Myasthenia GravisNCT04101578
Clinical Prognosis and Progression of Myasthenia Gravis Patients
An observational study of Myasthenia Gravis, run by Da, Yuwei, M.D. at 1 site in China.
Trial summary
This study collects the clinical data of myasthenia gravis (MG) patients, assesses outcomes and adverse effects of different treatment regimens, and searches for risk factors of conversion to generalized MG. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)
- Type of treatment
- Injection or infusion
- How many people
- 2,000 participants
- Where
- 1 siteChina
- Study dates
- 8 Feb 2017to 31 Dec 2024
- Phase
- Observational
- 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Myasthenia Gravis
Who may be able to join
- Age >14.
- Clinical Diagnosis of MG with supporting evidence:
- unequivocal clinical response to pyridostigmine
- positive antibody testing
- decrement >10% in repetitive nerve stimulations study (RNS) .
- Willingness to sample collection, imaging study and other disease-related examinations and assessments.
- Patients with informed consent.
Who cannot join
- History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
- Age ≤14 years.
- Severe anxiety, depression or schizophrenia.
- Cognitive impairment or mini-mental state examination (MMSE) score ≤24.
- Severe systemic illness with life-expectancy less than 4 years.
- Unwillingness to consent for collection of biological samples.
- Inability to provide informed consent.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Xuan Wu Hospital, Capital Medical University
Beijing, Beijing Municipality, 100053
yuwei Da, M.D., 00-86-010-83198492, dayuwei1000@163.com
About this study
Official title: Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients
This study collects the clinical data of myasthenia gravis (MG) patients, assesses outcomes and adverse effects of different treatment regimens, and searches for risk factors of conversion to generalized MG.
Read the detailed description
This is a multicenter, observational cohort trial in the real-world clinical setting recruiting MG patients from Neurology Departments of 6 hospitals in different regions of China. Clinical manifestations, laboratory test results, chest imaging and history of thymectomy are recorded. Patients will be classified by clinical manifestation as well as antibody status, and treatment regimens are determined according to the physician's judgment and preferences of the patients. Patients are followed up prospectively on regular to assess the outcomes of treatments and monitor any side effects. Peripheral blood samples are collected annually. Patients' clinical records are uploaded to an online database. The investigators plan to recruit a final sample of 2000 patients for analysis.
- Conditions
- Myasthenia Gravis
- Being tested
- Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)Drug
Also called Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate
Treatment regimens are determined according to the physician's judgment and preferences of the patients.
| Study group | Role | What they receive |
|---|---|---|
| Ocular MG | Patients with autoimmune MG whose symptoms restricted to extraocular muscles Receives: Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg) | |
| Generalized MG | Patients not only suffer from extraocular muscles weakness but also from limb weakness, bulbar symptoms, or even respiratory failure Receives: Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Conversion rates from ocular to generalized MG at the last visit and risk factors.
Ocular MG patients are followed up to determine the ratio of conversion to generalized disease at the end of follow-up. The clinical records will be retrospectively analyzed to search for risk factors of progressing.
Measured: Baseline, 48 months
Change in Quantitative Myasthenia Gravis (QMG) Scores from Baseline to 48 months.
The QMG is a 13-item scale which measures ocular, bulbar, limb function and respiratory function. The total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) obtained by summing the responses to each individual item (None=0, Mild=1, Moderate=2, Severe=3).
Measured: Baseline, 12 months, 24 months, 36 months, 48 months
Change in MG-specific Activities of Daily Living scale (MG-ADL).
The MG-ADL is an 8-item scale to assess symptoms of myasthenia gravis patients obtained by summing the responses to each individual item (Grades: 0,1,2,3). The score ranges from 0 to 24.
Measured: Baseline,3months, 6 months, 9 months, 12 months, 18 months, 24 months, 30months, 36 months, 42 months, 48 months
The proportion of patients reaching minimal manifestations (MM) or better.
Clinical statuses of patients are assessed and categorized according to Myasthenia Gravis Foundation of America (MGFA) postintervention status (PIS). MM or better includes Minimal Manifestation (MM), Pharmacologic Remission (PR) or Complete Remission (CR).
Measured: 48 months
Secondary outcomes (2)
Proportion of Patients with Treatment-related Adverse Experiences.
Treatment-Related Adverse Events (AEs) are evaluated in patients of different regimens.
Measured: 3 months, 6 months, 12 months, 24 months, 36 months, 48 months
Changes in titers of MG antibodies.
MG antibodies are detected at enrollment and the titers of antibodies will be monitored annually.
Measured: Baseline, 12 months, 24 months, 36 months, 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2019
- Study start8 Feb 2017
- Primary completion1 Jun 2023 (estimated)
- Study completion31 Dec 2024 (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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Da, Yuwei, M.D.
