Actively Recruiting
A Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Severe Aplastic Anemia Patients With COH-MC-17: A Non-Myeloablative/ Reduced-Intensity Conditioning Regimen and CD4+ T-Cell-Depleted Haploidentical Hematopoietic Transplant
Led by City of Hope Medical Center ยท Updated on 2026-01-05
6
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
C
City of Hope Medical Center
Lead Sponsor
N
National Cancer Institute (NCI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating a reduced-intensity treatment regimen including cyclophosphamide, pentostatin, and anti-thymocyte globulin before a CD4+ T-cell depleted haploidentical hematopoietic cell transplant (haploHCT) for patients with severe aplastic anemia that is refractory or recurrent. This phase I trial aims to assess the safety and feasibility of this approach, including the ability to prepare the required cell product and reduce risks such as graft versus host disease. Participants receive cyclophosphamide both orally and intravenously, pentostatin intravenously, and anti-thymocyte globulin intravenously before undergoing the CD4+ T-cell depleted haploHCT. The study includes procedures like bone marrow aspirate and biopsy, along with blood sample collection at screening and follow-up. The regimen is designed to prepare the patient's body for the transplant by making room for healthy cells and modulating the immune response. During the study, participants will be monitored for adverse events, blood cell recovery, infections, graft versus host disease, survival rates, and donor cell engraftment over periods extending up to three years. Assessments include lab tests, bone marrow evaluations, and questionnaires. Researchers will also study immune cell profiles and bone marrow environment changes for up to two years after transplant to better understand treatment effects and long-term outcomes.
CONDITIONS
Brief Title
A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented informed consent of the participant.
- Age 40 to 75 years at enrollment.
- Failed at least one trial of immunosuppressive therapy due to refractory or relapsed severe aplastic anemia.
- Karnofsky performance score of 60% or higher.
- Confirmed diagnosis of severe aplastic anemia with specific bone marrow and blood count criteria.
- No suitable fully matched related sibling donor available.
- Available relative who is a haploidentical match and meets defined genetic matching criteria.
- Patient and donor/legal guardians able to provide informed consent.
- Donor willing and able to donate bone marrow and weighs at least 20 kg.
- Liver function within specified limits, allowing Gilbert's Disease exception.
- Kidney function meeting creatinine clearance or GFR requirements.
- Negative pregnancy test for women of childbearing potential.
- Heart function meeting specified echocardiogram or MUGA criteria.
- Lung function meeting specified pulmonary test criteria or equivalent oxygenation status.
- Negative for HIV, hepatitis B and C, and syphilis by specified tests.
- Antibodies to donor red blood cell antigens meet institutional requirements.
- Agreement to use adequate contraception before and after transplant.
- Donor age younger than 60 years.
- Donor selection based on HLA typing and other compatibility factors.
- Donor infectious disease screening negative as specified.
- Donor pregnancy testing for women of childbearing potential.
- Donor approved and completed evaluation per institutional guidelines.
You will not qualify if you...
- Inherited or acquired bone marrow failure syndromes other than aplastic anemia.
- Clonal cytogenetic abnormalities or diagnosis of myelodysplastic syndrome.
- Presence of certain somatic mutations except PIG-A or BCOR.
- Positive anti-donor HLA antibodies.
- Prior allogeneic stem cell or solid organ transplant.
- Known life-threatening reaction to Thymoglobulin.
- Uncontrolled active infections.
- Seropositive for HIV or active hepatitis B or C with detectable viral load.
- Pregnant or breastfeeding women.
- Prior malignancies except certain treated cancers beyond five years.
- Alemtuzumab or ATG treatment within two weeks before enrollment.
- Any condition contraindicating study participation due to safety concerns.
- Inability to comply with study procedures.
- Donor history of transplantation or receiving investigational or conflicting therapies.
- Donor active infection or medical reasons preventing tolerance of growth factor therapy.
- Donor pregnant or breastfeeding within six months if female of childbearing potential.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 3 weeks
Participants receive cyclophosphamide orally and intravenously, pentostatin intravenously, and anti-thymocyte globulin intravenously as a reduced-intensity conditioning regimen.
Multiple visits during conditioning period
Duration - Day 0
Participants undergo CD4+ T-cell depleted haploidentical hematopoietic stem cell transplantation (haploHCT).
1 transplant procedure visit (in-person)
Duration - Up to 3 years
Participants are monitored for safety, hematologic recovery, graft-versus-host disease, chimerism, and survival outcomes with blood samples and bone marrow assessments.
Regular follow-up visits with blood sample and bone marrow aspirate/biopsy assessments at specified intervals
Trial Site Locations
Total: 1 location
1
City of Hope Medical Center
Duarte, California, United States, 91010
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here