Refractory Myasthenia GravisPhase 3NCT05332587
Efficacy and Safety of Low-dose Rituximab in the Treatment of Refractory Myasthenia Gravis
A Phase 3 study testing Rituximab in people with Refractory Myasthenia Gravis and Rituximab, run by First Affiliated Hospital, Sun Yat-Sen University at 1 site in China.
Trial summary
This study is designed to evaluate the effectiveness and safety of low-dose rituximab in patients with refractory myasthenia gravis. The traditional treatment of MG is immunosuppressive therapy, usually beginning with corticosteroids. Read more about this study
- Who is it for?
- Ages Up to 80 years
- Conditions the study is for
- Refractory Myasthenia Gravis, Rituximab
- What is being tested?
- Rituximab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2020to 1 Jul 2022
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 Up to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged 80 and under;
- Myasthenia gravis:
- Patients who are diagnosed as generalized or ocular myasthenia gravis
- Patients must have disease refractory to treatment: the condition did not improve (QMG score decreased by less than 3 points after treatment) or even deteriorated after treatment with sufficient prednisone or other immunosuppressive agents.
- Patients sign informed consent forms
Who cannot join
- Over the age of 80
- Patients with serious complications such as infection
- Patients with active TB (during the screening period, a chest X-ray or chest CT would be performed unless the patient can provide chest X-ray or chest CT reports in the last month); or patients with active HBV, HBV DNA> 200
- Patients suffering from cardiomyopathy, acute coronary events, or severe arrhythmia.
- Patients who were allergic to rituximab
- Pregnant or suckling period woman
- Patients accompanied with mental disorders and have difficult to communication
- Patients with a significant abnormality in white blood cells, hemoglobin, and platelet count.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
First Affiliated Hospital, Sun Yat-Sen University
Guangzhou, 510080
Liu Weibin, 86+13826121596, liuwb@mail.sysu.edu.cn
About this study
This study is designed to evaluate the effectiveness and safety of low-dose rituximab in patients with refractory myasthenia gravis.
The traditional treatment of MG is immunosuppressive therapy, usually beginning with corticosteroids. However, up to 70% of treated patients show an incomplete response, including 10 - 30% who are unresponsive. Corticosteroids and other immunosuppressive therapies presented also many side effects. The investigators propose to evaluate in a pilot, open, prospective, single central study, the interest of rituximab (RTX) in the treatment of patients with refractory MG. Fifty patients with refractory MG will be included in the study and divided into two stages: 14 patients in the first stage were followed up after using RTX according to the study protocol. The study will move into the second stage on if the number of effective cases is greater than 3, otherwise, the study will be discontinued (based on Simon's Optimal Two-stage Design). The remaining 36 patients were enrolled in the second stage. In the first and second stages, the treatment plan and follow-up plan were consistent. The therapeutical schema is rituximab 100-200 mg/week for a total of 2-4 times, followed by adequate organ function, laboratory parameters and assessment of MG after each injection and end of follow up for 24 weeks.
- Conditions
- Refractory Myasthenia Gravis, Rituximab
- Being tested
- Rituximab
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RituximabDrug
The therapeutical schema is rituximab 100-200 mg/week for a total of 2-4 times
| Study group | Role | What they receive |
|---|---|---|
| Rituximab Treated | Experimental | rituximab Receives: Drug: Rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to reach MM-1, the proportion of drug remission.
Mild clinical Manifestations（MM-1）was the primary efficacy endpoint observed. Record the time the patient appears from the time of medication to the time of MM-1，and calculate the percentage of Pharmacologic Remission (PR). Criteria for PR: The patient has had no symptoms or signs of MG and continues to take some form of therapy for MG. There is no weakness of any muscle on careful examination by someone skilled in the evaluation of neuromuscular disease. Isolated weakness of eyelid closure is accepted. Patients taking cholinesterase inhibitors are excluded from this category because their use suggests the presence of weakness.
Measured: 27 weeks
Secondary outcomes (1)
The changes of the disease severity
The magnitude by which the Quantitative Myasthenia Gravis (QMG) (0-39), the Myasthenia Gravis-specific Activities of Daily Living scale (MG-ADL) (0-24) , the MG Composite (MGC) (0-46) or the MG 15-item Quality of Life scale (MG-QOL15) (0-60) scores are changed. Higher scores mean a worse outcome.
Measured: 27 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2022
- Study start1 Aug 2020
- Primary completion1 Jul 2022 (estimated)
- Study completion1 Jul 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by First Affiliated Hospital, Sun Yat-Sen UniversityThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Liu Weibin, First Affiliated Hospital, Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05332587
- Sponsor's protocol ID
- [2020]414-1
- Status last verified
- Apr 2022
- First submitted
- 4 Dec 2021
- Last updated
- 2 May 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Low-dose Rituximab in the Treatment of Refractory Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT05332587
- Acronym / sponsor ID
- [2020]414-1
- Conditions
- Refractory Myasthenia Gravis, Rituximab
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- First Affiliated Hospital, Sun Yat-Sen UniversityUniversity, hospital or other
- Study start
- 1 Aug 2020 (actual)
- Primary completion
- 1 Jul 2022 (estimated)
- Study completion
- 1 Jul 2022 (estimated)
- First posted
- 18 Apr 2022 (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.
This study is designed to evaluate the effectiveness and safety of low-dose rituximab in patients with refractory myasthenia gravis.
The traditional treatment of MG is immunosuppressive therapy, usually beginning with corticosteroids. However, up to 70% of treated patients show an incomplete response, including 10 - 30% who are unresponsive. Corticosteroids and other immunosuppressive therapies presented also many side effects. The investigators propose to evaluate in a pilot, open, prospective, single central study, the interest of rituximab (RTX) in the treatment of patients with refractory MG. Fifty patients with refractory MG will be included in the study and divided into two stages: 14 patients in the first stage were followed up after using RTX according to the study protocol. The study will move into the second stage on if the number of effective cases is greater than 3, otherwise, the study will be discontinued (based on Simon's Optimal Two-stage Design). The remaining 36 patients were enrolled in the second stage. In the first and second stages, the treatment plan and follow-up plan were consistent. The therapeutical schema is rituximab 100-200 mg/week for a total of 2-4 times, followed by adequate organ function, laboratory parameters and assessment of MG after each injection and end of follow up for 24 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rituximab Treated | Experimental | Drug: Rituximabrituximab |
Who may qualify
- Ages
- Up to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Aged 80 and under;
- Myasthenia gravis:
- Patients sign informed consent forms
Key exclusion criteria (8 listed)
- Over the age of 80
- Patients with serious complications such as infection
- Patients with active TB (during the screening period, a chest X-ray or chest CT would be performed unless the patient can provide chest X-ray or chest CT reports in the last month); or patients with active HBV, HBV DNA> 200
- Patients suffering from cardiomyopathy, acute coronary events, or severe arrhythmia.
- Patients who were allergic to rituximab
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 |
|---|---|---|
| RituximabThe therapeutical schema is rituximab 100-200 mg/week for a total of 2-4 times | Drug | Rituximab Treated |
Primary outcome
Time to reach MM-1, the proportion of drug remission.
Time frame: 27 weeks
Mild clinical Manifestations（MM-1）was the primary efficacy endpoint observed. Record the time the patient appears from the time of medication to the time of MM-1，and calculate the percentage of Pharmacologic Remission (PR). Criteria for PR: The patient has had no symptoms or signs of MG and continues to take some form of therapy for MG. There is no weakness of any muscle on careful examination by someone skilled in the evaluation of neuromuscular disease. Isolated weakness of eyelid closure is accepted. Patients taking cholinesterase inhibitors are excluded from this category because their use suggests the presence of weakness.
1 secondary outcome
The changes of the disease severity
Time frame: 27 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2022.
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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. This study is not recruiting at the moment.
First Affiliated Hospital, Sun Yat-Sen University
Guangzhou, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Rituximab in people with Refractory Myasthenia Gravis and Rituximab, run by First Affiliated Hospital, Sun Yat-Sen University at 1 site in China.
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 5 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 2 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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 phase mean?
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to reach MM-1, the proportion of drug remission. (time frame: 27 weeks). 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 2 May 2022.