A study to assess the efficacy and safety of Empasiprubart versus IVIg in adults with Multifocal Motor Neuropathy

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] - Musculoskeletal Diseases [C05], Diseases [C] - Nervous System Diseases [C10]

Trial duration

31 Jul 2025 → ongoing

Decision date (initial)

2025-07-21

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Argenx BV, Belgium

External identifiers

EU CT number

2024-516473-72-00

ClinicalTrials.gov

NCT06742190

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacodynamic, Pharmacokinetic, Others, Safety

Part A: To demonstrate the efficacy of empasiprubart compared to IVIg in improving muscle strength

Secondary objectives 11

1. Part A - To demonstrate the efficacy of empasiprubart compared to IVIg on functional ability
2. Part A - To demonstrate the efficacy of empasiprubart compared to IVIg on motor function
3. Part A - To demonstrate the efficacy of empasiprubart compared to IVIg on manual dexterity
4. Part A - To evaluate the safety and tolerability of empasiprubart compared to IVIg
5. Part A - To evaluate the PK, PD, and immunogenicity of empasiprubart
6. Part A - To evaluate the efficacy of empasiprubart compared to IVIg on muscle strength, motor function, manual dexterity, and patient-reported outcomes
7. Part A - To evaluate the effect on health-related quality of life for empasiprubart compared to IVIg
8. Part B - To evaluate the long-term efficacy of empasiprubart in improving functional ability, muscle strength, motor function, manual dexterity, quality of life, and patient-reported outcomes
9. Part B - To evaluate the long-term safety and tolerability of empasiprubart
10. Part B - To evaluate the PK, PD, and immunogenicity of empasiprubart
11. Part B - To evaluate the effect of empasiprubart on long-term health-related quality of life

Conditions and MedDRA coding

Multifocal Motor Neuropathy

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-777632-PIP02-40

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Is at least 18 years of age and the local legal age of consent for clinical studies
2. Has a confirmed diagnosis of definite or probable MMN at screening according to the EFNS/PNS 2010 guidelines
3. Has responded to IVIg in the past 5 years.
4. Is receiving IVIg at a treatment interval of once every 2, 3, 4, or 5 weeks, and a dose of 0.4 to 2.0 g/kg body weight per cycle
5. Is receiving a maintenance regimen (no change in frequency, and no change in dose >10%) of IVIg for at least 8 weeks before screening (or at least 10 weeks for participants receiving IVIg once every 5 weeks)
6. Minimum converted weekly IVIg dose of ≥0.125 g/kg
7. Has documented immunization against encapsulated bacterial pathogens (N meningitidis and S pneumoniae) within 5 years of screening or is willing to receive immunization at least 14 days before first study drug administration

Exclusion criteria 2

1. Besides the indication under study, known autoimmune disease (eg, SLE) or any other medical condition that would confound the study results or put the participant at undue risk
2. Clinical signs or symptoms suggestive of neuropathies other than MMN, such as motor neuron disease (eg, bulbar signs, brisk reflexes) or other inflammatory neuropathies (eg, sensory neuropathy)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline in GS (3 day moving average) in the most affected hand at week 24

Secondary endpoints 30

1. Part A - Change from baseline in MMN-RODS centile score at week 24
2. Part A - Change from baseline in mMRC-14 sum score at week 24
3. Part A - PGI-C actual value over time
4. Part A - Change from baseline in CAP-PRI total score over time
5. Part A - Percentage change from baseline in time to complete the 9-HPT with the dominant hand at week 24
6. Part A - Incidence and severity of AEs and AESIs, Incidence of SAEs
7. Part A - Clinically meaningful changes in laboratory parameters, vital signs, and ECG results
8. Part A - Serum concentrations over time and PK parameters of empasiprubart
9. Part A - Values and percentage change from baseline in free C2 and total C2 over time
10. Part A - Incidence and prevalence of anti-drug antibodies (ADA) against empasiprubart in serum
11. Part A - Incidence and prevalence of NAb against empasiprubart in serum
12. Part A - Change from baseline in GS (3-day moving average) of the least affected hand over time and AUC of change from baseline in GS (daily average) for both hands; percentage change from baseline in GS (3-day moving average) for both hands
13. Part A - Percentage change from baseline in time to complete the 9-HPT with the nondominant hand over time
14. Part A - Change from baseline in sum scores for mMRC-10 and mMRC-14 restricted to the 2 most affected muscle groups over time
15. Part A - Proportion of participants and shift from baseline over time by level of severity on PGI-S
16. Part A - Change from baseline in Rasch-Transformed Fatigue Severity Scale (RT-FSS) score over time
17. Part A - Change from baseline in physical component and mental component scores of 12-Item Short Form Survey (SF-12) over time
18. Part A - Proportion of participants and shift from baseline by each dimension of the EQ5D-5L scale, change from baseline in the EQ-5D-5L visual analog scale over time, and change from baseline in EQ-5D-5L valuation index
19. Part B - Actual values of and changes from baseline in MMN-RODS centile score, CAPPRI total score, grip strength (daily average; both hands), mMRC-10 sum score, mMRC-14 sum score, and mMRC-14 sum score restricted to the 2 most affected muscle groups over time
20. Part B - Actual values of and percentage change from baseline in time to complete the 9-HPT with the dominant and nondominant hands over time
21. Part B - Actual values of PGI-C and PGI-S over time
22. Part B - Incidence and severity of AEs and AESIs, Incidence of SAEs
23. Part B - Clinically meaningful changes in laboratory parameters, vital signs, and ECG results
24. Part B - Serum concentrations over time and PK parameters of empasiprubart
25. Part B - Values and percentage change from baseline in free C2 and total C2 over time
26. Part B - Incidence and prevalence of ADA against empasiprubart in serum
27. Part B - Incidence and prevalence of NAb against empasiprubart in serum
28. Part B - Change from baseline in RT-FSS score over time
29. Part B - Change from baseline in physical component and mental component scores of SF-12 over time
30. Part B - Proportion of participants and shift from baseline by each dimension of the EQ5D-5L scale and change from baseline in the EQ-5D-5L visual analog scale over time

Investigational products

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

Test 1

Comparator 1

Placebo 2

Auxiliary 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 15

Locations

19 EU/EEA countries · 71 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 208 files

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

Application history

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