Immunosuppression Clinical Trial
Official title:
A Phase II Trial of Non-Myeloablative (NMA) Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Donor (MUD) Bone Marrow for Newly Diagnosed Patients With Severe Aplastic Anemia
Verified date | June 2022 |
Source | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Our primary objective is to determine if it is feasible for previously untreated severe aplastic anemia (SAA) patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide.
Status | Completed |
Enrollment | 21 |
Est. completion date | July 2021 |
Est. primary completion date | July 2021 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria: - Confirmed diagnosis of inherited or acquired severe aplastic anemia (SAA) - One of the following available donors: 1. HLA-haploidentical relative 2. If recipient is >= 40 years old, may use HLA-matched related donor 3. For recipients with inherited bone marrow failure syndromes (IBMFS) with clear evidence of same disorder in potential related donors, may use 10/10 matched unrelated donor - Recipient and/or legal guardian must sign protocol informed consent - Donor must be willing to donate bone marrow - Left ventricular ejection fraction (LVEF) >= 40%. For recipients < 13 years old, shortening fraction >= 26% may be used instead. - Bilirubin < 3 x upper limit of normal (ULN) for age, unless patient has Gilbert's disease - aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 5 x ULN for age - For patients >= 13 years old: estimated creatinine clearance > 50 mL/min using Cockcroft-Gault formula and actual body weight - For patients >= 1 but < 13 years old: glomerular filtration rate (GFR) estimated by updated Schwartz formula >= 90 mL/min/1.73 m^2. If estimated GFR is < 90 mL/min/1.73 m^2, 24-hour measured creatinine clearance must be > 50 mL/min/1.73 m^2. - For patients >= 8 years old, diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) > 40%; forced expiratory volume at one second (FEV1) > 50%; forced vital capacity (FVC) > 50% - For patients < 8 years old or unable to undergo pulmonary function testing: no evidence of dyspnea at rest; no need for supplemental oxygen; oxygen saturation > 92% on room air - Karnofsky/Lansky status (depending on age) >= 70% - Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time. If unwilling, they must agree to complete abstinence. Exclusion Criteria: - Previous administration of immunosuppressive therapy for SAA. - Fanconi anemia. At minimum, this diagnosis must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow in patients < 30 years old. - Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on bone marrow examination - Presence of anti-donor antibodies - Prior allogeneic stem cell transplant - Prior solid organ transplant - Uncontrolled bacterial, viral, or fungal infection - HIV seropositivity - Active hepatitis B or C infection determined by serology and/or nucleic acid testing (NAT) - Pregnancy or active breastfeeding - Prior malignancies except: resected basal carcinoma or treated cervical carcinoma in situ; cancer treated with curative intent > 5 years previously. Other prior cancers will not be allowed unless approved by the PI. |
Country | Name | City | State |
---|---|---|---|
United States | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Baltimore | Maryland |
Lead Sponsor | Collaborator |
---|---|
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Overall Survival and Engraftment at One Year | Number of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post bone marrow transplant (BMT). | 1 year | |
Secondary | Overall Survival at One Year | Number of participants alive at one year after BMT. | 1 year | |
Secondary | Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts | Probability of neutrophil recovery will be assessed by the number of participants who have recovered neutrophil counts at 1 year (>500 ANC). | 1 year | |
Secondary | Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts | Probability of platelet recovery will be assessed by the number of participants who have recovered platelet counts at 1 year. | 1 year | |
Secondary | Number of Participants Who Experience Primary Graft Failure | Number of participants who experience primary graft failure by one year after BMT. | 1 year | |
Secondary | Number of Participants Who Experience Secondary Graft Failure | Number of participants who experience secondary graft failure by one year after BMT. | 1 year | |
Secondary | Number of Participants Who Experience Grades II-IV Acute GVHD | Number of participants who experience grade II, III, or IV acute GVHD by Day 100. | Day 100 | |
Secondary | Number of Participants Who Experience Grades III-IV Acute GVHD | Number of participants who experience grade III or IV acute GVHD by Day 100. | Day 100 | |
Secondary | Number of Participants Who Experience Chronic GVHD | Number of participants who experience chronic GVHD by two years after BMT. | 2 years | |
Secondary | Number of Participants With Full Donor Chimerism | Number of participants with full donor chimerism at Day 60. | Day 60 | |
Secondary | GVHD-free Relapse-free Survival (GRFS) | Number of participants alive, without relapse, and without GVHD at 1 year. | 1 year | |
Secondary | Transplant-related Mortality | Number of participants deceased for reasons related to BMT at 1 year. | 1 year |
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