Open-label Study of ELA026 in Participants With Secondary Hemophagocytic Lymphohistiocytosis (sHLH)

NCT ID: NCT05416307

Last Updated: 2025-12-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

156 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-05-19

Study Completion Date

2029-06-30

Brief Summary

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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.

Detailed Description

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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 TN adult and pediatric sHLH participants. All participants are diagnosed with HLH-2004 criteria unless indicated. Cohort A (primary cohort) will enroll 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 TN mHLH diagnosed by biomarker criteria but not meeting HLH-2004 diagnostic criteria; and 6 to 17 year old participants with 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.

Conditions

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Secondary Hemophagocytic Lymphohistiocytosis (sHLH)

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part 1 ELA026

Cohort 1: Single dose escalation up to 3.0 mg/kg IV or SC.

Cohort 2: priming dose: 0.1 mg/kg IV on Day 1; 0.3 mg/kg IV on Day 2 - 4, followed by weekly maintenance doses of 1 mg/kg IV/SC from Day 8 to Day 81.

Cohort 3: priming dose: 0.1 mg/kg IV on Day 1; 0.3 mg/kg IV on Days 2 - 4, followed by twice weekly maintenance doses of 0.5 mg/kg IV/SC from Day 8 to Day 81.

Group Type EXPERIMENTAL

ELA026

Intervention Type DRUG

Multiple doses of ELA026

Part 2 ELA026

Cohort A and Cohort B: priming dose: 0.1 mg/kg IV on Day 1; loading dose 0.3 mg/kg IV on Days 2- 4, followed by twice weekly maintenance doses of 0.5 mg/kg (IV/SC) from Day 8 to Day 81.

Group Type EXPERIMENTAL

ELA026

Intervention Type DRUG

Multiple doses of ELA026

Interventions

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ELA026

Multiple doses of ELA026

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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.


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).

Exclusion Criteria

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


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.
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Electra Therapeutics Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Electra Therapeutics Inc.

Locations

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University of Alabama at Birmingham School of Medicine

Birmingham, Alabama, United States

Site Status RECRUITING

Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status ACTIVE_NOT_RECRUITING

University of California, Los Angeles

Los Angeles, California, United States

Site Status ACTIVE_NOT_RECRUITING

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, United States

Site Status RECRUITING

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Site Status RECRUITING

Emory University

Atlanta, Georgia, United States

Site Status RECRUITING

Columbia University

New York, New York, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status RECRUITING

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Texas Children's Hospital

Houston, Texas, United States

Site Status RECRUITING

University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

Huntsman Cancer Institute

Salt Lake City, Utah, United States

Site Status RECRUITING

Medizinische Universität Innsbruck

Innsbruck, , Austria

Site Status WITHDRAWN

Medical University of Vienna

Vienna, , Austria

Site Status COMPLETED

Universitätsklinikum Jena

Jena, Thuringia, Germany

Site Status RECRUITING

Azienda Ospedaliero Universitaria Meyer

Florence, , Italy

Site Status RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, , Italy

Site Status RECRUITING

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Milan, , Italy

Site Status COMPLETED

Bambino Gesu' Roma

Roma, , Italy

Site Status WITHDRAWN

Amsterdam Universitair Medische Centra - Locatie Academisch Medisch Centrum

Amsterdam, Noord-Holland - NET, Netherlands

Site Status RECRUITING

Erasmus UMC

Rotterdam, , Netherlands

Site Status ACTIVE_NOT_RECRUITING

Wilhelmina Kinderziekenhuis

Utrecht, , Netherlands

Site Status RECRUITING

Clinica Universidad de Navarra - Madrid

Madrid, Madrid - SPA, Spain

Site Status RECRUITING

Clinica Universidad de Navarra - Pamplona

Pamplona, Navarre - SPA, Spain

Site Status RECRUITING

Hospital Universitario Virgen del Rocio

Seville, Sevilla - SPA, Spain

Site Status RECRUITING

Hospital Universitari i Politecnic La Fe

Valencia, Valencia - SPA, Spain

Site Status RECRUITING

Hospital 12 de Octubre, Madrid

Madrid, , Spain

Site Status WITHDRAWN

Hospital Ramon y Cajal

Madrid, , Spain

Site Status COMPLETED

Hospital La Fe Valencia

Valencia, , Spain

Site Status WITHDRAWN

University College London Hospitals NHS Foundation Trust

London, England - UK, United Kingdom

Site Status RECRUITING

Countries

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United States Austria Germany Italy Netherlands Spain United Kingdom

Central Contacts

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Clinical Trials

Role: CONTACT

Phone: Please email

Email: [email protected]

Other Identifiers

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ELA026-CP002

Identifier Type: -

Identifier Source: org_study_id