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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06558279




Registration number
NCT06558279
Ethics application status
Date submitted
14/08/2024
Date registered
16/08/2024

Titles & IDs
Public title
A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis
Scientific 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
Secondary ID [1] 0 0
2024-514133-38-00
Secondary ID [2] 0 0
ARGX-113-2315
Universal Trial Number (UTN)
Trial acronym
ADAPT oculus
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myasthenia Gravis, Ocular 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Neurological 0 0 0 0
Other neurological disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Efgartigimod PH20 SC
Other interventions - Placebo PH20 SC

Experimental: Efgartigimod PH20 SC in part A+B - Participants receiving efgartigimod PH20 SC during part A and part B

Experimental: Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B - Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B


Other interventions: Efgartigimod PH20 SC
Subcutaneous efgartigimod PH20 SC given by prefilled syringe

Other interventions: Placebo PH20 SC
Subcutaneous placebo PH20 SC given by prefilled syringe
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
MGII (PRO) ocular score change from baseline to day 29 in part A
Timepoint [1] 0 0
Up to 29 days
Secondary outcome [1] 0 0
MGII (PRO+PE) ocular score change from baseline to day 29 in part A
Timepoint [1] 0 0
Up to 29 days
Secondary outcome [2] 0 0
MG-ADL ocular domain score change from baseline to day 29 in part A
Timepoint [2] 0 0
Up to 29 days
Secondary outcome [3] 0 0
MGII total score change from baseline to day 29 in part A
Timepoint [3] 0 0
Up to 29 days
Secondary outcome [4] 0 0
MGII (PE) ocular score change from baseline to day 29 in part A
Timepoint [4] 0 0
Up to 29 days
Secondary outcome [5] 0 0
MG-ADL total score change from baseline to day 29 in part A
Timepoint [5] 0 0
Up to 29 days
Secondary outcome [6] 0 0
MGII ocular scores changes (PRO) from baseline in part A+B
Timepoint [6] 0 0
Up to 111 weeks
Secondary outcome [7] 0 0
Incidence of (serious) adverse events
Timepoint [7] 0 0
Up to 111 weeks
Secondary outcome [8] 0 0
MG-QoL15r total score changes from baseline in part A+B
Timepoint [8] 0 0
Up to 111 weeks
Secondary outcome [9] 0 0
NEI VFQ-25 score changes from baseline in part A+B
Timepoint [9] 0 0
Up to 111 weeks
Secondary outcome [10] 0 0
Percent change from baseline in total IgG levels over time in part A+B
Timepoint [10] 0 0
Up to 111 weeks
Secondary outcome [11] 0 0
Percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A+B
Timepoint [11] 0 0
Up to 111 weeks
Secondary outcome [12] 0 0
Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B
Timepoint [12] 0 0
Up to 111 weeks
Secondary outcome [13] 0 0
Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B
Timepoint [13] 0 0
Up to 111 weeks

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/09/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2027
Actual
Sample size
Target
124
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Box Hill Hospital - Box Hill
Recruitment hospital [2] 0 0
Gold Coast University Hospital - Southport
Recruitment postcode(s) [1] 0 0
3128 - Box Hill
Recruitment postcode(s) [2] 0 0
4215 - Southport
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Texas
Country [3] 0 0
Belgium
State/province [3] 0 0
Leuven
Country [4] 0 0
Belgium
State/province [4] 0 0
Liège
Country [5] 0 0
Canada
State/province [5] 0 0
Halifax
Country [6] 0 0
Canada
State/province [6] 0 0
Ottawa
Country [7] 0 0
China
State/province [7] 0 0
Chengdu
Country [8] 0 0
China
State/province [8] 0 0
Guangzhou
Country [9] 0 0
China
State/province [9] 0 0
Jinan
Country [10] 0 0
China
State/province [10] 0 0
Shanghai
Country [11] 0 0
Cyprus
State/province [11] 0 0
Egkomi
Country [12] 0 0
Czech Republic
State/province [12] 0 0
Pardubice
Country [13] 0 0
Denmark
State/province [13] 0 0
Aarhus N
Country [14] 0 0
Denmark
State/province [14] 0 0
København
Country [15] 0 0
Finland
State/province [15] 0 0
Turku
Country [16] 0 0
Georgia
State/province [16] 0 0
Tbilisi
Country [17] 0 0
Japan
State/province [17] 0 0
Chuo-Ku
Country [18] 0 0
Japan
State/province [18] 0 0
Fukuoka
Country [19] 0 0
Japan
State/province [19] 0 0
Hanamaki-shi
Country [20] 0 0
Japan
State/province [20] 0 0
Hiroshima
Country [21] 0 0
Japan
State/province [21] 0 0
Kyoto-Shi
Country [22] 0 0
Japan
State/province [22] 0 0
Narita
Country [23] 0 0
Japan
State/province [23] 0 0
Sapporo-Shi
Country [24] 0 0
Japan
State/province [24] 0 0
Shinjuku-Ku
Country [25] 0 0
Japan
State/province [25] 0 0
Suita-Shi
Country [26] 0 0
Korea, Republic of
State/province [26] 0 0
Daegu
Country [27] 0 0
Korea, Republic of
State/province [27] 0 0
Seoul
Country [28] 0 0
Poland
State/province [28] 0 0
Bydgoszcz
Country [29] 0 0
Poland
State/province [29] 0 0
Katowice
Country [30] 0 0
Poland
State/province [30] 0 0
Kraków
Country [31] 0 0
Poland
State/province [31] 0 0
Lublin
Country [32] 0 0
Serbia
State/province [32] 0 0
Belgrade
Country [33] 0 0
Serbia
State/province [33] 0 0
Nis
Country [34] 0 0
Spain
State/province [34] 0 0
Barcelona
Country [35] 0 0
Spain
State/province [35] 0 0
Madrid
Country [36] 0 0
Spain
State/province [36] 0 0
Valencia
Country [37] 0 0
Sweden
State/province [37] 0 0
Göteborg

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
argenx
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Sabine Coppieters, MD
Address 0 0
Country 0 0
Phone 0 0
857-350-4834
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06558279