- Responsible party
- Sponsor
- Investigators
- yuwei Da, M.D., Study chair, Xuan Wu Hospital, Capital Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04101578
- Sponsor's protocol ID
- 2017YFC0907705
- Status last verified
- Aug 2021
- First submitted
- 16 Sep 2019
- Last updated
- 23 Aug 2021
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients
- ClinicalTrials.gov ID
- NCT04101578
- Acronym / sponsor ID
- 2017YFC0907705
- Conditions
- Myasthenia Gravis
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- Da, Yuwei, M.D.
- Study start
- 8 Feb 2017 (actual)
- Primary completion
- 1 Jun 2023 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 24 Sep 2019 (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 collects the clinical data of myasthenia gravis (MG) patients, assesses outcomes and adverse effects of different treatment regimens, and searches for risk factors of conversion to generalized MG.
Detailed description
This is a multicenter, observational cohort trial in the real-world clinical setting recruiting MG patients from Neurology Departments of 6 hospitals in different regions of China. Clinical manifestations, laboratory test results, chest imaging and history of thymectomy are recorded. Patients will be classified by clinical manifestation as well as antibody status, and treatment regimens are determined according to the physician's judgment and preferences of the patients. Patients are followed up prospectively on regular to assess the outcomes of treatments and monitor any side effects. Peripheral blood samples are collected annually. Patients' clinical records are uploaded to an online database. The investigators plan to recruit a final sample of 2000 patients for analysis.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ocular MG | – | Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)Patients with autoimmune MG whose symptoms restricted to extraocular muscles |
| Generalized MG | – | Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)Patients not only suffer from extraocular muscles weakness but also from limb weakness, bulbar symptoms, or even respiratory failure |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Myasthenia Gravis
Key inclusion criteria (7 listed)
- Age >14.
- Clinical Diagnosis of MG with supporting evidence:
- Willingness to sample collection, imaging study and other disease-related examinations and assessments.
- Patients with informed consent.
Key exclusion criteria (7 listed)
- History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
- Age ≤14 years.
- Severe anxiety, depression or schizophrenia.
- Cognitive impairment or mini-mental state examination (MMSE) score ≤24.
- Severe systemic illness with life-expectancy less than 4 years.
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 |
|---|---|---|
| Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)Also: Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, MethotrexateTreatment regimens are determined according to the physician's judgment and preferences of the patients. | Drug | Generalized MG, Ocular MG |
Primary outcome
Conversion rates from ocular to generalized MG at the last visit and risk factors.
Time frame: Baseline, 48 months
Ocular MG patients are followed up to determine the ratio of conversion to generalized disease at the end of follow-up. The clinical records will be retrospectively analyzed to search for risk factors of progressing.
Change in Quantitative Myasthenia Gravis (QMG) Scores from Baseline to 48 months.
Time frame: Baseline, 12 months, 24 months, 36 months, 48 months
The QMG is a 13-item scale which measures ocular, bulbar, limb function and respiratory function. The total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) obtained by summing the responses to each individual item (None=0, Mild=1, Moderate=2, Severe=3).
Change in MG-specific Activities of Daily Living scale (MG-ADL).
Time frame: Baseline,3months, 6 months, 9 months, 12 months, 18 months, 24 months, 30months, 36 months, 42 months, 48 months
The MG-ADL is an 8-item scale to assess symptoms of myasthenia gravis patients obtained by summing the responses to each individual item (Grades: 0,1,2,3). The score ranges from 0 to 24.
The proportion of patients reaching minimal manifestations (MM) or better.
Time frame: 48 months
Clinical statuses of patients are assessed and categorized according to Myasthenia Gravis Foundation of America (MGFA) postintervention status (PIS). MM or better includes Minimal Manifestation (MM), Pharmacologic Remission (PR) or Complete Remission (CR).
2 secondary outcomes
Proportion of Patients with Treatment-related Adverse Experiences.
Time frame: 3 months, 6 months, 12 months, 24 months, 36 months, 48 months
Changes in titers of MG antibodies.
Time frame: Baseline, 12 months, 24 months, 36 months, 48 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2021.
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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.
yuwei Da, M.D. study chair
Xuan Wu Hospital, Capital Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myasthenia Gravis, run by Da, Yuwei, M.D. 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 7 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 23 Aug 2021. 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.
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?
Contact yuwei Da, M.D. (00-86-010-83198492, dayuwei1000@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Conversion rates from ocular to generalized MG at the last visit and risk factors. (time frame: Baseline, 48 months). The study lists 4 primary outcomes in all. 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 23 Aug 2021.