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

April 10, 2024 updated by: Genentech, Inc.

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

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

18

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Duarte, California, United States, 91010
        • City of Hope
      • Los Angeles, California, United States, 90095
        • Ronald Reagan UCLA Medical Center
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami Miller School of Medicine; Clinical Reseach Building
    • Illinois
      • Chicago, Illinois, United States, 60637
        • University of Chicago
    • Kansas
      • Kansas City, Kansas, United States, 66160-7350
        • University of Kansas Med Ctr; Int med/Allgy/Immun/Rheum
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Dana Farber Cancer Institute
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • Buffalo, New York, United States, 14263
        • Roswell Park Cancer Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort A: Efmarodocokin Alfa Dosage Level 1
Participants undergoing allogeneic hematopoietic stem cell transplantation will receive Efmarodocokin Alfa dosage level 1 in combination with standard of care prophylaxis treatment for acute graft versus-host disease (aGVHD), consisting of tacrolimus plus methotrexate per institutional practices.
Drug: Efmarodocokin Alfa
Efmarodocokin Alfa will be administered intravenously (IV) per the dosage specified in each dose escalation cohort.
Other Names:
  • UTTR1147A
  • RG7880
  • RO7021610
  • IL-22Fc
Experimental: Cohort B: Efmarodocokin Alfa Dosage Level 2
Participants undergoing allogeneic hematopoietic stem cell transplantation will receive Efmarodocokin Alfa dosage level 2 in combination with standard of care prophylaxis treatment for acute graft versus-host disease (aGVHD), consisting of tacrolimus plus methotrexate per institutional practices.
Drug: Efmarodocokin Alfa
Efmarodocokin Alfa will be administered intravenously (IV) per the dosage specified in each dose escalation cohort.
Other Names:
  • UTTR1147A
  • RG7880
  • RO7021610
  • IL-22Fc
Experimental: Cohort C: Efmarodocokin Alfa Dosage Level 3
Participants undergoing allogeneic hematopoietic stem cell transplantation will receive Efmarodocokin Alfa dosage level 3 in combination with standard of care prophylaxis treatment for acute graft versus-host disease (aGVHD), consisting of tacrolimus plus methotrexate per institutional practices.
Drug: Efmarodocokin Alfa
Efmarodocokin Alfa will be administered intravenously (IV) per the dosage specified in each dose escalation cohort.
Other Names:
  • UTTR1147A
  • RG7880
  • RO7021610
  • IL-22Fc

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
From Baseline up to 365 days
Change from Baseline in Respiratory Rate Over Time
Time Frame: From Baseline up to 139 days
From Baseline up to 139 days
Change from Baseline in Oxygen Saturation Over Time
Time Frame: From Baseline up to 139 days
From Baseline up to 139 days
Change from Baseline in Pulse Rate Over Time
Time Frame: From Baseline up to 139 days
From Baseline up to 139 days
Change from Baseline in Systolic Blood Pressure Over Time
Time Frame: From Baseline up to 139 days
From Baseline up to 139 days
Change from Baseline in Diastolic Blood Pressure Over Time
Time Frame: From Baseline up to 139 days
From Baseline up to 139 days
Change from Baseline in Body Temperature Over Time
Time Frame: From Baseline up to 139 days
From Baseline up to 139 days
Number of Participants with Laboratory Abnormalities in Hematology Tests
Time Frame: From Baseline up to 139 days
From Baseline up to 139 days
Number of Participants with Laboratory Abnormalities in Blood Chemistry Tests
Time Frame: From Baseline up to 139 days
From Baseline up to 139 days

Secondary Outcome Measures

Outcome Measure
Time Frame
Serum Concentration of Efmarodocokin Alfa at Specified Timepoints
Time Frame: At predefined timepoints from Baseline until Day 139
At predefined timepoints from Baseline until Day 139
Number of Participants with Anti-Drug Antibodies (ADAs) at Baseline and During the Study
Time Frame: At predefined timepoints from Baseline until Day 139
At predefined timepoints from Baseline until Day 139

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genentech, Inc.

Investigators

  • Study Director: Clinical Trials, Genentech, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 19, 2020

Primary Completion (Actual)

February 24, 2023

Study Completion (Actual)

February 24, 2023

Study Registration Dates

First Submitted

August 31, 2020

First Submitted That Met QC Criteria

August 31, 2020

First Posted (Actual)

September 7, 2020

Study Record Updates

Last Update Posted (Actual)

April 11, 2024

Last Update Submitted That Met QC Criteria

April 10, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GA41825

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Qualified researchers may request access to individual patient level data through the request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/).

For further details on Roche's Global Policy on Sharing of Clinical Study Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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