status (ECOG 0 or 1; Karnofsky and Lansky 70-100)
- Patients and donors must be able to sign consent forms (or if a minor the parent will
sign). Donors should be willing to donate.
- Patients must be geographically accessible and willing to participate in all stages of
treatment.
- Adequate end-organ function as measured by:
1. Left ventricular ejection fraction > or = to 35%, or shortening fraction > 25%
(For pediatric patients, a normal ejection fraction is required)
2. Bilirubin = 3.0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT
and AST = 5 x ULN
3. FEV1 and FVC > or = to 40% of predicted; or in pediatric patients, if unable to
perform pulmonary function tests due to young age, oxygen saturation >92% on room
air
Exclusion Criteria:
- Patients will not be excluded on the basis of sex, racial or ethnic background.
- Prior transfusions from selected donor (as this could have cause recipient
alloimmunization against the donor)
- Women of childbearing potential who currently are pregnant (HCG+) or who are not
practicing adequate contraception.
- Patients who have any debilitating medical or psychiatric illness that would preclude
their giving informed consent or their receiving optimal treatment and follow up.
- Uncontrolled viral, bacterial, or fungal infections (HIV infection permitted if viral
load undetectable)
Age minimum:
N/A
Age maximum:
73 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Severe Aplastic Anemia
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Bone Marrow Failure Syndromes
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Intervention(s)
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Drug: Fludarabine
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Drug: Mycophenolic acid mofetil
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Drug: Cyclophosphamide
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Drug: Mesna
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Drug: Tacrolimus
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Drug: Thymoglobulin
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Radiation: TBI
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Procedure: Bone marrow transplant
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Primary Outcome(s)
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Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe?
[Time Frame: 1 year]
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Secondary Outcome(s)
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Number of Patients With Primary or Secondary Graft Failure Following Transplant
[Time Frame: 1 year]
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Participants That Were GVHD Free, Relapse Free Survival (GRFS)
[Time Frame: 1 year]
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Length of Time Required for Patients to Recover ANC and Platelet Counts After Transplant
[Time Frame: 1 year]
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Number of Participants With Major Toxicities Related to Transplant
[Time Frame: 1 year]
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Participants With Chronic GVHD at One Year
[Time Frame: 1 year]
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Number of Patients That Have Survived at One Year
[Time Frame: 1 year]
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Number of Participants With Grade II-IV or Grade III-IV Acute GVHD
[Time Frame: 1 year]
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Number of Patients That Expired Due to Non-relapsed-related Mortality Following Transplant
[Time Frame: 1 year]
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Number of Patients That Expired Due to Transplant Related Mortality
[Time Frame: 1 year]
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Number of Patients That Have Acheived Full Donor Chimerism by Day 60 After Transplant
[Time Frame: 60 days]
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Secondary ID(s)
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IRB00031590
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J1424
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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