Myasthenia GravisNCT04768465
Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis
An observational study of Myasthenia Gravis, run by Da, Yuwei, M.D. at 1 site in China.
Trial summary
This study is designed to evaluate the effectiveness and safety of tacrolimus combined with low-dose prednisone in the management of myasthenia gravis patients, compared to tacrolimus as initial immune monotherapy. 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?
- Pyridostigmine, Prednisone, Tacrolimus and Pyridostigmine, Tacrolimus
- Type of treatment
- Pill or oral medicine
- How many people
- 160 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2021to 31 Oct 2024
- How long will I take part?
- 6 Months
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with Myasthenia Gravis
Who may be able to join
- Age ≥18
- Clinical Diagnosis of MG is confirmed based on typical clinical features of fluctuating muscle weakness, with at least 1 of the following supporting evidence:
- positive clinical response to acetylcholinesterase inhibitor
- positive AchR-Ab or MuSK-Ab testing
- decrement >10% in repetitive nerve stimulations study (RNS) or increased jitter on single-fibre electromyography (SFEMG)
- MGFA clinical classification: I - IV
Show all 10Show fewer
- Baseline MG-ADL ≥ 3
- Disease course from onset to enrollment ≤ 12 months
- Cooperation to followup
- Written informed consent
Who cannot join
- Initiation of immunosuppressant for MG prior to screening, including Prednisone, Methylprednisolone, Azathioprine, Methotrexate, Cyclosporine A, Mycophenolate Mofetil, Tacrolimus and Cyclophosphamide
- Treatment of immunosuppressant for other concomitant disease 6 months prior to recruitment
- Rapid immunosuppressive treatments like Intravenous immunoglobulin or plasma exchange 1 month prior to recruitment
- Thymectomy within 3 months prior to Screening
- Concomitant chronic degenerative, psychiatric, or neurologic disorder that can cause weakness or fatigue
- Consciousness, dementia or schizophrenia
Show all 9Show fewer
- Pregnancy or lactation, unwillingness to avoid pregnancy
- Uncontrolled hypertension or diabetes, Liver or kidney dysfunction, Cataract, Severe osteoporosis, Femoral head necrosis; Hyperkalemia, HIV, Acute or chronic infection
- Other conditions that would preclude participation
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Yuwei Da
Beijing, Beijing Municipality, 100053
Yuwei Da, M.D., 00-86-010-83198492, dayuwei1000@163.com
About this study
Official title: Effectiveness and Safety of Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis: A Real-world Study
This study is designed to evaluate the effectiveness and safety of tacrolimus combined with low-dose prednisone in the management of myasthenia gravis patients, compared to tacrolimus as initial immune monotherapy.
Read the detailed description
This is a single center, observational real-world study recruiting myasthenia gravis patients from Neurology Departments of Xuanwu Hospital, aiming to compare effectiveness and safety of 2 different inmunotherapy for MG. The study plans to recruit 160 MG participants and divides into 2 treatment groups according to physician's judgment and preferences of patients, one is combined immunotherapy group in which tacrolimus added with low-dose prednisone (0.25mg/kg/d), and the other is tacrolimus monotherapy group. Both groups can be treated with pyridostigmine to relieve symptoms. Patients are followed up at 1, 3 and 6 month after treatment initiation to assess the efficacy of both regimen. The primary outcome is the change of MG-ADL scores. Also, liver and renal functions are tested to monitor any side effects. Patients' clinical records are uploaded to an online database.
- Conditions
- Myasthenia Gravis
- Being tested
- Pyridostigmine, Prednisone, Tacrolimus, Pyridostigmine, Tacrolimus
- Keywords
- myasthenia gravis, treatment, tacrolimus, low-dose prednisone
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pyridostigmine, Prednisone, TacrolimusDrug
Also called Pyridostigmine Bromide, Deltacortone/Meticorten/Prednisone Acetate Tablets, Prograf/FK506
Dose of tacrolimus should be initiated based on CYP3A5*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Prednisone is administrated with an initial dose of 0.25mg/kg/d and started to tamper with the achievement of MMS or the presence of any intolerable side effects. The rate of tampering is considered by the physician, usually no more than 5mg/month. If the participants failed to maintain MMS, dose of prednisone should be increased 5mg/week to 0.25mg/kg/d and maintained until MMS reached again. After MMS sustained for 1 month, prednisone dose would be tapered again with 2.5mg/month. Calcium and potassium supplements and gastric mucosa protectors could be addressed to avoid any adverse effects of prednisone. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients.
Pyridostigmine, TacrolimusDrug
Also called Pyridostigmine Bromide, Prograf/FK506
Dose of tacrolimus should be initiated based on CYP3A5*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients.
| Study group | Role | What they receive |
|---|---|---|
| Combined Immunotherapy | MG patients are treated with tacrolimus combined with low-dose prednisone (0.25mg/kg/d).
Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d). Receives: Drug: Pyridostigmine, Prednisone, Tacrolimus | |
| Tacrolimus monotherapy | MG patients are treated with tacrolimus as initial immune monotherapy. Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d). Receives: Drug: Pyridostigmine, Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of MG-specific Activities of Daily Living scale (MG-ADL) from Baseline
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, 1 month, 3 months, 6 months
Secondary outcomes (8)
Time to achievement of minimal manifestations (MMS) or better
The time duration from treatment initiation to the achievement of MMS or better. Clinical statuses of patients are assessed and categorized according to Myasthenia Gravis Foundation of America (MGFA) post-intervention status (PIS). MM or better includes Minimal Manifestation (MM), Pharmacologic Remission (PR) or Complete Remission (CR).
Measured: From Baseline to 6 months
Time to achievement of Patient-Acceptable Symptom States
The Patient-Acceptable Symptom States question is a simple yes or no query that asked: "Considering all the ways you are affected by Myasthenia, if you had to stay in your current state for the next month, would you say that your current disease state status is satisfactory?" This question reflects the patients assessment of their own health.
Measured: From Baseline to 6 months
Change of Quantitative Myasthenia Gravis (QMG) Scores from Baseline
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, 1 month, 3 months, 6 months
Change of Myasthenia Gravis Quantity-of-Life Scale (MG-QoL15) from Baseline
The MG-QOL15 is helpful in informing the clinician about the patient's perception of the extent of and dissatisfaction with myasthenia gravis (MG)-related dysfunction. MG-QOL15 evaluates patients' aspects about physical status, social adaptation and mental well-being.
Measured: Baseline, 1 month, 3 months, 6 months
Changes of MG-ADL subscores from baseline
Subscores of ADL items can reflect patients' assessment about different MG-related dysfunctions, including ptosis, diplopia, talking, chewing, swallowing, breathing and limbs function.
Measured: Baseline, 1 month, 3 months, 6 months
Serum IL-2 level
Tacrolimus exerts its immunosuppressive effect by inhibiting the early phase gene expression during T-cell activation, including the pro-inflammation IL-2 gene. Thus serum IL-2 is tested as a target of tacrolimus and also a marker of in vivo auto-immune status after treatment.
Measured: Baseline, 1 month, 3 months, 6 months
Treatment Failure
Treatment failure is defined as discontinuation of tacrolimus therapy in patients who failed to achieve MMS or better or suffered from exacerbations (MG-ADL or QMG scores increase 50%) or myasthenia crisis.
Measured: Baseline to 6 months
Withdrawal
Participants quit the clinical trial for any reason including unsatisfied response, economic burden or poor compliance to treatment protocol. Patients may quit at any time they want.
Measured: 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 posted24 Feb 2021
- Study start1 Jan 2021
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Oct 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
- 160 participants (estimated)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- Da, Yuwei, M.D.
- Responsible party
- Sponsor
- Investigators
- Yuwei Da, M.D., Study chair, Xuanwu Hospital, Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04768465
- Sponsor's protocol ID
- 1.0
- Status last verified
- Feb 2021
- First submitted
- 21 Feb 2021
- Last updated
- 24 Feb 2021
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness and Safety of Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis: A Real-world Study
- ClinicalTrials.gov ID
- NCT04768465
- Acronym / sponsor ID
- 1.0
- Conditions
- Myasthenia Gravis
- Keywords
- myasthenia gravis, treatment, tacrolimus, low-dose prednisone
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 160 participants (estimated)
- Lead sponsor
- Da, Yuwei, M.D.
- Study start
- 1 Jan 2021 (actual)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Oct 2024 (estimated)
- First posted
- 24 Feb 2021 (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 tacrolimus combined with low-dose prednisone in the management of myasthenia gravis patients, compared to tacrolimus as initial immune monotherapy.
Detailed description
This is a single center, observational real-world study recruiting myasthenia gravis patients from Neurology Departments of Xuanwu Hospital, aiming to compare effectiveness and safety of 2 different inmunotherapy for MG. The study plans to recruit 160 MG participants and divides into 2 treatment groups according to physician's judgment and preferences of patients, one is combined immunotherapy group in which tacrolimus added with low-dose prednisone (0.25mg/kg/d), and the other is tacrolimus monotherapy group. Both groups can be treated with pyridostigmine to relieve symptoms. Patients are followed up at 1, 3 and 6 month after treatment initiation to assess the efficacy of both regimen. The primary outcome is the change of MG-ADL scores. Also, liver and renal functions are tested to monitor any side effects. Patients' clinical records are uploaded to an online database.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 6 Months
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Combined Immunotherapy | – | Drug: Pyridostigmine, Prednisone, TacrolimusMG patients are treated with tacrolimus combined with low-dose prednisone (0.25mg/kg/d). Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d). |
| Tacrolimus monotherapy | – | Drug: Pyridostigmine, TacrolimusMG patients are treated with tacrolimus as initial immune monotherapy. Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with Myasthenia Gravis
Key inclusion criteria (10 listed)
- Age ≥18
- Clinical Diagnosis of MG is confirmed based on typical clinical features of fluctuating muscle weakness, with at least 1 of the following supporting evidence:
- MGFA clinical classification: I - IV
- Baseline MG-ADL ≥ 3
- Disease course from onset to enrollment ≤ 12 months
Key exclusion criteria (9 listed)
- Initiation of immunosuppressant for MG prior to screening, including Prednisone, Methylprednisolone, Azathioprine, Methotrexate, Cyclosporine A, Mycophenolate Mofetil, Tacrolimus and Cyclophosphamide
- Treatment of immunosuppressant for other concomitant disease 6 months prior to recruitment
- Rapid immunosuppressive treatments like Intravenous immunoglobulin or plasma exchange 1 month prior to recruitment
- Thymectomy within 3 months prior to Screening
- Concomitant chronic degenerative, psychiatric, or neurologic disorder that can cause weakness or fatigue
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 |
|---|---|---|
| Pyridostigmine, Prednisone, TacrolimusAlso: Pyridostigmine Bromide, Deltacortone/Meticorten/Prednisone Acetate Tablets, Prograf/FK506Dose of tacrolimus should be initiated based on CYP3A5*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Prednisone is administrated with an initial dose of 0.25mg/kg/d and started to tamper with the achievement of MMS or the presence of any intolerable side effects. The rate of tampering is considered by the physician, usually no more than 5mg/month. If the participants failed to maintain MMS, dose of prednisone should be increased 5mg/week to 0.25mg/kg/d and maintained until MMS reached again. After MMS sustained for 1 month, prednisone dose would be tapered again with 2.5mg/month. Calcium and potassium supplements and gastric mucosa protectors could be addressed to avoid any adverse effects of prednisone. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients. | Drug | Combined Immunotherapy |
| Pyridostigmine, TacrolimusAlso: Pyridostigmine Bromide, Prograf/FK506Dose of tacrolimus should be initiated based on CYP3A5*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients. | Drug | Tacrolimus monotherapy |
Primary outcome
Change of MG-specific Activities of Daily Living scale (MG-ADL) from Baseline
Time frame: Baseline, 1 month, 3 months, 6 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.
8 secondary outcomes
Time to achievement of minimal manifestations (MMS) or better
Time frame: From Baseline to 6 months
Time to achievement of Patient-Acceptable Symptom States
Time frame: From Baseline to 6 months
Change of Quantitative Myasthenia Gravis (QMG) Scores from Baseline
Time frame: Baseline, 1 month, 3 months, 6 months
Change of Myasthenia Gravis Quantity-of-Life Scale (MG-QoL15) from Baseline
Time frame: Baseline, 1 month, 3 months, 6 months
Changes of MG-ADL subscores from baseline
Time frame: Baseline, 1 month, 3 months, 6 months
Serum IL-2 level
Time frame: Baseline, 1 month, 3 months, 6 months
Treatment Failure
Time frame: Baseline to 6 months
Withdrawal
Time frame: Baseline to 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2021.
- Individual participant dataSharing planned: Undecided
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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
Xuanwu Hospital, Beijing
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 10 inclusion and 9 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 24 Feb 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. (00-86-010-83198493, dayuwei1000@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of MG-specific Activities of Daily Living scale (MG-ADL) from Baseline (time frame: Baseline, 1 month, 3 months, 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 24 Feb 2021.