Blinatumomab for Treatment of Refractory Myasthenia Gravis

NCT06836973 | Not Yet Recruiting Phase 2

• 1 other identifier: LYS[2024]408-002
• Study Type: interventional
• Target: 2
• Locations: 0 countries
• Sites: N/A

Brief Summary

The goal of this clinical trial is to evaluate the efficacy and safety of Blinatumomab in the treatment of refractory myasthenia gravis, with the expectation of offering a new therapeutic option for refractory patients. The main questions it aims to answer are:

• Does Blinatumomab improve patients' clinical symptoms?
• Is Blinatumomab safe for the treatment of myasthenia gravis? Participants will:
• Receive two cycles of intravenous Blinatumomab infusion, each lasting 5 days, with a 1-week interval between cycles.
• Visit the clinic once every 4 weeks for checkups and tests.
• Keep a diary of their symptoms and the types and dosages of medications.

Conditions

Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• Change of Myasthenia Gravis Activities of Daily Living (MG-ADL) Score from baseline

  The MG-ADL scale is an 8-item questionnaire. It is completed by trained interviewers based on the patients' subjective responses. The total score ranges from 0 to 24, with higher scores indicating greater impact of the disease on daily living activities.

  From baseline to 6 months

Secondary Outcomes (6)

• Change of Quantitative Myasthenia Gravis (QMG) Scores from baseline

  From baseline to 6 months

• Time to achievement of minimal symptom expression (MSE)

  From baseline to 6 months

• Change of Myasthenia Gravis Quantity-of-Life Scale (MG-QoL15) from baseline

  From baseline to 6 months

• Change of Myasthenia Gravis Composite (MGC) scores from baseline

  From baseline to 6 months

• Change of antibody titers from baseline

  From baseline to 6 months

Study Arms (1)

Blinatumomab

EXPERIMENTAL

Drug: Blinatumomab

Interventions

Blinatumomab DRUG

Blinatumomab is used at its starting dose of 9 µg per day and administered as continuous infusion for 5 days (total dose of 38.5 µg). After a 1-week pause, the patients receive a second 5-day infusion with blinatumomab of total 38.5 µg of the drug.

Blinatumomab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age at onset \> 18 years old
• The diagnosis of MG was based on the presence of typical myasthenic symptoms and supported by positive autoantibodies, electrophysiological studies, and/or the neostigmine test.
• Positive or negative for anti-AChR, and/or anti-MuSK, and/or anti-LRP4 antibodies.
• Refractory myasthenia gravis (MG) patients are defined as those who meet any of the following criteria: For patients with ocular MG, the condition is defined as having no significant improvement in disease symptoms (QMG score improvement \<25%) after adequate dosing and duration of existing immunosuppressive drugs and targeted biologics, with no change or worsening in the post-intervention status (PIS), or if the PIS improves but disease symptoms worsen or relapse during the regular tapering of immunosuppressive treatment, severely affecting daily quality of life. For generalized MG, the patient must meet the following conditions: no improvement or worsening in PIS after adequate dosing and duration of existing immunosuppressive drugs and targeted biologics; improvement in PIS, but with an MG-ADL score ≥6 persisting for at least six months; remission or improvement in PIS, but with ≥2 episodes of disease exacerbation (MG-ADL ≥6) per year during tapering of immunotherapy medications; patients who, after experiencing a myasthenic crisis, undergo multiple immunotherapies including intravenous efgartigimod, eculizumab, immunoglobulin, plasma exchange, and high-dose intravenous methylprednisolone, and active infection control, but still cannot be weaned off the ventilator due to respiratory muscle weakness from MG for more than 14 days. (Note: This includes patients who cannot tolerate existing treatment drugs due to contraindications, comorbidities, or adverse drug reactions.)
• Receiving stable doses of medication prior to enrollment
• Written informed consent

You may not qualify if:

• Patients who have thymoma or have undergone thymectomy within six months
• Patients who have used other biologics prior to enrollment that may affect the efficacy assessment of blinatumomab.
• Severe cardiovascular, hepatic, renal, respiratory, or endocrine diseases, malignancies, or uncontrolled acute or chronic infections
• Pregnancy or lactation, unwillingness to avoid pregnancy
• Patients with other diseases that may affect the assessment of muscle strength
• Other conditions that would preclude participation

Sponsors & Collaborators

• Da, Yuwei, M.D.: lead

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

blinatumomab

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Yuwei Da, M.D.

CONTACT

00-86-010-83198493; dayuwei100@hotmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDIV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 16, 2025
• First Posted: February 20, 2025
• Study Start: April 1, 2025
• Primary Completion (Estimated): March 31, 2028
• Study Completion (Estimated): June 30, 2028
• Last Updated: March 25, 2025

Record last verified: 2025-02