NCT02224872

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Academic / Other
• Sex: All
• Age: 73 Years
• Healthy volunteers: Not accepted

Summary

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.

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.

Conditions

• Severe Aplastic Anemia
• Bone Marrow Failure Syndromes

Interventions

Type	Name	Description
PROCEDURE	Bone marrow transplant	Day 0
DRUG	Thymoglobulin	0.5 mg/kg IV on Day -9 2 mg/kg IV on Days -8, -7
DRUG	Fludarabine	30 mg/M2 IV on days -6 to -2
DRUG	Cyclophosphamide	14.5 mg/kg IV on days -6, -5, 3, 4
RADIATION	TBI	200 cGy on day -1
DRUG	Mesna	40 mg/kg IV on days 3, 4
DRUG	Tacrolimus	For patients 18 years or older, tacrolimus will be given per institutional standards; may be increased or later changed to a PO BID schedule. Treatment to continue until Day 365 or longer if GVHD present
DRUG	Mycophenolic acid mofetil	15 mg/kg PO/IV TID beginning on day 5 through day 35

Timeline

• Start date: 2014-08-01
• Primary completion: 2021-12-01
• Completion: 2021-12-01
• First posted: 2014-08-25
• Last updated: 2023-03-10
• Results posted: 2023-03-10

Locations

2 sites across 1 country: United States