NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
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ClinicalTrials.gov Identifier: NCT02833805 |
Recruitment Status :
Active, not recruiting
First Posted : July 14, 2016
Last Update Posted : August 6, 2020
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Condition or disease | Intervention/treatment | Phase |
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Severe Aplastic Anemia Aplastic Anemia Bone Marrow Failure Immunosuppression | Drug: Thymoglobulin Drug: Fludarabine Drug: Cyclophosphamide Radiation: Total body irradiation Drug: Tacrolimus Drug: Mycophenolate mofetil | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 21 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | September 2016 |
Estimated Primary Completion Date : | June 2021 |
Estimated Study Completion Date : | June 2021 |

Arm | Intervention/treatment |
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Experimental: Bone marrow transplant
Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
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Drug: Thymoglobulin
Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily
Other Names:
Drug: Fludarabine Days -6 through -2: 30 mg/m^2 IV daily
Other Name: Fludara Drug: Cyclophosphamide Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Other Names:
Radiation: Total body irradiation Day -1: 200 centigray (cGy) in a single fraction
Other Name: TBI Drug: Tacrolimus Start on Day 5 through Day 365
Other Names:
Drug: Mycophenolate mofetil Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
Other Names:
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- Overall survival and engraftment at one year [ Time Frame: 1 year ]Percentage of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post BMT.
- Overall survival at one year [ Time Frame: 1 year ]Percentage of participants alive at one year after BMT.
- Probability of neutrophil recovery [ Time Frame: 1 year ]Percentage of participants who have recovered neutrophil counts at 1 year.
- Probability of platelet recovery [ Time Frame: 1 year ]Percentage of participants who have recovered platelet counts at 1 year.
- Incidence of primary graft failure [ Time Frame: 1 year ]Percentage of participants who experience primary graft failure by one year after BMT.
- Incidence of secondary graft failure [ Time Frame: 1 year ]Percentage of participants who experience secondary graft failure by one year after BMT.
- Incidence of grades II-IV acute GVHD, Day 100 [ Time Frame: Day 100 ]Percentage of participants who experience grade II, III, or IV acute GVHD by Day 100.
- Incidence of grades III-IV acute GVHD, Day 100 [ Time Frame: 1 year ]Percentage of participants who experience grade III or IV acute GVHD by Day 100.
- Incidence of chronic GVHD, one year [ Time Frame: 1 year ]Percentage of participants who experience chronic GVHD by one year after BMT.
- Estimate full donor chimerism [ Time Frame: Day 60 ]Percentage of participants with full donor chimerism at Day 60.
- Estimate GVHD-free relapse-free survival (GRFS) [ Time Frame: 1 year ]Percentage of participants alive, without relapse, and without GVHD at 1 year.
- Estimate transplant-related mortality [ Time Frame: 1 year ]Percentage of participants deceased for reasons related to BMT at 1 year.

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Confirmed diagnosis of inherited or acquired severe aplastic anemia (SAA)
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One of the following available donors:
- HLA-haploidentical relative
- If recipient is >= 40 years old, may use HLA-matched related donor
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02833805
United States, Maryland | |
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | |
Baltimore, Maryland, United States, 21287 |
Principal Investigator: | Amy E DeZern, MD | Johns Hopkins University |
Responsible Party: | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
ClinicalTrials.gov Identifier: | NCT02833805 |
Other Study ID Numbers: |
J1688 IRB00107139 ( Other Identifier: JHMIRB ) |
First Posted: | July 14, 2016 Key Record Dates |
Last Update Posted: | August 6, 2020 |
Last Verified: | August 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
immunosuppression nonmyeloablative non-myeloablative allogeneic tacrolimus bone marrow |
bone marrow transplant cyclophosphamide thymoglobulin ATG fludarabine |
Anemia Anemia, Aplastic Pancytopenia Hematologic Diseases Bone Marrow Diseases Mycophenolic Acid Cyclophosphamide Fludarabine Tacrolimus Thymoglobulin Antilymphocyte Serum Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |
Antirheumatic Agents Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Calcineurin Inhibitors Enzyme Inhibitors Antibiotics, Antineoplastic Antibiotics, Antitubercular Antitubercular Agents Anti-Bacterial Agents Anti-Infective Agents |