Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia
2 other identifiers
interventional
22
1 country
2
Brief Summary
Patients have been diagnosed with severe Aplastic Anemia that have not responded to treatment with immunosuppressive therapy (drugs that suppress the immune system, for example Steroids). The immune system is the system in the body that helps protect the body and fights bacterial, viral and fungal infections. Research studies have shown that patients with Aplastic Anemia have improved survival (may live longer) after receiving a HLA (Human Leukocyte Antigen) identical sibling (brother and sister) stem cell transplants. Patients who do not have matched siblings can undergo immunosuppressive therapy, which has also shown to improve outcome. Unfortunately patients who do not respond to immunosuppressive therapy usually die. The best chance of survival for these patients is an HLA matched unrelated or mismatched related stem cell transplant as described below. Stem cells are created in the bone marrow. They mature into different types of blood cells that people need including red blood cells which carry oxygen around the body, white blood cells which help fight infections, and platelets which help the blood to clot and prevent bleeding. For a matched unrelated stem cell transplant, stem cells are collected from a person (donor) who is not related to the patient but who has the same type of stem cells. For a mismatched related stem cell transplant, stem cells are collected from a donor who is related to the patient and whose stem cells are almost the same as those of the patient but not exactly. The patient then receives high dose chemotherapy. This chemotherapy kills the stem cells in the patient's bone marrow. Stem cells that have been collected from the donor are then given to the patient to replace the stem cells that have been killed. The major problems associated with these types of stem cell transplants are graft rejection (where the patient's immune system rejects the donor stem cells) and severe graft versus host disease (GVHD), where the donors stem cell reacts against the patient's tissues in the body.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Feb 2002
Longer than P75 for phase_2
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2002
CompletedFirst Submitted
Initial submission to the registry
December 19, 2007
CompletedFirst Posted
Study publicly available on registry
December 21, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2012
CompletedResults Posted
Study results publicly available
October 11, 2013
CompletedApril 22, 2016
March 1, 2016
10.4 years
December 19, 2007
August 4, 2013
March 24, 2016
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Subjects Alive at 100 Days Post Transplant
100 days
Secondary Outcomes (5)
Number of Patients With Engraftment Rate at 100 Days Post Transplant
100 days post transplant
Number of Patients With Acute GVHD at 100 Days Post Transplant
100 days
Number of Patients With Chronic GVHD at 2 Years Post Transplant
2 years
Number of Subjects Alive at 1 Year Post Transplant
1 year
Number of Subjects Alive at 2 Years Post Transplant
2 years
Study Arms (1)
Patients
EXPERIMENTALPatients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member. Cytoxan, Campath, TBI-Total Body Irradiation, FK-506, Methotrexate, Stem Cell Infusion
Interventions
Cytoxan will be given at 50 mg/kg per dose for 4 successive days.
Campath will be given at a dose of 3 mg for patients whose weight is between 5 and 15 kg; at a dose of 5 mg for patients whose weight is between 16 and 30 kg; and at a dose of 10 mg for patients whose weight is greater than 30 kg. The last dose of Campath should be 24 hours or more before stem cell infusion.
TBI will be given at a dose of 200 cGy for 6/6 HLA match and at a dose of 400 cGy in two fractions of 200 cGy each for 5/6 HLA matched donor.
FK-506 will be given at a dose of 0.03 mg/kg/day via continuous infusion over 24 hours from 4pm on day -2 until engraftment or when patient is able to take by mouth (PO), then 0.03 mg/kg PO every 12 hours.
Methotrexate will be administered on day +1, day +3, day +6 and day +11 at a dose of 5 mg/m2. The day +11 dose may be omitted at the discretion of the bone marrow transplant (BMT) in-patient attending physician.
Where possible patients will receive bone marrow. Marrow will be collected as per National Marrow Donor Program (NMDP) guidelines to provide a volume of 15-20 ml/kg of marrow and/or 2-4 X 10\^8 nucleated cells/kg. In case marrow cannot be collected, peripheral blood stem cell (PBSC) will be substituted. A minimum of 5-6 X 10\^6 CD 34+ cells/kg should be collected, with a target of 10 X 10\^6/kg.
Eligibility Criteria
You may qualify if:
- Diagnosis of Severe Aplastic Anemia (SAA) based on bone marrow aspirate and biopsy results.
- Failure to respond to immunosuppressive therapy.
- Lack of an Human Leukocyte Antigen (HLA) identical family member.
- A 6/6 or 5/6 HLA matched unrelated donor or a 5/6 matched related donor available after high resolution HLA typing.
- Age from birth to 60 years.
You may not qualify if:
- Severe disease other than aplastic anemia that would limit the probability of survival during the graft procedure. Patients who present with active infection must be treated to maximally resolve this problem before beginning the conditioning regimen.
- Human immunodeficiency virus (HIV) seropositive patients
- Patients who have clonal cytogenetic abnormalities or a myelodysplastic syndrome.
- Patient greater than 60 years of age.
- Women who are pregnant or nursing.
- Patients with active hepatitis
- Patients with severe cardiac dysfunction defined as shortening fraction \< 25%.
- Patients with severe renal dysfunction defined as creatinine clearance \< 40 ml/mim/1.73m2.
- Patient with severe pulmonary dysfunction with forced expiratory volume in the first second (FEV1), forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLCO) 40% of predicted or 3 standard deviations (SD) below normal.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Texas Children's Hospital
Houston, Texas, 77030, United States
The Methodist Hospital
Houston, Texas, 77030, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Kathryn Leung
- Organization
- Baylor College of Medicine
Study Officials
- PRINCIPAL INVESTIGATOR
Kathryn Leung, MD
Baylor College of Medicine
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal Investigator
Study Record Dates
First Submitted
December 19, 2007
First Posted
December 21, 2007
Study Start
February 1, 2002
Primary Completion
July 1, 2012
Study Completion
July 1, 2012
Last Updated
April 22, 2016
Results First Posted
October 11, 2013
Record last verified: 2016-03