Clinical trial NCT04119050: Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia
Official title: Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia: A Multicenter, Randomized, Double-blind, Placebo-controlled Study With a Long-term Open-label Extension
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Warm Autoimmune Hemolytic Anemia
Keywords: Warm Autoimmune Hemolytic Anemia
M281 (Nipocalimab)
wAIHA
JNJ-80202135
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2019-10-08
Enrollment: 111
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 efficacy and safety of M281 in participants with warm autoimmune hemolytic anemia (wAIHA).

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Percentage of Participants Achieving Durable Response of Improvement in Hemoglobin (Hgb)
timeFrame: Up to Week 20 of the double-blind period
Secondary outcomes:
measure: 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
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Change From Baseline in the Total Score From the FACIT-Fatigue Scale at the end of the Double-blind Period (Week 24)
timeFrame: Baseline (Day 1, Week 0) through Week 24 of the double-blind period
measure: Change from Baseline in Average Daily Dose of Prednisone or Equivalent
timeFrame: Baseline (Day 1, Week 0) and at Week 24
measure: Number of Participants That Simultaneously Attain Normal Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin Levels at a Minimum of 3 Consecutive Visits After Baseline
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: 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
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: 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
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: 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
timeFrame: Up to 24 weeks
measure: Change From Baseline in Hgb Concentration
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Change From Baseline in Reticulocyte Count
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Change From Baseline in Hemolytic Marker - Lactate Dehydrogenase
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Change From Baseline in Hemolytic Marker - Haptoglobin
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Change From Baseline in Hemolytic Marker - Indirect Bilirubin
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Time to Hgb Response
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Mean Time During Which the Primary Endpoint is Maintained
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Change From Baseline in the Total Score, Item Scores, and Impact and Experience Domains From the FACIT-Fatigue Scale
timeFrame: Baseline (Day 1, Week 0) through Week 24 of the double-blind period
measure: Change From Baseline in EuroQol 5-dimension 5-level ( EQ-5D-5L) Scale Score
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Change From Baseline in Medical Outcomes Study Short Form 36 Item Health Survey Version 2 Acute (SF-36v2) Score
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Change From Baseline in Patient Global Impression of Severity (PGIS)
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Patient-reported Status As Assessed by Patient Global Impression of Change (PGIC) Scale Score
timeFrame: At Week 24
measure: Hgb Range at Steady State
timeFrame: Baseline (Day 1, Week 0) through Week 24
measure: Absolute Change from Baseline in Average Daily Dose of Prednisone or Equivalent
timeFrame: Baseline (Day 1, Week 0) and at Week 24
measure: 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
timeFrame: At Week 24
Other outcomes:
Locations:
city: Fountain Valley
country: United States
facility: MemorialCare Medical Group
state: California
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Riverside
country: United States
facility: Compassionate Cancer Care
state: California
city: Whittier
country: United States
facility: American Institute of Research
state: California
city: Aurora
country: United States
facility: University of Colorado Anschutz Medical Campus
state: Colorado
city: Cooper City
country: United States
facility: GNP Research
state: Florida
city: Gainesville
country: United States
facility: University of Florida College of Medicine
state: Florida
city: Jacksonville
country: United States
facility: 21st Century Oncology
state: Florida
city: Miami Lakes
country: United States
facility: Lakes Research
state: Florida
city: Orlando
country: United States
facility: AdventHealth Cancer Institute
state: Florida
city: Saint Petersburg
country: United States
facility: Children's Research Institute
state: Florida
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Merriam
country: United States
facility: Alliance for Multispeciality Research
state: Kansas
city: New Orleans
country: United States
facility: Tulane University School of Medicine
state: Louisiana
city: Detroit
country: United States
facility: Henry Ford Medical Center
state: Michigan
city: Kansas City
country: United States
facility: Research Medical Center
state: Missouri
city: Lake Success
country: United States
facility: Montefiore Medical Center
state: New York
city: Lake Success
country: United States
facility: Monter Cancer Center
state: New York
city: Nyack
country: United States
facility: Hematology Oncology Associates of Rockland
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Cleveland
country: United States
facility: Taussig Cancer Insititute Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University- James Cancer Hospital
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: West Penn Hospital
state: Pennsylvania
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Tacoma
country: United States
facility: Northwest Medical Specialists
state: Washington
city: Madison
country: United States
facility: University of Wisconsin Carbone Cancer Center
state: Wisconsin
