Myasthenia Gravis, OcularPhase 3ADAPT oculusNCT06558279
A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis
A Phase 3 study testing Efgartigimod PH20 SC in people with Myasthenia Gravis, Ocular, run by argenx at 102 sites in 25 countries.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis, Ocular
- What is being tested?
- Efgartigimod PH20 SC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 141 participants
- Where
- 102 sitesin 25 countries
- Study dates
- 18 Sep 2024to Dec 2027
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Is at least 18 years of age and the local legal age of consent for clinical studies
- Has been diagnosed with myasthenia gravis and supported by seropositivity for AChR-Ab; or abnormal neuromuscular transmission demonstrated by abnormal neurophysiology testing and history on positive edrophonium chloride testing or demonstrated improvement on MG therapy"
- Is MGFA Class I (any ocular muscle weakness)
- Has a screening and baseline MGII (PRO) ocular score of at least 6 with at least 2 ocular items with a score of at least 2
Who cannot join
- Other diseases that lead to eyelid drooping, peripheral muscle weakness, or diplopia
- Known autoimmune disease or any medical condition other than indication under study that would interfere with an accurate assessment of clinical symptoms of ocular myasthenia gravis or puts the participant at undue risk
Trial locations and contacts
102 sites in 25 countries.
102 sites in 25 countries
United States20 sites
National Neuromuscular Research Institute
Austin, Texas, 78759-8402
Neurology Offices of South Florida
Boca Raton, Florida, 33428-4221
SFM Clinical Research, LLC
Boca Raton, Florida, 33487-2768
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514
Northwestern Memorial Hospital
Chicago, Illinois, 60611
Cleveland Clinic
Cleveland, Ohio, 44195
University of California Irvine
Columbia, Maryland, 21044
Martha Morehouse Medical plaza
Columbus, Ohio, 43221
Henry Ford Health System
Detroit, Michigan, 48202
Duke Neurological Disorders Clinic
Durham, North Carolina, 27710
Houston Methodist Neurological Institute
Houston, Texas, 77030
Loma Linda University Health
Loma Linda, California, 92354
USC Roski Eye Institute - Los Angeles
Los Angeles, California, 90033
Neurology Associates PA
Maitland, Florida, 32751
Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
Penn Medicine University City
Philadelphia, Pennsylvania, 19104
University of California San Francisco
San Francisco, California, 94143
HonorHealth Neurology - Bob Bové Neuroscience Institute
Scottsdale, Arizona, 85251
University of South Florida
Tampa, Florida, 33620
China12 sites
Xiangya Hospital Central South University
Changsha, 410008
Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital
Chengdu, 610072
The First Affiliated Hospital of Chongqing Medical University
Chongqing, 400016
Fujian Medical University Union Hospital
Fuzhou, 350001
Guangdong Province Traditional Chinese Medical Hospital
Guangzhou, 510120
The First Affiliated Hospital of Guangzhou University Chinese Medicine
Guangzhou, 510405
Qilu Hospital of Shandong University
Jinan, 250012
The First Affiliated Hospital of Nanchang University - Xianghu Campus
Nanchang, 330000
Huashan Hospital Fudan University
Shanghai, 200040
Shijiazhuang People's Hospital - Fangbeilu Campus
Shijiazhuang, 050011
Tongji Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan, 430030
Renmin Hospital of Wuhan University - Main Campus
Wuhan, 430060
Japan11 sites
Chiba University Hospital
Chūōku, 260-8677
Kyushu University Hospital
Fukuoka, 812-8582
General Hanamaki Hospital
Hanamaki-shi, 025-0075
Hiroshima University Hospital
Hiroshima, 734-8551
Saitama Medical Center
Kawagoe, 350-0844
National Hospital Organization Utano National Hospital
Kyoto, 616-8255
IUHW Narita Hospital
Narita, 286-8520
National Hospital Organization Hokkaido Medical Center
Sapporo, 063-0005
Kindai University Hospital
Sayama, 589-8511
Tokyo Medical University Hospital
Shinjuku-Ku, 160-0023
Osaka University Hospital
Suita-Shi, 565-0871
Canada6 sites
Maritime Neurology
Halifax, B3R 1V9
London Health Sciences Centre
London, N6A 5A5
Centre Hospitalier de l'Universite de Montreal
Montreal, H2X 0A9
Ottawa Hospital Research Institute
Ottawa, K1Y 4E9
Hopital de L'enfant Jesus
Québec, G1J 1Z4
Toronto General Hospital
Toronto, M5G 2C4
Italy6 sites
ASST Papa Giovanni XXIII - Ospedale Papa Giovanni XXIII
Bergamo, 24127
IRCCS Istituto dell Scienze Neurologiche di Bologna
Bologna, 40139
Ospedale San Raffaele S.r.l.
Milan, 20132
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
AORN Antonio Cardarelli
Naples, 80131
Azienda Ospedaliero-Universitaria Sant'Andrea
Roma, 00189
Spain6 sites
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital del Mar
Barcelona, 8003
Hospital Clinico San Carlos
Madrid, 28040
Hospital Universitario Ramon y Cajal
Madrid, 28304
Hospital Universitario de Donostia
San Sebastián, 20014
Hospital Clinico Universitario de Valencia
Valencia, 46010
Poland5 sites
MICS Centrum Medyczne Bydgoszcz
Bydgoszcz, 85-065
Centrum Medyczne Neurologia Slaska
Katowice, 40-689
SP ZOZ Szpital Uniwersytecki
Krakow, 31-503
Krakowska Akademia Neurologii
Krakow, 31-505
CLINIREM Sp z o.o.
Lublin, 20-064
United Kingdom5 sites
Royal Sussex County Hospital
Brighton, BN2 5BE
Panthera Biopartners - Glasgow
Glasgow, G51 4TY
Leeds General Infirmary
Leeds, LS1 3EX
Dementech Neurosciences Clinical Academic Centre
London, W1G 9ST
Southampton General Hospital
Southampton, SO16 6YD
Georgia4 sites
Petre Sarajishvili Institute of Neurology
Tbilisi, 112
New Hospitals
Tbilisi, 114
Pineo Medical Ecosystem
Tbilisi, 114
LLC Caucasus Medical Centre
Tbilisi, 186
Australia3 sites
Box Hill Hospital
Box Hill, 3128
The Alfred Hospital
Melbourne, 3004
Gold Coast University Hospital
Southport, 4215
South Korea3 sites
Kyungpook National University Chilgok hospital
Daegu, 41404
Korea University Anam Hospital
Seoul, 2841
Severance Hospital Yonsei University Health System
Seoul, 3722
Czechia2 sites
Fakultni nemocnice Brno
Brno, 625 00
Nemocnice Pardubickeho kraje, a.s., Pardubicka nemocnice
Pardubice, 532 03
Denmark2 sites
Aarhus Universitetshospital
Aarhus N, 8200
Rigshospitalet
Copenhagen, 2100
Germany2 sites
Charité - Universitätsmedizin Berlin
Berlin, 13353
Universität Georg August
Göttingen, 37075
Greece2 sites
Eginitio Hospital
Athens, 115 28
University General Hospital of Patras
Pátrai, 265 04
Portugal2 sites
ULS de Santa Maria, EPE - Hospital de Santa Maria
Lisbon, 1649-035
ULS de Santo António, EPE - Hospital de Santo António
Porto, 4099-001
Serbia2 sites
University Clinical Center of Serbia
Belgrade, 11000
University Clinical Center Nis
Niš, 18 000
About this study
Official title: A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Ocular Myasthenia Gravis
The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.
- Conditions
- Myasthenia Gravis, Ocular
- Being tested
- Efgartigimod PH20 SC, Placebo PH20 SC
- Keywords
- Myasthenia Gravis, Ocular myasthenia
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efgartigimod PH20 SCCombination product
Subcutaneous efgartigimod PH20 SC given by prefilled syringe
Placebo PH20 SCOther
Subcutaneous placebo PH20 SC given by prefilled syringe
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod PH20 SC in part A+B | Experimental | Participants receiving efgartigimod PH20 SC during part A and part B Receives: Combination Product: Efgartigimod PH20 SC |
| Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B | Experimental | Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B Receives: Combination Product: Efgartigimod PH20 SC; Other: Placebo PH20 SC |
What is being measured
The primary outcome is the main question the study is designed to answer.
MGII (PRO) ocular score change from baseline to day 29 in part A
The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The higher the score, the more severe the disease.
Measured: Up to 29 days
Secondary outcomes (13)
MGII (PRO+PE) ocular score change from baseline to day 29 in part A
The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The ocular PE score varies between 0 and 5. The higher the score, the more severe the disease.
Measured: Up to 29 days
MG-ADL ocular domain score change from baseline to day 29 in part A
The Myasthenia Gravis Activities of Daily Living (MG-ADL) scale assesses MG symptoms and their effects on daily activities. The ocular score varies between 0 and 16 (with 16 the most severe).
Measured: Up to 29 days
MGII total score change from baseline to day 29 in part A
The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The score varies between 0 and 84. The higher the score, the more severe the disease.
Measured: Up to 29 days
MGII (PE) ocular score change from baseline to day 29 in part A
The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 6 physical examinations (PE). The score varies between 0 and 5. The higher the score, the more severe the disease.
Measured: Up to 29 days
MG-ADL total score change from baseline to day 29 in part A
The Myasthenia Gravis Activities of Daily Living (MG-ADL) scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe).
Measured: Up to 29 days
MGII ocular scores changes (PRO) from baseline in part A+B
The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The ocular PE score varies between 0 and 5. The higher the score, the more severe the disease.
Measured: Up to 111 weeks
Incidence of (serious) adverse events
Measured: Up to 111 weeks
MG-QoL15r total score changes from baseline in part A+B
The Myasthenia Gravis Quality of Life 15-item scale revised (MG-QoL15r) questionnaire is a patient-reported instrument that measures the impact of MG symptoms on Quality of Life. The score varies between 0 and 30 (with 30 the lowest quality of life).
Measured: Up to 111 weeks
NEI VFQ-25 score changes from baseline in part A+B
The 25-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) is a 25-item vision-targeted questionnaire. The score varies between 0 and 100 (with 0 being the worst score)
Measured: Up to 111 weeks
Percent change from baseline in total IgG levels over time in part A+B
Measured: Up to 111 weeks
Percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A+B
Measured: Up to 111 weeks
Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B
Measured: Up to 111 weeks
Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B
Measured: Up to 111 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2024
- Study start18 Sep 2024
- Primary completion12 Jan 2026
- Study completionDec 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, OcularStudies of the same condition, open and under way.
- More trials run by argenxThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 141 participants (actual)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06558279
- Sponsor's protocol ID
- ARGX-113-2315
- Other IDs
- 2024-514133-38-00
- Acronym
- ADAPT oculus
- Status last verified
- Jul 2026
- First submitted
- 14 Aug 2024
- Last updated
- 2 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Ocular Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT06558279
- Acronym / sponsor ID
- ADAPT oculus / ARGX-113-2315
- Conditions
- Myasthenia Gravis, Ocular
- Keywords
- Myasthenia Gravis, Ocular myasthenia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 141 participants (actual)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Study start
- 18 Sep 2024 (actual)
- Primary completion
- 12 Jan 2026 (actual)
- Study completion
- Dec 2027 (estimated)
- First posted
- 16 Aug 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.
The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Efgartigimod PH20 SC in part A+B | Experimental | Combination Product: Efgartigimod PH20 SCParticipants receiving efgartigimod PH20 SC during part A and part B |
| Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B | Experimental | Combination Product: Efgartigimod PH20 SC, Other: Placebo PH20 SCParticipants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Is at least 18 years of age and the local legal age of consent for clinical studies
- Has been diagnosed with myasthenia gravis and supported by seropositivity for AChR-Ab; or abnormal neuromuscular transmission demonstrated by abnormal neurophysiology testing and history on positive edrophonium chloride testing or demonstrated improvement on MG therapy"
- Is MGFA Class I (any ocular muscle weakness)
- Has a screening and baseline MGII (PRO) ocular score of at least 6 with at least 2 ocular items with a score of at least 2
Key exclusion criteria (2 listed)
- Other diseases that lead to eyelid drooping, peripheral muscle weakness, or diplopia
- Known autoimmune disease or any medical condition other than indication under study that would interfere with an accurate assessment of clinical symptoms of ocular myasthenia gravis or puts the participant at undue risk
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 |
|---|---|---|
| Efgartigimod PH20 SCSubcutaneous efgartigimod PH20 SC given by prefilled syringe | Combination product | Efgartigimod PH20 SC in part A+B, Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B |
| Placebo PH20 SCSubcutaneous placebo PH20 SC given by prefilled syringe | Other | Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B |
Primary outcome
MGII (PRO) ocular score change from baseline to day 29 in part A
Time frame: Up to 29 days
The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The higher the score, the more severe the disease.
13 secondary outcomes
MGII (PRO+PE) ocular score change from baseline to day 29 in part A
Time frame: Up to 29 days
MG-ADL ocular domain score change from baseline to day 29 in part A
Time frame: Up to 29 days
MGII total score change from baseline to day 29 in part A
Time frame: Up to 29 days
MGII (PE) ocular score change from baseline to day 29 in part A
Time frame: Up to 29 days
MG-ADL total score change from baseline to day 29 in part A
Time frame: Up to 29 days
MGII ocular scores changes (PRO) from baseline in part A+B
Time frame: Up to 111 weeks
Incidence of (serious) adverse events
Time frame: Up to 111 weeks
MG-QoL15r total score changes from baseline in part A+B
Time frame: Up to 111 weeks
NEI VFQ-25 score changes from baseline in part A+B
Time frame: Up to 111 weeks
Percent change from baseline in total IgG levels over time in part A+B
Time frame: Up to 111 weeks
Percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A+B
Time frame: Up to 111 weeks
Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B
Time frame: Up to 111 weeks
Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B
Time frame: Up to 111 weeks
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 2 Jul 2026.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
102 sites in 25 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Efgartigimod PH20 SC in people with Myasthenia Gravis, Ocular, run by argenx at 102 sites in 25 countries.
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 4 inclusion and 2 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: under way, not looking for participants, as of the sponsor's last update on 2 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 102 sites in United States, China, Japan, Canada, Italy and 20 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
MGII (PRO) ocular score change from baseline to day 29 in part A (time frame: Up to 29 days). 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 2 Jul 2026.