Efficacy and Safety Study of Nipocalimab for Adults with CIDP

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

24 Oct 2022 → ongoing

Decision date (initial)

2024-05-14

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

2023-508425-28-00

EudraCT number

2021-003234-37

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Efficacy, Safety, Pharmacokinetic

To evaluate the efficacy of nipocalimab compared to placebo in delaying relapse in participants with CIDP who initially respond to nipocalimab in Stage A

Secondary objectives 7

1. Stage A - To assess improvement of symptoms on nipocalimab
2. Stage A - To assess improvement in disease severity and progression on nipocalimab
3. Stage B - To evaluate the efficacy of nipocalimab compared to placebo on time to CIDP disease progression from Stage B baseline
4. Stage B - To evaluate the efficacy of nipocalimab compared to placebo on improved functional level compared to Stage B baseline
5. Other secondary: Stage A: To assess the safety and tolerability of open-label nipocalimab Stage B: To assess the safety and tolerability of nipocalimab compared to placebo
6. Other secondary - To assess the PK and immunogenicity of nipocalimab
7. Other secondary - To evaluate the PD of nipocalimab

Conditions and MedDRA coding

Chronic Inflammatory Demyelinating Polyneuropathy

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. Adults ≥18 years of age at the time of consent and as applicable, must also meet the legal age of consent and in the jurisdiction in which the study is taking place.
2. Diagnosed with CIDP according to criteria of the EAN/PNS 2021, progressing or relapsing forms. CIDP diagnosis to be adjudicated by independent committee during screening period.
3. INCAT disability score between 2 and 9 at the Run-In Baseline visit for participants entering Run-In, or Stage A Baseline visit for participants directly entering Stage A. Participants with an INCAT score of 2 at trial entry must have this score exclusively from the leg disability score.

Exclusion criteria 3

1. Has a history of severe and/or uncontrolled hepatic (e.g. viral/alcoholic/ autoimmune hepatitis/cirrhosis and or metabolic liver disease), gastrointestinal, renal, pulmonary, cardiovascular, psychiatric, neurological or musculoskeletal disorder, hypertension and/ or any other medical or uncontrolled autoimmune disorder(s) (e.g. diabetes mellitus) or clinically significant abnormalities in screening laboratory that might interfere with the patient's full participation in the study, or might jeopardize the safety of the participant or the validity of the study results. Note: Any condition for which, in the opinion of the investigator, participation would not be in the best interest of the participants (e.g., compromise well-being) or that could prevent, limit, or confound the protocol-specified assessments.
2. Pure sensory CIDP or CISP (EAN/PNS definition).
3. Polyneuropathy of other causes, including the following: Multifocal motor neuropathy (MMN); Monoclonal gammopathy of uncertain significance with antimyelin associated glycoprotein (anti-MAG) immunoglobulin M (IgM) antibodies; Hereditary motor neuropathy; Hereditary neuropathy with liability to pressure palsies (HNPP); Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin change syndromes; Lumbosacral radiculoplexus neuropathy; Polyneuropathy most likely due to diabetes mellitus; Polyneuropathy most likely due to systemic illnesses; Drug- or toxin-induced polyneuropathy. Note: A concomitant polyneuropathy of other causes (e.g. a mild, stable diabetic polyneuropathy) is not necessarily exclusionary if CIDP is confirmed as the main diagnosis, as determined by the investigator and confirmed by the adjudication committee.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Time to first occurrence of a relapse event, where relapse is defined by the deterioration in either adjusted INCAT disability score relative to Stage B baseline or the switch to IVIg or other SoC as a result of investigator-assessed lack of efficacy as confirmed by an independent RAC

Secondary endpoints 22

1. Time to initial confirmed ECI
2. Percentage of responders as determined by ECI
3. Change from Stage A baseline over time in adjusted INCAT disability score
4. Change from Stage A baseline over time in MRC Muscle Grading Scale Sum score
5. Change from Stage A baseline over time in I-RODS centile score
6. Change from Stage A baseline over time in mean grip strength (dominant hand)
7. Change from Stage A baseline over time in mean grip strength (non-dominant hand)
8. Time to first adjusted INCAT disability score deterioration relative to Stage B baseline
9. Time to first switch to IVIg or other SoC as a result of investigator-assessed lack of efficacy as confirmed by an independent RAC relative to Stage B baseline
10. Change from Stage B baseline over time in adjusted INCAT disability score
11. Change from Stage B baseline over time in MRC Muscle Grading Scale Sum score
12. Change from Stage B baseline over time in I-RODS centile score
13. Change from Stage B baseline over time in mean grip strength (dominant hand)
14. Change from Stage B baseline over time in mean grip strength (non-dominant hand)
15. Binary response endpoint satisfying all 4 conditions: (1) an improved adjusted INCAT disability score compared to Stage B baseline, (2) not relapsing, (3) not switching to SoC, (4) not discontinuing treatment
16. Percentage of participants with TEAEs and SAEs
17. Change in ECG, vital signs and clinical laboratory values over time
18. Incidence of clinically significant ECG, vital signs and clinical laboratory abnormalities
19. Percentage of participants with suicidal ideation or suicidal behavior based on the C-SSRS
20. Serum nipocalimab concentrations over time in participants receiving active study intervention
21. Incidence and titers of ADA to nipocalimab and the presence of NAb to nipocalimab
22. Changes in total serum IgG concentrations over time

Investigational products

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

Test 2

Placebo 1

Auxiliary 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 13

Locations

8 EU/EEA countries · 43 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 132 files

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

Application history

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