Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

NCT01383434 | Terminated Phase 2 DMC

• 2 other identifiers: J10117NA_00042991
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

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.

Conditions

Severe Aplastic Anemia

Outcome Measures

Primary Outcomes (2)

• 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

  1 year

• 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

  1 year

Interventions

Bone Marrow Transplant PROCEDURE

Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy

Eligibility Criteria

• Age: 6 Months - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: lead

Study Sites (1)

The Sidney Kimmel Comprehensive Cancer Center

Baltimore, Maryland, 21031, United States

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Bone Marrow Transplantation

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Tissue Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Robert A Brodsky, MD

  The Johns Hopkins University

  PRINCIPAL INVESTIGATOR

• Amy Dezern, MD

  The JOhns Hopkins Sydney Kimmel Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 22, 2011
• First Posted: June 28, 2011
• Study Start: June 1, 2011
• Primary Completion: August 1, 2014
• Study Completion: August 1, 2014
• Last Updated: October 13, 2016

Record last verified: 2016-10

Locations

US (1)