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

Information source: Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Severe Aplastic Anemia; Bone Marrow Failure Syndromes

Intervention: Bone marrow transplant (Procedure); Thymoglobulin (Drug); Fludarabine (Drug); Cyclophosphamide (Drug); TBI (Radiation); Mesna (Drug); Tacrolimus (Drug); Mycophenolic acid mofetil (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Sidney Kimmel Comprehensive Cancer Center

Official(s) and/or principal investigator(s):
Amy DeZern, MD, Principal Investigator, Affiliation: Sidney Kimmel Comprehensive Cancer Center

Overall contact:
Amy DeZern, MD, Phone: 410-502-7208, Email: adezern1@jhmi.edu


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.

Clinical Details

Official title: A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Is this type of transplantation for SAA feasible and safe?

Secondary outcome:

Number of patients that have survived at one year

Number of patients that have acheived full donor chimerism by day 60 after transplant

Number of patients that expired due to non-relapsed-related mortality following transplant

Major toxicities related to transplant

Number of patients that expired due to transplant related mortality

Number of patients with primary or secondary graft failure following transplant

Number of patients with grade II-IV or grade III-IV acute GVHD

Number of patients with chronic GVHD

Length of time required for patients to recover ANC and platelet counts after transplant

Length of GVHD free, relapse free survival (GRFS) in patients

Detailed description: This research is being done to find out if bone marrow transplantation (BMT) followed by chemotherapy will help people with aplastic anemia who have failed other treatments. You have a severe, life threatening disease (severe aplastic anemia) in your bone marrow. Your disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed by bone marrow transplantation (BMT) has been used to treat blood diseases like yours but complications from Graft vs. Host disease (GVHD) and graft failure have limited the survival for those people. A small study done at Johns Hopkins has shown that in subjects with other diseases (blood cancers) some immunosuppressive drugs given after the BMT have decreased how often subjects had complications of GVHD and engraftment failure. People with aplastic anemia who have refractory disease (not responding to standard treatment) may join.


Minimum age: N/A. Maximum age: 73 Years. Gender(s): Both.


Inclusion Criteria:

- Patients with relapsed or refractory SAA or very SAA defined:

- Bone marrow (< 25% cellular)

- Peripheral cytopenias (at least 2 of 3)

- ANC < 500 per ml

- Platelets < 20,000 per ml

- Absolute retic < 60,000 or corrected retic < 1%

- Very severe: as above, but ANC < 200

- Disease may be designated as acquired or inherited if previous counts known

(these other bone marrow failure disorders that are characterized by aplastic anemia may go by additional names such as dyskeratosis congenita or PNH)

- Failed at least one course of immunosuppressive therapy (if presumed acquired

disease). Patients with inherited disease will be characterized as refractory and do not require immunosuppressive first.

- Age 0- upper age limit as determined by current institutional standards

- Good performance status (ECOG 0 or 1; Karnofsky and Lansky 70-100)

- Patients and donors must be able to sign consent forms (or if a minor the parent will

sign). Donors should be willing to donate.

- Patients must be geographically accessible and willing to participate in all stages

of treatment.

- Adequate end-organ function as measured by:

1. Left ventricular ejection fraction > or = to 35%, or shortening fraction > 25% (For pediatric patients, a normal ejection fraction is required) 2. Bilirubin ≤ 3. 0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST ≤ 5 x ULN 3. 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)

Locations and Contacts

Amy DeZern, MD, Phone: 410-502-7208, Email: adezern1@jhmi.edu

The Sidney Kimmel Comprehensive Cancer Center, Baltimore, Maryland 21287, United States; Recruiting
Amy DeZern, MD, Phone: 410-502-7208, Email: adezern1@jhmi.edu
Clinical Trials Office- SKCCC, Phone: 410-955-8804, Email: jhcccro@jhmi.edu
Amy DeZern, MD, Principal Investigator

Medical College of Wisconsin/Children's Hospital of Wisconsin, Milwaukee, Wisconsin 53226, United States; Not yet recruiting
David Margolis, MD, Phone: 414-337-7066, Email: dmargoli@mcw.edu
Monica Thakar, MD, Phone: 414-955-7546, Email: mthakar@mcw.edu

Additional Information

Starting date: August 2014
Last updated: February 26, 2015

Page last updated: August 23, 2015

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