Phase II Trial of Efprezimod Alfa (CD24Fc, MK-7110) for the Prevention of Acute Graft-Versus-Host Disease (GVHD) Following Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) (MK-7110-002)

NCT ID: NCT02663622

Last Updated: 2024-01-23

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

44 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-09-19

Study Completion Date

2021-05-18

Brief Summary

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This is a multicenter prospective phase IIa dose escalation and phase IIa expansion cohort clinical trial designed to evaluate the safety and tolerability of efprezimod alfa for acute GVHD prophylaxis.

Detailed Description

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The first part of this study was a phase IIa randomized, double blind, placebo controlled, multi-center study to investigate adding efprezimod alfa to standard of care tacrolimus and methotrexate in acute graft-versus host disease (GVHD) prophylaxis for allogeneic hematopoietic stem cell transplantation (HCT) with matched unrelated donors in treatment of leukemia and myelodysplastic syndrome. The primary objective was to evaluate the safety, tolerability and dose-limiting toxicities (DLTs) of efprezimod alfa in participants undergoing matched unrelated donor myeloablative allogeneic HCT for malignant hematologic disorders. Three dose cohorts were planned with 240 mg at day -1 (one day prior to HCT), 480 mg at day -1, and the multi-dose cohort of 480-240-240 mg at day -1, day 14 and day 28. The efprezimod alfa : placebo randomization ratio was 3:1.

The second part was a prospective open label phase IIa expansion cohort trial investigating the addition of efprezimod alfa to standard acute graft-versus host disease (GVHD) prophylaxis for allogeneic hematopoietic stem cell transplantation (HCT). Based on the first part's safety results and the pharmacokinetic data, the phase IIa expansion dose was the multi-dose 480-240-240 mg regimen administered on day -1, day 14 and day 28, respectively. The primary objective of phase IIa expansion was to determine if the addition of efprezimod alfa to standard GVHD prophylaxis improves 180 days post-HCT grade III-IV acute GVHD-free survival (AGFS) when compared to Center for International Blood and Marrow Transplant Research (CIBMTR) database registered control participants who had standard GVHD prophylaxis alone. Eligible participants were those requiring allogeneic HCT for malignant hematologic conditions and receiving a myeloablative conditioning regimen.

Conditions

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Graft Versus Host Disease Hematopoietic Stem Cell Transplantation Leukemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Study was double blind for the first 6 participants in each arm. At that point, the recommended phase II dose (RP2D) was determined, and the selected arm enrolled additional participants in an open label fashion.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Efprezimod alfa 240 mg

Efprezimod alfa in 240 mg as intravenous (IV) infusion at Day -1 + Tacrolimus (begin on day -3. IV \[0.03 mg/kg/day\] or PO \[0.045 mg/kg/dose\] dosing is permitted) + Methotrexate (given intravenously at a dose of 15 mg/square meter/dose once daily on Day 1 after HCT, and at a dose of 10 mg/square meter/dose on days 3, 6, and 11 after HCT)

Group Type EXPERIMENTAL

Efprezimod alfa

Intervention Type DRUG

Acute GVHD prophylaxis

Methotrexate

Intervention Type DRUG

Acute GVHD prophylaxis

Tacrolimus

Intervention Type DRUG

Acute GVHD prophylaxis

Efprezimod alfa 480 mg

Efprezimod alfa in 480 mg as intravenous (IV) infusion at Day -1 + Tacrolimus (begin on day -3. IV \[0.03 mg/kg/day\] or PO \[0.045 mg/kg/dose\] dosing is permitted) + Methotrexate (given intravenously at a dose of 15 mg/square meter/dose once daily on Day 1 after HCT, and at a dose of 10 mg/square meter/dose on days 3, 6, and 11 after HCT)

Group Type EXPERIMENTAL

Efprezimod alfa

Intervention Type DRUG

Acute GVHD prophylaxis

Methotrexate

Intervention Type DRUG

Acute GVHD prophylaxis

Tacrolimus

Intervention Type DRUG

Acute GVHD prophylaxis

Efprezimod alfa 960 mg

Efprezimod alfa (480 mg (day -1), 240 mg (day +14) and 240 mg (day +28)) + Tacrolimus (begin on day -3. IV \[0.03 mg/kg/day\] or PO \[0.045 mg/kg/dose\] dosing is permitted) + Methotrexate (given intravenously at a dose of 15 mg/square meter/dose once daily on Day 1 after HCT, and at a dose of 10 mg/square meter/dose on days 3, 6, and 11 after HCT)

