NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-09-30

Study Completion Date

2021-07-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia

NCT02224872

Severe Aplastic Anemia Bone Marrow Failure Syndromes

COMPLETED PHASE2

Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

NCT01383434

Severe Aplastic Anemia

TERMINATED PHASE2

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

NCT02828592

Severe Aplastic Anemia

RECRUITING PHASE2

Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301)

NCT00326417

Anemia, Aplastic

COMPLETED PHASE1/PHASE2

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

NCT06517641

Severe Aplastic Anemia

RECRUITING PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This is a clinical trial of upfront bone marrow transplantation for patients with SAA who do not have a fully human leukocyte antigen (HLA) matched donor. The trial uses a conditioning regimen which has been successful in the refractory and relapsed setting to maximize engraftment and post transplant therapy to minimize graft versus host disease (GVHD). This would be used here in patients who have not yet undergone immunosuppressive therapy for their SAA or are thought to be unlikely to respond to immunosuppressive therapy for SAA.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Severe Aplastic Anemia Aplastic Anemia Bone Marrow Failure Immunosuppression

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

Thymoglobulin

Intervention Type DRUG

Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine

Intervention Type DRUG

Days -6 through -2: 30 mg/m\^2 IV daily

Cyclophosphamide

Intervention Type DRUG

Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation

Intervention Type RADIATION

Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus

Intervention Type DRUG

Start on Day 5 through Day 365

Mycophenolate mofetil

Intervention Type DRUG

Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Thymoglobulin

Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Intervention Type DRUG

Fludarabine

Days -6 through -2: 30 mg/m\^2 IV daily

Intervention Type DRUG

Cyclophosphamide

Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Intervention Type DRUG

Total body irradiation

Day -1: 200 centigray (cGy) in a single fraction

Intervention Type RADIATION

Tacrolimus

Start on Day 5 through Day 365

Intervention Type DRUG

Mycophenolate mofetil

Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Anti-thymocyte globulin ATG Fludara Cytoxan Cy CTX TBI FK-506 FK506 Prograf MMF CellCept

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Eligible Sex

ALL

Accepts Healthy Volunteers

No