Acute Graft-versus-host DiseasePhase 1NCT04539470
Study to Evaluate the Safety and Pharmacokinetics of Efmarodocokin Alfa in Combination With Standard of Care in Participants Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
A Phase 1 study testing Efmarodocokin Alfa in people with Acute Graft-versus-host Disease, run by Genentech, Inc. at 8 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Graft-versus-host Disease
- What is being tested?
- Efmarodocokin Alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 8 sitesUnited States
- Study dates
- 19 Nov 2020to 24 Feb 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Roswell Park Cancer Institute
Buffalo, New York, 14263
University of Chicago
Chicago, Illinois, 60637
City of Hope
Duarte, California, 91010
University of Kansas Med Ctr; Int med/Allgy/Immun/Rheum
Kansas City, Kansas, 66160-7350
Ronald Reagan UCLA Medical Center
Los Angeles, California, 90095
University of Miami Miller School of Medicine; Clinical Reseach Building
Miami, Florida, 33136
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
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
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).
- Conditions
- Acute Graft-versus-host Disease
- Being tested
- Efmarodocokin Alfa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efmarodocokin AlfaDrug
Also called UTTR1147A, RG7880, RO7021610, IL-22Fc
Efmarodocokin Alfa will be administered intravenously (IV) per the dosage specified in each dose escalation cohort.
| Study group | Role | What they receive |
|---|---|---|
| Cohort A: Efmarodocokin Alfa Dosage Level 1 | Experimental | 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. Receives: Drug: Efmarodocokin Alfa |
| Cohort B: Efmarodocokin Alfa Dosage Level 2 | Experimental | 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. Receives: Drug: Efmarodocokin Alfa |
| Cohort C: Efmarodocokin Alfa Dosage Level 3 | Experimental | 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. Receives: Drug: Efmarodocokin Alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: From Baseline up to 365 days
Change from Baseline in Respiratory Rate Over Time
Measured: From Baseline up to 139 days
Change from Baseline in Oxygen Saturation Over Time
Measured: From Baseline up to 139 days
Change from Baseline in Pulse Rate Over Time
Measured: From Baseline up to 139 days
Change from Baseline in Systolic Blood Pressure Over Time
Measured: From Baseline up to 139 days
Change from Baseline in Diastolic Blood Pressure Over Time
Measured: From Baseline up to 139 days
Change from Baseline in Body Temperature Over Time
Measured: From Baseline up to 139 days
Number of Participants with Laboratory Abnormalities in Hematology Tests
Measured: From Baseline up to 139 days
Number of Participants with Laboratory Abnormalities in Blood Chemistry Tests
Measured: From Baseline up to 139 days
Secondary outcomes (2)
Serum Concentration of Efmarodocokin Alfa at Specified Timepoints
Measured: At predefined timepoints from Baseline until Day 139
Number of Participants with Anti-Drug Antibodies (ADAs) at Baseline and During the Study
Measured: At predefined timepoints from Baseline until Day 139
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Sep 2020
- Study start19 Nov 2020
- Primary completion24 Feb 2023
- Study completion24 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Graft-versus-host DiseaseStudies of the same condition, open and under way.
- More trials run by Genentech, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genentech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Genentech, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04539470
- Sponsor's protocol ID
- GA41825
- Status last verified
- Apr 2024
- First submitted
- 31 Aug 2020
- Last updated
- 11 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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).
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04539470
- Acronym / sponsor ID
- GA41825
- Conditions
- Acute Graft-versus-host Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Genentech, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Nov 2020 (actual)
- Primary completion
- 24 Feb 2023 (actual)
- Study completion
- 24 Feb 2023 (actual)
- First posted
- 7 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort A: Efmarodocokin Alfa Dosage Level 1 | Experimental | Drug: Efmarodocokin AlfaParticipants 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 2 | Experimental | Drug: Efmarodocokin AlfaParticipants 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 3 | Experimental | Drug: Efmarodocokin AlfaParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Efmarodocokin AlfaAlso: UTTR1147A, RG7880, RO7021610, IL-22FcEfmarodocokin Alfa will be administered intravenously (IV) per the dosage specified in each dose escalation cohort. | Drug | Cohort A: Efmarodocokin Alfa Dosage Level 1, Cohort B: Efmarodocokin Alfa Dosage Level 2, Cohort C: Efmarodocokin Alfa Dosage Level 3 |
Primary outcome
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
2 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2024.
- Individual participant dataSharing planned: No. 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).
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Genentech, Inc.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Efmarodocokin Alfa in people with Acute Graft-versus-host Disease, run by Genentech, Inc. at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Apr 2024.