Clinical trial NCT05716035: Open-Label Extension of Tocilizumab in Subjects With Generalized Myasthenia Gravis
Official title: A Phase III, Open-label Extension Trial of tMG to Evaluate the Safety and Efficacy of Tocilizumab in Subjects With Generalized Myasthenia Gravis (gMG)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Myasthenia Gravis, Generalized
Keywords: Generalized Myasthenia Gravis
Tocilizumab
Open-label
Lead sponsor: Tang-Du Hospital
Sponsor class: OTHER
First posted: 2023-02-08
Enrollment: 64
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate the safety and efficacy of tocilizumab in the treatment of generalized myasthenia gravis (gMG) as an extension study for the participants who previously completed Study tMG(NCT05067348).

Detailed description: tMG-E was an extension study designed to provide the participants who completed Study tMG an opportunity to receive tocilizumab and collect clinical data to provide long-term safety and efficacy information on tocilizumab in participants with gMG.

After receiving blinded study treatment (tocilizumab or placebo) in Study tMG for 16 weeks, participants were eligible to enroll in the tMG-E extension study. Participants were to enter Study tMG-E within 8 weeks after completing their Week 16 visit in Study tMG.

Study tMG-E consisted of an Open-Label treatment Phase up to 16 weeks.

Eligibility criteria: Inclusion Criteria:

1. Participant has completed Study tMG.
2. Participant has given written informed consent.
3. MG-ADL score ≥ 5 points, or a decrease of ≤ 3 points relative to the baseline MG-ADL of the tMG study

Exclusion Criteria:

1. Participants had clinically relevant active infections (such as sepsis, pneumonia, or abscess) or severe infections (resulting in hospitalization or requiring antibiotic treatment) in the past 4 weeks;
2. Those with high-risk tuberculosis infection, acquired tuberculosis infection, and chronic hepatitis after the tMG study;
3. planned thymectomy during RCP;
4. Received IVIG or plasma exchange in the past 4 weeks;
5. Any medical condition or circumstances that, in the opinion of the investigator, might have interfered with the participant's participation in the study, posed any added risk for the participant, or confounded the assessment of the participants.

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs)
timeFrame: From Baseline (Day 1) to Safety Follow-Up Visit (up to 16 weeks)
Secondary outcomes:
measure: Change in Quantitative Myasthenia Gravis (QMG) scores.
timeFrame: 16 weeks
measure: Proportion of subjects with both (1) ≥ 3-point improvement in QMG and (2) lasts ≥4 weeks
timeFrame: 16 weeks
measure: Proportion of subjects with both (1) ≥ 2-point improvement in MG-ADL and (2) lasts ≥4 weeks
timeFrame: 16 weeks
measure: Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Profile score
timeFrame: 16 weeks
measure: Change in Myasthenia Gravis Composite (MGC) score
timeFrame: 16 weeks
measure: Change in Myasthenia Gravis Quality of Life-15, revised (MG-QOL15r) score.
timeFrame: 16 weeks
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Tiantan Hospital, Capital Medical University
state: Beijing
city: Changsha
country: China
facility: Xiangya Hospital Central South University
state: Hunan
city: Xi'an
country: China
facility: Tangdu Hospital, The Fourth Military Medical University
state: Shaanxi
city: Shanghai
country: China
facility: Huashan Hospital
state: Shanghai
city: Chengdu
country: China
facility: West China Hospital of Sichuan University
state: Sichuan
city: Tianjin
country: China
facility: Tianjin medical university general hospital
state: Tianjin
Study officials:
References: