Efficacy and Safety Study of Nipocalimab for Adults with Active Inflammatory Myopathies

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Janssen - Cilag International

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

20 Jan 2023 → ongoing

Decision date (initial)

2024-02-06

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Janssen Research & Development, LLC United States

External identifiers

EU CT number

2023-505314-20-00

EudraCT number

2021-005202-98

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacogenetic, Efficacy, Safety, Pharmacokinetic

To evaluate the efficacy of nipocalimab vs placebo in participants with active IIM.

Conditions and MedDRA coding

Active Idiopathic Inflammatory Myopathies (IIM)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

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

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Disease classification criteria: Participant meets the diagnostic criteria of probable or definite idiopathic inflammatory myopathies (IIM) based on 2017 The European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) classification criteria for adult IIM at least 6 weeks prior to first administration of the study intervention
2. If a participant is on regular or as needed treatment with low potency topical glucocorticoids (GC) that are allowed in the study or topical tacrolimus (TAC) to treat skin lesions, the dose and frequency should be stable for greater than or equal to (>=) 4 weeks prior to first administration of the study intervention as well as maintained at the same dose until Week 52 of the study
3. Antibody positivity criteria: Any 1 of the myositis-specific antibodies (MSAs) positive: dermatomyositis (DM): anti-Mi-2 (Mi-2/nucleosome remodeling and deacetylase [NuRD] complex), anti-transcription intermediary factor 1-Gamma (TIF1-Gamma), anti- nuclear matrix protein 2 (NXP-2), anti-serious adverse event (SAE); anti- antimelanoma differentiation-associated gene 5 (MDA-5) antibodies. Or immunemediated necrotizing myopathy (IMNM): anti- signal recognition particle (SRP) and anti- 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) antibodies. Or anti-synthetase syndrome (ASyS): anti- histidylribonucleic acid [tRNA] synthetase (Jo-1), anti- threonyl-tRNA synthetase (PL7), anti- alanyl-tRNA synthetase (PL12), anti- isoleucyltRNA synthetase (OJ), and anti- glycyl-tRNA synthetase (EJ) antibodies. If all MSAs are negative or more than 1 MSA is positive within different subtypes (defined by the central laboratory) at screening, the tests should be repeated during the screening period. If the same results are observed at retesting, the participant should not be enrolled in the study.

Exclusion criteria 5

1. Has a juvenile myositis diagnosis and now ≥18 years old
2. Has cancer-associated myositis defined as cancer diagnosis within 3 years of myositis diagnosis except for cervical carcinoma in situ and nonmelanoma skin cancer (squamous cell carcinoma, basal cell carcinoma of the skin)
3. Has comorbidities (example, asthma, chronic obstructive pulmonary disease [COPD]) which have required 3 or more courses of oral GC within 1 year prior to screening
4. Has a history of primary immunodeficiency or secondary immunodeficiency not related to the treatment of the participants IIM.
5. Has experienced myocardial infarction (MI), unstable ischemic heart disease, or stroke within 12 weeks of screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion Percentage of participants who achieve at least minimal improvement (≥ 20-point improvement) in IMACS TIS (International myositis assessment and clinical studies total improvement score) at Week 52 and on ≤5 mg/day of oral prednisone (or equivalent) from week 44 through week 52.

Investigational products

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

Test 2

Placebo 1

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 12

Locations

7 EU/EEA countries · 13 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 151 files

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

Application history

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