for people ages 18 years and up (full criteria)
study started
estimated completion



This is a Phase Ib, open-label, multicenter, dose-escalation study to evaluate the safety, tolerability, and pharmacokinetics of Efmarodocokin Alfa and to make a preliminary assessment of activity of Efmarodocokin Alfa in combination with standard-of-care (SOC) in the prevention of acute graft-versus-host disease (aGVHD) in participants undergoing allogeneic hematopoietic stem cell transplantation (HSCT).

Official Title

A Phase Ib, Open-Label, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Efmarodocokin Alfa in Combination With Standard of Care in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation


Acute Graft-versus-host Disease, Graft vs Host Disease, Efmarodocokin Alfa


You can join if…

Open to people ages 18 years and up

  • Eligible for hematopoietic stem cell transplantation (HSCT)
  • Donor meeting human leukocyte antigen (HLA) matching criteria of HLA-matched related or HLA-matched unrelated (HLA-A, HLA-B, HLA-C, and HLA-DRB1, eight out of eight) from either peripheral blood or bone marrow stem cells and meeting donor-eligibility criteria as outlined by the U.S. Food and Drug Administration (FDA) in 21 CFR 1271 (including screening for Zika and SARS-CoV-2 exposure or infection)
  • Planned HLA (HLA-A, HLA-B, HLA-C, and HLA-DRB1)-matched (eight out of eight) related or planned HLA-matched (eight out of eight) unrelated HSCT, from either peripheral blood or bone marrow stem cells, for patients with acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL) in first complete remission (per institutional criteria) or patients with intermediate or high-risk myelodysplastic syndrome (MDS)
  • Planned myeloablative conditioning regimen per institutional guidelines
  • Planned aGvHD prophylaxis consisting of tacrolimus and methotrexate; in cases of tacrolimus intolerance, cyclosporine or sirolimus may be used as a substitute

You CAN'T join if...

  • Prior receipt of autologous or allogeneic HSCT
  • Diagnosis of myelofibrosis or myelodysplastic/myeloproliferative overlap syndrome
  • Treatment with investigational biologic or non-biologic therapy within 5 drug elimination half-lives (or within 90 days or 30 days, respectively, if half-life is unknown) prior to initiation of study drug
  • Positive hepatitis B virus (HBV) or hepatitis C virus (HCV) serologies
  • History of Grade >1 cervical intraepithelial neoplasia
  • A marked baseline prolongation of QT/QTc interval
  • Risk factors for torsades de pointes
  • Pregnant or breastfeeding
  • Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study


  • Ronald Reagan UCLA Medical Center
    Los Angeles California 90095 United States
  • City of Hope
    Duarte California 91010 United States


in progress, not accepting new patients
Start Date
Completion Date
Genentech, Inc.
Phase 1 research study
Study Type
About 18 people participating
Last Updated