MP: ADAPT Forward - Master protocol of a platform study to evaluate the safety and efficacy of multiple regimens in participants with Myasthenia Gravis. ISA1: ADAPT Forward 1 - ISA1 – a study to evaluate empasiprubart IV as add-on therapy to efgartigimod IV in participants with AChR-Ab seropositive generalized myasthenia gravis with a partial clinical response to efgartigimod.

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

13 Mar 2026 → ongoing

Decision date (initial)

2026-02-18

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

argenx BV

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy, Others

MP - To determine the safety and tolerability of multiple regimens (monotherapy or add-on therapy to backbone therapy) in MG subtypes.

ISA 1 - To determine the safety and tolerability of empasiprubart as add-on therapy to efgartigimod.

Secondary objectives 2

1. MP - To evaluate the efficacy of multiple regimens (monotherapy or add-on therapy to backbone therapy) in MG subtypes
2. ISA 1 - To evaluate the efficacy of empasiprubart as add-on therapy to efgartigimod compared with efgartigimod alone

Conditions and MedDRA coding

AChR-Ab Seropositive Generalized Myasthenia Gravis, Myasthenia Gravis, MG, gMG

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

IPD plan description

n/a

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. MP Main inclusion criteria: o Is at least 18 years of age and the local legal age of consent for clinical studies o Has been diagnosed with MG consistent with MG's clinical features and with symptom severity appropriate for currently enrolling ISAs, per the investigator’s clinical judgment o If receiving MG therapy, including nonsteroidal immunosuppressive drugs (NSIDs), corticosteroids, or acetylcholinesterase (AChE) inhibitors either in combination or alone, the participant should receive a stable dosage before master protocol screening
2. ISA 1 Main inclusion criteria:- Is seropositive for anti-acetylcholine receptor antibodies (AChR-Ab). - Has confirmed diagnosis of gMG and is Myasthenia Gravis Foundation of America (MGFA) Class II, III, IVa, or IVb. - Has documented immunization against encapsulated bacterial pathogens (Neisseria meningitidis and Streptococcus pneumoniae) within 5 years of ISA screening or is willing to receive immunization at least 14 days before the first study drug administration.

Exclusion criteria 2

1. MP Main exclusion criteria: - Known autoimmune disease or any medical condition other than the indication under study that would interfere with an accurate assessment of clinical symptoms of MG or puts the participant at undue risk. - Is MGFA (Myasthenia Gravis Foundation of America) Class V
2. ISA 1 Main exclusion criteria: - Clinical diagnosis of systemic lupus erythematosus (SLE). - Any known complement deficiency. - Current administration of a complement inhibitor or received zilucoplan or eculizumab <2 months or ravulizumab <6 months before the first study drug administration. - Patients proven to be refractory to efgartigimod (ie, not achieving a clinically meaningful improvement in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score defined as an improvement of ≥2 points).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. MP - 1. Incidence, severity, and relatedness of AEs and SAEs
2. MP - 2. Clinically meaningful changes in vital signs, ECGs, and laboratory parameters
3. ISA 1 - 1. Incidence, severity, and relatedness of adverse events and serious adverse events in parts A and B
4. ISA 1 - 2. Clinically meaningful changes in vital signs, electrocardio grams, and laboratory parameters in parts A and B

Secondary endpoints 9

1. MP - 1. Change in MG disease severity as measured by change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score or Myasthenia Gravis Impairment Index (MGII) total score
2. MP - 2. Proportion of participants reaching minimal symptom expression (MSE) and other percentage improvements at variable time points.
3. ISA1 - 1. Myasthenia Gravis Activities of Daily Living (MG-ADL) total score change from baseline at week 18 in part B (cycle 2 day 29) compared with MG-ADL total score change from baseline at week 4 in part A
4. ISA 1 - 2. Proportion of participants reaching minimal symptom expression (MSE) at any point in part B cycles 1 and 2, and part B cycles 1 or 2
5. ISA 1 - 3. MG-ADL total score actual values and changes from baseline over time in part B compared with part A
6. ISA 1 - 4. Quantitative Myasthenia Gravis (QMG) total score change from baseline at week 18 in part B (cycle 2 day 29) compared with QMG total score change from baseline at week 4 in part A
7. ISA 1 - 5. QMG total score actual values and changes from baseline over time in part B compared with part A
8. ISA 1 - 6. Proportion of participants who have a 50% MG-ADL total score improvement in part B cycles 1 and 2
9. ISA 1 - 7. Proportion of participants who have positive patient acceptable symptom state (PASS) in part B cycles 1 and 2, and part B cycles 1 or 2

Investigational products

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

Test 2

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 25

Locations

7 EU/EEA countries · 19 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 121 files

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

Application history

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