Group Type EXPERIMENTAL

Efprezimod alfa

Intervention Type DRUG

Acute GVHD prophylaxis

Methotrexate

Intervention Type DRUG

Acute GVHD prophylaxis

Tacrolimus

Intervention Type DRUG

Acute GVHD prophylaxis

Placebo

Placebo to efprezimod alfa (saline IV injection solution) on day -1 or days -1, 14, and 28 + Tacrolimus (begin on day -3. IV \[0.03 mg/kg/day\] or PO \[0.045 mg/kg/dose\] dosing is permitted) + Methotrexate (given intravenously at a dose of 15 mg/square meter/dose once daily on Day 1 after HCT, and at a dose of 10 mg/square meter/dose on days 3, 6, and 11 after HCT)

Group Type PLACEBO_COMPARATOR

Methotrexate

Intervention Type DRUG

Acute GVHD prophylaxis

Tacrolimus

Intervention Type DRUG

Acute GVHD prophylaxis

Placebo

Intervention Type DRUG

100 ml saline IV infusion.

Interventions

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Efprezimod alfa

Acute GVHD prophylaxis

Intervention Type DRUG

Methotrexate

Acute GVHD prophylaxis

Intervention Type DRUG

Tacrolimus

Acute GVHD prophylaxis

Intervention Type DRUG

Placebo

100 ml saline IV infusion.

Intervention Type DRUG

Other Intervention Names

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CD24 Fusion Protein MK-7110 Trexall FK506 Prograf Saline Solution

Eligibility Criteria

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Inclusion Criteria

4.1.1 A prospective participant for allogeneic hematopoietic stem cell transplantation (HCT) for a malignant hematologic disorder.

4.1.2 The donor and recipient must have a human leukocyte antigen (HLA)-8/8 allelic match at the HLA-A, -B, -C, and - DRB1 loci. High-resolution typing is required for all alleles for unmatched donors. Only matched unrelated donors are acceptable for this trial.

4.1.3 The following diagnoses are to be included:

1. Acute Myeloid Leukemia (AML) or Acute Lymphoblastic Leukemia (ALL) in first or second remission. Remission is defined as the absence of blasts in the peripheral circulation at the time of enrollment, \< 5% blasts in the bone marrow and absence of extramedullary disease including central nervous system (CNS) involvement.
2. Chronic Myelogenous Leukemia (CML) in first or subsequent chronic phase failing to respond (or intolerant) to at least two different tyrosine kinase inhibitors. CML in accelerated or blast phase (CML-AP/BP) are eligible without requirement to fail tyrosine kinase inhibitor therapy, but must be in remission at time of enrollment. Remission is defined as the absence of blasts in the peripheral circulation at the time of enrollment, \< 5% blasts in the bone marrow and absence of extramedullary disease including CNS involvement.
3. Myelodysplastic syndrome (MDS) with intermediate or high-risk International Prognostic Scoring System (IPSS) or equivalent Revised IPSS (IPSS-R) score with \< 10% blasts in the bone marrow.
4. Chronic Myelomonocytic Leukemia (CMML) with \< 10% blasts in the bone marrow.

4.1.4 Males or non-pregnant, non-lactating females, ≥ 18 years of age. Note there is no defined upper age limited, so long as deemed appropriate candidate for myeloablative conditioning.

4.1.5 Karnofsky Performance Status \>70%.

4.1.6 Participants must have normal or near normal organ function as defined by their treating institutions bone marrow transplantation (BMT) program clinical practice guidelines. In addition, for purposes of this protocol minimum organ function criteria within 21 days of beginning conditioning include:

TABLE 1: Eligibility According to Pre HCT Organ Function Total bilirubin ≤2.5 mg% (unless from Gilbert's disease or disease-related) Aspartate aminotransferase (serum glutamic-oxaloacetic transaminase) (AST\[SGOT\])/ alanine aminotransferase (serum glutamic-pyruvic transaminase) (ALT\[SGPT\]) \<3.0 X institutional upper limit of normal Estimated or actual glomerular filtration rate (GFR) \>50 mL/min/1.73 m2 for participants with creatinine levels above institutional normal (GFR should be corrected for body surface area \[BSA\]) Pulmonary Function Tests\* diffusing capacity of the lung for carbon monoxide (DLCO), forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC) \> 50% DLCO should be corrected for hemoglobin Ejection Fraction\* \>50% Hematopoietic Cell Transplantation-Specific Comorbidity Index (HCT-CI) ≤ 5

\*May be assessed up to 6 weeks prior to the start of conditioning therapy

4.1.7 Ability to understand and the willingness to sign a written informed consent document.

4.1.8 Women of child bearing potential and men must agree to use contraception prior to study entry and through day 100 post HCT (hormonal or barrier method of birth control; abstinence). Should a woman become pregnant or suspect she is pregnant while she or her partner is on treatment in this study, she should inform her study physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study until day 100 post HCT.

