Myasthenia GravisPhase 3NCT01727193
The Efficacy and Safety of Leflunomide or Azathioprine Therapy in Myasthenia Gravis Patients After Expand Thymectomy
A Phase 3 study testing Azathioprine and Leflunomide in people with Myasthenia Gravis, run by First Affiliated Hospital, Sun Yat-Sen University at 1 site in China.
Trial summary
This is a randomized controlled clinical study. The investigators screen of eligible patients, randomized divide into the following two groups: corticosteroids + azathioprine group, corticosteroids + leflunomide group. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- Azathioprine and Leflunomide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 290 participants
- Where
- 1 siteChina
- Study dates
- Sep 2012to 18 Nov 2021
- 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 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 12 to 65 years；
- Myasthenia gravis:
- Patients who are diagnosed as generalized or ocular myasthenia gravis
- have experienced extended thymectomy (including thymic hyperplasia and thymoma)， no significant complications in 6 months after operation , and does not received any immunosuppressants or glucocorticoids treatments.
- do not applied in plasmapheresis or immunoglobulins treatment during 3 months .
- women of child-bearing period do not have a plan of pregnant for at least 3 year.
- Written consent of the patient, after informing
Who cannot join
- The liver , kidney or glycometabolic function is abnormal
- Seriously complications, such as infection or symptom in central nervous system,
- The patients who suffering from malignancy or a history of malignancy, a variety of sexually transmitted diseases and HIV infection, tuberculosis infection, and other condition which need to prohibit the use of immunosuppressive patients.
- Be allergic to leflunomide, azathioprine
- Pregnant or suckling period woman
- Accompanied with mental disorders and have difficult to communication
Show all 10Show fewer
- Experienced myasthenia crisis in 3 months.
- suffering from clear cardiopulmonary functional and brain abnormalities
- Have a history of refractory hypertension or peptic ulcer .
- One of the white blood cells, hemoglobin, and platelet count obvious abnormalities
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital, SUN YAT-SEN UNIVERSITY
Guangzhou, Guangdong, 510080
About this study
This is a randomized controlled clinical study. The investigators screen of eligible patients, randomized divide into the following two groups: corticosteroids + azathioprine group, corticosteroids + leflunomide group. The investigators treat the enrolled patients, estimate efficacy and observed the side effects according to the requirements of program. The investigators establish a clinical database for recording patients date and statistical analysis. Evaluation of short-term and long-term efficacy of thymectomized myasthenia gravis patients in the different group prove that what kind of treatment can improve the cure rate. The investigators will evaluate the acute toxicity (gastrointestinal side effects, liver and kidney dysfunction) and long-term toxicity (immune dysfunction, gonadal suppression) when the investigators apply these therapy in the treatment of different clinical types of myasthenia gravis.
- Conditions
- Myasthenia Gravis
- Being tested
- Azathioprine, Leflunomide
- Keywords
- myasthenia gravis, leflunomide
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzathioprineDrug
The basic therapy of each group is cholinesterase inhibitors (pyridostigmine 36mg / kg / d). Group 1 glucocorticoid + Azathioprine: Glucocorticoid (0.25mg/kg/d )plus Azathioprine at a starting dose of 50mg/d for 14 days, then increase up to 2mg/kg in 2 weeks.If no adverse events occurred , maintain the dose until 48 weeks.
LeflunomideDrug
The basic therapy of each group is cholinesterase inhibitors (pyridostigmine 36mg / kg / d). Group 2 glucocorticoid + Leflunomide: Glucocorticoid (0.25mg/kg/d )plus Leflunomide 20mg/d for adult, 10mg/d for child. if no adverse events occurred , maintain the dose until 48 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Azathioprine | Active comparator | cholinesterase inhibitors+Glucocorticoid +Azathioprine Receives: Drug: Azathioprine |
| Leflunomide | Active comparator | cholinesterase inhibitors+glucocorticoid+Leflunomide Receives: Drug: Leflunomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
the percent of achieving good response
Pharmacologic Remission (PR) was the primary efficacy endpoint observed. Record the time the patient appears from the time of medication to the time of PR, and calculate the percentage of PR patients in each arm. Criteria for PR: The patient has had no symptoms or signs of MG for at least 1 year 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: 144 weeks
Secondary outcomes (1)
The changes of the disease severity and the antibody titer during the follow-up
The magnitude by which QMG or ADL scores are reduced. Total amount of steroid used during the follow-up. Level of the acetylcholine receptor antibody.
Measured: 144 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2012 (estimated)
- Study startSep 2012
- Primary completion18 Oct 2021
- Study completion18 Nov 2021
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.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 290 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Liu Weibin, First Affiliated Hospital, Sun Yat-Sen University
- Investigators
- Weibin Liu, Doctor, Principal investigator, First Affiliated Hospital, Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01727193
- Sponsor's protocol ID
- ZS-LEF
- Status last verified
- Apr 2022
- First submitted
- 8 Nov 2012
- Last updated
- 14 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy and Safety of Leflunomide or Azathioprine Therapy in Myasthenia Gravis Patients After Expand Thymectomy
- ClinicalTrials.gov ID
- NCT01727193
- Acronym / sponsor ID
- ZS-LEF
- Conditions
- Myasthenia Gravis
- Keywords
- myasthenia gravis, leflunomide
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 290 participants (actual)
- Lead sponsor
- First Affiliated Hospital, Sun Yat-Sen UniversityUniversity, hospital or other
- Study start
- Sep 2012 (actual)
- Primary completion
- 18 Oct 2021 (actual)
- Study completion
- 18 Nov 2021 (actual)
- First posted
- 15 Nov 2012 (estimated)
- 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 is a randomized controlled clinical study. The investigators screen of eligible patients, randomized divide into the following two groups: corticosteroids + azathioprine group, corticosteroids + leflunomide group. The investigators treat the enrolled patients, estimate efficacy and observed the side effects according to the requirements of program. The investigators establish a clinical database for recording patients date and statistical analysis. Evaluation of short-term and long-term efficacy of thymectomized myasthenia gravis patients in the different group prove that what kind of treatment can improve the cure rate. The investigators will evaluate the acute toxicity (gastrointestinal side effects, liver and kidney dysfunction) and long-term toxicity (immune dysfunction, gonadal suppression) when the investigators apply these therapy in the treatment of different clinical types of myasthenia gravis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Azathioprine | Active comparator | Drug: Azathioprinecholinesterase inhibitors+Glucocorticoid +Azathioprine |
| Leflunomide | Active comparator | Drug: Leflunomidecholinesterase inhibitors+glucocorticoid+Leflunomide |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 12 to 65 years；
- Myasthenia gravis:
Key exclusion criteria (10 listed)
- The liver , kidney or glycometabolic function is abnormal
- Seriously complications, such as infection or symptom in central nervous system,
- The patients who suffering from malignancy or a history of malignancy, a variety of sexually transmitted diseases and HIV infection, tuberculosis infection, and other condition which need to prohibit the use of immunosuppressive patients.
- Be allergic to leflunomide, azathioprine
- Pregnant or suckling period woman
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 |
|---|---|---|
| AzathioprineThe basic therapy of each group is cholinesterase inhibitors (pyridostigmine 36mg / kg / d). Group 1 glucocorticoid + Azathioprine: Glucocorticoid (0.25mg/kg/d )plus Azathioprine at a starting dose of 50mg/d for 14 days, then increase up to 2mg/kg in 2 weeks.If no adverse events occurred , maintain the dose until 48 weeks. | Drug | Azathioprine |
| LeflunomideThe basic therapy of each group is cholinesterase inhibitors (pyridostigmine 36mg / kg / d). Group 2 glucocorticoid + Leflunomide: Glucocorticoid (0.25mg/kg/d )plus Leflunomide 20mg/d for adult, 10mg/d for child. if no adverse events occurred , maintain the dose until 48 weeks. | Drug | Leflunomide |
Primary outcome
the percent of achieving good response
Time frame: 144 weeks
Pharmacologic Remission (PR) was the primary efficacy endpoint observed. Record the time the patient appears from the time of medication to the time of PR, and calculate the percentage of PR patients in each arm. Criteria for PR: The patient has had no symptoms or signs of MG for at least 1 year 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 and the antibody titer during the follow-up
Time frame: 144 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 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.
Weibin Liu, Doctor principal investigator
First Affiliated Hospital, Sun Yat-Sen University
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 Azathioprine and Leflunomide in people with Myasthenia Gravis, 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 7 inclusion and 10 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: finished, as of the sponsor's last update on 14 Apr 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
the percent of achieving good response (time frame: 144 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 14 Apr 2022.