Clinical trial NCT05327114: Efficacy and Safety Study of Nipocalimab for Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2022-04-14
Enrollment: 201
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The main purpose of this study is to evaluate the safety and efficacy of nipocalimab compared to placebo in delaying relapse in adults with chronic inflammatory demyelinating polyneuropathy (CIDP) who initially respond to nipocalimab in Stage A.


Eligibility criteria: Inclusion Criteria:

* Adults greater than or equal to (\>=) 18 years of age at the time of consent and as applicable, must also meet the legal age of consent in the jurisdiction in which the study is taking place
* Diagnosed with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) according to criteria of the European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) 2021, progressing or relapsing forms. CIDP diagnosis to be adjudicated by independent committee during screening period
* Inflammatory Neuropathy Cause and Treatment (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
* Fulfilling any of the following treatment conditions: a) Currently treated with oral corticosteroids (CS) less than or equal to (\<=) 20 milligrams (mg)/day; or b) Currently treated with pulsed CS, and/or intravenous immunoglobulin (IVIg) or subcutaneous immunoglobulin (SCIg) and the participant is willing to discontinue no later than the run-in baseline visit; or c) Currently treated with oral CS greater than (\>) 20 mg/day and the participant is willing to taper to \<=20 mg/day during the run-in period; or d) Without previous treatment (treatment naive); or treatment with CS and/or IVIg or SCIg discontinued at least 3 months prior to screening (untreated)
* Active disease as determined by CIDP Disease Activity Status (CDAS) score \>= 3
* Other protocol-defined inclusion criteria will apply

Exclusion Criteria:

* Has a history of severe and/or uncontrolled hepatic (example, 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) (example, diabetes mellitus) or clinically significant abnormalities in screening laboratory that, might interfere with the participants full participation in the study, or might jeopardize the safety of the participant or the validity of the study results
* Pure sensory CIDP or chronic immune sensory polyradiculopathy (CISP) (EAN/PNS definition)
* Any other disease that could better explain the participant'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
* 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 (example, a mild, stable diabetic polyneuropathy) is not necessarily exclusionary if chronic inflammatory demyelinating polyneuropathy (CIDP) is confirmed as the main diagnosis, as determined by the investigator and confirmed by the adjudication committee
* Has known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients
* Other protocol-defined exclusion criteria will apply

