ATG Dosing for Blood Cancer

Recruiting in Palo Alto (17 mi)

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: University of Alabama at Birmingham

No Placebo Group

Prior Safety Data

Approved in 3 jurisdictions

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

This trial is for individuals with acute leukemia, myeloproliferative disorders, preleukemia, or myelodysplastic syndromes who are undergoing allogeneic stem cell transplant. The study aims to prevent severe graft versus host disease (GVHD). Specific eligibility criteria details were not provided.

Inclusion Criteria

My lung function tests show at least half the normal capacity.

My liver tests are within the required range.

I agree to use birth control or abstain from sex.

I am between 18 and 75 years old.

I am able to care for myself but may not be able to do active work.

I have been diagnosed with AML, MDS, or Myelofibrosis.

I have a stem cell donor who matches the required HLA criteria.

My heart pumps well, with an ejection fraction over 40%.

Exclusion Criteria

I had a stem cell transplant using my own cells less than 3 months ago.

I have had a stem cell transplant from a donor.

I do not have uncontrolled heart problems or abnormal heart rhythms.

I have an infection that is not under control.

I have been diagnosed with cirrhosis.

My leukemia is active with more than 5% of immature blood cells.

Participant Groups

The trial tests a new way of giving anti-thymocyte globulin (ATG) in two steps before the transplant to reduce GVHD while improving immune response and survival without relapse or GVHD at one year post-transplant. It's combined with standard tacrolimus and mini methotrexate.

1Treatment groups

Experimental Treatment

Group I: phase II single arm study of 2 step ATG dosing in prevention of aGVHDExperimental Treatment1 Intervention

The primary outcome for the study is GRFS rate at one-year post transplant. GRFS will be estimated using Kaplan Meier method The reported GRFS with recent phase III trial of PTCY/tac/MMF in transplant from matched related and unrelated donors at 1 year follow up was 52%. We hypothesize that with 2 step ATG/Tac/Mini MTX regimen, we can achieve a one year GRFS of 69%.

ATG is already approved in United States, European Union, Canada for the following indications:

🇺🇸 Approved in United States as Thymoglobulin for:

Prevention and treatment of acute rejection in patients undergoing kidney transplantation
Treatment of aplastic anemia in patients who are not candidates for bone marrow transplantation

🇪🇺 Approved in European Union as Thymoglobulin for:

Prevention and treatment of acute rejection in patients undergoing kidney transplantation
Treatment of aplastic anemia in patients who are not candidates for bone marrow transplantation

🇨🇦 Approved in Canada as Thymoglobulin for:

Prevention and treatment of acute rejection in patients undergoing kidney transplantation
Treatment of aplastic anemia in patients who are not candidates for bone marrow transplantation