Myasthenia GravisPhase 2/3NCT06836973
Blinatumomab for Treatment of Refractory Myasthenia Gravis
A Phase 2/3 study testing Blinatumomab in people with Myasthenia Gravis, run by Da, Yuwei, M.D..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- Blinatumomab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2025to 30 Jun 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Efficacy and Safety of Blinatumomab for Treatment of Refractory Myasthenia Gravis.
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
- Being tested
- Blinatumomab
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BlinatumomabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Blinatumomab | Experimental | Not described Receives: Drug: Blinatumomab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline to 6 months
Secondary outcomes (6)
Change of Quantitative Myasthenia Gravis (QMG) Scores from baseline
The QMG scale is a 13-item scale used to objectively assess muscle strength and fatigue in patients with myasthenia gravis. The total score ranges from 0 to 39, with higher scores indicating greater disease severity.
Measured: From baseline to 6 months
Time to achievement of minimal symptom expression (MSE)
The MSE is a quantifiable, patient-reported outcome measure that assesses treatment goals in myasthenia gravis. It is defined as achieving an MG-ADL score of ≤1, indicating that clinical symptoms of MG are at their minimal level.
Measured: From baseline to 6 months
Change of Myasthenia Gravis Quantity-of-Life Scale (MG-QoL15) from baseline
The MG-QOL15 scale is a 15-item questionnaire designed to assess the impact of myasthenia gravis on patients' daily life, physical function, social well-being, and mental health. The total score ranges from 0 to 60, with higher scores indicating poorer quality of life.
Measured: From baseline to 6 months
Change of Myasthenia Gravis Composite (MGC) scores from baseline
The MGC scale is a 10-item scale that combines patient self-assessment and physician examination results. Each item is assigned a weight based on factors such as health risks, quality of life, and prognosis. The total score of the scale is 50 points, with higher scores indicating more severe disease.
Measured: From baseline to 6 months
Change of antibody titers from baseline
MG antibodies are detected at enrollment and the titers of antibodies will be monitored monthly.
Measured: From baseline to 6 months
Treatment-Related Adverse Events (AEs)
Record all AEs reported by patients during the study period.
Measured: From baseline to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2025
- Study start1 Apr 2025 (estimated)
- Primary completion31 Mar 2028 (estimated)
- Study completion30 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia GravisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Da, Yuwei, M.D.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06836973
- Sponsor's protocol ID
- LYS[2024]408-002
- Status last verified
- Feb 2025
- First submitted
- 16 Feb 2025
- Last updated
- 25 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Blinatumomab for Treatment of Refractory Myasthenia Gravis.
- ClinicalTrials.gov ID
- NCT06836973
- Acronym / sponsor ID
- LYS[2024]408-002
- Conditions
- Myasthenia Gravis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 2 participants (estimated)
- Lead sponsor
- Da, Yuwei, M.D.
- Study start
- 1 Apr 2025 (estimated)
- Primary completion
- 31 Mar 2028 (estimated)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 20 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Blinatumomab | Experimental | Drug: Blinatumomab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BlinatumomabBlinatumomab 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. | Drug | Blinatumomab |
Primary outcome
Change of Myasthenia Gravis Activities of Daily Living (MG-ADL) Score from baseline
Time frame: From baseline to 6 months
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.
6 secondary outcomes
Change of Quantitative Myasthenia Gravis (QMG) Scores from baseline
Time frame: From baseline to 6 months
Time to achievement of minimal symptom expression (MSE)
Time frame: From baseline to 6 months
Change of Myasthenia Gravis Quantity-of-Life Scale (MG-QoL15) from baseline
Time frame: From baseline to 6 months
Change of Myasthenia Gravis Composite (MGC) scores from baseline
Time frame: From baseline to 6 months
Change of antibody titers from baseline
Time frame: From baseline to 6 months
Treatment-Related Adverse Events (AEs)
Time frame: From baseline to 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Blinatumomab in people with Myasthenia Gravis, run by Da, Yuwei, M.D..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 25 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yuwei Da, M.D. (00-86-010-83198493, dayuwei100@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of Myasthenia Gravis Activities of Daily Living (MG-ADL) Score from baseline (time frame: From baseline to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2025; AzTrials last refreshed its copy on 8 Nov 2026.