A Study to Evaluate the Safety, Tolerability, PK, PD, and Clinical Activity of EQ001 in Subjects With aGVHD (EQUATE)

A Phase 1b/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of EQ001 in Subjects With Newly Diagnosed Acute Graft Versus Host Disease

This is a multi-center study to evaluate the safety, tolerability, PK, PD, and clinical activity of EQ001 in subjects with Acute Graft Versus Host Disease (aGVHD).

Study Overview

• Status: Completed
• Conditions: GVHD; GVHD, Acute; aGVHD; Acute-graft-versus-host Disease
• Intervention / Treatment: Biological: EQ001 Placebo; Biological: EQ001

Detailed Description

The study will enroll approximately 100 subjects in two (2) parts:

Part A is an open label study and will enroll approximately 40 evaluable subjects with aGVHD across 4 cohorts. The total number of patients will depend on the number of dose escalations necessary to enable a decision to be made on the recommended dose to take forward into Part B of the study. The planned dose escalation will start with cohort 1, where subjects will receive EQ001 administered intravenously every two weeks for a total of 5 doses.

Part B is a randomized, double-blind, placebo-controlled study and will enroll approximately 60 additional subjects, randomized in a 2:1 ratio to either active treatment EQ001 (40) or placebo (20). Subjects will receive either EQ001 or placebo administered intravenously every two weeks for a total of 5 doses.

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Duarte, California, United States, 91010
      • City of Hope Comprehensive Cancer Center
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida Health Shands Hospital
    • Miami, Florida, United States, 33136
      • University of Miami - Miller School of Medicine
    • Tampa, Florida, United States, 33612
      • H. Lee Moffitt Cancer Center & Research Institute
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan - C.S. Mott Children's Hospital
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University and Barnes Jewish Heart & Vascular Center
  • New York
    • Buffalo, New York, United States, 14263
      • Roswell Park Cancer Institute
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • University of North Carolina Lineberger Comprehensive Cancer Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania, Abramson Cancer Center
    • Pittsburgh, Pennsylvania, United States, 15232
      • University of Pittsburgh Cancer Institute
  • Tennessee
    • Nashville, Tennessee, United States, 53719
      • TriStar Centennial Medical Center (SCRI)
  • Utah
    • Salt Lake City, Utah, United States, 84103
      • Intermountain Healthcare
  • Washington
    • Seattle, Washington, United States, 98109-4433
      • Fred Hutchinson Cancer Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female subject at least 18 years of age for Part A, and at least 12 years of age for Part B.
2. Recipients of allogeneic hematopoietic stem cell transplantation (alloHSCT) using myeloablative or non myeloablative conditioning regimens.
3. Have a clinical diagnosis of acute GVHD requiring systemic immune suppressive therapy.
4. Deemed by the investigator to be likely to comply with the planned procedure as required by the protocol for the duration of the study

Exclusion Criteria:

1. Presence of morphologic relapsed primary malignancy, treatment for relapse after alloHSCT was performed, or requirement for rapid immunosuppressive treatment withdrawal for early malignancy relapse.
2. Evidence of graft failure based on cytopenia(s), and as determined by the investigator.
3. Evidence of post-transplant lymphoproliferative disease.
4. Any prior therapy for acute GVHD, except for alloHSCT prophylaxis regimens or systemically administered corticosteroids.
5. As determined by the investigator, any medical, psychiatric, or other condition or circumstance that is likely to negatively affect: the subject's participation in this clinical study, the subject's safety, or the reliability of the study data.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: EQ001 Dose Escalation (Part A); Open label EQ001 administered by intravenous infusion every two weeks for a total of 5 doses.	Biological: EQ001; Itolizumab [Bmab 600]); Other Names: Bmab600; Itolizumab
Experimental: EQ001 (Part B); EQ001 administered in a blinded fashion using the optimal dose selected from Part A by intravenous infusion every two weeks for a total of 5 doses.	Biological: EQ001; Itolizumab [Bmab 600]); Other Names: Bmab600; Itolizumab
Placebo Comparator: EQ001 Placebo (Part B); Placebo administered in a blinded fashion by intravenous infusion every two weeks for a total of 5 doses.	Biological: EQ001 Placebo; EQ001 Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Treatment Emergent Adverse Events	Number of participants with treatment-related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.	Study Day 85
Overall Response Rate	Overall Response Rate (ORR) is defined as the number of subjects with a partial response (PR), very good partial response (VGPR), or complete response (CR) who are alive at Day 29. Subjects must not have received new systemic therapy for aGVHD before the Day 29 Visit.	Study Day 29

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clearance, Cl	Clearance, Cl	Study Day 337
Inflammatory Markers	Including but not limited to: IL-1β, IL-2, IL-6, IL-17, IL-21, IL-22, IL-23, IFN-γ, and TGF-β, C-reactive protein	Study Day 337
Time to Maximum EQ001serum Concentration, Tmax	Time to maximum EQ001 serum concentration, Tmax	Day 337
Maximum EQ001 Serum Drug Concentration, Cmax	Maximum EQ001 serum drug concentration, Cmax	Study Day 337
Minimum EQ001 Serum Drug Concentration, Cmin	Minimum EQ001 serum drug concentration prior to next dose, Cmin	Study Day 337
Total EQ001 Exposure Across Time, AUC (From Zero to Infinity)	Total EQ001 exposure across time, AUC (from zero to infinity)	Study Day 337
Half Life of EQ001, t1/2	Half life of EQ001, t1/2	Study Day 337
Volume of Distribution of EQ001, Vd	Volume of distribution of EQ001, Vd	Study Day 337
CD6 Receptor Expression Levels	CD6 receptor expression levels - percent of baseline	Study Day 85

Collaborators and Investigators

• Sponsor: Equillium
• Collaborators: Biocon Limited
• Investigators: Study Director: Joel Rothman, Equillium

Publications and helpful links

General Publications

• Rambaldi B, Kim HT, Arihara Y, Asano T, Reynolds C, Manter M, Halpern M, Weber A, Koreth J, Cutler C, Gooptu M, Nikiforow S, Ho VT, Antin JH, Romee R, Ampudia J, Ng C, Connelly S, Soiffer RJ, Ritz J. Phenotypic and functional characterization of the CD6-ALCAM T-cell co-stimulatory pathway after allogeneic cell transplantation. Haematologica. 2022 Nov 1;107(11):2617-2629. doi: 10.3324/haematol.2021.280444.

Helpful Links

• Company website

Study record dates

Study Major Dates

• Study Start (Actual): July 15, 2019
• Primary Completion (Actual): November 21, 2022
• Study Completion (Actual): November 21, 2022

Study Registration Dates

• First Submitted: November 19, 2018
• First Submitted That Met QC Criteria: November 30, 2018
• First Posted (Actual): December 4, 2018

Study Record Updates

• Last Update Posted (Actual): April 18, 2025
• Last Update Submitted That Met QC Criteria: April 1, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: EQ001-aGVHD-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No