Clinical trial for Severe Aplastic Anemia
Official title Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
ClinicalTrials.gov ID: NCT06517641
What this study is testing
What is Haploidentical donor bone marrow transplant?
This is an investigational procedure being studied as a potential treatment for severe aplastic anemia. It is designed to replace the patient's bone marrow with healthy stem cells from a donor who shares some, but not all, genetic markers.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study will look at whether bone marrow transplant can restore normal blood cell production in these patients.
- Who it's for
- Adults with severe aplastic anemia that has not been treated before.
- What's involved
- Participants will receive a bone marrow transplant from a donor and will have follow‑up visits and tests to monitor recovery.
- Time commitment
- The study lasts about a year, with regular clinic visits after the transplant.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 75
You may be able to join if
- Age 3 years to 75 years
- Confirmed diagnosis of acquired SAA defined as: a. Bone marrow cellularity \< 25% or variable marrow cellularity but with \< 30% residual...
- No suitable fully matched related donor as per Investigator's discretion (6/6 match for HLA A and B at intermediate or high-resolution and DRB1 at...
- Available donor as defined in the protocol.
- Participant and/or legal guardian must sign informed consent.
You likely can't join if
- Inherited bone marrow failure syndromes such as Fanconi anemia and short telomere syndromes must be ruled out according to center standards.
- Clonal cytogenetic abnormalities consistent with pre-MDS or MDS on marrow examination (e.g., monosomy 7 and other MDS-defining changes per recent...
- Formal diagnosis of MDS by World Health Organization (WHO) 2022 or International Consensus Classification (ICC).
- Recipient positive for HLA antibodies against a mismatched HLA in the selected donor determined by the presence of donor specific HLA antibodies...
- Prior desensitization attempt for HLA antibodies to chosen donor. Any intervention with the sole intent to reduce the level of HLA DSA, (e.g...
- Prior treatment for SAA (e.g., immunosuppressive therapy using ATG, calcineurin inhibitors [CNIs], thrombopoietin receptor agonists or androgens).
See the full eligibility criteria
- Age 3 years to 75 years
- Confirmed diagnosis of acquired SAA defined as: a. Bone marrow cellularity \< 25% or variable marrow cellularity but with \< 30% residual hematopoietic cells deemed HYPOcellular for age AND b.
- No suitable fully matched related donor as per Investigator's discretion (6/6 match for HLA A and B at intermediate or high-resolution and DRB1 at high-resolution using deoxyribonucleic acid [DNA]-based typing)...
- Available donor as defined in the protocol.
- Participant and/or legal guardian must sign informed consent.
- Cardiac: Left ventricular ejection fraction (LVEF) at rest \> 40% with no clinical signs of cardiac failure.
- Hepatic: Total bilirubin \< 2.0 mg/dL unless Gilbert's disease is present
- Renal: For participants \> 13.0 years of age at the time of enrollment: estimated creatinine clearance (CrCl) \> 60 mL/minute (per institutional standard). For participants \ 50 mL/min/1.73 m2.
- Pulmonary: i. For participants \> 13.0 years of age: Diffusing capacity of the lung for carbon monoxide (DLCO, corrected/adjusted for hemoglobin [Hb]) \> 50%, or Spirometry with forced expiratory volume 1 (FEV1) \> 50%...
- Karnofsky or Lansky performance status ≥ 60%.
- Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence.
- Inherited bone marrow failure syndromes such as Fanconi anemia and short telomere syndromes must be ruled out according to center standards.
- Clonal cytogenetic abnormalities consistent with pre-MDS or MDS on marrow examination (e.g., monosomy 7 and other MDS-defining changes per recent pathology guidelines).
- Formal diagnosis of MDS by World Health Organization (WHO) 2022 or International Consensus Classification (ICC).
- Recipient positive for HLA antibodies against a mismatched HLA in the selected donor determined by the presence of donor specific HLA antibodies (DSA) to any mismatched HLA allele/antigen at any of the following loci...
- Prior desensitization attempt for HLA antibodies to chosen donor. Any intervention with the sole intent to reduce the level of HLA DSA, (e.g., plasmapheresis, intravenous immunoglobulin [IVIG], MMF, etc.) would...
- Prior treatment for SAA (e.g., immunosuppressive therapy using ATG, calcineurin inhibitors [CNIs], thrombopoietin receptor agonists or androgens).
- Prior allogeneic stem cell transplant.
- Prior solid organ transplant.
- Known life-threatening reaction (i.e., anaphylaxis) to Thymoglobulin® (Sanofi) that would prohibit use for the participant as this study requires use of the Thymoglobulin® (Sanofi) preparation of ATG.
- Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment.
- Female participants who are pregnant, as detected using a pregnancy test as per institutional practice, or breast-feeding.
- Prior infections must be controlled
- HepB participants are eligible if on effective suppressive therapy and otherwise meet inclusion/exclusion criteria
- HepC participants are eligible if otherwise meet inclusion/exclusion criteria
The study team makes the final eligibility decision.
Where it is enrolling
- Birmingham, Alabama, United States
- Duarte, California, United States
- Los Angeles, California, United States
- Stanford, California, United States
- Tampa, Florida, United States
- Atlanta, Georgia, United States
- Westwood, Kansas, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Detroit, Michigan, United States
- Rochester, Minnesota, United States
- St Louis, Missouri, United States
- New York, New York, United States
- Chapel Hill, North Carolina, United States
- Charlotte, North Carolina, United States
- Durham, North Carolina, United States
- Columbus, Ohio, United States
- Oklahoma City, Oklahoma, United States
- Philadelphia, Pennsylvania, United States
- Nashville, Tennessee, United States
+ 3 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Birmingham, Alabama, United States; Duarte, California, United States; Los Angeles, California, United States; Stanford, California, United States; Tampa, Florida, United States; Atlanta, Georgia, United States and 17 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.