Clinical trial NCT01383434: Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia
Official title: A Phase II Trial of Myeloablative Conditioning and Transplantation of HLA-matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated (MUD) Bone Marrow for Patients With Refractory Severe Aplastic Anemia
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Severe Aplastic Anemia
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Sponsor class: OTHER
First posted: 2011-06-28
Enrollment: 4
Sex: ALL
Minimum age (years): 6
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Patients with severe, refractory aplastic anemia have a severe, life threatening disease in their bone marrow. Refractory disease means that 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 aplastic anemia but complications from Graft vs Host disease (GVHD) and graft failure have limited the survival for those patients.

Another study done here at Johns Hopkins has shown that in patients with other diseases (blood cancers) some immunosuppressive drugs given after the BMT has decreased how often patients had complications of GVHD and engraftment failure.

This research is being done to find if this approach will help patients with aplastic anemia who have failed other treatments will have better outcomes.


Eligibility criteria: Inclusion Criteria:

* Patients who have refractory or relapsed SAA following treatment with one or more immunosuppressive regimens.
* Age 6 months - 70 years
* Patients must meet medical criteria for myeloablative BMT
* Patients or their parents/guardians and donors must be able to sign consent forms.
* Patients must be geographically accessible and willing to participate in all stages of treatment.

Exclusion Criteria:

* Poor cardiac function: left ventricular ejection fraction \<45% as determined by MUGA or ECHO.
* Poor pulmonary function: FEV1 and FVC \<50% predicted.
* Poor renal function
* Positive leukocytotoxic crossmatch
* Women of childbearing potential who currently are pregnant (Β-HCG+) or who are not practicing adequate contraception
* Uncontrolled viral, bacterial, or fungal infections

Primary outcomes:
measure: To estimate the overall survival (OS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
timeFrame: 1 year
measure: To estimate the event-free survival (EFS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
timeFrame: 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: The Sidney Kimmel Comprehensive Cancer Center
state: Maryland
Study officials:
affiliation: The Johns Hopkins University
name: Robert A Brodsky, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: The JOhns Hopkins Sydney Kimmel Comprehensive Cancer Center
name: Amy Dezern, MD
role: PRINCIPAL_INVESTIGATOR
References: