What is this study trying to find out about severe aplastic anemia?

This study is evaluating a bone marrow transplant from partially matched donors for patients with severe aplastic anemia who lack fully matched related donors. Researchers want to see if this transplant method can improve engraftment and reduce complications. The study is sponsored by Northside Hospital, Inc.

Who may be eligible for this severe aplastic anemia clinical trial?

People aged 1 to 75 years with severe aplastic anemia may be eligible. Participants must meet specific blood and bone marrow criteria and have adequate heart, lung, liver, and kidney function. Both males and females can join. Please review eligibility criteria for more details.

Is there any cost to participate in this severe aplastic anemia clinical trial?

Study-related care, including the transplant and all trial visits, is provided at no cost. Personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly.

Actively Recruiting

Phase 2

Age: 1Year - 75Years

All Genders

ID02828592

Haploidentical Bone Marrow Transplant with Post-Transplant Cyclophosphamide for Severe Aplastic Anemia Patients Without HLA-Matched Sibling Donors

Led by Northside Hospital, Inc. · Updated on 2026-04-20

Participants Needed

Research Sites

52 weeks

Total Duration

AI-Summary

What this Trial Is About

Severe aplastic anemia is a rare and serious bone marrow failure caused by an immune-related problem, leading to very low blood cell counts and high risks of infections and bleeding. This research is studying a type of bone marrow transplant using partly matched donors for patients who do not have fully matched related donors. The study aims to evaluate the success of this transplant approach and reduce complications like graft failure and graft versus host disease. Patients will receive a preparative treatment combining Fludarabine, Cyclophosphamide, and low-dose radiation before undergoing a haploidentical bone marrow transplant. After transplant, Cyclophosphamide is given on Days 3 and 4 to help prevent rejection, followed by immunosuppressive medications Tacrolimus and Mycophenolate Mofetil starting Day 5. Mycophenolate Mofetil is stopped on Day 35, while Tacrolimus continues until Day 180. This treatment plan is designed to support successful engraftment and reduce complications. During the study, participants will have monthly tests to measure donor cell engraftment for up to two years after transplant. Researchers will also monitor for treatment-related side effects, acute and chronic graft versus host disease, and overall survival at 100 days and one year. The total participation time includes close follow-up and evaluations to assess how well the transplant works and to track safety outcomes.

CONDITIONS

Brief Title

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

How This Trial Compares With Other Options

This table compares participation in this trial with other options available for this condition. The information is drawn from published research and the trial’s study details and is provided for educational purposes only.

Factors	Haploidentical (partially matched related donor) bone marrow transplant with conditioning and post-transplant cyclophosphamide	Allogeneic bone marrow transplant (matched unrelated or HLA-identical sibling donor), standard conditioning
What it is	A bone marrow transplantation procedure using a haploidentical (partially related) donor, with a conditioning regimen of fludarabine, cyclophosphamide, low-dose total body irradiation, and rabbit ATG, followed by post-transplant cyclophosphamide to help prevent graft-versus-host disease.	An allogeneic bone marrow transplant from either an HLA-identical sibling or a matched unrelated donor, usually with a conventional myeloablative or reduced-intensity conditioning regimen based on guideline recommendations.
Role in the trial	Investigational approach for patients lacking a suitable matched related donor, assessed for sustained engraftment, survival, and transplant-related complications.	Current standard of care recommended for eligible patients with a suitable matched unrelated or sibling donor.
Primary use	Potential curative therapy for severe aplastic anemia in patients without a fully matched related donor.	Established curative therapy for patients with severe aplastic anemia who have a matched unrelated or sibling donor.
How it is given or performed	Patients receive preparative chemotherapy and low-dose total body irradiation, undergo transplantation of donor bone marrow, then receive post-transplant cyclophosphamide and supportive care.	Transplantation is performed after conditioning chemotherapy (sometimes with irradiation), using stem cells from a fully matched unrelated or sibling donor; supportive care and immunosuppression are provided post-transplant.
Evidence status	Ongoing clinical trials and recent cohort studies show promising engraftment rates and survival among patients lacking matched donors, but further comparative data are still being collected.	Multiple large studies and guidelines recognize high rates of sustained engraftment and durable cure for severe aplastic anemia with matched unrelated or sibling donor transplantation.
Important limitations	Limited long-term follow-up data in severe aplastic anemia; potential risks include graft failure, graft-versus-host disease, and regimen-related toxicity. Post-transplant cyclophosphamide may lower some risks but effectiveness and late complications relative to standard transplantation are not fully established.	Not all patients have an available matched unrelated or sibling donor. Procedure risks include graft failure, graft-versus-host disease, infection, and regimen-related toxicity; outcomes depend on patient and donor factors.

Disclaimer: This comparison is created with the help of artificial intelligence(AI) and may contain errors or missing information. It is not medical advice and does not mean one option is better or safer than another. Clinical trials are still being studied, and joining a trial is your choice. Please consult your healthcare professional before making any decisions.

Who Can Participate

Age: 1Year - 75Years

All Genders

Eligibility Criteria

You may qualify if you...

Availability of a 3/6 to 5/6 matched related donor with negative HLA cross-match in host vs. graft direction
Age 65 years or younger if previously treated, and 75 years or younger if previously untreated
Karnofsky Performance Status of 70% or higher
Diagnosis of aplastic anemia with at least two of: neutrophils below 500/mm3, platelets below 20,000/mm3, or absolute reticulocyte count below 40,000/microL
Bone marrow with marked hypocellularity (less than 25% normal) or moderate hypocellularity with 70% non-myeloid precursors plus peripheral blood criteria

You will not qualify if you...

Poor heart function with left ventricular ejection fraction below 40%
Poor lung function with FEV1 and FVC less than 50% predicted
Poor liver function with bilirubin 2 mg/dL or higher
Poor kidney function with creatinine 2.0 mg/dL or higher or creatinine clearance less than 40 mL/min
Prior allogeneic transplant

Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Run-in Period

Duration - 6 days

Participants undergo a preparative regimen including Fludarabine, Cyclophosphamide, Rabbit ATG, and Total Body Irradiation before transplantation.

Daily visits from Day -6 to Day -1

Treatment

Duration - Approximately 180 days

Participants receive haploidentical bone marrow transplantation followed by post-transplant Cyclophosphamide and immunosuppression with Tacrolimus and MMF.

Hospitalization for transplant and treatment; visits on Days +3 and +4 for Cyclophosphamide dosing; immunosuppression monitoring visits until Day +180

Follow-up

Duration - Up to 2 years

Participants are monitored for engraftment, graft-versus-host disease, survival, and other post-transplant outcomes.

Monthly visits for chimerism tests and assessments up to 2 years