Clinical trial • Phase II/III • Immunology|Rare Disease
ELA026 for Secondary Hemophagocytic Lymphohistiocytosis (sHLH)
Phase II/III trial of ELA026 for Secondary Hemophagocytic Lymphohistiocytosis (sHLH).
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Secondary Hemophagocytic Lymphohistiocytosis (sHLH)
- Trial Stage
- Phase II/III
- Drug Modality
- Peptide/protein/enzyme
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 09-04-2025
- First CTIS Authorization Date
- 05-08-2025
Trial design
open-label, none; single-arm study with historical control (historical control used as comparator; no active comparator arm specified). Phase II/III trial in Germany, Spain, Italy and others.
- Open Label
- Yes
- Comparator
- None; single-arm study with historical control (historical control used as comparator; no active comparator arm specified).
- Real World Control
- Yes
- Target Sample Size
- 60
- Trial Duration For Participant
- 120
Eligibility
Recruits 60 paediatric patients.
- Vulnerable Population
- The protocol selects vulnerable populations (isVulnerablePopulationSelected = true). Consent and assent handling: participants or legally authorized representatives (LAR) must be capable of giving signed informed consent and the ICF must be signed prior to study activities (Inclusion criterion 4). For minors (Cohort B) minor participants must be capable of giving written assent as appropriate per applicable age/local regulations and the participant or LAR must provide signed informed consent (Inclusion criterion 5). Country-specific ICF and assent documents are provided (examples in Spanish, Italian, German, Dutch as listed in the document set).
Inclusion criteria
- {"criterion_text":"- 1. Age and disease"}
- {"criterion_text":"- 2. Adequate organ function that in consideration of the underlying disease trigger and HLH would support enrollment in a clinical trial"}
- {"criterion_text":"- 3. Must be Eastern Cooperative Oncology Group (ECOG) ≤3"}
- {"criterion_text":"- 4.Cohort A and Cohort B: Participant or legally authorized representative(s) (LAR) capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. ICF must be signed prior to initiation of any study related activities unless assessments are conducted as part of routine SOC as indicated in the screening SoA"}
- {"criterion_text":"- 5.Cohort B: Minor participants must be capable of giving written assent as appropriate per the applicable age (per local regulatory requirements). Participant or LAR capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. ICF must be signed prior to initiation of any study related activities."}
Exclusion criteria
- {"criterion_text":"- 1.\tRefractory sHLH (exception is the safety lead-in cohort for 6 to 12 year olds with refractory sHLH in Cohort B)"}
- {"criterion_text":"- 2.\tKnown or suspected primary or hereditary HLH"}
- {"criterion_text":"- 3.\tSevere organ dysfunction (see protocol for the list)"}
- {"criterion_text":"- 4.\tAny other significant concurrent, uncontrolled medical condition that contraindicates participation in this study or prohibits completion of study procedures"}
- {"criterion_text":"- 5.\tEnd-stage malignancy for which no suitable therapies are available to treat the malignancy triggering the HLH (participants with refractory malignancy with ongoing therapeutic options are not excluded)"}
- {"criterion_text":"- 6.\tHistory of hypersensitivity or allergy to dexamethasone"}
- {"criterion_text":"- 7.\tHistory of hypersensitivity or allergy to any components of ELA026"}
- {"criterion_text":"- 8.\tOngoing social, psychological, religious or spiritual beliefs, or logistical issues that would prevent following study procedures or receiving supportive care to receive study treatment safely please see the protocol for whole list."}
Endpoints
Primary endpoints
- {"endpoint_text":"- 56-day survival from the time of diagnosis","definition_or_measurement_approach":"56-day survival from time of diagnosis; measured as survival status at Day 56 from diagnosis (primary efficacy endpoint for Cohort A as described in main objective)."}
Secondary endpoints
- {"endpoint_text":"- 30-day, 45-day, 56-day (any type of cancer), 90-day survival (early survival) from time of diagnosis; Survival status at hospital discharge for initial hospital admission for sHLH; Number of participants achieving HLH disease response by Day 29 (modified HLH-2004 criteria and biomarker criteria).","definition_or_measurement_approach":"Survival measured at specified timepoints from time of diagnosis; survival status at initial hospital discharge; number achieving HLH disease response by Day 29 using modified HLH-2004 criteria and biomarker criteria."}
- {"endpoint_text":"- HLH disease response is defined as a participant who achieves complete response, modified CR, partial response, or HLH improvement; Incidence of treatment-emergent adverse events","definition_or_measurement_approach":"HLH disease response defined as achievement of complete response (CR), modified CR (mCR), partial response (PR), or HLH improvement (HI) by Day 29 per modified HLH-2004 criteria and biomarker criteria; safety assessed by incidence of treatment-emergent adverse events (TEAEs)."}
Recruitment
- Planned Sample Size
- 60
- Recruitment Window Months
- 35
- Consent Approach
- Informed consent must be provided by the participant or legally authorized representative (LAR) and the ICF must be signed prior to initiation of study-related activities (unless assessments are part of routine SOC). Minors (Cohort B) must provide written assent as appropriate for age per local regulations, and consent is provided by the participant or LAR. Country- and age-specific ICF and assent documents are provided (examples listed in the document set: Spanish, Italian, German, Dutch ICFs and assent forms for ages 6-11 and 15-17).