Primary outcomes:
measure: Stage B: Time to First Occurrence of a Relapse Event
timeFrame: Up to 52 weeks
Secondary outcomes:
measure: Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI)
timeFrame: 12 weeks
measure: Stage A: Percentage of Responders as Determined by ECI
timeFrame: 12 weeks
measure: Stage A: Change from Baseline Over Time in Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale Score
timeFrame: Baseline to 12 weeks
measure: Stage A: Change from Stage A Baseline Over Time in Medical Research Council (MRC) Muscle Grading Scale Sum Score
timeFrame: Baseline to 12 weeks
measure: Stage A: Change from Baseline Over Time in Inflammatory Rasch-Built Overall Disability Scale (I-RODS) Centile Score
timeFrame: Baseline to 12 weeks
measure: Stage A: Change from Baseline Over Time in Mean Grip Strength (Dominant Hand)
timeFrame: Baseline to 12 weeks
measure: Stage A: Change from Baseline Over Time in Mean Grip Strength (Non-Dominant Hand)
timeFrame: Baseline to 12 weeks
measure: Stage B: Time to First Adjusted INCAT Disability Scale Score Deterioration Relative to Baseline
timeFrame: Up to 52 weeks
measure: Stage B: Time to First Switch to Intravenous Immunoglobulin (IVIg) or Other Standard of Care (SoC) as a Result of Investigator-assessed Lack of Efficacy as Confirmed by an Independent RAC Relative to Baseline
timeFrame: Up to 52 weeks
measure: Stage B: Change from Baseline Over Time in Adjusted INCAT Disability Score
timeFrame: Up to 52 weeks
measure: Stage B: Change from Baseline Over Time in MRC Muscle Grading Scale Sum Score
timeFrame: Up to 52 weeks
measure: Stage B: Change from Baseline Over Time in I-RODS Centile Score
timeFrame: Up to 52 weeks
measure: Stage B: Change from Baseline Over Time in Mean Grip Strength (Dominant Hand)
timeFrame: Up to 52 weeks
measure: Stage B: Change from Baseline Over Time in Mean Grip Strength (Non-Dominant Hand)
timeFrame: Up to 52 weeks
measure: Stage B: Number of Participants with Binary Response Endpoint Satisfying all 4 Conditions: a) An Improved Adjusted INCAT Disability Score Compared to Baseline; b) Not Relapsing; c) Not Switching to SoC; d) Not Discontinuing Treatment
timeFrame: Up to 52 weeks
measure: Percentage of Participants with Treatment-emergent Adverse Events (TEAEs)
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Percentage of Participants with Serious Adverse Events (SAEs)
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Number of Participants with Change in Electrocardiogram (ECG) Values Over Time
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Number of Participants with Change in Vital Signs Values Over Time
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Number of Participants with Change in Clinical Laboratory Values Over Time
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Number of Participants with Clinically Significant ECG Abnormalities
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Number of Participants with Clinically Significant Vital Signs Abnormalities
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Number of Participants with Clinically Significant Clinical Laboratory Abnormalities
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Percentage of Participants with Suicidal Ideation or Suicidal Behavior based on the Columbia-Suicide Severity Rating Scale (C-SSRS)
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Serum Nipocalimab Concentrations Over Time in Participants Receiving Active Study Intervention
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Number of Participants with Anti-drug Antibodies (ADA) to Nipocalimab
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Titers of ADA to Nipocalimab
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Number of Participants with Neutralizing Antibodies (NAb) to Nipocalimab
timeFrame: Stage A: 12 weeks; Stage B: Up to 52 weeks
measure: Change from Baseline in Total Serum Immunoglobulin (IgG) Concentrations Levels Over Time
timeFrame: Stage A: Baseline to 12 weeks; Stage B: Baseline up to 52 weeks
Other outcomes:
Locations:
city: Centennial
country: United States
facility: IMMUNOe Health and Research Centers
state: Colorado
city: Maitland
country: United States
facility: Neurology Associates PA
state: Florida
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Boston Clinical Trials
state: Massachusetts
city: Royal Oak
country: United States
facility: Beaumont Hospital Royal Oak
state: Michigan
city: New York
country: United States
facility: The Neurological Institute of New York
state: New York
city: Patchogue
country: United States
facility: South Shore Neurologic Associates - Patchogue
state: New York
city: Charlotte
country: United States
facility: The Neurological Institute, PA
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Main Campus
state: Ohio
city: Austin
country: United States
facility: Austin Neuromuscular Center
state: Texas
city: Milwaukee
country: United States
facility: Advocate Health - Aurora St. Luke's Medical Center
state: Wisconsin
city: Melbourne
country: Australia
facility: The Alfred Hospital
city: Parkwood
country: Australia
facility: Gold Coast University Hospital
city: Beijing
country: China
facility: Peking University First Hospital
city: Beijing
country: China
facility: Xuanwu Hospital ,Capital Medical University
