Myasthenia Gravis, GeneralizedPhase 4OPTIMAGENCT07072988
Evaluate the Benefit of Corticoid Sparing in Elderly With Generalized AntiRAch Myasthenia Gravis Treated With IV or SC Efgartigimod
A Phase 4 study testing Efgartigimod in people with Myasthenia Gravis, Generalized, run by Centre Hospitalier Universitaire de Nice at 9 sites in France.
Trial summary
Generalized Myasthenia Gravis (gMG) is a rare chronic autoimmune disorder causing muscle weakness and fatigue, primarily due to autoantibodies that disrupt neuromuscular junction function. Read more about this study
- Who is it for?
- Ages 65 years and older
- Conditions the study is for
- Myasthenia Gravis, Generalized
- What is being tested?
- Efgartigimod
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 9 sitesFrance
- Study dates
- 19 Sep 2025to 1 Oct 2027
- 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 65 years and older
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Male or female subjects aged ≥65 years
- Diagnosed with gMG with confirmed documentation and supported by a physical exam and confirmed seropositivity for AChR-Abs.
- Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MFGA) for generalized MG class IIa, Iib, IIIa, IIIb.
- Patient suitable for efgartigimod treatment
- MG-ADL score ≥ 2
- Treated with high (>50mg/daily) or intermediate (20-50mg/daily) dose of oral corticosteroids (prednisone or prednisolone) and in stable dose of concomitant treatments for at least three months (changes of anticholinesterase therapy are permitted).
Show all 10Show fewer
- Patient that has developed at least one of the following iatrogenic comorbidities after the introduction of intermediate or high dose of oral corticosteroids: type 2 diabetes; arterial hypertension; overweight; sleep disorders; irritability and/or Patient experiencing a documented worsening of at least one of the following comorbidities after the introduction of intermediate or high dose of corticosteroids, including type 2 diabetes; arterial hypertension; overweight; sleep disorders; irritability
- Capable of understanding the written informed consent, and providing signed, dated, and witnessed written informed consent
- Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures
- Patient affiliated to a European social security system
Who cannot join
- Any current mental condition (psychiatric disorder, senility, or dementia) that, in the opinion of the investigator, may affect study compliance or prevent understanding of the aims, investigational procedures, or possible consequences of the study; and/or patient with a Mini-mental State Examination (MMSE) <28.
- History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
- Ongoing malignancy including solid tumors, hematologic malignancies and in situ carcinoma (except basal cell and squamous cell carcinomas of the skin, or in situ carcinoma of the cervix uteri that have been completely excised and cured)
- Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
- Vaccination with live or live-attenuated vaccines within the 6 weeks
- Patient already treated with anti-complement (wash-out period 3 months) or anti-FcRN (wash-out period 3 months), anti-CD20 (wash-out period 6 months) and PLEX (wash-out period 1 month)
- Patient with end-stage disease or with a disease that will enable him to be evaluated and/or treated
- Patient protected by law, under guardianship or curator ship, or not able to participate in a clinical study according to the article L.1121-16 of the French Public Health Code.
Trial locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
9 sites in 1 country
France9 sites, 9 recruiting
- Recruiting
Hôpital Henri Mondor
Créteil, 94000
Edoardo MALFATI, PU-PH, +33149812111, malfati.e@aphp.fr
- Not yet recruiting
Hôpital Raymond Pointcarré
Garches, 92380
Djillali ANNANE, PU-PH, +33147107787, djillali.annane@aphp.fr
- Recruiting
CHU de Grenoble
Grenoble, 38700
Emmeline LAGRANGE, PH, elagrange@chu-grenoble.fr
- Recruiting
Hôpital Roger Salengro
Lille, 59037
Céline TARD, PH, +00320444145, celine.tard@chu-lille.fr
- Recruiting
CHU de la Timone
Marseille, 13385
Shahram ATTARIAN, PU-PH, shahram.attarian@ap-hm.fr
- Recruiting
CHRU de Nancy
Nancy, 54000
Maud Michaud, ph, +33383851688, m.michaud@chru-nancy.fr
- Recruiting
CHU de Nice
Nice, Alpes-Maritimes, 06000
Sabrina SACCONI, pu-ph, +33 (4) 92 03 57 57
Sabrina SACCONI, pu-ph, +33 (4) 92 03 57 57, sacconi.s@chu-nice.fr
- Recruiting
CHU Charles Nicolle
Rouen, 76031
Linda Zourdani, PH, linda.zourdani@chu-rouen.fr
- Recruiting
CHU de Toulouse
Toulouse, 31059
Blandine ACKET, PH, +33561779989, acket.b@chu-toulouse.fr
About this study
Official title: A Multicenter, Prospective, Observational, Open-label Study to Evaluate the Benefit of Corticoid Sparing in Elderly With Generalized AntiRAch Myasthenia Gravis Treated With IV or SC Efgartigimod
