Phase 2
Completed N=18
Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia
severe aplastic anemia · Bone Marrow Failure Syndromes
Source: ClinicalTrials.gov NCT02224872 ↗
Enrolled (actual)
18
Serious AEs
38.9%
Results posted
Mar 2023
Primary outcomePrimary: Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe? — 18 Participants
Summary
Our primary objective is to determine if it is feasible for SAA patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide with partially HLA-mismatched donors.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe? |
18 | — |
| SECONDARY Number of Patients That Have Survived at One Year |
18 | — |
| SECONDARY Number of Patients That Have Acheived Full Donor Chimerism by Day 60 After Transplant |
7 | — |
| SECONDARY Number of Patients That Expired Due to Non-relapsed-related Mortality Following Transplant |
— | — |
| SECONDARY Number of Participants With Major Toxicities Related to Transplant |
— | — |
| SECONDARY Number of Patients That Expired Due to Transplant Related Mortality |
— | — |
| SECONDARY Number of Patients With Primary or Secondary Graft Failure Following Transplant |
17 | — |
| SECONDARY Number of Participants With Grade II-IV or Grade III-IV Acute GVHD |
17 | — |
| SECONDARY Participants With Chronic GVHD at One Year |
3 | — |
| SECONDARY Length of Time Required for Patients to Recover ANC and Platelet Counts After Transplant |
18.85 | — |
| SECONDARY Participants That Were GVHD Free, Relapse Free Survival (GRFS) |
14 | — |
Eligibility Criteria
Inclusion Criteria
- Patients with relapsed or refractory SAA or very SAA defined:
- Bone marrow ( or = to 35%, or shortening fraction > 25% (For pediatric patients, a normal ejection fraction is required)
- Bilirubin ≤ 3.0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST ≤ 5 x ULN
- 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)
Data sourced from ClinicalTrials.gov (NCT02224872). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.