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

• Register: EUCTR
• Last refreshed on: 15 April 2024
• Main ID: EUCTR2021-004998-32-NL
• Date of registration: 19/09/2022
• Prospective Registration: Yes
• Primary sponsor: argenx BV
• Public title: 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
• Scientific title: A Long-Term Extension of the ARGX-117-2002 Trial to Evaluate the Long-term Safety and Tolerabillity, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 in Adults with Multifocal Motor Neuropathy - ARDA+
• Date of first enrolment: 05/12/2022
• Target sample size: 48
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2021-004998-32
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: Double - blinded rollover treatment (DTP) followed by Open - label treatment period (OTP) If controlled, specify comparator, Other Medicinial Product: yes Placebo: yes Other: no Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States

Contacts

• Name: Regulatory
• Affiliation: argenx BV

• Name: Regulatory
• Affiliation: argenx BV

Key inclusion & exclusion criteria

Inclusion criteria:
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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 36
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 12

Exclusion criteria:
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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Multifocal Motor Neuropathy
MedDRA version: 21.1 Level: PT Classification code 10065579 Term: Multifocal motor neuropathy System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: ARGX-117 IV
Product Code: ARGX-117 IV
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: not defined
Other descriptive name: ARGX-117
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 120-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use

Product Name: ARGX-117 IV
Product Code: ARGX-117 IV
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: not defined
Other descriptive name: ARGX-117
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 165-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

Main Objective: To evaluate the long-term safety and tolerability of ARGX-117 in adult participants with MMN

Primary end point(s): • Safety outcomes based on AE monitoring and other safety assessments

Secondary Objective: • To evaluate the long-term efficacy of ARGX-117 on muscle strength and/or motor function in adult participants with MMN
• 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
• To evaluate the long-term effect of ARGX-117 on health-related productivity and work productivity
• To assess the PK, PD, and immunogenicity of ARGX-117

Timepoint(s) of evaluation of this end point: continuously

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Please refer to section 1.3 of protocol

Secondary end point(s): • 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
- Proportion of participants showing a deterioration of 1 or more points in at least 2 muscle groups as assessed by the mMRC-14 sum score
- Proportion of participants with no deterioration in 2 or more muscle groups as assessed by mMRC-14 sum score
- Value and change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC-14 sum score
• Grip strength (GS)
- Values, change, and percent change from baseline in GS
- Proportion of participants with a decline of >30% in GS
- Proportion of participants with a GS decrease of 8 kilopascals (kPa) or more
• Values and change from baseline in the Rasch-built overall disability scale for MMN (MMN-RODS)
• Values and 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)
• Proportion of participants by level of severity on each dimension of EQ-5D-5L
• Value and change from baseline in EQ-5D-5L visual analog scale (VAS)
• Values and change from baseline in the Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI)
• Values and change from baseline in the 9-item Fatigue Severity Scale (FSS)
• Values of the Patient Global Impression of Change (PGIC) scale
• Proportion of participants by level of severity of MMN as assessed by the Patient Global Impression of Severity (PGIS)
• Values for work-related and household chore activities of the Health-Related Productivity Questionnaire (HRPQ)
• Serum concentrations and PK parameters for ARGX-117
• Values and change from baseline in free C2, total C2, and functional complement activity (CH50) over time
• Incidence and prevalence of antidrug antibodies (ADA) against ARGX-117

Secondary ID(s)

ARGX-117-2003

2021-004998-32-ES

Source(s) of Monetary Support

argenx BV

Ethics review

Status: Approved
Approval date: 05/12/2022
Contact: