A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SATRALIZUMAB IN PATIENTS WITH GENERALIZED MYASTHENIA GRAVIS

JRCT ID: jRCT2021210024

Registered date:30/07/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Myasthenia Gravis
• Date of first enrollment: 15/02/2022
• Target sample size: 240
• Countries of recruitment: United States,Japan,Argentina,Japan,Australia,Japan,Brazil,Japan,Canada,Japan,China,Japan,Denmark,Japan,France,Japan,Germany,Japan,Italy,Japan,Republic of Korea,Japan,Netherlands,Japan,Poland,Japan,Russian Federation,Japan,Spain,Japan,Taiwan,Japan,Turkey,Japan
• Study type: Interventional
• Intervention(s): Satralizumab: 120 mg or 180mg SC injection every 4 weeks (Q4W)

Outcome(s)

Primary Outcome

Efficacy
To evaluate the efficacy of satralizumab versus placebo on function in daily life in the acetylcholine receptor antibody seropositive (AChR+) population

Secondary Outcome

Safety, Efficacy, Exploratory, Phamacokinetics, Phamacodynamics, Other
- To evaluate the efficacy of satralizumab versus placebo on function in daily life in the overall population (OP)
- To evaluate the efficacy of satralizumab versus placebo in the AChR+ and OP on QMG, QoL, Fatigue, Clinical status and Disease severity
- To evaluate the durability of the efficacy of satralizumab versus placebo in the AChR+ population and the OP
- To evaluate the safety of satralizumab versus placebo
- To confirm target engagement and pathway inhibition in response to satralizumab
- To investigate the pharmacokinetics (PK) of satralizumab
- To evaluate the immune response to satralizumab

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Signed Informed Consent Form
2. Age >= 12 years at time of signing Informed Consent Form
3. Confirmed diagnosis of gMG
- Documented history of myasthenic weakness
- MG severity of MGFA Class II, III, or IV at screening
- The confirmation of the diagnosis has to be supported by positive serologic test for one of the three antibody types: anti-AChR, anti-MuSK or anti-LRP4 at screening
4. A total MG ADL score of >= 5 points at screening with more than 50% of this score attributed to non-ocular items
5. Ongoing gMG standard treatment at a stable dose
6. No contraindication to at least one of the rescue treatments: IVIg, PE, or high dose corticosteroids

Exclude criteria

1. History of thymic cysts, thymoma, thymic carcinoma or other neoplasm of the thymus as defined by the 2015 WHO classification of tumors of the thymus (Marx et al. 2015) unless deemed cured by adequate treatment with no evidence of recurrence for >= 5 years before screening.
2. History of thymectomy within 12 months prior to screening
3. Ocular MG (MGFA Class I)
4. Myasthenic crisis within the last 3 months prior to screening (MGFA Class V)
5. Known disease other than gMG that would interfere with the course and conduct of the study (such as severe RA or symptomatic [overt] hyperthyroidism or hypothyroidism)
6. Patients receiving with prohibited therapy or prohibited medication

Related Information

• Primary Sponsor: Ivana Vodopivec
• Secondary ID(s): NCT04963270,2020-004436-21