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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04119050
Other study ID # CR108987
Secondary ID 2019-000720-17MO
Status Recruiting
Phase Phase 2/Phase 3
First received
Last updated
Start date August 15, 2019
Est. completion date April 10, 2028

Study information

Verified date June 2024
Source Janssen Research & Development, LLC
Contact Study Contact
Phone 844-434-4210
Email Participate-In-This-Study@its.jnj.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The main purpose of this study is to evaluate the efficacy and safety of M281 in participants with warm autoimmune hemolytic anemia (wAIHA).


Description:

The study consists of a 24-week double-blind, placebo control period, a 144-week open-label extension period and follow-up period of 8 weeks after last study drug administration. Eligible participants will be randomized to placebo or nipocalimab (2 dose levels) during the double-blind period and nipocalimab (2 dose levels) during the open-label extension period.


Recruitment information / eligibility

Status Recruiting
Enrollment 111
Est. completion date April 10, 2028
Est. primary completion date March 20, 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion criteria: - Participants greater than or equal to (>=)18 years of age - Have been diagnosed with warm autoimmune hemolytic anemia (wAIHA) for at least 3 months, and are currently receiving treatment for wAIHA or have previously received treatment for wAIHA (treatment-naive participants are not eligible) - Participants must be able to understand and voluntarily provide written informed consent to participate in the study and comply with all study procedures Exclusion criteria: - Participants must not be pregnant or breastfeeding - Participants must not have other clinically relevant abnormalities currently or in their history that the Investigator would deem them ineligible to participate - Have been diagnosed with cold antibody autoimmune hemolytic anemia (AIHA), cold agglutinin syndrome, mixed type (that is, warm and cold) AIHA, or paroxysmal cold hemoglobinuria

Study Design


Intervention

Drug:
M281
M281 injection administered as intravenous infusion
Placebo
Placebo administered as intravenous infusion