Geography
- Total Number Of Sites
- 16
- Total Number Of Participants
- 40
Germany
- Earliest CTIS Part Ii Submission Date
- 18-07-2025
- Latest Decision Or Authorization Date
- 09-04-2026
- Processing Time Days
- 265
- Number Of Sites
- 2
- Number Of Participants
- 10
Sites
- Site Name
- Universitaetsklinikum Jena KöR
- Department Name
- Hämatologische und onkologische Intensivmedizin
- Contact Person Name
- Jakob Hammersen
- Contact Person Email
- jakob.hammersen@med.uni-jena.de
- Site Name
- Schwarzwald-Baar Klinikum Villingen-Schwenningen GmbH
- Department Name
- Hämatologie, Onkologie und Palliativmedizin
- Contact Person Name
- Paul Graf La Rosée
- Contact Person Email
- paul.larosee@sbk-vs.de
Spain
- Earliest CTIS Part Ii Submission Date
- 18-07-2025
- Latest Decision Or Authorization Date
- 13-04-2026
- Processing Time Days
- 269
- Number Of Sites
- 4
- Number Of Participants
- 10
Sites
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Department of Hematology
- Contact Person Name
- Jose Antonio Perez Simon
- Contact Person Email
- josea.perez.simon.sspa@juntadeandalucia.es
- Site Name
- Clinica Universidad De Navarra (Madrid)
- Department Name
- Department of Hematology
- Contact Person Name
- Sara Villar Fernández
- Contact Person Email
- svillar@unav.es
- Site Name
- Clinica Universidad De Navarra (Pamplona)
- Department Name
- Department of Hematology
- Contact Person Name
- Sara Villar Fernández
- Contact Person Email
- svillar@unav.es
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Oncología pediátrica
- Contact Person Name
- Jose Maria Fernández Navarro
- Contact Person Email
- fernandez_jma@gva.es
Italy
- Earliest CTIS Part Ii Submission Date
- 06-05-2025
- Latest Decision Or Authorization Date
- 08-04-2026
- Processing Time Days
- 337
- Number Of Sites
- 6
- Number Of Participants
- 10
Sites
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- Clinica di Ematologia and Immunologia
- Contact Person Name
- Gianni Binotto
- Contact Person Email
- gianni.binotto@unipd.it
- Site Name
- Fondazione IRCCS San Gerardo Dei Tintori (Monza - Adult)
- Department Name
- Divisione di Ematologia - Adult
- Contact Person Name
- Luisa Verga
- Contact Person Email
- l.verga@hsgerardo.org
- Site Name
- Fondazione IRCCS San Gerardo Dei Tintori (Monza - Satellite)
- Department Name
- Clinica Pediatrica Satellite Site Dr Luisa Verga
- Contact Person Name
- Luisa Verga
- Contact Person Email
- a.balduzzi@hsgerardo.org
- Site Name
- Azienda Ospedaliera Universitaria Meyer IRCCS
- Department Name
- Tumori Pediatrici e Trapianto di Cellule Staminali
- Contact Person Name
- Elena Sieni
- Contact Person Email
- elena.sieni@meyer.it
- Site Name
- IRCCS Istituto Giannina Gaslini
- Department Name
- Dipartimento di Scienze Pediatriche
- Contact Person Name
- Elena Palmisani
- Contact Person Email
- elenapalmisani@gaslini.org
- Site Name
- Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
- Department Name
- Materno Infantile
- Contact Person Name
- Marco Rabusin
- Contact Person Email
- marco.rabusin@burlo.trieste.it
Netherlands
- Earliest CTIS Part Ii Submission Date
- 18-07-2025
- Latest Decision Or Authorization Date
- 07-04-2026
- Processing Time Days
- 263
- Number Of Sites
- 4
- Number Of Participants
- 10
Sites
- Site Name
- Radboud universitair medisch centrum Stichting
- Department Name
- Department of Hematology
- Contact Person Name
- Suzanne van Dorp
- Contact Person Email
- suzanne.vandorp@radboudumc.nl
- Site Name
- Wilhelmina Childrens Hospital
- Department Name
- Department of Pediatric Immunology & Rheumatology
- Contact Person Name
- Sebastiaan J. (Bas) Vastert
- Contact Person Email
- b.vastert@umcutrecht.nl
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Klinische Immunologie
- Contact Person Name
- Jan van Laar
- Contact Person Email
- j.vanlaar@erasmusmc.nl
- Site Name
- Amsterdam UMC Stichting
- Department Name
- Hematology Clinic
- Contact Person Name
- Sanne H Tonino
- Contact Person Email
- s.h.tonino@amsterdamumc.nl
Sponsor
Primary sponsor
- Full Name
- Electra Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Precision For Medicine Inc.
- Responsibilities
- sponsorDuties codes: 4
- Name
- Almac Clinical Services LLC
- Responsibilities
- sponsorDuties codes: 14
- Name
- Medidata Solutions Inc.
- Responsibilities
- sponsorDuties codes: 7
- Name
- Icon Clinical Research Limited
- Responsibilities
- sponsorDuties codes: 3,4
- Name
- Scout Clinical
- Responsibilities
- sponsorDuties codes: 15 (ex-US Patient Reimbursement)
- Name
- PPD Development LP
- Responsibilities
- sponsorDuties codes: 4
- Name
- Bioforum C.D.M.C Ltd.
- Responsibilities
- sponsorDuties codes: 10
- Name
- Almac Clinical Services (Ireland) Limited
- Responsibilities
- sponsorDuties codes: 14
Third parties
- {"country":"United States","full_name":"Precision For Medicine Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Almac Clinical Services LLC","duties_or_roles":"sponsorDuties codes: 14","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties codes: 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"sponsorDuties codes: 3,4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"sponsorDuties codes: 15 (value: ex-US Patient Reimbursement)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
- {"country":"Israel","full_name":"Bioforum C.D.M.C Ltd.","duties_or_roles":"sponsorDuties codes: 10","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Almac Clinical Services (Ireland) Limited","duties_or_roles":"sponsorDuties codes: 14","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- ELA026
- Active Substance
- ELA026
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
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