Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis
- Conditions
- Generalized Myasthenia Gravis
- Interventions
- Biological: efgartigimod PH20 SC
- Registration Number
- NCT04818671
- Lead Sponsor
- argenx
- Brief Summary
The purpose of this study is to evaluate the long-term safety and tolerability of efgartigimod PH20 SC 1000 mg, and the clinical efficacy, PD, pharmacokinetics (PK), immunogenicity, impact on the quality of life (QoL) of the participants, treatment satisfaction, and administration method preference, and the feasibility of self- and caregiver-supported administration of the SC injection.
Treatment duration: 3-week treatment periods, repeated as needed with at least 28 days in between treatment periods
Health measurements: total levels of immunoglobulin G (IgG), Acetylcholine receptor binding autoantibodies (AChR-Ab) levels, Myasthenia Gravis Activities of Daly Living (MG-ADL).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 183
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Must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
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Previously participated in antecedent studies ARGX-113-2001 or ARGX-113-1705 and are eligible for roll over.
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Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:
- Women of Child bearing potential (WOCBP) must have a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered.
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The participant was discontinued early from studies ARGX-113-2001 or ARGX-113-1705, unless the reason for discontinuation from study ARGX-113-1705 was to roll over into study ARGX-113-2002.
a. Participants who, in the investigator's judgment, are not benefiting from efgartigimod IV in study ARGX-113-1705 Part B are not eligible for roll over into ARGX-113-2002.
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Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of investigational medicinal product (IMP)
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Has any of the following medical conditions:
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Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at roll-over
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Any other known autoimmune disease that, in the opinion of the investigator, would interfere with accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk
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History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence for ≥3 years before the first administration of investigational medicinal product (IMP).
Participants with the following cancers can be included at any time:
- adequately treated basal cell or squamous cell skin cancer
- carcinoma in situ of the cervix
- carcinoma in situ of the breast
- incidental histological findings of prostate cancer (TNM classification of malignant tumors stage T1a or T1b)
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Clinical evidence of other significant serious diseases, or the participant has had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the participant at undue risk
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Received a live-attenuated vaccine within 28 days prior to study entry or plan to receive a live-attenuated vaccine during the study
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A known hypersensitivity reaction to efgartigimod, rHuPH20, or any of its excipients
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description efgartigimod PH20 SC efgartigimod PH20 SC Patients receiving efgartigimod PH20 subcutaneous (SC) treatment
- Primary Outcome Measures
Name Time Method Incidence of Adverse Events of Special Interest (AESI) Up to 3.5 years Incidence and severity of Adverse Events (AEs) Up to 3.5 years Incidence of Serious Adverse Events (SAEs) Up to 3.5 years
- Secondary Outcome Measures
Name Time Method Percentage of caregivers who administered the injection to the participant at home over time by cycle Up to 3.5 years Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC Up to 3.5 years Myasthenia Gravis Activities of Daily Living (MG-ADL) total score changes from baseline Up to 3.5 years the higher the score, the more impairment
Percentage change in levels of total immunoglobulin G (IgG) from baseline Up to 3.5 years Cycle baseline over time by cycle (for total immunoglobulin G (IgG) Up to 3.5 years Percentage change of anti-acetylcholine receptor antibodies (AChR-Ab) from baseline Up to 3.5 years Cycle baseline over time by cycle (for MG-ADL) Up to 3.5 years Prevalence of neutralizing antibodies (NAbs) against efgartigimod over time Up to 3.5 years Number of participants who performed self-administration at home over time by cycle Up to 3.5 years Prevalence of ADAs to rHuPH20 over time Up to 3.5 years Cycle baseline over time by cycle in AChR-Ab seropositive participants (for acetylcholine receptor binding autoantibodies (AChR-Ab)) Up to 3.5 years Prevalence of anti-drug antibodies (ADAs) to efgartigimod over time Up to 3.5 years Incidence of neutralizing antibodies (NAbs) against efgartigimod over time Up to 3.5 years Incidence of ADAs to rHuPH20 over time Up to 3.5 years Incidence of NAbs against rHuPH20 over time Up to 3.5 years EQ-5D-5L responses over time by cycle Up to 3.5 years Percentage of self- or caregiver-supported study drug administration among all study treatment visits at home Up to 3.5 years Efgartigimod serum concentrations Up to 3.5 years Prevalence of NAbs against rHuPH20 over time Up to 3.5 years Number of self- or caregiver-supported study drug administration among all study treatment visits at home Up to 3.5 years Incidence of anti-drug antibodies (ADAs) to efgartigimod over time Up to 3.5 years Cycle baseline by cycle (for MG-QoL15r) Up to 3.5 years Changes in EuroQoL 5 Dimensions 5-Level (EQ-5D-5L) visual analog scale (VAS) score from baseline Up to 3.5 years Cycle baseline by cycle (for EQ-5D-5L) Up to 3.5 years Number of caregivers who administered the injection to the participant at home over time by cycle Up to 3.5 years Changes in total Myasthenia Gravis Quality of Life Questionnaire (15-item scale revised) (MG-QoL15r) from baseline Up to 3.5 years Percentage of participants who performed self-administration at home over time by cycle Up to 3.5 years
Trial Locations
- Locations (47)
Investigator Site 26 - DE490009
🇩🇪Münster, Germany
Investigator Site 1 - GEO9950001
🇬🇪Tbilisi, Georgia
Investigator Site 33 - GEO9950016
🇬🇪Tbilisi, Georgia
Investigator site 3 - GEO9950003
🇬🇪Tbilisi, Georgia
Investigator Site 28- JP0810059
🇯🇵Ota-Ku, Tokyo, Japan
Investigator site 2 - GEO9950002
🇬🇪Tbilisi, Georgia
Investigator Site 32 - GEO9950004
🇬🇪Tbilisi, Georgia
Investigator Site 45 - US0010108
🇺🇸Boca Raton, Florida, United States
Investigator site 4 - US0010110
🇺🇸Port Charlotte, Florida, United States
Investigator Site 44 - US0010077
🇺🇸Durham, North Carolina, United States
Investigator Site 42 - US0010019
🇺🇸Cleveland, Ohio, United States
Investigator Site 40 - US0010009
🇺🇸San Antonio, Texas, United States
Investigator site 6 - US0010032
🇺🇸Carlsbad, California, United States
Investigator Site 47 - US0010021
🇺🇸Palo Alto, California, United States
Investigator Site 41 - US0010015
🇺🇸Kansas City, Kansas, United States
Investigator Site 46 - US0010111
🇺🇸Amherst, New York, United States
Investigator Site 39 - US0010006
🇺🇸Tampa, Florida, United States
Investigator Site 38 - US0010003
🇺🇸Chapel Hill, North Carolina, United States
Investigator Site 43 - US0010066
🇺🇸Austin, Texas, United States
Investigator site 7 - US0010008
🇺🇸Cordova, Tennessee, United States
Investigator site 5 - BE0320007
🇧🇪Gent, Belgium
Investigator Site 25 - DE490006
🇩🇪Berlin, Germany
Investigator site 24 - CZ4200005
🇨🇿Brno, Czechia
Investigator site 10 - HU0360013
🇭🇺Budapest, Hungary
Investigator site 9 - HU0360012
🇭🇺Budapest, Hungary
Investigator Site 34 - IT0390007
🇮🇹Napoli, Italy
Investigator Site 35 - IT0390008
🇮🇹Roma, Italy
Investigator site 11 - IT0390003
🇮🇹Milano, Italy
Investigator site 12 - JP0810002
🇯🇵Chiba, Chiba-Shi, Japan
Investigator site 8 - JP0810004
🇯🇵Hanamaki, Iwate, Japan
Investigator Site 36 - JP0810055
🇯🇵Sapporo, Hokkaido, Japan
Investigator site 14 - JP0810007
🇯🇵Osaka, Japan
Investigator site 21 - PL0480022
🇵🇱Warsaw, Poland
Investigator Site 27 - JP0810008
🇯🇵Sapporo, Japan
Investigator site 15 - JP0810009
🇯🇵Tokyo, Japan
Investigator site 17 - PL0480001
🇵🇱Gdańsk, Poland
Investigator site 16 - NL0310001
🇳🇱Leiden, Netherlands
Investigator site 13 - JP0810005
🇯🇵Sendai-shi, Japan
Investigator site 22 - PL0480065
🇵🇱Kraków, Poland
Investigator site 18 - PL0480005
🇵🇱Kraków, Poland
Investigator site 20 - PL0480018
🇵🇱Lublin, Poland
Investigator Site 37 - ES0340021
🇪🇸Barcelona, Spain
Investigator Site 29- RU0070002
🇷🇺Novosibirsk, Russian Federation
Investigator Site 30 - RU0070014
🇷🇺Saint Petersburg, Russian Federation
Investigator Site 31 - ES0340038
🇪🇸Barcelona, Spain
Investigator site 23 - ES0340039
🇪🇸Valencia, Spain
Investigator site 19 - PL0480007
🇵🇱Katowice, Poland