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Efmarodocokin Alfa

Phase 1

Acute Graft-versus-host Disease | Small molecule | Immunology |Roche Holding AG|Last Updated: Apr 11, 2024

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment18

FDA Designations

No designations recorded

Clinical trial landscape

Efmarodocokin Alfa · 1 trial · 1 indication

Phase 1 1
NCT04539470Study to Evaluate the Safety and Pharmacokinetics of Efmarodocokin Alfa in Combination With Standard of Care in Participants Undergoing Allogeneic Hematopoietic Stem Cell TransplantationAcute Graft-versus-host Disease
COMPLETED18 Analytics
PHASE1COMPLETED
Study to Evaluate the Safety and Pharmacokinetics of Efmarodocokin Alfa in Combination With Standard of Care in Participants Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Acute Graft-versus-host Disease
NCT0453947018
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Study Endpoints

Primary Endpoints

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

Secondary Endpoints

Serum Concentration of Efmarodocokin Alfa at Specified Timepoints
At predefined timepoints from Baseline until Day 139
Number of Participants with Anti-Drug Antibodies (ADAs) at Baseline and During the Study
At predefined timepoints from Baseline until Day 139
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposePREVENTION

Treatment Arms

ArmTypeDescription
Cohort A: Efmarodocokin Alfa Dosage Level 1EXPERIMENTALParticipants 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.
Cohort B: Efmarodocokin Alfa Dosage Level 2EXPERIMENTALParticipants 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.
Cohort C: Efmarodocokin Alfa Dosage Level 3EXPERIMENTALParticipants 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.

Interventions

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

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites8

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 ...

Countries:United States
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Frequently asked questions about Efmarodocokin Alfa

What is Efmarodocokin Alfa used for?

Efmarodocokin Alfa is an investigational small molecule being studied for the treatment of Acute Graft-versus-host Disease, a condition that can occur after allogeneic hematopoietic stem cell transplantation. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes Efmarodocokin Alfa?

Efmarodocokin Alfa is being developed by Roche Holding AG, which trades under the ticker RHHBY. The company is conducting clinical trials to evaluate the safety and pharmacokinetics of this investigational drug in patients with Acute Graft-versus-host Disease.

What phase is Efmarodocokin Alfa in?

Efmarodocokin Alfa is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The drug is being studied for its safety and pharmacokinetics in patients with Acute Graft-versus-host Disease.

What clinical trials is Efmarodocokin Alfa in?

Efmarodocokin Alfa has one completed clinical trial, NCT04539470, which evaluated the safety and pharmacokinetics of the drug in combination with standard of care in participants undergoing allogeneic hematopoietic stem cell transplantation. The trial enrolled 18 participants in the United States and was a controlled, non-randomized study.

Is Efmarodocokin Alfa the same as any other drug?

Efmarodocokin Alfa is a distinct investigational drug being developed by Roche Holding AG. No alternative names for this drug have been identified in the available clinical trial information. It is being studied specifically for Acute Graft-versus-host Disease.