Myasthenia GravisPhase 4NCT06723548
Telitacicept and Low-dose Steroids in Refractory Myasthenia Gravis
A Phase 4 study testing Telitacicept in people with Myasthenia Gravis and Autoimmune Diseases, run by First Affiliated Hospital of Wenzhou Medical University.
Trial summary
This study is designed to explore the efficacy and safety of Telitacicept combined with low-dose steroids for the treatment of refractory MG, and to investigate related biomarkers such as immunoglobulins, BlyS/APRIL, and AChR-Ab titers, in order to clarify whether Telitacicept can rapidly and effectively help achieve… Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Myasthenia Gravis, Autoimmune Diseases
- What is being tested?
- Telitacicept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- Dec 2024to Dec 2026
- Phase
- Phase 4
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age between 18-85 years, both genders included；
- Meet the diagnostic criteria of the 2020 Chinese MG guidelines, with positive serological testing for AChR-Ab;
- Clinical classification of Type I to Type IVa according to the MGFA;
- Meet the criteria for refractory MG in the 2022 Japanese MG guidelines: poor response to standard treatment, intolerance to standard treatment drugs due to adverse reactions, frequent relapses/exacerbations after reduction of standard treatment drugs, frequent need for rescue treatment due to disease fluctuations, frequent myasthenic crises, and comorbidities that limit the use of standard treatment;
- Patients with unstable symptoms (MG-ADL score ≥6 or QMG ≥8) despite treatment with standard therapeutic regimens before enrollment, defined as follows:
- Patients on monotherapy with corticosteroids: a corticosteroid dose ≤60mg/d, and a stable dose for at least 1 month before enrollment;
- Patients on combination therapy with corticosteroids and other immunosuppressants: a corticosteroid dose ≤60mg/d, and a stable dose for at least 1 month before enrollment, while other immunosuppressants such as azathioprine, methotrexate, tacrolimus, and mycophenolate mofetil have been stable for 6 months prior to the study start and will remain stable during the study period;
- Patients must provide written informed consent.
Who cannot join
- Patients with active infections, such as herpes zoster, HIV, active pulmonary tuberculosis, or active hepatitis;
- Patients with thymic tumors or those who have undergone thymectomy within the past 6 months;
- Patients with coexisting malignant tumors;
- Patients with severe hepatic or renal insufficiency;
- Patients who have received intravenous immunoglobulin or undergone plasmapheresis within the last 2 months;
- Patients who have received any live vaccines within the last 3 months or plan to receive any vaccines during the study period;
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- Women who are currently pregnant or breastfeeding, and patients who plan to conceive during the trial period;
- Patients with allergies to human-derived biological products;
- Patients who have participated in any clinical trial within the last 28 days or within 5 half-lives of the study medication (whichever is longer);
- Any other patients deemed unsuitable for enrollment by the investigator (e.g., severe psychiatric disorders).
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: The Efficacy and Safety of Telitacicept Combined With Low-dose Steroids in Patients With Refractory Myasthenia Gravis
This study is designed to explore the efficacy and safety of Telitacicept combined with low-dose steroids for the treatment of refractory MG, and to investigate related biomarkers such as immunoglobulins, BlyS/APRIL, and AChR-Ab titers, in order to clarify whether Telitacicept can rapidly and effectively help achieve MG treatment goals and assist in steroid reduction.
- Conditions
- Myasthenia Gravis, Autoimmune Diseases
- Being tested
- Telitacicept
- Medical subject headings
- Myasthenia Gravis, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TelitaciceptDrug
Also called Pyridostigmine Bromide, NSISTs, prednisone
Patients with MG who fulfill the inclusion criteria will receive Telitacicept 240mg weekly as an adjunct to their medication. Upon reaching MMS, significant symptom improvement, or a QMG score reduction of at least 6 points, Telitacicept is reduced to biweekly doses. Pyridostigmine and NSISTs are tapered based on patient response. Following this, Prednisone is tapered, starting with rapid reduction early and slowing later. If a patient on 60mg Prednisone daily achieves treatment goals within 6-8 weeks of Telitacicept initiation, the tapering sequence is 60mg every other day for 4 weeks, then 30mg daily for 2-4 weeks, and so on, until reaching 5mg daily or 10mg every other day. At this point, Telitacicept may be reduced to 160mg biweekly.
| Study group | Role | What they receive |
|---|---|---|
| Patients with refractory MG treated with Telitacicept combined with low-dose steroids | Experimental | This is an open-label, single-arm exploratory study of Telitacicept (240mg weekly, then every two weeks after achieving MMS or QMG reduction of ≥ 6 points) combined with a gradual reduction of steroids and other immunosuppressants. When the steroid dose is reduced to 5mg/day or 10mg/every other day, Telitacicept can be reduced to 160mg, administered subcutaneously every two weeks. Receives: Drug: Telitacicept |
What is being measured
The primary outcome is the main question the study is designed to answer.
The change in patients' ADL scores
The variation in ADL scores at 52 weeks compared to baseline in patients. Total MG -ADL scores range from 0 (normal) to 24 (severe).
Measured: from baseline to week 52
The change in patients' QMG scores
The variation in QMG scores at 52 weeks compared to baseline in patients. Total QMG scores range from 0 (none) to 39 (severe).
Measured: from baseline to week 52
Secondary outcomes (10)
MGFA-PIS
Change in MGFA-PIS grading from baseline to week 52
Measured: from baseline to week 52
the average daily corticosteroid usage
Change in the average daily corticosteroid usage from baseline to week 24 during follow-up；Change in the average daily corticosteroid usage from baseline to week 52 during follow-up
Measured: from baseline to week 24；from baseline to week 52
the usage of traditional non-steroidal immunosuppressants
Change in the usage of traditional non-steroidal immunosuppressants from baseline to week 24 during follow-up; Change in the usage of traditional non-steroidal immunosuppressants from baseline to week 52 during follow-up. Medication usage logs or medical record reviews to document the changes in the use of traditional non-steroidal immunosuppressants (such as Azathioprine, Mycophenolate Mofetil, Tacrolimus, Methotrexate, Cyclosporine, etc.) from baseline to week 52. The change in the use of traditional non-steroidal immunosuppressants will be assessed by comparing the medication usage at baseline and at weeks 24 and 52. This includes details on the type of medication, dosage, frequency, and any adjustments or discontinuations.
Measured: from baseline at weeks 24 and 52
Number of relapses
Number of relapses in both groups by the end of treatment (clinical relapse is defined as an increase in QMG score of ≥3 points);
Measured: week 52
AUDTC
Area under the corticosteroid dose-time curve (AUDTC) at week 52
Measured: week 52
Proportion of patients with a corticosteroid dose ≤10mg/d
Proportion of patients with a corticosteroid dose ≤10mg/d at weeks 24 and 52 during follow-up;
Measured: at weeks 24 and 52
Proportion of patients achieving MMS with a corticosteroid dose ≤ 5mg/d
Proportion of patients achieving MMS with a corticosteroid dose ≤5mg/d at week 24;
Measured: week 24
MG QoL15r scores
Change in MG QoL15r scores from baseline at weeks 24 and 52 during follow-up
Measured: from baseline at weeks 24 and 52
safety（Incidence, severity, and outcome of adverse events）
Incidence, severity, and outcome of adverse events
Measured: 52 weeks
relevant biomarkers
relevant biomarkers, such as immunoglobulins,lymphocyte subpopulation changes, BlyS/APRIL, and AChR-Ab titers.
Measured: from baseline at week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2024
- Study startDec 2024 (estimated)
- Primary completionDec 2025 (estimated)
- Study completionDec 2026 (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.
- More trials run by First Affiliated Hospital of Wenzhou Medical 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06723548
- Sponsor's protocol ID
- 2024-ps-195
- Status last verified
- Nov 2024
- First submitted
- 15 Nov 2024
- Last updated
- 9 Dec 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy and Safety of Telitacicept Combined With Low-dose Steroids in Patients With Refractory Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT06723548
- Acronym / sponsor ID
- 2024-ps-195
- Conditions
- Myasthenia Gravis, Autoimmune Diseases
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Wenzhou Medical UniversityUniversity, hospital or other
- Study start
- Dec 2024 (estimated)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 9 Dec 2024 (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 explore the efficacy and safety of Telitacicept combined with low-dose steroids for the treatment of refractory MG, and to investigate related biomarkers such as immunoglobulins, BlyS/APRIL, and AChR-Ab titers, in order to clarify whether Telitacicept can rapidly and effectively help achieve MG treatment goals and assist in steroid reduction.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with refractory MG treated with Telitacicept combined with low-dose steroids | Experimental | Drug: TelitaciceptThis is an open-label, single-arm exploratory study of Telitacicept (240mg weekly, then every two weeks after achieving MMS or QMG reduction of ≥ 6 points) combined with a gradual reduction of steroids and other immunosuppressants. When the steroid dose is reduced to 5mg/day or 10mg/every other day, Telitacicept can be reduced to 160mg, administered subcutaneously every two weeks. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age between 18-85 years, both genders included；
- Meet the diagnostic criteria of the 2020 Chinese MG guidelines, with positive serological testing for AChR-Ab;
- Clinical classification of Type I to Type IVa according to the MGFA;
- Meet the criteria for refractory MG in the 2022 Japanese MG guidelines: poor response to standard treatment, intolerance to standard treatment drugs due to adverse reactions, frequent relapses/exacerbations after reduction of standard treatment drugs, frequent need for rescue treatment due to disease fluctuations, frequent myasthenic crises, and comorbidities that limit the use of standard treatment;
- Patients with unstable symptoms (MG-ADL score ≥6 or QMG ≥8) despite treatment with standard therapeutic regimens before enrollment, defined as follows:
Key exclusion criteria (10 listed)
- Patients with active infections, such as herpes zoster, HIV, active pulmonary tuberculosis, or active hepatitis;
- Patients with thymic tumors or those who have undergone thymectomy within the past 6 months;
- Patients with coexisting malignant tumors;
- Patients with severe hepatic or renal insufficiency;
- Patients who have received intravenous immunoglobulin or undergone plasmapheresis within the last 2 months;
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 |
|---|---|---|
| TelitaciceptAlso: Pyridostigmine Bromide, NSISTs, prednisonePatients with MG who fulfill the inclusion criteria will receive Telitacicept 240mg weekly as an adjunct to their medication. Upon reaching MMS, significant symptom improvement, or a QMG score reduction of at least 6 points, Telitacicept is reduced to biweekly doses. Pyridostigmine and NSISTs are tapered based on patient response. Following this, Prednisone is tapered, starting with rapid reduction early and slowing later. If a patient on 60mg Prednisone daily achieves treatment goals within 6-8 weeks of Telitacicept initiation, the tapering sequence is 60mg every other day for 4 weeks, then 30mg daily for 2-4 weeks, and so on, until reaching 5mg daily or 10mg every other day. At this point, Telitacicept may be reduced to 160mg biweekly. | Drug | Patients with refractory MG treated with Telitacicept combined with low-dose steroids |
Primary outcome
The change in patients' ADL scores
Time frame: from baseline to week 52
The variation in ADL scores at 52 weeks compared to baseline in patients. Total MG -ADL scores range from 0 (normal) to 24 (severe).
The change in patients' QMG scores
Time frame: from baseline to week 52
The variation in QMG scores at 52 weeks compared to baseline in patients. Total QMG scores range from 0 (none) to 39 (severe).
10 secondary outcomes
MGFA-PIS
Time frame: from baseline to week 52
the average daily corticosteroid usage
Time frame: from baseline to week 24；from baseline to week 52
the usage of traditional non-steroidal immunosuppressants
Time frame: from baseline at weeks 24 and 52
Number of relapses
Time frame: week 52
AUDTC
Time frame: week 52
Proportion of patients with a corticosteroid dose ≤10mg/d
Time frame: at weeks 24 and 52
Proportion of patients achieving MMS with a corticosteroid dose ≤ 5mg/d
Time frame: week 24
MG QoL15r scores
Time frame: from baseline at weeks 24 and 52
safety（Incidence, severity, and outcome of adverse events）
Time frame: 52 weeks
relevant biomarkers
Time frame: from baseline at week 52
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 9 Dec 2024.
- Individual participant dataSharing planned: No
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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 4 study testing Telitacicept in people with Myasthenia Gravis and Autoimmune Diseases, run by First Affiliated Hospital of Wenzhou Medical University.
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 8 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: opening soon, as of the sponsor's last update on 9 Dec 2024. 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 phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 Jia Li, Master's Degree (19016577562, lijia@wzhospital.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change in patients' ADL scores (time frame: from baseline to week 52). The study lists 2 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 9 Dec 2024.