Stem Cell Transplant for Severe Aplastic Anemia

Phase 2

Recruiting

Led By Richard W Childs, M.D.

Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Recipient: Patients diagnosed with one of the following hematologic diseases which are associated with reasonable longevity, shown to be curable by allogeneic BMT but where concern for a high procedural mortality with conventional BMT may delay or prevent such treatment: 1) Paroxysmal nocturnal hemoglobinuria (PNH) associated with life-threatening thrombosis, and/or cytopenia, and/or transfusion dependence and/or recurrent and debilitating hemolytic crisis 2) Severe aplastic anemia (SAA) or pure red cell aplasia (PRCA [acquired or congenital]) associated with transfusion dependence and/or neutropenia in patients who are not candidates for, or who have failed immunosuppressive therapy 3) Refractory anemia (RA) or RARS MDS patients who have associated transfusion dependence and/or neutropenia. Ages 4 to 80 (both inclusive), and weight >18kg Availability of HLA identical or single HLA locus mismatched family donor or 10/10 matched unrelated donor at the allelic level (HLA alleles A, B, C, DR, and DQ). 9/10 donors where all the HLA sequences have the same antigen/peptide binding domains in key exons to the patient. This can result in identical protein sequences between patient and donor. Allele mismatches in p and g groups can be considered acceptable due to the exact matching which exists in the binding domains. Telomere Length Testing Germline/Inherited gene panel in patients where a suspicion for a familial bone marrow failure syndrome (BMFS) exists, hTERC and hTERT, GATA2 mutation testing will be performed on protocol 04-H-0012 or performed elsewhere prior to enrolling on 04-H-0012.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new method of stem cell transplantation that may reduce the likelihood of severe side effects or transplant rejection.

Who is the study for?

This trial is for people aged 4-80 with severe blood diseases treatable by stem cell transplants, like severe anemia. They must weigh over 18kg and have a related donor or a matched unrelated donor available. It's not suitable for those who could be at high risk from traditional bone marrow transplants.

What is being tested?

The study tests a new stem cell transplant method using G-CSF mobilized CD34+ cells combined with fewer non-mobilized white blood cells to potentially reduce complications. Donors give white blood cells and then stem cells after filgrastim injections; recipients get chemotherapy before the infusion.

What are the potential side effects?

Possible side effects include reactions to the infusion, immune system suppression leading to increased infection risk, graft-versus-host disease where donated cells attack the body, and potential complications from chemotherapy drugs used.