Locations

Country Name City State
Brazil Hospital das Clínicas da Faculdade de Medicina de Botucatu Botucatu
Brazil Impar Servicos Hospitalares S/A - Hospital Brasilia Brasilia
Brazil CTO Centro de Tratamento Oncologico Unidade Belem Campinas
Brazil Hospital Das Clinicas Da Universidade Federal De Goias Goiânia
Brazil Complexo Hospitalar de Niteroi Niterói
Brazil Oncoclinicas - Unidade OC Oncoclinicas Multihemo Ilha do Leite Recife
Brazil Hospital Sao Rafael Salvador
Brazil Nucleo de Oncologia da Bahia Salvador
Brazil Hospital Santa Marcelina Santo Andre
Brazil CEPHO - Centro de Estudos e Pesquisa de Hematologia e Oncologia Santo André
Brazil Centro Integrado de Pesquisa -Hospital de Base de São José do Rio Preto Sao Jose Do Rio Preto
Brazil Hospital Das Clinicas Da Faculdade De Medicina Da USP Sao Paulo
Brazil Hospital Sirio Libanes Sao Paulo Sao Paulo
Brazil Universidade Federal de Sao Paulo Sao Paulo
China Beijing Chao-yang Hospital, Capital Medical University Beijing Shi
China Peking University People s Hospital Beijing Shi
China The First Hospital of Jilin University Changchun
China West China Hospital of Sichuan University Chengdu
China Guangzhou First Municipal People's Hospital Guangzhou Shi
China Hainan General Hospital Haikou
China First Affiliated Hospital Medical School of Zhejiang University Hangzhou
China Lanzhou University Second Hospital Lanzhou
China Jiangsu Province Hospital Nanjing
China Affiliated Hospital of Nantong University Nantong
China Huashan Hospital affiliated to Fudan University Shanghai
China Shanghai Zhongshan Hospital ShangHai
China The First Affiliated Hospital of Soochow University Suzhou
China Tianjin Medical University General Hospital Tianjin
China Union Hospital Tongji Medical College of Huazhong University of Science and Technology Wuhan
China The First Affiliated Hospital of Xian Jiaotong University Xi'An
Czechia Fakultni nemocnice Brno Brno
Czechia Fakultni nemocnice Kralovske Vinohrady Praha 10
Czechia Ustav Hematologie A Krevni Transfuze Praha 2
Egypt Alexandria University Hospital Alexandria
Egypt Ain Shams University Hospital Cairo
Egypt Cairo university Cairo
Egypt Nasser institute Hospital Cairo
Egypt National Cancer Institute Cairo
Egypt Mansoura University Hospital Mansoura
France ICH Hopital A. Morvan Brest Cedex 2
France CHU Dijon Bourgogne Dijon
France CHU Grenoble La Tronche
France CHU Nantes - Hotel Dieu Nantes
France CHU De Poitiers Poitiers
France CHRU Hopital Sud Rennes
Germany Onkologische Schwerpunktpraxis Berlin
Germany Universitatsklinikum Essen Essen
Germany Rotkreuzklinikum München München
Greece Attikon University General Hospital of Attica Athens
Greece General Hospital of Athens 'G. Gennimatas' Athens
Greece Laiko General Hospital of Athens Athens
Greece University Hospital Of Larissa Larissa
Greece University General Hospital of Rio Patras Patras
Greece University General Hospital of Thessaloniki Thessaloniki
Hungary Del Pesti Centrumkorhaz Orszagos Hematologiai es Infektologiai Intezet Szent Laszlo Telephely Budapest
Hungary Semmelweis Egyetem Budapest
Hungary Debreceni Egyetem Klinikai Kozpont Debrecen
Hungary Petz Aladar Megyei Oktato Korhaz Gyõr
Hungary Somogy Megyei Kaposi Mor Oktato Korhaz Kaposvar
Israel Ha'Emek Medical Center Afula
Israel Soroka Medical Center Beer Sheva
Israel Rambam Health Care Campus Haifa
Israel Hadassah Medical Center Jerusalem
Israel Shaare Zedek Medical Center Jerusalem
Israel Hospital Sanz Medical Center Laniado Hospital Kiryat Tzanz
Israel Rabin Medical Center, Beilinson Hospital Petach Tikvah
Israel Tel Aviv Sourasky Medical Center Tel Aviv
Italy PO C e G Mazzoni AST Ascoli Piceno Ascoli Piceno
Italy ASST Spedali Civili di Brescia Brescia
Italy AOU Policlinico G Rodolico S Marco Catania
Italy Azienda Ospedaliera di Rilievo Nazionale e di Alta Specializzazione Garibaldi Catania
Italy Azienda Ospedaliero Universitaria Careggi Firenze
Italy Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan
Italy Fondazione IRCCS San Gerardo dei Tintori Monza
Italy Azienda Ospedaliero Universitaria Maggiore della Carita di Novara Novara
Italy Fondazione Policlinico Universitario A Gemelli IRCCS Roma
Italy Umberto I Policlinico di Roma Roma
Italy Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino Torino
Italy Azienda Ulss 8 Berica- Ospedale Di Vicenza Vicenza
Japan Fukushima Medical University Hospital Fukushima
Japan Saitama Medical University Hospital Iruma-gun
Japan St Marianna University Hospital Kanagawa
Japan Japan Community Health care Organization Kyushu Hospital Kitakyusyu-Shi
Japan Yamanashi Prefectural Central Hospital Kofu-Shi
Japan Ogaki Municipal Hospital Ogaki
Japan Sapporo Medical University Hospital Sapporo
Japan Tohoku University Hospital Sendai
Japan Osaka University Hospital Suita
Japan Toyama Prefectural Central Hospital Toyama
Japan Wakayama Medical University Hospital Wakayama
Japan Tottori University Hospital Yonago
Korea, Republic of Kyungpook National University Hospital Daegu
Korea, Republic of Chungnam National University Hospital Daejeon
Korea, Republic of Asan Medical Center Seoul
Korea, Republic of Samsung Medical Center Seoul
Malaysia Hospital Ampang Ampang
Malaysia Hospital Sultanah Aminah Johor Bahru
Malaysia Hospital Queen Elizabeth Kota Kinabalu
Malaysia University Malaya Medical Centre Kuala Lumpur
Malaysia Hospital Tengku Ampuan Afzan Kuantan
Malaysia Hospital Umum Sarawak Kuching
Malaysia Sunway Medical Centre Petaling Jaya
Netherlands Academic Medical Centre Amsterdam Amsterdam
Netherlands Amsterdam Universitair Medische Centra Locatie Vrije Universiteit Medisch Centrum Amsterdam
Netherlands Maastricht Universitair Medisch Centrum Maastricht
Poland Interhem Bialystok
Poland Klinika Hematologii i Transplantologii, UCK Gdansk
Poland Pratia Onkologia Katowice Katowice
Poland Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im M Kopernika w Lodzi Lódz
Poland Szpital Wojewodzki w Opolu Opole
Poland MTZ Clinical Research Powered by Pratia Warszawa
Spain Hosp. Univ. Principe de Asturias Alcalá de Henares
Spain Hosp. Univ. Germans Trias I Pujol Badalona
Spain Hosp Clinic de Barcelona Barcelona
Spain Complejo Asistencial Univ. de Burgos Burgos
Spain Hosp Reina Sofia Córdoba
Spain Hosp. Univ. de Gran Canaria Dr. Negrin Las Palmas de Gran Canaria
Spain Hosp. Univ. 12 de Octubre Madrid
Spain Hosp. Univ. Infanta Leonor Madrid
Spain Hosp. Univ. La Paz Madrid
Spain Hosp. Univ. Ramon Y Cajal Madrid
Spain Hospital Clinico San Carlos Madrid
Spain Hospital Universitario Puerta de Hierro Majadahonda Majadahonda
Spain Hosp. Regional Univ. de Malaga Málaga
Spain Hosp. Virgen de La Victoria Málaga
Spain Hosp. Virgen Del Rocio Málaga
Spain Hosp. Son Llatzer Palma de Mallorca
Spain Clinica Univ. de Navarra Pamplona
Spain Hosp. Quiron Madrid Pozuelo Pozuelo de Alarcon
Spain Hosp. Univ. I Politecni La Fe Valencia
Spain Hosp. Univ. Miguel Servet Zaragoza
Ukraine Cherkassy Regional Oncology Dispensary, Department of Hematology Cherkassy
Ukraine Dnipropetrovsk State Medical Academy, Dnipropetrovsk City Multifield Clinical Hospital # 4 Dnipro
Ukraine Kyiv City Clinical Hospital #9, Department of infectious diseases Kyiv
Ukraine Medical Center 'Ok Clinic' of LLC 'International Institute of Clinical Studies' Kyiv
Ukraine Ternopil University Hospital of Ternopil Regional Council Ternopil
United Kingdom Leeds Teaching Hospitals NHS Trust Leeds
United Kingdom Liverpool University Hospitals NHS Foundation Trust Liverpool
United Kingdom Barts Health NHS Trust London
United Kingdom Oxford University Hospitals NHS Foundation Trust Oxford
United Kingdom University Hospitals Plymouth NHS Trust Plymouth
United Kingdom University Hospital Southampton NHS Foundation Trust Southampton
United States University of Colorado Anschutz Medical Campus Aurora Colorado
United States Rush University Medical Center Chicago Illinois
United States Taussig Cancer Insititute - Cleveland Clinic Cleveland Ohio
United States The Ohio State University- James Cancer Hospital Columbus Ohio
United States GNP Research Cooper City Florida
United States Henry Ford Medical Center Detroit Michigan
United States Duke University Medical Center Durham North Carolina
United States MemorialCare Medical Group Fountain Valley California
United States University of Florida College of Medicine Gainesville Florida
United States East Carolina University Greenville North Carolina
United States 21st Century Oncology Jacksonville Florida
United States Research Medical Center Kansas City Missouri
United States Montefiore Medical Center Lake Success New York
United States Monter Cancer Center Lake Success New York
United States University of Southern California Los Angeles California
United States University of Wisconsin Carbone Cancer Center Madison Wisconsin
United States Alliance for Multispeciality Research Merriam Kansas
United States Lakes Research Miami Lakes Florida
United States Tulane University School of Medicine New Orleans Louisiana
United States Hematology Oncology Associates of Rockland Nyack New York
United States AdventHealth Cancer Institute Orlando Florida
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States Hospital of the University of Pennsylvania Philadelphia Pennsylvania
United States West Penn Hospital Pittsburgh Pennsylvania
United States Compassionate Cancer Care Riverside California
United States Children's Research Institute Saint Petersburg Florida
United States University of Utah Salt Lake City Utah
United States Northwest Medical Specialists Tacoma Washington
United States American Institute of Research Whittier California

