Stem Cell Transplantation for Blood Cancers with Germline RUNX1 Mutations
This study is for people aged 4 to 70 who have blood cancers like leukemia or lymphoma, and also have a specific inherited gene change called a germline RUNX1 mutation. It's testing a stem cell transplant approach that aims to improve outcomes by carefully adjusting the doses of chemotherapy and supportive care medications for each person. The chemotherapy drugs used are Cyclophosphamide, Busulfan, and Fludarabine, along with Total Body Irradiation (radiation therapy). You would then receive Hematopoietic stem cells. The main goal is to see how many participants are free of disease one year after the transplant. The study is currently unclear on its recruitment status.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 98 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for at least one year after the stem cell transplant to measure disease-free survival.
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NCT07524530
Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers
Not Yet Recruiting
PHASE2Ages 4–70InterventionalTreatmentNational Cancer Institute (NCI)Government-funded
~102 participants
Updated 2026-08-28 on ClinicalTrials.gov
What's tested:CyclophosphamideBusulfanFludarabineTotal Body IrradiationHematopoietic stem cellsTacrolimus
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To determine the proportion of participants with disease free survival at 1 year post haploidentical transplantation.
Measured over 1 year post HSCT
Conditions
Where it's being run
1 sites across 1 statesMaryland1
Study leadership
- Lea C Cunningham, M.D. · PRINCIPAL_INVESTIGATOR · National Cancer Institute (NCI)
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Affected participants (Recipients)
History (review of lab test result primary source) of deleterious or suspected deleterious (defined as P/LP or VUS with RUNX1 phenotype) germline RUNX1 mutation as defined by ClinVar (nih.gov)
Histological confirmation of a myeloid malignancy - acute or chronic leukemia (\<5% marrow blasts preferred) or myelodysplastic syndrome/myeloproliferative neoplasms (MDS/MPN) (\<10% marrow blasts preferred). Participants may be treated on this study to achieve preferred blast cutoffs. Participants with poorly responsive or relapsed disease remain eligible and may proceed as the graft-versus-leukemia (GVL) effect may produce cures.
Availability of a haploidentical donor (HLA haploidentical-match only).
Age \>= 4 and \<= 70 years
Karnofsky (\>=16 years) or Lansky (\<16 years) \>=60%
For human immunodeficiency virus (HIV)-infected participants, participant must be on effective anti-retroviral therapy, without uncontrolled opportunistic infection and have approval via Transplant Infectious Disease consultation. Consider donor with CCR5(delta)32 homozygosity for these participants.
For individuals with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load (VL) must be undetectable on suppressive therapy, if indicated.
Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. Participants with active HCV infection who are currently on treatment must have an undetectable HCV VL.
Contraception as follows:
Women of child-bearing potential (WOCBP) must agree to use highly effective contraception (hormonal, intrauterine device \[IUD\], abstinence, surgical sterilization) at the study entry and up to and 12 months post conditioning and/or post-transplant.
Men that can father a child must agree to use an effective method of contraception (barrier, surgical sterilization, abstinence) at the study entry and up to 12 months posttransplant or 4 months after conditioning if transplant is not done. We also will recommend men that can father children with partners that can bear children ask their partners to be on highly effective birth control (hormonal, IUD, surgical sterilization). Men that can father children must not freeze or donate sperm within the same period.
Breastfeeding participants must be willing to discontinue breastfeeding during the study and for 12 months post-transplant or 1 week after conditioning if transplant is not done.
Willingness to remain in the NIH hospital or, if discharged, stay close to the NIH (30 minutes drive), for a minimum of 100 days after transplant or longer if there are complications. The participants must commit to having an adult caregiver with them during the first 100 days after the transplant
Participants or parent/guardian/legally authorized representative must be able to understand and willing to sign a written informed consent document.
Additional criteria for recipients suitable for MAC
Age \<= 65 years
HCT-CI \<4
Pulmonary function tests (PFTs): Forced expiratory volume in the first second (FEV1) and adjusted diffusion capacity of carbon monoxide (DLCO) \>=66%, without dyspnea at rest or oxygen requirement. If too young to cooperate with PFTs, must have \>=92% oxygen saturation on room air and no dyspnea at rest.
Left ventricular ejection fraction (LVEF) \>=50% by echocardiogram (ECHO) or Multigated Acquisition (MUGA) scan (101)
Recipients must have adequate organ function as defined below:
Total bilirubin \<1.5 x institutional upper limit of normal (iULN) (unless Gilbert disease, hemolysis)
Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) \<=2.5 x iULN (unless therapy related and will improve in discussion with the National Institute of Diabetes and Digestive and Kidney Diseases \[NIDDK\])
Creatinine within normal institutional limits or 24-hour urine or calculated creatinine clearance \>=60 mL/min/1.73 m\^2 for individuals with creatinine levels above institutional normal (calculated using the Chronic Kidney Disease Epidemiology Collaboration \[CKD-EPI\] equation)
Additional criteria for recipients suitable for RIC
Age \<=70 years
PFTs: FEV1 and DLCO \>=50%, without dyspnea at rest or oxygen requirement. If too young to cooperate with PFTs, must have \>=92% oxygen saturation on room air and no dyspnea at rest
LVEF \>=40% by ECHO or MUGA obtained within 2 months of HSCT (Children s Oncology Group \[COG\] criteria).
Recipients must have adequate organ function as defined below:
Total bilirubin \<2.5 x iULN (unless Gilbert disease, hemolysis)
AST/ALT \<3.5 x iULN (unless therapy related and will improve in discussion with NIDDK)
Creatinine within normal institutional limits or 24-hour urine or calculated creatinine clearance \>=50 mL/min/1.73 m\^2 for individuals with creatinine levels above institutional normal (calculated using the CKD-EPI equation)
Unaffected participants
Haploidentical donors
Age \>=4 years
Participants or parent/guardian must be able to understand and willing to sign a written informed consent document.
Unaffected family members
Age \>=18 years
If the participant is a blood relative of the recipient, participant must be negative for RUNX1 mutations by molecular testing
Participants must be able to understand and willing to sign a written informed consent document.
Exclusion
All participants
Recipients who are receiving any investigational agent except virus specific T cells (VST)
Active non-hematologic malignancies
History of allergic reactions attributed to compounds of similar chemical or biologic composition to the drugs used in study.
Participants with the following cardiac conditions: symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia (except atrial fibrillation if cleared by cardiology consultation)
Participants without access to medical care at home.
Positive serum or urine beta-human chorionic gonadotropin (beta-hCG) test at screening
Uncontrolled intercurrent illness evaluated by history, physical exam, and laboratory studies or situations that would limit compliance with study requirements, interpretation of results or that could increase risk to the participant
What this trial measures
- To determine the proportion of participants with disease free survival at 1 year post haploidentical transplantation.1 year post HSCT
The proportion of participants with disease-free survival (DFS) at one year post transplantation will be reported separately by treatment arm along with 80% and 95% two-sided confidence intervals.