A study to assess the efficacy and safety of empasiprubart in adults with CIDP

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Argenx

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

12 Mar 2026 → ongoing

Decision date (initial)

2026-02-16

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

argenx BV, Belgium

External identifiers

EU CT number

2025-520805-10-00

ClinicalTrials.gov

NCT07091630

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

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

To demonstrate the efficacy of empasiprubart IV compared with placebo in improving functional ability

Secondary objectives 14

1. To demonstrate the efficacy of empasiprubart IV compared with placebo and the long-term efficacy of empasiprubart IV on activity and social limitations
2. To demonstrate the efficacy of empasiprubart IV compared with placebo on muscle strength and function
3. To demonstrate the effect of empasiprubart IV compared with placebo on the time to improvement
4. To assess the efficacy of empasiprubart IV compared with placebo on the time to clinical deterioration
5. To assess the efficacy of empasiprubart IV compared with placebo and long-term efficacy of empasiprubart IV on muscle strength and function
6. To assess the efficacy of empasiprubart IV compared with placebo on functional ability
7. To assess the effect of empasiprubart IV compared with placebo and the long-term effect of empasiprubart IV on health-related quality of life and patient-reported outcome measures
8. To assess the immunogenicity of empasiprubart IV in participants with CIDP
9. To assess the safety and tolerability of empasiprubart IV compared with placebo and the long-term safety and tolerability of empasiprubart IV in participants with CIDP
10. To assess the PD effect of empasiprubart IV in participants with CIDP
11. To assess the PK of empasiprubart IV in participants with CIDP
12. To evaluate long-term efficacy of empasiprubart IV in improving functional ability
13. To assess long-term efficacy of empasiprubart IV in preventing clinical deterioration
14. To demonstrate the efficacy of empasiprubart IV compared with placebo and the long-term efficacy of empasiprubart IV on gait impairment

Conditions and MedDRA coding

Chronic inflammatory demyelinating polyneuropathy (CIDP)

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Meets criteria for CIDP based on EAN/PNS Task Force CIDP guidelines, second revision (2021)
2. Has 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
3. Has residual disability and active disease
4. Has not received previous treatment for CIDP; or has stopped receiving CIDP treatment; or is receiving CIDP treatment (pulsed or oral corticosteroids, immunoglobulins, PLEX, or FcRn inhibitors).

Exclusion criteria 5

1. Meets the criteria for possible CIDP based on EAN/PNS Task Force CIDP guidelines, second revision (2021)
2. Sensory CIDP (including sensory-predominant CIDP)
3. Polyneuropathy of other causes
4. Clinical diagnosis of systemic lupus erythematosus (SLE)
5. Use of other long-acting immunomodulatory treatment or prior treatment (at any time) with total lymphoid irradiation or bone marrow transplantation

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Reduction of ≥1 point compared with baseline in aINCAT score at week 24

Secondary endpoints 23

1. Change from baseline in I-RODS centile points score - up to 24 weeks (part A)
2. Change from baseline in MRC-SS at week 24
3. Change from baseline in grip strength (3-day moving average) in the dominant hand at week 24
4. Time to reduction of ≥1 point from baseline in aINCAT score - up to 24 weeks
5. Time to increase of ≥1 point compared with baseline in aINCAT score up to week 24
6. Change from baseline in grip strength (3-day moving average) of both hands over time and change from baseline in grip strength (daily average) for both hands
7. Change from baseline in MRC-SS over time. Up to 96 weeks (Part B)
8. Change from baseline in aINCAT score over time
9. Change from baseline in EQ-5D-5L over time
10. Change from baseline in RT-FSS over time
11. Change from baseline in BPI-SF over time
12. PGI-S values over time
13. PGI-C values over time
14. Incidence and prevalence of ADA against empasiprubart in serum
15. Incidence and prevalence of NAb against empasiprubart in serum
16. Incidence and severity of AEs
17. Incidence of SAEs
18. Clinically meaningful changes in laboratory parameters, ECG results, and vital signs
19. Absolute values and percentage change from baseline in free C2 and total C2 over time
20. Serum concentrations of empasiprubart over time
21. Reduction of ≥1 point in aINCAT over time
22. Increase of ≥1 point from baseline in aINCAT over time
23. Change from baseline in TUG at week 24

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Argenx

Sponsor organisation

Argenx

Address

Industriepark-Zwijnaarde 7

City

Gent

Postcode

9052

Country

Belgium

Scientific contact point

Organisation

Argenx

Contact name

Chief Scientific Officer

Public contact point

Organisation

Argenx

Contact name

Vice President Clinical Development

Third parties 22

Locations

15 EU/EEA countries · 46 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 172 files

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

Application history

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