A Study to Assess the Correct Dose, Safety and Efficacy of Empasiprubart in Adolescent Participants Aged 12 to Less Than 18 Years With Chronic Inflammatory Demyelinating Polyradiculoneuropathy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Empasiprubart IV.
- Who it may be relevant to
- Registry conditions: Chronic Inflammatory Demyelinating Polyradiculoneuropathy, CIDP (Chronic Inflammatory Demyelinating Polyradiculoneuropathy), CIDP, Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). Basic parameters: 12 years — 17 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-Label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Empasiprubart in Adolescent Participants Aged 12 to Less Than 18 Years With Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Overview
The main purpose of the study is to determine the correct dose of empasiprubart in adolescent participants. It also aims to evaluate if empasiprubart may work and how safe it is for the use in children living with CIDP. The study consists of an open label treatment phase where participants will receive empasiprubart for up to 27 months approximately. After the final dose of empasiprubart, participants will enter a safety follow-up period for up to 14 months approximately. The overall study duration for each participant is up to 43 months. More information can be found here: clinicaltrials.argenx.com/emlight
Interventions
- Biological Empasiprubart IV
Intravenous infusions of empasiprubart
Primary outcome measures
- Empasiprubart serum concentrations as input for a population PK-driven analysis to determine the effect of age and body size on CL and Vd [Time frame: Up to 8 weeks]
- Free and total C2 levels as input for PK/PD modeling analysis [Time frame: Up to 8 weeks]
Secondary outcome measures (4)
- Incidence of AEs, SAEs and AESIs [Time frame: Up to 180 weeks]
- Empasiprubart serum concentrations over time [Time frame: Up to 180 weeks]
- Percentage reductions from baseline of free and total C2 levels over time [Time frame: Up to 180 weeks]
- Incidence of ADA and NAb against empasiprubart in serum [Time frame: Up to 180 weeks]
Eligibility criteria
Inclusion criteria
- Is aged 12 to <18 years.
- Meets criteria for CIDP based on EAN/PNS Task Force CIDP guidelines, second revision (2021).
- Has a diagnosis of either typical CIDP or 1 of the following CIDP variants: motor CIDP (including motor-predominant CIDP), multifocal CIDP (also known as Lewis-Sumner syndrome), focal CIDP, or distal CIDP.
Exclusion criteria
- Possible CIDP based on EAN/PNS Task Force CIDP guidelines, second revision (2021).
- Sensory CIDP (including sensory-predominant CIDP).
- Besides the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of CIDP, or that puts the participant at undue risk.
- Prior use of other long-acting immunomodulatory treatment.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07638566 · ARGX-117-6-CIDP-2001 · 2025-524153-14-00