Exclusion Criteria

4.2.1 Participants may not have presence of active CNS disease or extramedullary disease.

4.2.2 Prior cytotoxic chemotherapy within 21 days from the initiation of HCT conditioning (i.e. intensive induction / consolidation for AML). Note, certain low intensity treatments not intended to induce remission but rather stabilize disease are acceptable up to 24 hrs prior to initiation of HCT conditioning (i.e. Tyrosine Kinase Inhibitor, sorafenib).

4.2.3 Cord blood and haploidentical donors are not eligible.

4.2.4 HLA-mismatch at the HLA-A, -B, -C, and - DRB1 loci. Note, HLA-DQ mismatches are permissible.

4.2.5 Pregnant and nursing mothers are excluded from this study. This is because the risk to the fetus is unknown.

4.2.6 Any physical or psychological condition that, in the opinion of the investigator, would pose unacceptable risk to the participant or raise concern that the participant would not comply with protocol procedures.

4.2.7 Uncontrolled infections. Participants still under therapy for presumed or proven infection are eligible provided there is clear evidence (radiologic, clinical and/or culture) that the infection is well controlled.

4.2.8 Participants seropositive or polymerase chain reaction (PCR) positive for the human immunodeficiency virus (HIV). Participants with evidence of Hepatitis B or Hepatitis C PCR positivity.

4.2.9 Prior HCT (allograft or prior autograft).

4.2.10 Use of T cell depletion either ex vivo or in vivo (i.e. anti-thymocyte globulin \[ATG\], alemtuzumab) is prohibited.

4.2.11 Current or prior diagnosis of antecedent Myelofibrosis is excluded.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ohio State University

OTHER

Sponsor Role collaborator

University of Michigan Rogel Cancer Center

OTHER

Sponsor Role collaborator

Indiana University School of Medicine

OTHER

Sponsor Role collaborator

Barbara Ann Karmanos Cancer Institute

OTHER

Sponsor Role collaborator

National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Oncoimmune, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Merck Sharp & Dohme LLC

Locations

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Indiana University School of Medicine

Indianapolis, Indiana, United States

Site Status

The University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, United States

Site Status

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Ohio State University

Columbus, Ohio, United States

Site Status

Countries

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United States

References

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Toubai T, Rossi C, Oravecz-Wilson K, Zajac C, Liu C, Braun T, Fujiwara H, Wu J, Sun Y, Brabbs S, Tamaki H, Magenau J, Zheng P, Liu Y, Reddy P. Siglec-G represses DAMP-mediated effects on T cells. JCI Insight. 2017 Jul 20;2(14):e92293. doi: 10.1172/jci.insight.92293. eCollection 2017 Jul 20.

Reference Type BACKGROUND
PMID: 28724800 (View on PubMed)

Toubai T, Hou G, Mathewson N, Liu C, Wang Y, Oravecz-Wilson K, Cummings E, Rossi C, Evers R, Sun Y, Wu J, Choi SW, Fang D, Zheng P, Liu Y, Reddy P. Siglec-G-CD24 axis controls the severity of graft-versus-host disease in mice. Blood. 2014 May 29;123(22):3512-23. doi: 10.1182/blood-2013-12-545335. Epub 2014 Apr 2.

Reference Type BACKGROUND
PMID: 24695850 (View on PubMed)

Toubai T, Mathewson ND, Magenau J, Reddy P. Danger Signals and Graft-versus-host Disease: Current Understanding and Future Perspectives. Front Immunol. 2016 Nov 29;7:539. doi: 10.3389/fimmu.2016.00539. eCollection 2016.

Reference Type BACKGROUND
PMID: 27965667 (View on PubMed)

Magenau J, Jaglowski S, Uberti J, Farag SS, Riwes MM, Pawarode A, Anand S, Ghosh M, Maciejewski J, Braun T, Devenport M, Lu S, Banerjee B, DaSilva C, Devine S, Zhang MJ, Burns LJ, Liu Y, Zheng P, Reddy P. A phase 2 trial of CD24Fc for prevention of graft-versus-host disease. Blood. 2024 Jan 4;143(1):21-31. doi: 10.1182/blood.2023020250.

Reference Type RESULT
PMID: 37647633 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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http://merckoncologyclinicaltrials.com

Merck Oncology Clinical Trials Information

Other Identifiers

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HUM00107805

Identifier Type: OTHER

Identifier Source: secondary_id

15-4789

Identifier Type: OTHER

Identifier Source: secondary_id

R44CA221513

Identifier Type: NIH

Identifier Source: secondary_id

View Link

R44CA246991

Identifier Type: NIH

Identifier Source: secondary_id

View Link

UMCC 2015.181

Identifier Type: OTHER

Identifier Source: secondary_id

MK-7110-002

Identifier Type: OTHER

Identifier Source: secondary_id

NCI-2017-00017

Identifier Type: OTHER

Identifier Source: secondary_id

CD24Fc-002

Identifier Type: OTHER

Identifier Source: secondary_id

7110-002

Identifier Type: -

Identifier Source: org_study_id

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