sHLH is a rare and life-threatening inflammatory syndrome characterized by dysregulated immune function. The disease is associated with a massive systemic inflammatory response for which patients require immediate and aggressive treatment with intensive care. Clinically, patients present with fever, hepatomegaly, pancytopenia, splenomegaly, hypotension, and coagulopathy. It is difficult to assess the true epidemiology of HLH due to the rarity of the condition. MedDRA version: 20.0 Level: SOC
Conditions
Interventions
Product Name: ELA026
Product Code: ELA026
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: None
CAS Number: None
Current Sponsor code: ELA026
Other descriptive name: ELA026
Concentratio
Sponsors
Electra Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Age 1. =12 years at the time of HLH diagnosis. Type of Participant and Disease Characteristics 1. Treatment naive OR 2. Relapsed or refractory HLH defined as: a. Participant has failed to respond to 2 weeks of non-etoposide, non-steroid therapy with a less than 50% decrease in serum ferritin, OR b. Participant has received 4 doses of etoposide with a less than 50% decrease in serum ferritin 72 hours after last dose, OR c. On a case-by-case basis as determined by the Medical Monitor 3. Participant is hospitalized with an HLH confirmed diagnosis based on fulfilling 5 out of 8 HLH-2004 criteria below (Henter, Horne, et al., 2007): Clinical Criteria 1. Fever =38.5C 2. Splenomegaly Laboratory Criteria 1. Cytopenia (affecting =2 of 3 lineages in the peripheral blood): Haemoglobin (=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Medical Conditions 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. Prior/Concurrent Therapy 1. Hemapoietic Stem Cell Transplant (HSCT) within 100 days of the first dose of ELA026. 2. Treatment with CAR T-Cell therapy within 3 months of the first dose of ELA026. 3. Ongoing administration of any investigational treatment or treatment for HLH (excluding dexamethasone) within 14 days prior to Screening or 5 drug half-lives, whichever is shorter. 4. Live or attenuated vaccine received within 6 weeks or bacille Calmette-Guerin (BCG) vaccine within 12 weeks prior to Screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To determine the safety of ELA026 administered IV and SC to adolescent and adult participants with sHLH. • To identify the RP3D and schedule for ELA026.;Secondary Objective: • To characterize the pharmacokinetic (PK) profile of ELA026 administered IV and SC to adolescent and adult participants with sHLH. • To determine the efficacy of ELA026 administered IV and SC to adolescent and adult participants with sHLH. • To characterize the pharmacodynamic (PD) effect of ELA026 administered IV and SC to adolescent and adult participants with sHLH. • To assess the immunogenicity of ELA026 administered IV and SC to adolescent and adult participants with sHLH.;Primary end point(s): • Incidence of adverse events (AEs) including dose-limiting toxicities (DLTs), serious adverse events (SAEs), deaths, AEs leading to withdrawal from study. • Safety, efficacy, PD.;Timepoint(s) of evaluation of this end point: Evaluated on an ongoing basis. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Best response by week 4 defined as either complete response (CR) modified complete response (mCR) or partial response (PR) evaluated by objective clinical and laboratory parameters. • Plasma concentrations and PK parameters of ELA026. • Change from baseline in monocytes and T lymphocytes. • Incidence of Anti-drug antibodies (ADAs) to ELA026.;Timepoint(s) of evaluation of this end point: After the given period is completed. | — |
Countries
Austria, Italy, Spain, United Kingdom
Contacts
Public ContactMichelle A. Carpenter
Electra Therapeutics, Inc.
Outcome results
None listed