A study to assess efficacy and safety of efgartigimod PH20 SC in adults with ocular myasthenia gravis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Argenx

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

19 Mar 2025 → ongoing

Decision date (initial)

2025-03-10

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

argenx BV

External identifiers

EU CT number

2024-514133-38-00

ClinicalTrials.gov

NCT06558279

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Pharmacodynamic, Pharmacokinetic, Others, Pharmacoeconomic

To determine the efficacy of efgartigimod PH20 SC compared to placebo PH20 SC (placebo) in participants with oMG in part A

Secondary objectives 6

1. 1. To determine the efficacy of efgartigimod PH20 SC compared to placebo PH20 SC in participants with oMG in part A
2. 2. To determine the efficacy of efgartigimod PH20 SC compared to placebo and the long-term efficacy of efgartigimod PH20 SC in participants with oMG
3. 3. To assess the safety and tolerability of efgartigimod PH20 SC compared to placebo and the long-term safety and tolerability of efgartigimod PH20 SC in participants with oMG
4. 4. To evaluate the impact of efgartigimod PH20 SC on Quality of Life in participants with oMG
5. 5. To assess the pharmacodynamics effect of efgartigimod PH20 SC in participants with oMG
6. 6. To assess the immunogenicity of efgartigimod PH20 SC in participants with oMG

Conditions and MedDRA coding

Ocular Myasthenia Gravis

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. 1. Is at least 18 years of age and the local legal age of consent for clinical studies
2. 2. 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
3. 3. Is MGFA Class I (any ocular muscle weakness)
4. 4. 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 2

1. 1. Other diseases that lead to eyelid drooping, peripheral muscle weakness, or diplopia
2. 2. 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. MGII (PRO) ocular score change from baseline to day 29 in part A

Secondary endpoints 14

1. • MGII (PRO+PE) ocular score change from baseline to day 29 in part A
2. • MG-ADL ocular domain score change from baseline to day 29 in part A
3. • MGII total score change from baseline to day 29 in part A
4. • MGII (PE) ocular score change from baseline to day 29 in part A
5. • MG-ADL total score change from baseline to day 29 in part A
6. • MGII ocular scores (PRO, PRO+PE, and PE), generalized score, and total score; actual values and changes from baseline in part A and part A+B
7. • Incidence and severity of AEs and SAEs in part A and part A+B
8. • Clinically relevant changes in laboratory parameters, vital signs, and ECGs in part A and part A+B
9. • MG-QoL15r total score actual values and changes from baseline in part A and part A+B
10. • NEI VFQ-25 score actual values and changes from baseline in part A and part A+B
11. • Actual values and percent changes from baseline in total IgG levels over time in part A and part A+B
12. Actual values and percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A and part A+B
13. • Incidence and prevalence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A and part A+B
14. • Incidence and prevalence of antibodies and Neutralizing Antibodies against rHuPH20 in part A and part A+B

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Argenx

Sponsor organisation

Argenx

Address

Industriepark-Zwijnaarde 7

City

Gent

Postcode

9052

Country

Belgium

Scientific contact point

Organisation

Argenx

Contact name

Chief Scientific Officer

Public contact point

Organisation

Argenx

Contact name

Vice President Clinical Development

Third parties 12

Locations

15 EU/EEA countries · 38 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 180 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

13 submissions — initial application plus substantial / non-substantial modifications