Generalized Myasthenia Gravis (gMG) is a rare chronic autoimmune disorder causing muscle weakness and fatigue, primarily due to autoantibodies that disrupt neuromuscular junction function. The most common antibodies target nicotinic acetylcholine receptors (AChR), with others such as anti-MuSK and anti-LRP4 being less prevalent. The conventional gMG treatments include acetylcholinesterase inhibitors, corticosteroids, immunosuppressant and, in case of myasthenic crisis, plasma exchange (PLEX) and intravenous immunoglobulins (IVIG). Treatment aims to achieve minimal manifestation status (MMS), but many patients face persistent symptoms or side effects. Corticosteroids, while effective, carry significant risks, especially for long-term use, such as, increased infection and cardiovascular risks, chronic conditions like hypertension, diabetes, and osteoporosis and quality of life impacts, including weight gain and mood changes. Elderly patients, who form the majority of the gMG population, are particularly vulnerable due to age-related comorbidities, which limit treatment options and prolong corticosteroid reliance. This contributes to increased mortality, disability, and dependency. Efgartigimod (EFG), a novel therapeutic targeting the neonatal Fc receptor (FcRn), accelerates degradation of pathogenic IgG antibodies, including anti-AChR. Clinical trials demonstrated its efficacy and safety in reducing antibody levels, improving muscle strength, and enhancing quality of life. Both intravenous (IV) and subcutaneous (SC) forms are effective and well tolerated. Approved in the United States and subsequently in Japan and Europe, EFG became available in France in 2023. The present multicenter observational study aims to evaluate the real-life impact of EFG in elderly gMG patients struggling with corticosteroid side effects or comorbidity exacerbations. The objectives of this study include the assessing EFG's ability to enable corticosteroid reduction and monitoring improvements in gMG symptoms, quality of life, comorbidities, and overall health.
This approach highlights a shift towards targeted therapies that balance efficacy with reduced treatment-related burdens for vulnerable gMG populations.
- Conditions
- Myasthenia Gravis, Generalized
- Being tested
- Efgartigimod
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EfgartigimodDrug
Administration of efgartigimod, a neonatal Fc receptor (FcRn) antagonist, given either intravenously (IV) or subcutaneously (SC) to treat patients with generalized Anti-AChR Myasthenia Gravis.
| Study group | Role | What they receive |
|---|---|---|
| Elderly with Generalized Anti-AChR Myasthenia Gravis Treated with IV or SC Efgartigimod | Experimental | Not described Receives: Drug: Efgartigimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
evaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG
evaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG
Measured: 12 mois
Secondary outcomes (4)
change in percentage of patients in low dose of corticosteroid (<20mg) at 12 months
change from baseline of the percentage of patient treated with <20 mg of corticosteroids
Measured: 12 months
change in percentage of patients in intermediate dose of corticosteroid (≥20 mg - ≤50 mg) at 12 months
change from baseline of the percentage of patient treated with ≥20 mg - ≤50 mg of corticosteroids
Measured: 12 months
change in percentage of patients in high dose of corticosteroid (≥ 50mg) at 12 months
change from baseline of the percentage of patient treated with >50 mg of corticosteroids
Measured: 12 months
change in Glucocorticoid Toxicity Index (GTI) at 12 months
change from baseline in GTI
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2025
- Study start19 Sep 2025
- Primary completion1 Oct 2027 (estimated)
- Study completion1 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia Gravis, GeneralizedStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07072988
- Sponsor's protocol ID
- 25-PP-08
- Acronym
- OPTIMAGE
- Status last verified
- Sep 2026
- First submitted
- 9 Jul 2025
- Last updated
- 3 Sep 2026
- 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 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Prospective, Observational, Open-label Study to Evaluate the Benefit of Corticoid Sparing in Elderly With Generalized AntiRAch Myasthenia Gravis Treated With IV or SC Efgartigimod
- ClinicalTrials.gov ID
- NCT07072988
- Acronym / sponsor ID
- OPTIMAGE / 25-PP-08
- Conditions
- Myasthenia Gravis, Generalized
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- 19 Sep 2025 (actual)
- Primary completion
- 1 Oct 2027 (estimated)
- Study completion
- 1 Oct 2027 (estimated)
- First posted
- 18 Jul 2025 (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.
Generalized Myasthenia Gravis (gMG) is a rare chronic autoimmune disorder causing muscle weakness and fatigue, primarily due to autoantibodies that disrupt neuromuscular junction function. The most common antibodies target nicotinic acetylcholine receptors (AChR), with others such as anti-MuSK and anti-LRP4 being less prevalent. The conventional gMG treatments include acetylcholinesterase inhibitors, corticosteroids, immunosuppressant and, in case of myasthenic crisis, plasma exchange (PLEX) and intravenous immunoglobulins (IVIG). Treatment aims to achieve minimal manifestation status (MMS), but many patients face persistent symptoms or side effects. Corticosteroids, while effective, carry significant risks, especially for long-term use, such as, increased infection and cardiovascular risks, chronic conditions like hypertension, diabetes, and osteoporosis and quality of life impacts, including weight gain and mood changes. Elderly patients, who form the majority of the gMG population, are particularly vulnerable due to age-related comorbidities, which limit treatment options and prolong corticosteroid reliance. This contributes to increased mortality, disability, and dependency. Efgartigimod (EFG), a novel therapeutic targeting the neonatal Fc receptor (FcRn), accelerates degradation of pathogenic IgG antibodies, including anti-AChR. Clinical trials demonstrated its efficacy and safety in reducing antibody levels, improving muscle strength, and enhancing quality of life. Both intravenous (IV) and subcutaneous (SC) forms are effective and well tolerated. Approved in the United States and subsequently in Japan and Europe, EFG became available in France in 2023. The present multicenter observational study aims to evaluate the real-life impact of EFG in elderly gMG patients struggling with corticosteroid side effects or comorbidity exacerbations. The objectives of this study include the assessing EFG's ability to enable corticosteroid reduction and monitoring improvements in gMG symptoms, quality of life, comorbidities, and overall health.
This approach highlights a shift towards targeted therapies that balance efficacy with reduced treatment-related burdens for vulnerable gMG populations.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Elderly with Generalized Anti-AChR Myasthenia Gravis Treated with IV or SC Efgartigimod | Experimental | Drug: Efgartigimod |
Who may qualify
- Ages
- 65 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (10 listed)
- Male or female subjects aged ≥65 years
- Diagnosed with gMG with confirmed documentation and supported by a physical exam and confirmed seropositivity for AChR-Abs.
- Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MFGA) for generalized MG class IIa, Iib, IIIa, IIIb.
- Patient suitable for efgartigimod treatment
- MG-ADL score ≥ 2
Key exclusion criteria (8 listed)
- Any current mental condition (psychiatric disorder, senility, or dementia) that, in the opinion of the investigator, may affect study compliance or prevent understanding of the aims, investigational procedures, or possible consequences of the study; and/or patient with a Mini-mental State Examination (MMSE) <28.
- History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
- Ongoing malignancy including solid tumors, hematologic malignancies and in situ carcinoma (except basal cell and squamous cell carcinomas of the skin, or in situ carcinoma of the cervix uteri that have been completely excised and cured)
- Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
- Vaccination with live or live-attenuated vaccines within the 6 weeks
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 |
|---|---|---|
| EfgartigimodAdministration of efgartigimod, a neonatal Fc receptor (FcRn) antagonist, given either intravenously (IV) or subcutaneously (SC) to treat patients with generalized Anti-AChR Myasthenia Gravis. | Drug | Elderly with Generalized Anti-AChR Myasthenia Gravis Treated with IV or SC Efgartigimod |
Primary outcome
evaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG
Time frame: 12 mois
evaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG
4 secondary outcomes
change in percentage of patients in low dose of corticosteroid (<20mg) at 12 months
Time frame: 12 months
change in percentage of patients in intermediate dose of corticosteroid (≥20 mg - ≤50 mg) at 12 months
Time frame: 12 months
change in percentage of patients in high dose of corticosteroid (≥ 50mg) at 12 months
Time frame: 12 months
change in Glucocorticoid Toxicity Index (GTI) at 12 months
Time frame: 12 months
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 3 Sep 2026.
Related trials for Myasthenia Gravis, Generalized
Active, not recruitingPhase 3
An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Recruiting
Holistic Assessment and Remote Digital MONitoring of mYasthenia Gravis Via MyoSense 360
Myasthenia Gravis, Generalized
RecruitingPhase 3
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
Myasthenia Gravis
RecruitingPhase 2/3
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Not yet recruitingEarly Phase 1
A Clinical Study to Evaluate the Safety and Efficacy of GT802 Injection in the Treatment of Refractory Generalized Myasthenia Gravis
Generalized Myasthenia Gravis (gMG)
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.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Efgartigimod in people with Myasthenia Gravis, Generalized, run by Centre Hospitalier Universitaire de Nice at 9 sites in France.
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 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: looking for participants now, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in France. 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 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 Sabrina SACCONI, pu-ph (+33 (4) 92 03 57 57, sacconi.s@chu-nice.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
evaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG (time frame: 12 mois). 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 3 Sep 2026.