A clinical trial to investigate the Long-term Safety and Tolerability, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 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] - Nervous System Diseases [C10]

Trial duration

29 Mar 2023 → ongoing

Decision date (initial)

2024-03-07

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Argenx BV, Belgium

External identifiers

EU CT number

2023-507052-69-00

EudraCT number

2021-004998-32

ClinicalTrials.gov

NCT05405361

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Pharmacodynamic, Pharmacokinetic, Efficacy

To evaluate the long-term safety and tolerability of ARGX-117 in adult participants with Multifocal Motor Neuropathy (MMN)

Secondary objectives 4

1. 1. To evaluate the long-term efficacy of ARGX-117 on muscle strength and/or motor function in adult participants with MMN
2. 2. To evaluate the long-term efficacy of ARGX-117 on functional ability, arm and hand function, quality of life (QOL), and fatigue in adult participants with MMN
3. 3. To evaluate the long-term effect of ARGX-117 on health-related productivity and work productivity
4. 4. To assess the PK, PD, and immunogenicity of ARGX-117

Conditions and MedDRA coding

Multifocal Motor Neuropathy

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Participants are eligible to be included in the trial only if all of the following criteria apply: 1. Capable of providing signed informed consent, and complying with protocol requirements. Participants must be able to read and write. 2. Must have completed the double-blinded treatment period of the ARGX-117-2002 trial and considered to be eligible for treatment with ARGX-117 3. Agrees to use contraceptive measures consistent with local regulations and the following: a. Male participants: must use an acceptable contraceptive method that should be maintained at minimum until 15 months after last dose of Investigational Medicinal Product (IMP). b. Female participants (women) of childbearing potential must have a negative urine pregnancy test at baseline before IMP can be administered.

Exclusion criteria 1

1. Participants will be excluded from the trial if any of the following criteria apply: 1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection 2. Clinical evidence of other significant serious diseases, have had a recent major surgery, or who have any other condition, in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk. 3. Currently participating in another interventional clinical study 4. Pregnant or lactating or intend to become pregnant during the trial or within 15 months after last dose of the IMP.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety outcomes based on AE monitoring and other safety assessments (clinical laboratory tests)

Secondary endpoints 16

1. 1. Modified Medical Research Council (mMRC) − Value and change from baseline in the mMRC-10 sum score − Proportion of participants showing a deterioration of at least 2 points in mMRC-10 sum score − Value and change from baseline in the mMRC-14 sum score − Proportion of participants showing a deterioration of at least 2 points in the mMRC-14 sum score
2. − Value and change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC-14 sum score
3. 2. Grip strength (GS) − Values, change, and percent change from baseline in GS − Proportion of participants with a decline of >30% in GS
4. 3. Values and change from baseline in the Rasch-built overall disability scale for MMN (MMN‐RODS)
5. 4. Values, change, and percentage change from baseline in the average time for the upper extremity (arm and hand) function (9-Hole Peg Test [9-HPT], or timed pegboard test)
6. 5. Proportion of participants by level of severity on each dimension of EQ-5D-5L
7. 6. Value and change from baseline in EQ-5D-5L visual analog scale (VAS)
8. 7. Values and change from baseline in the Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI)
9. 8. Values and change from baseline in the 9-item Fatigue Severity Scale (FSS)
10. 9. Values of the Patient Global Impression of Change (PGIC) scale
11. 10. Proportion of participants by level of severity of MMN as assessed by the Patient Global Impression of Severity (PGIS)
12. 11. Values for work-related and household chore activities of the Health-Related Productivity Questionnaire (HRPQ) at each visit: − Hours lost because of absenteeism − Hours lost because of presenteeism
13. − Total hours lost − Percent of scheduled hours lost because of absenteeism − Percent of scheduled hours lost because of presenteeism − Percent of scheduled hours lost in total
14. 12. Serum concentrations and PK parameters for ARGX-117
15. 13.Values and change from baseline in free C2, total C2, and functional complement activity (CH50) over time
16. 14. Incidence and prevalence of antidrug antibodies (ADA) against ARGX-117

Investigational products

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

Test 2

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 13

Locations

8 EU/EEA countries · 17 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 43 files

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

Application history

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