A Study to Evaluate the Safety, Tolerability, PK, PD, and Clinical Activity of EQ001 in Subjects With aGVHD
Purpose
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).
Conditions
- Acute-graft-versus-host Disease
- aGVHD
- GVHD
- GVHD, Acute
Eligibility
- Eligible Ages
- Over 12 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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 Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential Assignment
- Intervention Model Description
- Part A is an open label 3+3 dose escalation Part B is blinded and randomized 2:1
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Masking Description
- Part B will be blinded to all study staff that has direct access to the subjects and the sponsor. The site's pharmacist or designee will be unblinded to prepare the study drug.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental EQ001 Dose Escalation (Part A) |
Open label EQ001 administered by intravenous infusion every two weeks for a total of 5 doses. |
|
|
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. |
|
|
Placebo Comparator EQ001 Placebo (Part B) |
Placebo administered in a blinded fashion by intravenous infusion every two weeks for a total of 5 doses. |
|
Recruiting Locations
More Details
- NCT ID
- NCT03763318
- Status
- Completed
- Sponsor
- Equillium
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.