ClinicalTrials.gov record NCT05416307
Open-label Study of ELA026 in Participants With Secondary Hemophagocytic Lymphohistiocytosis (sHLH)
A Multipart, Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of ELA026 in Participants With Secondary Hemophagocytic Lymphohistiocytosis (sHLH)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This interventional study tests ELA026, an antibody given by vein (IV) or under the skin (SC), in people with secondary hemophagocytic lymphohistiocytosis (sHLH). It has two parts: a Phase 1b dose‑finding phase with escalating and fixed-dose cohorts, and a Phase 2/3 single-arm registrational phase for newly diagnosed, treatment‑naïve cases. Key outcomes include safety, drug levels and activity, and early survival and HLH response measures.
What it is studying. Whether different doses and schedules of ELA026 are safe, how the body handles the drug (pharmacokinetics/pharmacodynamics), and early measures of disease response in sHLH.
What the researchers hope to learn. To evaluate the safety and tolerability of multiple ELA026 dosing regimens; to describe pharmacokinetics and pharmacodynamics; to identify a recommended Phase 3 dose and schedule; and to collect early clinical outcome data such as short-term survival and HLH disease response measures (for example 56-day survival and response by Day 29).
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- ELA026
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- for Part 1:
- ≥12 years at the time of HLH diagnosis (Cohort 1).
- ≥6 years at the time of HLH diagnosis (Cohort 2-3).
- Treatment naïve or relapsed/refractory (Cohorts 1 and 2).
- Treatment naïve or early refractory (Cohort 3).
- Participant with sHLH confirmed criteria based on fulfilling 5 out of 8 HLH-2004 diagnostic criteria.
- Key Inclusion Criteria for Part 2:
- Cohort A: Adults with newly diagnosed, treatment-naïve, malignancy-associated sHLH.
- Cohort B: Adults with newly diagnosed, treatment-naïve, non-malignancy-associated sHLH.
- Cohort B: Adults with newly diagnosed, treatment-naïve, malignancy-associated sHLH, diagnosed by OHI index.
- Cohort B: 13 to 17 years olds with newly diagnosed, treatment-naïve sHLH.
- Cohort B: 6 to 12 year olds, with refractory sHLH (safety lead-in cohort).
- 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:
- Known or previous treatment for primary HLH
- Any other significant concurrent, uncontrolled medical condition that in the opinion of the Investigator contraindicates participation in this study
- Unknown trigger for sHLH
- Active, relapsed/refractory malignancy for which no suitable therapies are available to treat the malignancy triggering the HLH
- Allogeneic hemopoietic stem cell transplant (HSCT) within 100 days of the first dose of ELA026.
- Ongoing administration of any therapies used to treat HLH (excluding dexamethasone)
- 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:
- Refractory sHLH (except for the safety lead-in cohort for 6-12 year olds in Cohort B).
- Known or suspected primary or hereditary HLH.
- Severe organ dysfunction.
- Any other significant concurrent, uncontrolled medical condition that contraindicates participation in this study or prohibits completion of study procedures.
- End-stage malignancy for which no suitable therapies are available to treat the malignancy triggering the HLH.
- Allogeneic hemopoietic stem cell transplant within 100 days prior to the first dose of ELA026.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive the study drug ELA026 by intravenous (IV) or subcutaneous (SC) injections with initial priming/loading doses followed by maintenance dosing through about 12 weeks (up to Day 81). They will attend regular clinic visits for safety monitoring, blood samples for pharmacokinetic and immune testing, and scheduled assessments of disease response and survival (including Day 29 and Day 56/90 checks noted in the study). Study staff will monitor for adverse events and perform any additional tests the investigators require.
How the study is structured. Open-label, non-randomized interventional trial with two parts. Part 1 (Phase 1b) uses dose-escalation and fixed-dose cohorts to evaluate safety, pharmacokinetics/pharmacodynamics and to select a Phase 3 dose; dosing is by IV or SC. Part 2 (Phase 2/3) is an open-label, single-arm, multicenter registrational study that evaluates the selected regimen in newly diagnosed, treatment‑naïve adult and pediatric participants, using historical controls for comparison.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in Austria, Germany, Italy, Netherlands, Spain, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on hemophagocytic lymphohistiocytosis
Important dates
- Study start
- May 19, 2022
- Primary completion
- June 2027 (estimated)
- Study completion
- June 2029 (estimated)
- Target enrollment
- 156 participants (estimated)
- Last updated on registry
- May 26, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Electra Therapeutics Inc.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Clinical Trials 📞 Please email ✉ clinicaltrials@electra-therapeutics.com
Participating sites (41)
- University of Alabama at Birmingham School of MedicineRecruiting
Birmingham, Alabama, 35233 · United States
- Phoenix Children's HospitalActive Not Recruiting
Phoenix, Arizona, 85016 · United States
- University of California, Los AngelesActive Not Recruiting
Los Angeles, California, 90095 · United States
- MedStar Georgetown University HospitalRecruiting
Washington D.C., District of Columbia, 20007 · United States
- Johns Hopkins All Children's HospitalRecruiting
St. Petersburg, Florida, 33701 · United States
- Children's Healthcare of Atlanta - Center for Advanced PediatricsRecruiting
Atlanta, Georgia, 30322 · United States
- Emory UniversityRecruiting
Atlanta, Georgia, 30322 · United States
- National Institute of Allergy and Infectious Diseases (NIAID)Recruiting
Rockville, Maryland, 20892 · United States
- Columbia UniversityRecruiting
New York, New York, 10032 · United States
- Cornell UniversityRecruiting
New York, New York, 10065 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- pharmacokinetics
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- pharmacodynamics
- What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
- Cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
- relapsed/refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- Key Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- treatment-naïve
- Has not previously received the treatment, or the type of treatment, that the study is about.
- stem cell transplant
- A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
- prior to Screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Key Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Phase 3
- A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- biomarker
- Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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