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

• ClinicalTrials.gov Identifier: NCT04539470
• Recruitment Status: Completed
• First Posted: September 7, 2020
• Last Update Posted: April 11, 2024
• Sponsor: Genentech, Inc.
• Information provided by (Responsible Party): Genentech, Inc.

Tracking Information

• First Submitted Date: August 31, 2020
• First Posted Date: September 7, 2020
• Last Update Posted Date: April 11, 2024
• Actual Study Start Date: November 19, 2020
• Actual Primary Completion Date: February 24, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 31, 2020)

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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 8, 2021)

• Serum Concentration of Efmarodocokin Alfa at Specified Timepoints [ Time Frame: 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 ]

Original Secondary Outcome Measures (submitted: August 31, 2020)

• Serum Concentration of UTTR1147A at Specified Timepoints [ Time Frame: 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 ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• 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).
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: Non-Randomized; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Prevention
• Condition: Acute Graft-versus-host Disease

Intervention

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

Study Arms

• 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.
  Intervention: Drug: Efmarodocokin Alfa
• 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.
  Intervention: Drug: Efmarodocokin Alfa
• 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.
  Intervention: Drug: Efmarodocokin Alfa

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 21, 2022): 18
• Original Estimated Enrollment (submitted: August 31, 2020): 24
• Actual Study Completion Date: February 24, 2023
• Actual Primary Completion Date: February 24, 2023 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04539470
• Other Study ID Numbers: GA41825
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

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).

• Current Responsible Party: Genentech, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Genentech, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Clinical Trials; Genentech, Inc.

• PRS Account: Genentech, Inc.
• Verification Date: April 2024