A safety and tolerability study of ARGX-113 in patients with myasthenia gravis who have generalized muscle weakness.

• Register: EUCTR
• Last refreshed on: 11 January 2021
• Main ID: EUCTR2018-002133-37-NL
• Date of registration: 26/09/2018
• Prospective Registration: Yes
• Primary sponsor: argenx BVBA
• Public title: A safety and tolerability study of ARGX-113 in patients with myasthenia gravis who have generalized muscle weakness.
• Scientific title: A Long-Term, Single-Arm, Open-Label, Multicenter, Phase 3 Follow-on Trial of ARGX-113-1704 to Evaluate the Safety and Tolerability of ARGX-113 in Patients with Myasthenia Gravis having Generalized Muscle Weakness
• Date of first enrolment: 06/06/2019
• Target sample size: 150
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-002133-37
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Canada, Czech Republic, Czechia, Denmark, France, Georgia, Germany, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Serbia, Sweden, United Kingdom, United States

Contacts

• Name: Regulatory affairs
• Affiliation: argenx BVBA

• Name: Regulatory affairs
• Affiliation: argenx BVBA

Key inclusion & exclusion criteria

Inclusion criteria:
Patients will roll over in this trial only if they meet all of the following criteria:
1. Patients with the ability to understand the requirements of the trial, provide written informed consent, and can comply with the trial protocol procedures.
2. Patients who participated in trial ARGX-113-1704 and are eligible for roll over, as specified in the protocol.

Other more specific inclusion criteria are further defined in the protocol.
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 130
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion criteria:
Patients will not roll over in this trial if they meet any of the following criteria:

1. Patients who discontinued early from trial ARGX-113-1704 or patients who discontinued early from randomized treatment for pregnancy or rescue reasons or an (S)AE that might jeopardize the safety of the patient in that trial.
2. Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. Women of childbearing potential (DEFINITION OF TERMS) should have a negative urine pregnancy test at SEB.
3. Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing.
4. Patients with known hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) seropositivity.

Other more specific exclusion criteria are further defined in the protocol.


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

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Myasthenia Gravis
MedDRA version: 21.1 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 21.1 Level: LLT Classification code 10028423 Term: Myasthenia gravis-like syndrome System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 20.0 Level: HLT Classification code 10071942 Term: Myasthenia gravis and related conditions System Organ Class: 100000004859

Intervention(s)

Product Name: Efgartigimod
Product Code: ARGX-113
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Efgartigimod
CAS Number: 1821402-21-4
Current Sponsor code: ARGX-113
Other descriptive name: ARGX-113
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)

Main Objective: To evaluate the long-term safety and tolerability of ARGX-113 in acetylcholine receptor antibody (AChR-Ab) seropositive patients.

Secondary Objective: To evaluate the long-term safety and tolerability of ARGX-113 in the overall population (AChR-Ab seropositive and AChR-Ab seronegative patients).

Timepoint(s) of evaluation of this end point: Up to maximum 3 years

Primary end point(s): Incidence and severity of AEs, serious adverse events (SAEs), vital signs,
electrocardiogram (ECG) and laboratory assessments over the duration of the trial in AChR-Ab seropositive patients.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: over the duration of the trial (up to maximum 3 years)

Secondary end point(s): Incidence and severity of AEs, SAEs, vital signs, ECG and laboratory assessments over the duration of the trial in the overall population (AChR-Ab seropositive patients and AChR-Ab
seronegative patients).

Secondary ID(s)

ARGX-113-1705

Source(s) of Monetary Support

argenx BVBA

Ethics review

Status: Approved
Approval date: 06/06/2019
Contact: