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Completed

NCT Number: NCT04539470

Study to Evaluate the Safety and Pharmacokinetics of Efmarodocokin Alfa in Combination With Standard of Care in Participants Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

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).

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Key information

Conditions

Acute Graft-versus-host Disease

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

City of Hope, Duarte, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

Efmarodocokin Alfa

Drug

Efmarodocokin Alfa will be administered intravenously (IV) per the dosage specified in each dose escalation cohort.

Other names: UTTR1147A, RG7880, RO7021610, IL-22Fc

Primary outcomes

  1. Number of Participants with Adverse Events by Severity, According to National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0 (NCI-CTCAE v5.0)

    Time frame: From Baseline up to 365 days

  2. Change from Baseline in Respiratory Rate Over Time

    Time frame: From Baseline up to 139 days

  3. Change from Baseline in Oxygen Saturation Over Time

    Time frame: From Baseline up to 139 days

  4. Change from Baseline in Pulse Rate Over Time

    Time frame: From Baseline up to 139 days

  5. Change from Baseline in Systolic Blood Pressure Over Time

    Time frame: From Baseline up to 139 days

  6. Change from Baseline in Diastolic Blood Pressure Over Time

    Time frame: From Baseline up to 139 days

  7. Change from Baseline in Body Temperature Over Time

    Time frame: From Baseline up to 139 days

  8. Number of Participants with Laboratory Abnormalities in Hematology Tests

    Time frame: From Baseline up to 139 days

  9. Number of Participants with Laboratory Abnormalities in Blood Chemistry Tests

    Time frame: From Baseline up to 139 days

Secondary outcomes

  1. Serum Concentration of Efmarodocokin Alfa at Specified Timepoints

    Time frame: At predefined timepoints from Baseline until Day 139

  2. Number of Participants with Anti-Drug Antibodies (ADAs) at Baseline and During the Study

    Time frame: At predefined timepoints from Baseline until Day 139

Sponsors and collaborators

Lead sponsor

Genentech, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Sep 7, 2020
Registry last updated
Apr 11, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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