Trial of 2 Step ATG for Prevention of Acute GVHD Post Allogeneic Stem Cell Transplant
Phase II Clinical Trial of 2 Step ATG Combined With Tacrolimus and Mini Methotrexate for Prevention of Acute GVHD Post Reduced Intensity Allogeneic Stem Cell Transplant
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years to 75 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests a two-step schedule of anti-thymocyte globulin (ATG), given with tacrolimus and methotrexate before and after a donor stem cell transplant. It aims to prevent graft-versus-host disease (GVHD), when donor immune cells attack the body, and improve survival without GVHD or cancer returning. It is for adults ages 18–75 with certain high-risk blood cancers or bone marrow diseases.
In an effort to reduce graft versus host disease (GVHD) and enhance graft versus leukemia (GVL) effect post allogenic hematopoietic stem cell transplantation (AHSCT), recent research has focused on host immune cell depletion. Frame shifting anti-thymocyte globulin (ATG) backwards to earlier days before days 0 can result in deeper host and less graft T-cell depletion, leading to better immune reconstitution. Preliminary data where 80% of the ATG dose is given on days -6,-5,-4 and 20% given on day -1, showed effective prevention of severe acute GVHD, chronic GVHD and favorable early immune reconstitution. We hypothesize that our 2 step ATG dosing platform when combined with standard tacrolimus and mini methotrexate can prevent grade III-IV acute GVHD and chronic GVHD, resulting in improvement of GVHD/relapse free survival at one year post transplant.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 18–75 and have certain forms of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or myelofibrosis. These are blood cancers or bone marrow diseases that must meet the study’s specific disease and genetic rules.
- Have a willing, eligible donor of stem cells from the blood: a sibling with a 6-of-6 tissue match or an unrelated donor with an 8-of-8 match.
- Be suited for a less intense treatment plan before transplant because you are 60 or older, have a health-risk score of at least 4, or your doctor thinks stronger treatment would be too risky.
- Have heart pumping strength above 40% and kidney clearance above 50 mL/minute, a measure of how well kidneys filter blood.
- Have both required lung test results at least 50%. Liver tests must meet study limits, with a special bilirubin limit for people with Gilbert’s disease.
- Have a daily activity score of at least 70, meaning you can care for yourself but may not be able to do normal activities or work.
- Follow birth control rules from consent through 12 months after transplant: women who can become pregnant must use two effective methods or avoid sex with men. Men with partners who can become pregnant must use barrier birth control or avoid sex with women, even after sterilization.
- Be willing and able to sign the consent form and follow study rules.
Who can't join
- Had a transplant using your own stem cells less than 3 months before enrollment, or ever had a donor stem cell transplant.
- Have AML with more than 5% immature cancer cells in the bone marrow or any in the blood.
- Have uncontrolled heart-related chest pain, severe uncontrolled abnormal heart rhythms, or certain serious heart test findings.
- Have a known allergy to any study drug.
- Used an experimental drug within 14 days before transplant preparation, or joined another study using an experimental product in the last 28 days.
- Are pregnant or breastfeeding.
- Have HIV, an uncontrolled bacterial, viral, or fungal infection, or cirrhosis, which is severe liver scarring.
- Have had a cancer other than a blood cancer within the past 3 years, with some skin and cervical cancer exceptions. Cancer treated to cure it at least 5 years ago is allowed; treatment less than 5 years ago needs approval from the study leader.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Adult male or female, age 18-75 years 2. Patients must have a related or unrelated peripheral blood stem cell donor. Sibling donor must be a 6/6 match for HLA-A and -B at intermediate (or higher) resolution, and -DRB1 at high resolution using DNA-based typing, and must be willing to donate peripheral blood stem cells and meet institutional criteria for donation. Unrelated donor must be 8/8 match at HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing. Unrelated donor must be willing to donate peripheral blood stem cells and be medically eligible to donate stem cells according to NMDP criteria. 3. A candidate for reduced intensity preparative regimen, based on age≥60, or HCT-CI of ≥4, or considered by the treating physician to have high risk for toxicity with myeloablative preparative regimen. 4. Cardiac function: Ejection fraction \>40% 5. Measured creatinine clearance greater than 50 mL/minute (using the Cockcroft-Gault formula and actual body weight) 6. Pulmonary function: DLCO ≥50% (adjusted for hemoglobin) and FEV1≥50% 7. Liver function: total bilirubin \< 1.5x the upper limit of normal and ALT/AST \< 2.5x the upper normal limit. Patients who have been diagnosed with Gilbert's Disease are allowed to exceed the defined bilirubin value of up to \<3mg/dl. 8. Female subjects (unless postmenopausal for at least 1 year before the screening visit, or surgically sterilized), agree to practice two effective methods of contraception or agree to complete abstain from heterosexual intercourse from the time of signing the informed consent through 12 months post-transplant. 9. Male subjects (even if surgically sterilized), of partners of women of childbearing potential must agree to practice effective barrier contraception or abstain from heterosexual intercourse from the time of signing the informed consent through 12 months post-transplant. 10. Karnofsky performance status KPS ≥ 70 11. Patients must have a diagnosis of one of the following: A-AML with either detectable AML on pre AHSCT bone marrow (microscopic ≤5, flow or cytogenetic), or adverse cytogenetic, or molecular features (≥ 4 clonal abnormalities, or monosomal karyotype, inv(3)/t(3;3) or, EV11+, FLT3-ITD (+) without MPN1, P53 mutation positive, ASXL1+, mutant RUNX1. B- MDS with the following features: Residual blasts \> 5% blasts in the bone marrow after hypomethylating agents +/- venetoclax, MDS with high IPSS-R and monosomal karyotype, MDS with P-53 or JAK2 mutation. C-Myelofibrosis with blasts in the peripheral blood. 12. The subject is willing and able to signed informed consent and abide by the protocol requirements. Exclusion Criteria: 1. Autologous hematopoietic stem cell transplant \< 3 months prior to enrollment. 2. Patients with florid residual AML with \> 5% blast in the marrow or circulating blast in the peripheral blood are not eligible for this study. 3. Previous allogeneic stem cell transplant. 4. Uncontrolled angina, severe uncontrolled ventricular arrhythmias, or EKG suggestive of acute ischemia or active conduction system abnormalities. 5. Known hypersensitivity to one or more of the study agents 6. Received any investigational drugs within the 14 days prior to the first day of transplant conditioning 7. Pregnant and/or breastfeeding 8. Evidence of HIV infection or known HIV positive serology. 9. Current uncontrolled bacterial, viral or fungal infection (currently taking medication with evidence of progression of clinical symptoms or radiologic findings). 10. Patient with documented cirrhosis 11. Non-hematologic malignancy within prior three (3) years, with the exception of squamous cell or basal cell skin carcinoma. Patients with prior malignancies except resected localized non-melanoma skin cancer or treated cervical carcinoma in situ. Cancer treated with curative intent ≥ 5 years previously will be allowed. Cancer treated with curative intent \< 5 years previously must be reviewed and approved by the PI 12. Participation in another clinical study with an investigational product during the last 28 days.
Study sites (1)
AL
- University of Alabama at Birmingham · Birmingham, AL 35294Zaid Al Kadhimi, MD · Study contact · (205) 975-1269 · zsalkadhimi@uabmc.eduZaid Al Kadhimi, MD · Principal investigatorManuel Espinoza-Gutarra, MD · Sub-investigatorDonna Salzman, MD · Sub-investigatorMore trials in Birmingham