Open-label Study of ELA026 in Participants with sHLH (short title)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Electra Therapeutics Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

19 Sep 2025 → ongoing

Decision date (initial)

2025-08-07

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Electra Therapeutics Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Therapy, Pharmacodynamic, Safety, Others

Cohort A: To determine 56-day survival with ELA026 treatment in participants with mHLH who are TN and have lymphoma as the cancer trigger

Secondary objectives 4

1. To determine 30-day, 45-day, 90-day and hospital discharge survival with ELA026 treatment in participants with mHLH who are TN and have lymphoma as the cancer trigger
2. To determine 30-day, 45-day, 56-day, 90-day and hospital discharge survival with ELA026 treatment in participants with mHLH who are TN and have any type of cancer as the cancer trigger
3. To determine the best response with ELA026 treatment in participants with mHLH who are TN and have lymphoma as the cancer trigger and in participants with any type of cancer trigger
4. To evaluate the safety of ELA026 in TN mHLH participants

Conditions and MedDRA coding

Secondary Hemophagocytic Lymphohistiocytosis (sHLH)

Study design 5 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 1. Age and disease
2. 2. Adequate organ function that in consideration of the underlying disease trigger and HLH would support enrollment in a clinical trial
3. 3. Must be Eastern Cooperative Oncology Group (ECOG) ≤3
4. 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
5. 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 8

1. 1. Refractory sHLH (exception is the safety lead-in cohort for 6 to 12 year olds with refractory sHLH in Cohort B)
2. 2. Known or suspected primary or hereditary HLH
3. 3. Severe organ dysfunction (see protocol for the list)
4. 4. Any other significant concurrent, uncontrolled medical condition that contraindicates participation in this study or prohibits completion of study procedures
5. 5. End-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)
6. 6. History of hypersensitivity or allergy to dexamethasone
7. 7. History of hypersensitivity or allergy to any components of ELA026
8. 8. Ongoing 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 and secondary outcome measures (English text)

Primary endpoints 1

1. 56-day survival from the time of diagnosis

Secondary endpoints 2

1. 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).
2. 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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Electra Therapeutics Inc.

Sponsor organisation

Electra Therapeutics Inc.

Address

201 Haskins Way Floor 5th

City

South San Francisco

Postcode

94080-6215

Country

United States

Scientific contact point

Organisation

Electra Therapeutics Inc.

Contact name

clinical Trials

Public contact point

Organisation

Electra Therapeutics Inc.

Contact name

Medical Director

Third parties 8

Locations

4 EU/EEA countries · 16 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 33 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications