Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruitment pending
Participants planned
59
Countries
4
Sites
16
generalized myasthenia gravis
To evaluate the efficacy of nipocalimab vs efgartigimod in participants initiating neonatal Fc receptor (FcRn) treatment for generalized myasthenia gravis (gMG).
Key facts
- Sponsor
- Janssen Cilag International
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20]
- Decision date (initial)
- 2025-12-14
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
To evaluate the efficacy of nipocalimab vs efgartigimod in participants initiating neonatal Fc receptor (FcRn) treatment for generalized myasthenia gravis (gMG).
Conditions and MedDRA coding
generalized myasthenia gravis
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | PT | 10028417 | Myasthenia gravis | 100000004852 |
Regulatory references
- Plan to share IPD
- Yes
- IPD plan description
- The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu
| EU CT number | Title | Sponsor |
|---|---|---|
| 2023-504152-97-00 | Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Nipocalimab Administered to Adults with Generalized Myasthenia Gravis | Janssen - Cilag International |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 7
- ≥18 years of age and <75 years of age
- AChR antibody positive
- MG-ADL ≥5 with ≥50% as non-ocular
- Has suboptimal response to current stable therapy for generalized Myasthenia Gravis according to the investigator, (specific to arms 1 and 2)
- Total IgG ≥6 g/L, (specific to arms 1 and 2)
- Treatment with efgartigimod intravenously or subcutaneously for ≥1 cycles, (specific to arm 3)
- Participant and HCP agree it is appropriate for the participant to switch to nipocalimab, (specific to arm 3)
Exclusion criteria 3
- Has received treatment for MG with an FcRn-targeting therapy, (specific to arms 1 and 2)
- Is currently taking IgG monoclonal antibody therapeutics, or Fc-conjugated therapeutic agents, including factor or enzyme replacement, (specific to arms 1 and 2)
- Current efgartigimod dosing is inconsistent with the label, (specific to arm 3)
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Averaged mean percent change from baseline in total IgG over Weeks 8 to 12 between Arms 1 and 2.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10565805 · Product
- Active substance
- Nipocalimab
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENUS USE
- Max daily dose
- 0 Other
- Max total dose
- 0 Other
- Max treatment duration
- 12 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
Comparator 1
SCP130522783 · ATC
- Active substance
- Efgartigimod Alfa
- Substance synonyms
- IMMUNOGLOBULIN G1, ANTI-(FCRN RECEPTOR) (HUMAN MONOCLONAL ARGX-113 FC FRAGMENT), ARGX-113
- Route of administration
- INTRAVENUS USE
- Max daily dose
- 0 Other
- Max total dose
- 0 Other
- Max treatment duration
- 4 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AA58 — EFGARTIGIMOD ALFA
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/18/1992
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Janssen Cilag International
- Sponsor organisation
- Janssen Cilag International
- Address
- Turnhoutseweg 30
- City
- Beerse
- Postcode
- 2340
- Country
- Belgium
Scientific contact point
- Organisation
- Janssen Cilag International
- Contact name
- CTIS Point of Contact
Public contact point
- Organisation
- Janssen Cilag International
- Contact name
- CTIS Point of Contact
Third parties 4
| Organisation | City, country | Duties |
|---|---|---|
| 4g Clinical LLC ORG-100042775
|
Wellesley, United States | Interactive response technologies (IRT) |
| Eresearchtechnology Inc. ORG-100013039
|
Philadelphia, United States | E-data capture |
| Labcorp Central Laboratory Services SARL ORG-100011524
|
Meyrin, Switzerland | Laboratory analysis |
| Iqvia Inc. ORG-100010622
|
Durham, United States | Other |
Locations
4 EU/EEA countries · 16 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Authorised, recruitment pending | 4 | 1 |
| Germany | Authorised, recruitment pending | 7 | 6 |
| Italy | Authorised, recruitment pending | 11 | 5 |
| Spain | Authorised, recruitment pending | 7 | 4 |
| Rest of world
Switzerland, United States, Israel
|
— | 30 | — |
Investigational sites
Universitaetsklinikum Erlangen AöR
Klinik für Neurologie, Schwabachanlage 6, Innenstadt, Erlangen
Berufsgenossenschaftliches Universitaetsklinikum Bergmannsheil gGmbH
Klinik für Neurologie, Buerkle De La Camp-Platz 1, Wiemelhausen, Bochum
Universitaet Muenster
Klinik für Neurologie, Albert-Schweitzer-Campus 1, Sentrup, Muenster
Klinikum Altenburger Land GmbH
Klinik für Neurologie, Am Waldessaum 10, 04600, Altenburg
Katholisches Klinikum Bochum gGmbH
Klinik für Neurologie, Gudrunstrasse 56, Grumme, Bochum
Medical Center - University Of Freiburg
Klinik für Neurologie und Neurophysiologie, Hugstetter Strasse 55, Stuehlinger, Freiburg Im Breisgau
Azienda Ospedaliero Universitaria Pisana
Unità di Neurologia, Dipartimento Neuroscienza, PO Santa Chiara, Via Roma 67, 56126, Pisa
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
UOC Neurologia, Largo Francesco Vito 1, 00168, Rome
Azienda Ospedaliero-Universitaria Sant Andre
UOC di Neurologia, Via Di Grottarossa 1035-1039, 00189, Rome
IRCCS Foundation Istituto Neurologico Carlo Besta
SSD Immunoterapia e Aferesi Terapeutica , Dip. Neuroscienze Cliniche, Via Giovanni Celoria 11, 20133, Milan
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
UOC di Neurofisiopatologia, Via Antonio Cardarelli 9, 80131, Naples
Hospital Clinic De Barcelona
Neurology, Calle Villarroel 170, 08036, Barcelona
Hospital Universitario Infanta Sofía
Neurology, Paseo De Europa 34, 28702, San Sebastian De Los Reyes
Hospital Universitario Virgen De La Macarena
Neurology, Avenida Del Doctor Fedriani 3, 41009, Sevilla
Hospital Clinico Universitario De Valencia
Neurology, Avenida Blasco Ibanez 17, 46010, Valencia
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 45 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | REDACTED_D1_Protocol_2025-521130-28 | PAM1 |
| Protocol (for publication) | REDACTED_D4_PF GAD-7_Multicountry_eng_2025-521130-28 | 1 |
| Protocol (for publication) | REDACTED_D4_PF Neuro-QoL_Multicountry_eng_2025-521130-28 | 1 |
| Protocol (for publication) | REDACTED_D4_PF PRO AE_Multicountry_eng_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K1_Recruitment Arrangements _DE_ENG_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K1_Recruitment arrangements_AT_ENG_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K1_Recruitment Arrangements_ES_ENG_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K1_Recruitment arrangements_IT_ENG_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material Digital Patient Brochure_AT_GER_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material Digital Patient Brochure_DE_GER_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment Material Digital Patient Brochure_ES_SPA_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment Material Digital Patient Brochure_IT_ITA_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material Dr to Patient Email_AT_GER_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material Dr to Patient Email_DE_GER_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment Material Dr to Patient Email_IT_ITA_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material Patient Brochure_AT_GER_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material Patient Brochure_DE_GER_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment Material Patient Brochure_ES_SPA_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment Material Patient Brochure_IT_ITA_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material Poster_AT_GER_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material Poster_DE_GER_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment Material Poster_ES_SPA_2025-521130-28 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment Material Poster_IT_ITA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Clinical_DE_GER_2025-521130-28 | 2 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Clinical_ES_SPA_2025-521130-28 | 3 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Main_AT_GER_2025-521130-28 | 5 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Main_IT_ITA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Patient Travel Reimbursement_IT_ITA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Pregnancy Partner_IT_ITA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Pregnant Partner_DE_GER_2025-521130-28 | 2 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Pregnant Partner_ES_SPA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Privacy Appendix Main_IT_ITA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Privacy Child exposed to IP_IT_ITA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Privacy Pregnancy Partner_IT_ITA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Withdrawal_IT_ITA_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF_ICF Contact Data Sheet_AT_ENG_2025-521130-28 | 2 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF_Pregnancy_AT_GER_2025-521130-28 | 3 |
| Subject information and informed consent form (for publication) | REDACTED_L2_Subject wallet card_AT_GER_2025-521130-28 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L2_Subject Wallet Card_DE_GER_2025-521130-28 | 2 |
| Subject information and informed consent form (for publication) | REDACTED_L2_Subject Wallet Card_ES_SPA_2025-521130-28 | 7 |
| Subject information and informed consent form (for publication) | REDACTED_L2_Subject wallet card_IT_ITA_2025-521130-28 | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Efgartigimod | 1 |
| Synopsis of the protocol (for publication) | REDACTED_D1_Protocol Synopsis_ES_SPA_2025-521130-28 | PAM1 |
| Synopsis of the protocol (for publication) | REDACTED_D1_Protocol Synopsis_IT_ITA_2025-521130-28 | PAM1 |
| Synopsis of the protocol (for publication) | REDACTED_D1_Protocol_Synopsis_AT_GER_2025-521130-28 | PAM1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-08-22 | Spain | Acceptable with conditions 2025-12-09
|
2025-12-10 |