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Open-label Study of ELA026 in Participants With Secondary Hemophagocytic Lymphohistiocytosis (sHLH)
Trial Status: active
Hemophagocytic lymphohistiocytosis is a rare, aggressive and life-threatening syndrome of
excessive immune activation. Secondary hemophagocytic lymphohistiocytosis (sHLH) is the
most common form of this disease and is typically associated with several other clinical
conditions (eg, malignancy associated HLH (mHLH), infection, or autoimmune disease).
ELA026 is a fully human immunoglobulin G1 (IgG1) signal regulatory protein
(SIRP)-directed monoclonal antibody designed to deplete the myeloid and T cells driving
the inflammation. The purpose of this study is to assess the safety, efficacy
pharmacokinetics and pharmacodynamics of ELA026 in participants with sHLH.
Inclusion Criteria
Key Inclusion Criteria for Part 1:
1. ≥12 years at the time of HLH diagnosis (Cohort 1).
2. ≥6 years at the time of HLH diagnosis (Cohort 2-3).
3. Treatment naïve or relapsed/refractory (Cohorts 1 and 2).
4. Treatment naïve or early refractory (Cohort 3).
5. Participant with sHLH confirmed criteria based on fulfilling 5 out of 8 HLH-2004
diagnostic criteria.
Key Inclusion Criteria for Part 2:
1. Cohort A: Adults with newly diagnosed, treatment-naïve, malignancy-associated sHLH.
2. Cohort B: Adults with newly diagnosed, treatment-naïve, non-malignancy-associated
sHLH.
3. Cohort B: Adults with newly diagnosed, treatment-naïve, malignancy-associated sHLH,
diagnosed by OHI index.
4. Cohort B: 13 to 17 years olds with newly diagnosed, treatment-naïve sHLH.
5. Cohort B: 6 to 12 year olds, with refractory sHLH (safety lead-in cohort).
6. Cohort B: 6 to 12 year olds, with newly diagnosed, treatment-naïve sHLH (after
completion of safety lead-in cohort).
Key Exclusion Criteria for Part 1:
1. Known or previous treatment for primary HLH
2. Any other significant concurrent, uncontrolled medical condition that in the opinion
of the Investigator contraindicates participation in this study
3. Unknown trigger for sHLH
4. Active, relapsed/refractory malignancy for which no suitable therapies are available
to treat the malignancy triggering the HLH
5. Allogeneic hemopoietic stem cell transplant (HSCT) within 100 days of the first dose
of ELA026.
6. Ongoing administration of any therapies used to treat HLH (excluding dexamethasone)
7. Live or attenuated vaccine received within 6 weeks or bacille Calmette-Guerin (BCG)
vaccine within 12 weeks prior to Screening
Key Exclusion Criteria for Part 2:
1. Refractory sHLH (except for the safety lead-in cohort for 6-12 year olds in Cohort
B).
2. Known or suspected primary or hereditary HLH.
3. Severe organ dysfunction.
4. Any other significant concurrent, uncontrolled medical condition that contraindicates
participation in this study or prohibits completion of study procedures.
5. End-stage malignancy for which no suitable therapies are available to treat the
malignancy triggering the HLH.
6. Allogeneic hemopoietic stem cell transplant within 30 days prior to enrollment.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT05416307.
Locations matching your search criteria
United States
Alabama
Birmingham
University of Alabama at Birmingham Cancer Center
Status: Active
Name Not Available
Georgia
Atlanta
Emory University Hospital/Winship Cancer Institute
Status: Active
Name Not Available
New York
New York
Memorial Sloan Kettering Cancer Center
Status: Active
Name Not Available
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
Status: Active
Name Not Available
Pennsylvania
Philadelphia
Thomas Jefferson University Hospital
Status: Active
Name Not Available
Texas
Dallas
UT Southwestern/Simmons Cancer Center-Dallas
Status: Active
Name Not Available
Houston
UT MD Anderson Cancer Center
Status: Active
Name Not Available
Utah
Salt Lake City
Huntsman Cancer Institute/University of Utah
Status: Active
Name Not Available
Washington
Seattle
Fred Hutch/University of Washington/Seattle Children's Cancer Consortium
Status: Active
Name Not Available
This study consists of two parts: Phase 1b (Part 1) and Phase 2/3 (Part 2).
Part 1 is designed to evaluate the safety, efficacy, pharmacodynamics, and
pharmacokinetics of ELA026 in pediatric and adult participants with treatment-naïve (TN)
and relapsed/refractory sHLH. The main objectives of Part 1 are to determine the safety
of ELA026 administered intravenously (IV) and subcutaneously (SC) to participants with
sHLH and to identify the recommended Phase 3 dose and schedule for ELA026. Participants
will be enrolled into a dose-escalating cohort (Cohort 1) followed by two fixed dose
cohorts (Cohorts 2-3) treated over 12-weeks.
Part 2 (SURPASS) is designed as an open-label, single-arm, multicenter, historical
control registrational study to evaluate ELA026 in newly diagnosed TN adult and pediatric
sHLH participants. All participants are diagnosed with HLH-2004 criteria unless
indicated. Cohort A (primary cohort) will enroll newly diagnosed TN participants ≥18
years old with mHLH. Cohort B (exploratory cohort) will enroll participants including ≥18
years old participants with TN sHLH not triggered by malignancy; ≥18 years old
participants with newly diagnosed TN mHLH diagnosed by biomarker criteria but not meeting
HLH-2004 diagnostic criteria; and 6 to 17 year old participants with newly diagnosed TN
sHLH (due to any trigger). For 6 to 12 year old participants, there is a safety lead-in
cohort with refractory sHLH.
Part 1 is closed to recruitment and Part 2 is recruiting for eligible participants.
