NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia

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Brief Title

NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia

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

Brief Summary

      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.
    

Detailed Description

      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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Overall survival and engraftment at one year

Secondary Outcome

 Overall survival at one year

Condition

Severe Aplastic Anemia

Intervention

Thymoglobulin

Study Arms / Comparison Groups

 Bone marrow transplant
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

21

Start Date

September 2016

Completion Date

July 2021

Primary Completion Date

July 2021

Eligibility Criteria

        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.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Amy E DeZern, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02833805

Organization ID

J1688

Secondary IDs

IRB00107139

Responsible Party

Sponsor

Study Sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins


Study Sponsor

Amy E DeZern, MD, Principal Investigator, Johns Hopkins University


Verification Date

September 2021