ARGX-113-1705 - ADAPT+
Research type
Research Study
Full 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
IRAS ID
250772
Contact name
Saiju Jacob
Contact email
Sponsor organisation
argenx BVBA
Eudract number
2018-002133-37
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 4 months, 1 days
Research summary
Research Summary
Generalised Myasthenia gravis (gMG) is a chronic, autoimmune condition (where the immune system attacks the body) that causes muscle weakness and excessive muscle fatigue. The muscle weakness fluctuates with activity, and symptoms may be relieved temporarily after periods of rest. The treatment of gMG is based on a variety of medications and medical procedures used either alone or in combination. Treatments include acetylcholine inhibitors (which help electrical signals travel between the nerves and muscles, providing temporary reduction in muscle weakness), corticosteroids, immunosuppressants, new treatments such as rituximab and eculizumab, therapeutic plasma exchange (PLEX) (which removes substances such as harmful antibodies from the blood), and surgery to remove the thymus gland. These treatments can help keep the symptoms of gMG under control, but some provide only short periods of relief, requiring several doses each day, and some are associated with frequent and often serious side effects.
The purpose of this clinical study is to evaluate the long-term safety and tolerability of the study drug, ARGX-113, in participants with gMG. Participants enrolling in this study will be “rolling over” from Study ARGX-113-1704. The maximum number of participants in this extension study will be the number of participants who took part in the first study.
Participants will be administered ARGX-113 at 4 weekly infusions at a dose of 10mg per kg of body weight (up to a maximum of 1200mg for participants with a body weight of 120kg or over), for a period of 3 weeks, on an “as needed basis” on top of their standard of care in Treatment Periods. The time between Treatment Periods is based on how long the treatment is effective, and may vary between participants. The study will last for approximately 1 year.
Summary of Results
The ARGX-113-1705 final analyses shows that the long-term safety and tolerability profiles of efgartigimod IV (intravenous) 10 mg/kg, administered in repeated cycles of 1 infusion per week for 4 infusions, remained favorable with no incidences of new events or more frequent occurrences or greater severity of previously reported events occurring with repeated efgartigimod cycles.
The efficacy data indicate that the clinical effect, evident by improvements in the MG-ADL (Myasthenia Gravis Activities of Daily Living) and QMG (Quantitative Myasthenia Gravis) scores, is maintained with repeated cycles of treatment in adult participants with gMG (generalized myasthenia gravis). Efgartigimod IV was safe and well tolerated, with most AEs (Adverse Events) being mild to moderate in severity. The overall benefit-risk profile was positive.
The AChR-Ab (Anti-acetylCholine Receptor Antibody) seropositive, AChR-Ab seronegative, and overall populations had consistent improvements in MG-ADL and QMG scores over repeated cycles.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
18/WM/0373
Date of REC Opinion
6 Feb 2019
REC opinion
Further Information Favourable Opinion