Sponsors (1)

Lead Sponsor Collaborator
Janssen Research & Development, LLC

Countries where clinical trial is conducted

United States,  Brazil,  China,  Czechia,  Egypt,  France,  Germany,  Greece,  Hungary,  Israel,  Italy,  Japan,  Korea, Republic of,  Malaysia,  Netherlands,  Poland,  Spain,  Ukraine,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Percentage of Participants Achieving Durable Response of Improvement in Hemoglobin (Hgb) Up to Week 20 of the double-blind period
Secondary Change From Baseline in the Total Score From the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale at the Time of Durable Response The FACIT-Fatigue is a self-administered 13-item questionnaire that assess patient-reported fatigue associated with chronic illness therapy. It assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement. Baseline (Day 1, Week 0) through Week 24
Secondary Change From Baseline in the Total Score From the FACIT-Fatigue Scale at the end of the Double-blind Period (Week 24) The FACIT-Fatigue is a self-administered 13-item questionnaire that assess patient-reported fatigue associated with chronic illness therapy. It assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement. Baseline (Day 1, Week 0) through Week 24 of the double-blind period
Secondary Change from Baseline in Average Daily Dose of Prednisone or Equivalent Change from baseline in average daily dose of prednisone or equivalent at week 24 among participants on prednisone or equivalent at baseline will be reported. Baseline (Day 1, Week 0) and at Week 24
Secondary Number of Participants That Simultaneously Attain Normal Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin Levels at a Minimum of 3 Consecutive Visits After Baseline Baseline (Day 1, Week 0) through Week 24
Secondary Percentage of Participants who Experience at Least a 2 g/dL Increase in Hgb From Baseline and Normalization of Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin at any Time During the Study Baseline (Day 1, Week 0) through Week 24
Secondary Percentage of Participants who Experience at Least a 2 g/dL Increase in Hgb From Baseline and Normalization of Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin at 3 Consecutive Visits Baseline (Day 1, Week 0) through Week 24
Secondary Percentage of Participants who Achieve the Durable Response in Improvement of Hgb During the Double-blind Period and Maintain that Response for Up to 24 Weeks, Without the Need of Rescue Therapy Percentage of participants who achieve the durable response in improvement of Hgb during the double-blind period and maintain that response for up to 24 weeks, without the need of rescue therapy will be reported. Up to 24 weeks
Secondary Change From Baseline in Hgb Concentration Baseline (Day 1, Week 0) through Week 24
Secondary Change From Baseline in Reticulocyte Count Baseline (Day 1, Week 0) through Week 24
Secondary Change From Baseline in Hemolytic Marker - Lactate Dehydrogenase Baseline (Day 1, Week 0) through Week 24
Secondary Change From Baseline in Hemolytic Marker - Haptoglobin Baseline (Day 1, Week 0) through Week 24
Secondary Change From Baseline in Hemolytic Marker - Indirect Bilirubin Baseline (Day 1, Week 0) through Week 24
Secondary Time to Hgb Response Baseline (Day 1, Week 0) through Week 24
Secondary Mean Time During Which the Primary Endpoint is Maintained Baseline (Day 1, Week 0) through Week 24
Secondary Change From Baseline in the Total Score, Item Scores, and Impact and Experience Domains From the FACIT-Fatigue Scale The FACIT-Fatigue scale is a 13-item self-administered questionnaire that assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement. Baseline (Day 1, Week 0) through Week 24 of the double-blind period
Secondary Change From Baseline in EuroQol 5-dimension 5-level ( EQ-5D-5L) Scale Score The EQ-5D-5L quality of life questionnaire will be used to assess health related quality of life status. The 5 dimensions are mobility, self-care, usual activities, pain/discomfort, and anxiety/depression; each dimension is rated by the patient on a 5 level scale (no problems, slight problems, moderate problems, severe problems, extreme problems). Baseline (Day 1, Week 0) through Week 24
Secondary Change From Baseline in Medical Outcomes Study Short Form 36 Item Health Survey Version 2 Acute (SF-36v2) Score The SF-36v2 will be used to assess general quality of life. The 36 items on the SF-36 health survey encompass the following 8 domains: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. The 8 domains can be aggregated into 2 summary scales that reflect physical and mental health: a physical component summary (PCS) and a mental component summary (MCS). Responses to all items are rated on a 3-, 5- or 6-point Likert scale. Higher scores indicate a higher level of functioning. A positive change from baseline score indicates an improvement. Baseline (Day 1, Week 0) through Week 24
Secondary Change From Baseline in Patient Global Impression of Severity (PGIS) The PGIS will be used to assess the severity of warm autoimmune hemolytic anemia (wAIHA) fatigue symptoms. The PGIS is a 5-point response scale. Participant will be asked to rate their fatigue over the past 7 days using the following 5-point scale: 1 = None, 2 = Mild, 3 = Moderate, 4 = Severe, and 5 = Very severe. Baseline (Day 1, Week 0) through Week 24
Secondary Patient-reported Status As Assessed by Patient Global Impression of Change (PGIC) Scale Score The PGIC will assess if there has been an improvement or decline in patient-reported status since the beginning of the treatment. The PGIC is a 7-point response scale. Participants will be asked to rate their current fatigue as compared to when they started the study, using the following 7-point scale: 1 = Much better, 2 = Moderately better, 3 = A little better, 4 = No change, 5 = A little worse, 6 = Moderately worse, and 7 = Much worse. At Week 24
Secondary Hgb Range at Steady State It will be estimated using a model-based longitudinal analysis of Hgb/hemolysis parameters in relationship to IgG level and dose regimen. Baseline (Day 1, Week 0) through Week 24
Secondary Absolute Change from Baseline in Average Daily Dose of Prednisone or Equivalent Absolute change from baseline in average daily dose of prednisone or equivalent at Week 24 among all participants will be reported. Baseline (Day 1, Week 0) and at Week 24
Secondary Percentage of participants who Achieve Corticosteroid Reduction to less than or equal to (<=) 7.5 milligrams per day (mg/day) of Oral Prednisone (or Equivalent), Among Participants with Prednisone or Equivalent greater than (>) 7.5 mg/day at Baseline Percentage of participants who achieve corticosteroid reduction to <= 7.5 mg/day of oral prednisone (or equivalent) at Week 24 of the double-blind period, among participants with prednisone or equivalent >7.5 mg/day at baseline will be reported. At Week 24
See also
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Temporarily not available NCT05221619 - Post-trial Access for Nipocalimab in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)