Clinical Trials Register

Summary
EudraCT Number:	2021-003234-37
Sponsor's Protocol Code Number:	80202135CDP3001
National Competent Authority:	Slovakia - SIDC (Slovak)
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2022-05-02
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Slovakia - SIDC (Slovak)

A.2

EudraCT number

2021-003234-37

A.3

Full title of the trial

Phase 2/3, Multistage, Multicenter, Randomized, Double-Blind, Placebo-Controlled Parallel Group Withdrawal Study to Evaluate the Efficacy and Safety of Nipocalimab Administered to Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Multicentrické, randomizované, dvojito zaslepené, placebom kontrolované klinické skúšanie fázy 2/3 s viacerými štádiami, paralelnými skupinami s ukončením užívania na vyhodnotenie účinnosti a bezpečnosti nipokalimabu podávaného dospelým osobám s chronickou zápalovou demyelinizačnou polyneuropatiou (CIDP)

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Efficacy and Safety Study of Nipocalimab for Adults with CIDP

Skúšanie účinnosti a bezpečnosti nipokalimabu u dospelých s CIDP

A.3.2

Name or abbreviated title of the trial where available

Efficacy and Safety Study of Nipocalimab for Adults with CIDP

A.4.1

Sponsor's protocol code number

80202135CDP3001

A.5.4

Other Identifiers

Name:

IND

Number:

156269

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Janssen-Cilag International NV
B.1.3.4	Country	Belgium
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Janssen Research & Development, LLC
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Janssen-Cilag International NV
B.5.2	Functional name of contact point	Clinical Registry Group
B.5.3	Address:
B.5.3.1	Street Address	Archimedesweg 29
B.5.3.2	Town/ city	Leiden
B.5.3.3	Post code	2333
B.5.3.4	Country	Netherlands
B.5.4	Telephone number	+31(0)71524 2166
B.5.5	Fax number	+31(0)71524 2110
B.5.6	E-mail	ClinicalTrialsEU@its.jnj.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Nipocalimab
D.3.2	Product code	M281/JNJ-80202135
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	nipocalimab
D.3.9.1	CAS number	2211985-36-1
D.3.9.2	Current sponsor code	JNJ-80202135
D.3.9.3	Other descriptive name	JNJ-80202135
D.3.9.4	EV Substance Code	SUB195356
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	30
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Chronic Inflammatory Demyelinating Polyneuropathy

E.1.1.1

Medical condition in easily understood language

Chronic Inflammatory Demyelinating Polyneuropathy

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10057645
E.1.2	Term	Chronic inflammatory demyelinating polyradiculoneuropathy
E.1.2	System Organ Class	10029205 - Nervous system disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

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

E.2.2

Secondary objectives of the trial

Stage A:
* To assess improvement of symptoms on nipocalimab
* To assess improvement in disease severity and progression on nipocalimab

Stage B:
* To evaluate the efficacy of nipocalimab compared to placebo on time to CIDP disease progression from Stage B baseline
* To evaluate the efficacy of nipocalimab compared to placebo on improved functional level compared to Stage B baseline

Other secondary:
* To assess the safety and tolerability of nipocalimab compared to placebo
* To assess the PK and immunogenicity of nipocalimab
* To evaluate the PD of nipocalimab

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

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. Adjusted INCAT disability score between 2 and 9 (a score of 2 has to be exclusively from leg disability).
4. Fulfilling any of the following treatment conditions:
a. Currently treated with pulsed corticosteroids, oral corticosteroids and/or IVIg or SCIg and the patient is willing to discontinue or taper this treatment at the first run-in visit;
or
b. Without previous treatment (treatment naïve); or
Treatment with corticosteroids and/or IVIg or SCIg discontinued at least 3 months prior to screening (untreated)
5. Active disease as determined by CIDP Disease Activity Status (CDAS) score ≥3.
6. Has sufficient venous access to allow drug administration by infusion and blood sampling as per the protocol.
7. It is recommended to be up to date on all age-appropriate vaccinations prior to screening per routine local medical guidelines. It is strongly recommended that participants will have completed a locally-approved (or emergency use-authorized) COVID-19 vaccination regimen at least 2 weeks prior to study-related visits or procedures. Study participants should follow applicable local vaccine labeling, guidelines, and standards-of-care for patients receiving immune-targeted therapy when determining an appropriate interval between vaccination and study enrollment.
8. A woman of childbearing potential must have a negative highly sensitive serum (b-human
chorionic gonadotropin [b-hCG]) at Screening and a negative urine pregnancy test at
Day 1 prior to administration of study intervention.
9. A woman must be:
- Not of childbearing potential
- Of childbearing potential and practicing a highly effective method of contraception while receiving study intervention and until 30 days after last dose-the end of relevant systemic exposure.
10. A woman must agree not to donate eggs (ova, oocytes) or freeze for future use for the purposes of assisted reproduction during the study and for a period of 30 days after the administration of study intervention.
11. A male participant must wear a condom when engaging in any activity that allows for passage of ejaculate to another person during the study and for at least 90 days after receiving the last administration of study intervention. In addition, male participants with partners who are a woman of childbearing potential are highly encouraged to inform their partner to use highly effective contraception methods that result in a ow failure rate (less than 1% per year). See Appendix 6.
12. A male participant must agree not to donate sperm for the purpose of reproduction during the study and for a minimum of 90 days after receiving the last dose of study intervention.
13. Must sign an ICF indicating that the participant understands the purpose of, and procedures required for, the study and is willing to voluntarily participate in the study and comply with all study procedures. Participants who initially provide consent and subsequently withdraw it prior to enrollment in Run-in or Stage A (as applicable as per Inclusion Criteria 4), will be deemed as having failed this inclusion criterion. (Must be legal age of consent in the jurisdiction in which the study is taking place, at the time of consent).
14. Must be able to read and write.

E.4

Principal exclusion criteria

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, in the opinion of the investigator, 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.
4. Any other disease that could better explain the patient's signs and symptoms, such as significant persisting neurological deficits from stroke or central nervous system (CNS) trauma or peripheral neuropathy from another cause such as connective tissue disease or systemic lupus erythematosus.
5. Any history of myelopathy or evidence of central demyelination.
6. Currently has a malignancy or has a history of malignancy within 3 years before screening (with the exception of localized basal cell carcinoma and/or squamous cell carcinoma skin cancer that has been adequately treated with no evidence of recurrence for at least 3 months [defined as a minimum of 12 weeks] before the first study intervention administration or cervical carcinoma in situ that has been treated with no evidence of recurrence for at least 3 months before the first study intervention administration).
7. Has known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients.
8. Has shown a previous severe immediate hypersensitivity reaction, such as anaphylaxis to therapeutic proteins (eg, mAbs).
9. Has experienced myocardial infarction, unstable ischemic heart disease, or stroke within 12 weeks of screening.
10. History of moderate or severe substance or alcohol use disorder according to Diagnostic and Statistical Manual of Mental Disorders (5th edition) (DSM-5) criteria, except nicotine or caffeine, within 1 year before screening.
11. Is (anatomically or functionally) asplenic.
12. Is currently breastfeeding, pregnant, intends to become pregnant during the study, or is planning egg donation during the study or within 30 days after the last dose of study intervention.
13. Is planning to father a child while enrolled in this study or within 90 days after the last dose of study intervention.
14. Treatment with the following:
- Within 3 months (or 5 half-lives of the drug, whichever is longer) before screening: plasma exchange or immunoadsorption, any concomitant Fccontaining therapeutic agents (including factor or enzyme replacement) or other biological, or any other investigational product.
- Within 6 months before screening: rituximab, alemtuzumab, any other monoclonal antibody, cyclophosphamide, interferon, tumor necrosis factor-alpha inhibitors, fingolimod, methotrexate, azathioprine, mycophenolate, and any other immunomodulating or immunosuppressive medications.
Note: Inhaled, intra-articular, topical or ocular corticosteroids are not exclusionary.
- Has previously received nipocalimab.
15. Has received, or is expected to receive, a live vaccine within 4 weeks prior to screening or has a known need to receive a live vaccine during the study or within at least 8 weeks after the last administration of study intervention in this study. Participants are allowed to receive a vaccine conditionally approved SARS-CoV-2, unless it is a live vaccine. Concomitant enrollment in an investigational study for any SARS-CoV-2 (COVID-19) vaccine while participating in this study is not permitted.
16. Has had a Bacille Calmette-Guérin (BCG) vaccination within 1 year of first administration of study intervention..
(For additional criteria, please see protocol)

E.5 End points

E.5.1

Primary end point(s)

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

E.5.1.1

Timepoint(s) of evaluation of this end point

Stage B

E.5.2

Secondary end point(s)

* Time to initial confirmed ECI
* Percentage of responders as determined by ECI
* Change from Stage A baseline over time in adjusted INCAT disability score
* Change from Stage A baseline over time in MRC Muscle Grading Scale Sum score
* Change from Stage A baseline over time in I-RODS centile score
* Change from Stage A baseline over time in mean grip strength (dominant hand)
* Change from Stage A baseline over time in mean grip strength (non-dominant hand)

* Time to first adjusted INCAT disability score deterioration relative to Stage B baseline
* 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
* Change from Stage B baseline over time in adjusted INCAT disability score
* Change from Stage B baseline over time in MRC Muscle Grading Scale Sum score
* Change from Stage B baseline over time in I-RODS centile score
* Change from Stage B baseline over time in mean grip strength (dominant hand)
* Change from Stage B baseline over time in mean grip strength (non-dominant hand)
* 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

* Percentage of participants with TEAEs and SAEs
* Change in ECG, vital signs and clinical laboratory values over time
* Incidence of clinically significant ECG, vital signs and clinical laboratory abnormalities
* Percentage of participants with suicidal ideation or suicidal behavior based on the C-SSRS

* Serum nipocalimab concentrations over time in participants receiving active study intervention
* Incidence and titers of ADA to nipocalimab and the presence of NAb to nipocalimab
* Changes in total serum IgG concentrations over time

E.5.2.1

Timepoint(s) of evaluation of this end point

See protocol

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Argentina

Colombia

Switzerland

Taiwan

Australia

Brazil

Canada

China

Japan

Korea, Republic of

Mexico

Turkey

United Kingdom

United States

Austria

Belgium

Czechia

Denmark

France

Germany

Greece

Italy

Poland

Portugal

Slovakia

Spain

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

260

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

175

F.4.2.2

In the whole clinical trial

300

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Local regulations on continued access will always take precedence.
Plans for continued access stated in the protocol may change if new information on the benefit-risk profile of nipocalimab becomes available during the study or program.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2022-09-09

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-05-03

P. End of Trial
P.	End of Trial Status	Prematurely Ended

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials