Chronic Inflammatory Demyelinating Polyradiculoneuropathy, Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), CIDP, CIDP (Chronic Inflammatory Demyelinating Polyradiculoneuropathy)
Conditions
Brief summary
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
BIOLOGICALEmpasiprubart IV
Intravenous infusions of empasiprubart
Sponsors
argenx
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 17 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Empasiprubart serum concentrations as input for a population PK-driven analysis to determine the effect of age and body size on CL and Vd | Up to 8 weeks | CL = Clearance; Vd = apparent volume of distribution. |
| Free and total C2 levels as input for PK/PD modeling analysis | Up to 8 weeks | C2 = complement component 2. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of AEs, SAEs and AESIs | Up to 180 weeks | AE = Adverse event ; SAE = Serious adverse event ; AESI = Adverse event of special interest. |
| Empasiprubart serum concentrations over time | Up to 180 weeks | — |
| Percentage reductions from baseline of free and total C2 levels over time | Up to 180 weeks | C2 = complement component 2. |
| Incidence of ADA and NAb against empasiprubart in serum | Up to 180 weeks | ADA = antidrug antibody(ies); NAb = neutralizing antibody(ies). |
Contacts
CONTACTSabine Coppieters, MD
Outcome results
None listed