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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 treatment-naïve, malignancy-associated sHLH.
2. Cohort B: Adults with treatment-naïve, non-malignancy-associated sHLH.
3. Cohort B: Adults with treatment-naïve, malignancy-associated sHLH, diagnosed by OHI
index.
4. Cohort B: 13 to 17 years olds with 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 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 100 days prior to the first dose
of ELA026.
Additional locations may be listed on ClinicalTrials.gov for NCT05416307.