city: Beijing
country: China
facility: Beijing Tiantan Hospital, Capital Medical University
city: Beijing
country: China
facility: Peking University Third Hospital
city: Changchun
country: China
facility: The First Hospital of Jilin University
city: Changsha
country: China
facility: The Third Xiangya Hospital of Central Sourth University
city: Changsha
country: China
facility: Xiangya Hospital Central South University
city: Chifeng
country: China
facility: Chifeng Municipal Hospital
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital
city: Guang Zhou
country: China
facility: The First Affiliated Hospital Sun Yat sen University
city: Jinan
country: China
facility: Qianfoshan hospital of Shandong Province
city: Nanchang
country: China
facility: The First Affiliated Hospital of NanChang University
city: Shanghai
country: China
facility: Huashan Hospital Fudan University
city: Shanghai
country: China
facility: Tong Ren Hospital Shanghai Jiao Tong University school of medicine
city: Xi'an
country: China
facility: Xi 'an GaoXin Hospital
city: Floridablanca
country: Colombia
facility: Fundación Cardiovascular de Colombia - Instituto del Corazón Floridablanca
city: Medellin
country: Colombia
facility: Instituto Neurológico de Colombia
city: Hradec Králové
country: Czechia
facility: Fakultni nemocnice Hradec Kralove
city: Ostrava
country: Czechia
facility: Fakultni Nemocnice Ostrava
city: Pardubice
country: Czechia
facility: Pardubicka krajska nemocnice a s
city: Bordeaux
country: France
facility: CHU Bordeaux
city: Bron
country: France
facility: Hospices Civils de Lyon HCL
city: Le Kremlin Bicêtre
country: France
facility: Hopital de Bicetre
city: Nice
country: France
facility: Hopital PASTEUR
city: Strasbourg
country: France
facility: CHRU Strasbourg
city: Bochum
country: Germany
facility: St. Josef-Hospital, Ruhr-Universität Bochum
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Leipzig
country: Germany
facility: Universitaetsklinikum Leipzig
city: Potsdam
country: Germany
facility: St Josefs Krankenhaus Potsdam Sanssouci
city: Athens
country: Greece
facility: Attikon General Hospital of Athens
city: Patras
country: Greece
facility: Patras University Hospital
city: Thessaloniki
country: Greece
facility: Papageorgiou General Hospital Of Thessaloniki
city: Asahikawa
country: Japan
facility: National Hospital Organization Asahikawa Medical Center
city: Bunkyo ku
country: Japan
facility: Institute of Science Tokyo Hospital
city: Chiba-shi
country: Japan
facility: Chiba University Hospital
city: Hamamatsu
country: Japan
facility: Seirei Hamamatsu General Hospital
city: Isehara
country: Japan
facility: Tokai University Hospital
city: Kobe City
country: Japan
facility: Kobe City Medical Center General Hospital
city: Kodaira-shi
country: Japan
facility: National Center of Neurology and Psychiatry
city: Koshigaya
country: Japan
facility: Saitama Medical Center
city: Kumamoto
country: Japan
facility: Kumamoto University Hospital
city: Nagoya-shi
country: Japan
facility: Nagoya University Hospital
city: Nagoya
country: Japan
facility: Chubu Rosai Hospital
city: Osaka Sayama shi
country: Japan
facility: Kindai University Hospital
city: Sendai-City
country: Japan
facility: National Hospital Organization Sendai Medical Center
city: Shimotsuga Gun
country: Japan
facility: Dokkyo Medical University Hospital
city: Shinjuku-ku
country: Japan
facility: Tokyo Women's Medical University Hospital
city: Tenri
country: Japan
facility: Tenri Hospital
city: Toon-shi
country: Japan
facility: Ehime University Hospital
city: Toyama-shi
country: Japan
facility: Toyama University Hospital
city: Ube
country: Japan
facility: Yamaguchi University Hospital
city: Seoul
country: Korea, Republic of
facility: Konkuk University Medical Center
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Monterrey
country: Mexico
facility: i Can Oncology Center
city: Tlalnepantla
country: Mexico
facility: Clinical Research Institute S.C.
city: Chorzow
country: Poland
facility: Centrum Medyczne
city: Krakow
country: Poland
facility: Specjalistyczne Gabinety Lekarskie
city: Lublin
country: Poland
facility: Prywatny Gabinet Lekarski
city: Poznan
country: Poland
facility: Clinical Research Center sp z o o MEDIC R s k
city: Almada
country: Portugal
facility: Hospital Garcia de Orta
city: Braga
country: Portugal
facility: Hospital de Braga
city: Porto
country: Portugal
facility: Centro Hospitalar de Sao Joao Epe
city: Alicante
country: Spain
facility: Hosp. Gral. Univ. de Alicante
city: Barcelona
country: Spain
facility: Hosp Univ Vall D Hebron
city: Bilbao
country: Spain
facility: Hosp. Univ. de Basurto
city: Madrid
country: Spain
facility: Hosp. Clinico San Carlos
city: Sevilla
country: Spain
facility: Hosp. Virgen Macarena
city: Kaohsiung
country: Taiwan
facility: Kaohsiung Chang Gung Memorial Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taipei
country: Taiwan
facility: Shin Kong Wu Ho Su Memorial Hospital
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust
city: Glasgow
country: United Kingdom
facility: NHS Greater Glasgow and Clyde
city: London
country: United Kingdom
facility: Royal Free Hospital
city: Sheffield
country: United Kingdom
facility: Sheffield Teaching Hospitals NHS Foundation Trust
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: