Blinatumomab in Refractory Myasthenia Gravis (BLINA-MG) (BLINA-MG)

Safety and Efficacy of Blinatumomab in Refractory Myasthenia Gravis

The goal of this clinical trial is to evaluating the efficacy and safety of Blinatumomab in the treatment of refractory myasthenia gravis and exploring the immunological mechanisms of Blinatumomab therapy in refractory myasthenia gravis. The main questions it aims to answer are:

Will Blinatumomab improve the symptoms of participants? What medical problems do participants have when using Blinatumomab?

Participants will:

Continuous intravenous infusion of Blinatumomab for 5 days, pause for one week, and continue continuous intravenous infusion for 5 days Visit the clinic once every 1 month for checkups and tests Keep a diary of their symptoms

Study Overview

• Status: Not yet recruiting
• Conditions: Myasthaenia Gravis
• Intervention / Treatment: Drug: Blinatumomab

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Zhaoyou Meng; Phone Number: 0086-023-68774449; Email: mengzhaoyou@tmmu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 18 years old ≤ Age ≤ 75 years old
• patients with a Myasthenia Gravis Activities of Daily Living (MG-ADL) scale score of 6 or higher, and an ocular muscle score constituting less than 50% of the total score
• after receiving an adequate course of at least two conventional immunotherapeutic drugs (including both steroidal and non-steroidal immunosuppressants), the Post-Intervention Status (PIS) remains unchanged or worsens; or after receiving an adequate course of at least two conventional immunotherapeutic drugs, the PIS improves, but the MG-ADL score is still ≥6 points and persists for at least half a year; or after receiving an adequate course of at least two conventional immunotherapeutic drugs, the PIS is in remission or improvement, yet during the regular tapering of immunotherapeutic drugs, there are still at least two annual exacerbations of disease symptoms (MG-ADL score ≥6 points); or after the occurrence of a crisis, despite multiple immunotherapies such as intravenous immunoglobulin, plasmapheresis, and high-dose intravenous methylprednisolone, and active infection control, the patient still cannot be weaned off the ventilator due to MG-induced respiratory muscle weakness for more than 14 days
• positive for anti-AChR, and/or anti-MuSK, and/or anti-LRP4 antibodies.
• patient or their legal representative signs an informed consent form in writing

Exclusion Criteria:

• pregnant or lactating women
• individuals with an allergy to Blinatumomab
• individuals who have undergone thymectomy within the 12 months prior to baseline or who are planning to have a thymectomy during the 12-week study period
• individuals who have received biologic agents targeting CD19 or CD20 within 6 months prior to baseline are eligible for enrollment (subjects with CD19 or CD20 positive B cell counts above the lower limit of normal are allowed to participate)
• within the 3 months prior to baseline, use of Ecuzumab and Tocilizumab
• received intravenous immunoglobulin or plasma exchange therapy within 4 weeks prior to baseline
• currently participating in other clinical studies

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment group	Drug: Blinatumomab; The initial dose is 9 µ g, administered intravenously continuously for 5 days, with a total dose of 38.5 µ g. After stopping the medication for one week, the patient received a second infusion of Blinatumomab for a total of 5 days, with a total dose of 38.5 µ g.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale	The minimum value is 0, and maximum value is 24. higher scores mean a worse outcome. The primary endpoint is the proportion of patients who achieved a reduction of at least 2 points in the MG-ADL score by week 12 and did not undergo rescue therapy.	At the 12th week

Collaborators and Investigators

• Sponsor: Zhongming Qiu

Study record dates

Study Major Dates

• Study Start (Estimated): November 30, 2024
• Primary Completion (Estimated): December 31, 2025
• Study Completion (Estimated): June 30, 2026

Study Registration Dates

• First Submitted: October 16, 2024
• First Submitted That Met QC Criteria: November 10, 2024
• First Posted (Estimated): November 12, 2024

Study Record Updates

• Last Update Posted (Estimated): November 12, 2024
• Last Update Submitted That Met QC Criteria: November 10, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis; Antineoplastic Agents; Blinatumomab
• Other Study ID Numbers: 2024-254-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes