Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT06558279

A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults 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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Myasthenia Gravis, Ocular Autoimmune Diseases Autoimmune Diseases of the Nervous System Immune System Diseases Myasthenia Gravis Neoplasms Neoplasms by Site Nervous System Diseases Nervous System Neoplasms Neurodegenerative Diseases Neuromuscular Diseases Neuromuscular Junction Diseases Paraneoplastic Syndromes Paraneoplastic Syndromes, Nervous System

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Box Hill Hospital, Box Hill, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

Efgartigimod PH20 SC

Combination Product

Subcutaneous efgartigimod PH20 SC given by prefilled syringe

Placebo PH20 SC

Other

Subcutaneous placebo PH20 SC given by prefilled syringe

Primary outcomes

  1. 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.

Secondary outcomes

  1. MGII (PRO+PE) 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 ocular PE score varies between 0 and 5. The higher the score, the more severe the disease.

  2. MG-ADL ocular domain score change from baseline to day 29 in part A

    Time frame: Up to 29 days

    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).

  3. MGII total 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 score varies between 0 and 84. The higher the score, the more severe the disease.

  4. MGII (PE) 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 6 physical examinations (PE). The score varies between 0 and 5. The higher the score, the more severe the disease.

  5. MG-ADL total score change from baseline to day 29 in part A

    Time frame: Up to 29 days

    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).

  6. MGII ocular scores changes (PRO) from baseline in part A+B

    Time frame: Up to 111 weeks

    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.

  7. Incidence of (serious) adverse events

    Time frame: Up to 111 weeks

  8. MG-QoL15r total score changes from baseline in part A+B

    Time frame: Up to 111 weeks

    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).

  9. NEI VFQ-25 score changes from baseline in part A+B

    Time frame: Up to 111 weeks

    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)

  10. Percent change from baseline in total IgG levels over time in part A+B

    Time frame: Up to 111 weeks

  11. 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

  12. Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B

    Time frame: Up to 111 weeks

  13. Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B

    Time frame: Up to 111 weeks

Sponsors and collaborators

Lead sponsor

argenx

Industry

Registry information

Official study 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

Acronym: ADAPT oculus

Important dates

Study start
2024
Primary completion
2026
Study completion
2027
First posted
Aug 16, 2024
Registry last updated
Jul 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.