Clinical trial NCT06456580: A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (RemeMG)
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study With an Open-label Extension Period to Evaluate the Efficacy and Safety of Telitacicept in Patients With Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Keywords: Myasthenia Gravis
gMG
Lead sponsor: RemeGen Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2024-06-13
Enrollment: 180
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.

Detailed description: Myasthenia gravis (MG) is an autoimmune disease that affects the neuromuscular junction on the postsynaptic membrane. The predominant manifestation is muscle weakness, which typically worsens with repeated muscle exertion, such that function is usually the best in the morning with more pronounced weakness at the end of the day. A major challenge in MG is the lack of therapies that cure the disease.

Telitacicept is a fully human TACI-Fc fusion protein that targets B-lymphocyte stimulator (BLyS) and A proliferating-inducing ligand (APRIL), neutralizing their interactions with receptors on B cells. The blockage of BLyS and APRIL interaction with their respective cell membrane receptors (transmembrane activator and CAML interactor \[TACI\], B-cell maturation antigen, and BLyS receptors) by telitacicept would inhibit B-cell proliferation and maturation. This suppression at the proximal portion of the immune response could alleviate autoimmune symptoms.

This study is a randomized, double-blind, placebo-controlled Phase 3 study with an open-label extension to evaluate the efficacy and safety of telitacicept in a global patient population with gMG.

Eligibility criteria: Key Inclusion Criteria:

1. Male or female patient aged ≥18 years at screening.
2. Patients have prior confirmed diagnosis of gMG with generalized muscle weakness (typical pattern of weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) clinical classification II-IV.
3. Patients have positive antibodies against AChR or MuSK at screening.
4. MG-ADL score ≥6 points at screening and baseline with ocular-related score \<50% of the total score.
5. QMG score ≥11 points at screening and baseline.

Key Exclusion Criteria:

1. Patients have been diagnosed with any other autoimmune disease.
2. Patients having acute or chronic infection.
3. Patients having thymoma within 5 years or received thymectomy ≤6 months prior to screening.
4. Patients having current or history of primary immunodeficiency.
5. Patients having history of malignancy within the last 5 years.
6. Patient having prior or continuing diagnosis of serious cardiovascular disease.

Primary outcomes:
measure: Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24
timeFrame: Week 24
Secondary outcomes:
measure: Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24
timeFrame: Week 24
measure: Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24
timeFrame: Week 24
measure: Proportion of patients with a decrease of ≥2 points from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24
timeFrame: Week 24
measure: Proportion of patients with a decrease of ≥3 points from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24
timeFrame: Week 24
measure: Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24
timeFrame: Week 24
Other outcomes:
Locations:
city: Orange
country: United States
facility: Orange Site
state: California
city: Boca Raton
country: United States
facility: Boca Raton, Florida Site
state: Florida
city: Miami
country: United States
facility: Miami, Florida Site
state: Florida
city: Port Charlotte
country: United States
facility: Port Charlotte, Florida Site
state: Florida
city: Lexington
country: United States
facility: Lexington Site
state: Kentucky
city: Foxboro
country: United States
facility: Foxboro Site
state: Massachusetts
city: Greenfield
country: United States
facility: Greenfield Site
state: Wisconsin
city: Bydgoszcz
country: Poland
facility: Bydgoszcz Site
state: Kujawsko-Pomorskie
city: Bydgoszcz
country: Poland
facility: Bydgoszcz Site
state: Kujawsko-Pomorskie
city: Lublin
country: Poland
facility: Lublin Site
state: Lubelskie
city: Lublin
country: Poland
facility: Lublin
state: Lubuskie
city: Kraków
country: Poland
facility: Kraków Site
state: Malopolskie
city: Katowice
country: Poland
facility: Katowice Site
state: Slaskie
city: Poznań
country: Poland
facility: Poznań Site
state: Wielkopolskie
Study officials:
References: