efgartigimod alfa for Generalized myasthenia gravis

Inclusion criteria

• {"criterion_text":"- Adult patients ≥ 18 years of age, of both sexes."}
• {"criterion_text":"- Confirmed diagnosis of myasthenia gravis by a neurologist with experience in the disease [clinical symptoms suggestive of MG and positive anti-AChR antibodies and/or an electrophysiological study suggestive of neuromuscular junction disorder]"}
• {"criterion_text":"- Myasthenia Gravis Foundation of America (MGFA) Class II, III, or IV at the time of the screening visit."}
• {"criterion_text":"- Clinical presentation with 5 or more points (more than 50% of the points for non-ocular symptoms) on the MG-ADL scale."}
• {"criterion_text":"- Not having received prior immunosuppressive treatment for MG with the exception of corticosteroids that will be started during the study(naïve patients)"}
• {"criterion_text":"- Women of childbearing potential must have a negative serum pregnancy test at screening, be required to use contraception during the study and for 90 days after the last dose of drug, and agree not to donate eggs during the same period."}
• {"criterion_text":"- Men, agree to use adequate contraception and not donate sperm until the end of the study (and/or until 90 days after the last drug infusion)."}
• {"criterion_text":"- Signed Informed Consent Form."}

Exclusion criteria

• {"criterion_text":"- MGFA Class I or V."}
• {"criterion_text":"- Active hepatitis B, or positivity for hepatitis C (unless treated and cured) and/or HIV at screening."}
• {"criterion_text":"- Known autoimmune or non-autoimmune disease that, in the opinion of the treating physician, would interfere with an accurate evaluation of the clinical symptoms of MG or place the patient at undue risk."}
• {"criterion_text":"- History of malignancy, unless considered cured with adequate treatment and no evidence of recurrence for ≥ 3 years. (Patients in whom adequate treatment of basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, carcinoma in situ of the breast, or incidental histological finding of prostate cancer -stage can be included at any time). TNM T1a or T1b-)."}
• {"criterion_text":"- Have received a live or attenuated vaccine during the month prior to the screening visit."}
• {"criterion_text":"- Be pregnant and/or breast-feeding or intend to become pregnant during the study or within 90 days of the final dose of efgartigimod."}
• {"criterion_text":"- Inability to understand informed consent"}
• {"criterion_text":"- Presence of symptoms that may endanger the patient's life if immediate rescue treatment is not instituted (intravenous immunoglobulins -I VIG- or plasma exchange/plasmapheresis), according to the treating physician's criteria."}
• {"criterion_text":"- Having received any immunosuppressive treatment for MG previously."}
• {"criterion_text":"- Treatment with IVIG or plasmapheresis within 4 weeks prior to the screening visit."}
• {"criterion_text":"- Treatment with rituximab or eculizumab within 6 months prior to the screening visit."}
• {"criterion_text":"- Treatment with any monoclonal antibody at the time of the screening visit."}
• {"criterion_text":"- History of thymectomy prior to the screening visit or having a thymectomy scheduled during the planned weeks of the study."}
• {"criterion_text":"- Have any surgical intervention scheduled during the planned weeks of the study."}
• {"criterion_text":"- Active or chronic uncontrolled and clinically significant bacterial, viral or fungal infection at the time of the screening visit."}

Primary endpoints

• {"endpoint_text":"- Proportion of patients with MGg and anti-AChR antibody responders in MG-ADL after a full course of efgartigimod in combination with the usual prednisone initiation regimen.","definition_or_measurement_approach":"Responder status measured using the MG-ADL (activities of daily living in myasthenia gravis) scale after a full course of efgartigimod combined with the usual prednisone initiation regimen."}

Secondary endpoints

• {"endpoint_text":"- Total score (per patient) and mean total score on the MG-ADL and mean change in the total score on the MG-ADL at weeks 4, 6, 8, 16 and 28, compared to the baseline score.","definition_or_measurement_approach":"MG-ADL total score and mean change at specified weeks vs baseline."}
• {"endpoint_text":"- Total score (per patient) and mean total QMG score and mean change in total QMG at weeks 4, 6, 8, 16 and 28, compared to baseline score.","definition_or_measurement_approach":"Quantitative Myasthenia Gravis (QMG) total score and mean change at specified weeks vs baseline."}
• {"endpoint_text":"- Proportion of responding patients in the QMG.","definition_or_measurement_approach":"Responder proportion defined by improvement on QMG scale (as per protocol-defined responder criteria)."}
• {"endpoint_text":"- Total score (per patient) and mean total MGC score and mean change in total MGC at weeks 4, 6, 8, 16 and 28, compared to baseline score.","definition_or_measurement_approach":"Myasthenia Gravis Composite (MGC) total score and mean change at specified weeks vs baseline."}
• {"endpoint_text":"- Proportion of responding patients in the MGC.","definition_or_measurement_approach":"Responder proportion based on MGC improvement."}
• {"endpoint_text":"- Median time to onset of efficacy of efgartigimod, determined by when an improvement of 2 or more points on the MG-ADL scale is achieved.","definition_or_measurement_approach":"Time-to-event (median time) until ≥2-point improvement on MG-ADL."}
• {"endpoint_text":"- Median time of onset of efficacy of efgartigimod, determined by the time at which an improvement of 3 or more points on the QMG scale is achieved.","definition_or_measurement_approach":"Time-to-event (median time) until ≥3-point improvement on QMG."}
• {"endpoint_text":"- Median time of onset of efficacy of efgartigimod, determined by the time at which an improvement of 3 or more points on the MGC scale is achieved.","definition_or_measurement_approach":"Time-to-event (median time) until ≥3-point improvement on MGC."}
• {"endpoint_text":"- Proportion of early responders on the MG-ADL scale.","definition_or_measurement_approach":"Proportion achieving early response on MG-ADL (protocol-defined timing)."}
• {"endpoint_text":"- Total score (per patient) and mean total score and mean change in total scores in the MG-QOL15r and in the EQ-5D-5L at weeks 4, 6, 8, 16 and 28, compared to the baseline score.","definition_or_measurement_approach":"Patient-reported outcomes: MG-QOL15r and EQ-5D-5L scores and changes at specified weeks vs baseline."}
• {"endpoint_text":"- Dose of steroids (prednisone) that patients are taking at 4, 6, 8, 16, and 28 weeks after starting treatment (day of first infusion of efgartigimod cycle/baseline visit).","definition_or_measurement_approach":"Recorded prednisone dose at specified visits (mg)."}
• {"endpoint_text":"- Global levels of IgG, and of the different IgG subclasses, at the screening visit and at weeks 2, 4, 8, 16 and 28 after starting treatment (day of the first infusion of the efgartigimod cycle/baseline visit ).","definition_or_measurement_approach":"Laboratory measurement of total IgG and IgG subclasses at screening and specified weeks."}
• {"endpoint_text":"- Levels of anti-AChR antibody titers at the screening visit and at weeks 2, 4, 6, 8, 16 and 28 after starting treatment (day of the first infusion of the efgartigimod cycle/baseline visit).","definition_or_measurement_approach":"Laboratory measurement of anti-AChR antibody titers at screening and specified weeks."}
• {"endpoint_text":"- Number and percentage of patients receiving more than one cycle of efgartigimod.","definition_or_measurement_approach":"Count and proportion of patients receiving >1 treatment cycle."}
• {"endpoint_text":"- Number and percentage of patients receiving rescue treatment with IVIG and/or plasmapheresis.","definition_or_measurement_approach":"Count and proportion receiving rescue IVIG and/or plasmapheresis."}
• {"endpoint_text":"- Number and percentage of patients requiring hospital admission (with description of the causes with number and percentage of them).","definition_or_measurement_approach":"Count and proportion of hospital admissions with cause descriptions."}
• {"endpoint_text":"- Number, percentage, description and severity of adverse events.","definition_or_measurement_approach":"Safety reporting: count, percent, description and severity grading of adverse events."}

Spain

Earliest CTIS Part Ii Submission Date

31-10-2024

Latest Decision Or Authorization Date

05-12-2024

Processing Time Days

35

Number Of Sites

1

Number Of Participants

20

Primary sponsor

Full Name

Bellvitge University Hospital

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Spain

Co-sponsors

• Fundacio Institut D'Investigacio Biomedica De Bellvitge IDIBELL