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

Primary Purpose

Acute Graft-versus-host Disease

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Efmarodocokin Alfa

About this trial

This is an interventional prevention trial for Acute Graft-versus-host Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

City of Hope
Ronald Reagan UCLA Medical Center
University of Miami Miller School of Medicine; Clinical Reseach Building
University of Chicago
University of Kansas Med Ctr; Int med/Allgy/Immun/Rheum
Dana Farber Cancer Institute
Washington University School of Medicine
Roswell Park Cancer Institute

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Cohort A: Efmarodocokin Alfa Dosage Level 1

Cohort B: Efmarodocokin Alfa Dosage Level 2

Cohort C: Efmarodocokin Alfa Dosage Level 3

Arm Description

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.

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.

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.

Outcomes

Primary Outcome Measures

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)

Change from Baseline in Respiratory Rate Over Time

Change from Baseline in Oxygen Saturation Over Time

Change from Baseline in Pulse Rate Over Time

Change from Baseline in Systolic Blood Pressure Over Time

Change from Baseline in Diastolic Blood Pressure Over Time

Change from Baseline in Body Temperature Over Time

Number of Participants with Laboratory Abnormalities in Hematology Tests

Number of Participants with Laboratory Abnormalities in Blood Chemistry Tests

Secondary Outcome Measures

Serum Concentration of Efmarodocokin Alfa at Specified Timepoints

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

Full Information

NCT ID

NCT04539470

First Posted

August 31, 2020

Last Updated

April 17, 2023

Sponsor

Genentech, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04539470

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Completed

Study Start Date

November 19, 2020 (Actual)

Primary Completion Date

February 24, 2023 (Actual)

Study Completion Date

February 24, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Genentech, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Acute Graft-versus-host Disease

7. Study Design

Primary Purpose

Prevention

Study Phase

Phase 1

Interventional Study Model

Sequential Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

18 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cohort A: Efmarodocokin Alfa Dosage Level 1

Arm Type

Experimental

Arm Description

Arm Title

Cohort B: Efmarodocokin Alfa Dosage Level 2

Arm Type

Experimental

Arm Description

Arm Title

Cohort C: Efmarodocokin Alfa Dosage Level 3

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Efmarodocokin Alfa

Other Intervention Name(s)

UTTR1147A, RG7880, RO7021610, IL-22Fc

Intervention Description

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

Primary Outcome Measure Information:

Title

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

Title

Change from Baseline in Respiratory Rate Over Time

Time Frame

From Baseline up to 139 days

Title

Change from Baseline in Oxygen Saturation Over Time

Time Frame

From Baseline up to 139 days

Title

Change from Baseline in Pulse Rate Over Time

Time Frame

From Baseline up to 139 days

Title

Change from Baseline in Systolic Blood Pressure Over Time

Time Frame

From Baseline up to 139 days

Title

Change from Baseline in Diastolic Blood Pressure Over Time

Time Frame

From Baseline up to 139 days

Title

Change from Baseline in Body Temperature Over Time

Time Frame

From Baseline up to 139 days

Title

Number of Participants with Laboratory Abnormalities in Hematology Tests

Time Frame

From Baseline up to 139 days

Title

Number of Participants with Laboratory Abnormalities in Blood Chemistry Tests

Time Frame

From Baseline up to 139 days

Secondary Outcome Measure Information:

Title

Serum Concentration of Efmarodocokin Alfa at Specified Timepoints

Time Frame

At predefined timepoints from Baseline until Day 139

Title

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

Time Frame

At predefined timepoints from Baseline until Day 139

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Trials

Organizational Affiliation

Genentech, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

City of Hope

City

Duarte

State/Province

California

ZIP/Postal Code

91010

Country

United States

Facility Name

Ronald Reagan UCLA Medical Center

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

University of Miami Miller School of Medicine; Clinical Reseach Building

City

Miami

State/Province

Florida

ZIP/Postal Code

33136

Country

United States

Facility Name

University of Chicago

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60637

Country

United States

Facility Name

University of Kansas Med Ctr; Int med/Allgy/Immun/Rheum

City

Kansas City

State/Province

Kansas

ZIP/Postal Code

66160-7350

Country

United States

Facility Name

Dana Farber Cancer Institute

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02215

Country

United States

Facility Name

Washington University School of Medicine

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

Facility Name

Roswell Park Cancer Institute

City

Buffalo

State/Province

New York

ZIP/Postal Code

14263

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing 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).

Learn more about this trial

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

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