city: Botucatu
country: Brazil
facility: Hospital das Clínicas da Faculdade de Medicina de Botucatu
city: Brasilia
country: Brazil
facility: Impar Servicos Hospitalares S/A - Hospital Brasilia
city: Campinas
country: Brazil
facility: CTO Centro de Tratamento Oncologico Unidade Belem
city: Goiânia
country: Brazil
facility: Hospital Das Clinicas Da Universidade Federal De Goias
city: Niterói
country: Brazil
facility: Complexo Hospitalar de Niteroi
city: Recife
country: Brazil
facility: Oncoclinicas - Unidade OC Oncoclinicas Multihemo Ilha do Leite
city: Salvador
country: Brazil
facility: Nucleo de Oncologia da Bahia
city: Salvador
country: Brazil
facility: Hospital Sao Rafael
city: Santo Andre
country: Brazil
facility: CEPHO Centro de Estudos e Pesquisa de Hematologia e Oncologia
city: Santo Andre
country: Brazil
facility: Hospital Santa Marcelina
city: Sao Jose Do Rio Preto
country: Brazil
facility: Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto Hospital de Base
city: Sao Paulo
country: Brazil
facility: Sociedade Beneficente de Senhoras Hospital Sirio Libanes
city: Sao Paulo
country: Brazil
facility: Universidade Federal de Sao Paulo
city: Sao Paulo
country: Brazil
facility: Hospital Das Clinicas Da Faculdade De Medicina Da USP
city: Beijing Shi
country: China
facility: Beijing Chao-yang Hospital, Capital Medical University
city: Beijing Shi
country: China
facility: Peking University People s Hospital
city: Changchun
country: China
facility: The First Hospital of Jilin University
city: Chengdu
country: China
facility: West China Hospital of Sichuan University
city: Guangzhou Shi
country: China
facility: Guangzhou First Municipal People's Hospital
city: Haikou
country: China
facility: Hainan General Hospital
city: Hangzhou
country: China
facility: First Affiliated Hospital Medical School of Zhejiang University
city: Lanzhou
country: China
facility: Lanzhou University Second Hospital
city: Nanjing
country: China
facility: Jiangsu Province Hospital
city: Nantong
country: China
facility: Affiliated Hospital of Nantong University
city: ShangHai
country: China
facility: Shanghai Zhongshan Hospital
city: Shanghai
country: China
facility: Huashan Hospital affiliated to Fudan University
city: Suzhou
country: China
facility: The First Affiliated Hospital of Soochow University
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
city: Wuhan
country: China
facility: Union Hospital Tongji Medical College of Huazhong University of Science and Technology
city: Xi'An
country: China
facility: The First Affiliated Hospital of Xian Jiaotong University
city: Brno
country: Czechia
facility: Fakultni nemocnice Brno
city: Praha 10
country: Czechia
facility: Fakultni nemocnice Kralovske Vinohrady
city: Praha 2
country: Czechia
facility: Ustav Hematologie A Krevni Transfuze
city: Alexandria
country: Egypt
facility: Alexandria University Hospital
city: Cairo
country: Egypt
facility: Cairo university
city: Cairo
country: Egypt
facility: Ain Shams University Hospital
city: Cairo
country: Egypt
facility: National Cancer Institute
city: Cairo
country: Egypt
facility: Nasser institute Hospital
city: Mansoura
country: Egypt
facility: Mansoura University Hospital
city: Brest Cedex 2
country: France
facility: ICH Hopital A. Morvan
city: Dijon
country: France
facility: CHU Dijon Bourgogne
city: La Tronche
country: France
facility: CHU Grenoble
city: Nantes
country: France
facility: CHU Nantes - Hotel Dieu
city: Poitiers
country: France
facility: CHU De Poitiers
city: Rennes
country: France
facility: CHRU Hopital Sud
city: Berlin
country: Germany
facility: Onkologische Schwerpunktpraxis
city: Essen
country: Germany
facility: Universitatsklinikum Essen
city: München
country: Germany
facility: Rotkreuzklinikum München
city: Athens
country: Greece
facility: General Hospital of Athens G Gennimatas
city: Athens
country: Greece
facility: Laiko General Hospital of Athens
city: Athens
country: Greece
facility: Attikon University General Hospital of Attica
city: Larissa
country: Greece
facility: University Hospital Of Larissa
city: Patras
country: Greece
facility: University General Hospital of Rio Patras
city: Thessaloniki
country: Greece
facility: University General Hospital of Thessaloniki
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Budapest
country: Hungary
facility: Del Pesti Centrumkorhaz Orszagos Hematologiai es Infektologiai Intezet Szent Laszlo Telephely
city: Debrecen
country: Hungary
facility: Debreceni Egyetem Klinikai Kozpont
city: Gyõr
country: Hungary
facility: Petz Aladar Megyei Oktato Korhaz
city: Kaposvar
country: Hungary
facility: Somogy Megyei Kaposi Mor Oktato Korhaz
city: Afula
country: Israel
facility: Ha'Emek Medical Center
city: Beer Sheva
country: Israel
facility: Soroka Medical Center
city: Haifa
country: Israel
facility: Rambam Health Care Campus
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
city: Jerusalem
country: Israel
facility: Hadassah Medical Center
city: Kiryat Tzanz
country: Israel
facility: Hospital Sanz Medical Center Laniado Hospital
city: Petach Tikvah
country: Israel
facility: Rabin Medical Center, Beilinson Hospital
city: Tel Aviv
country: Israel
facility: Tel Aviv Sourasky Medical Center
city: Ascoli Piceno
country: Italy
facility: PO C e G Mazzoni AST Ascoli Piceno
city: Brescia
country: Italy
facility: ASST Spedali Civili di Brescia
city: Catania
country: Italy
facility: Azienda Ospedaliera di Rilievo Nazionale e di Alta Specializzazione Garibaldi
city: Catania
country: Italy
facility: AOU Policlinico G Rodolico S Marco
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Careggi
city: Milan
country: Italy
facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
city: Monza
country: Italy
facility: Fondazione IRCCS San Gerardo dei Tintori
city: Novara
country: Italy
facility: Azienda Ospedaliero Universitaria Maggiore della Carita di Novara
city: Roma
country: Italy
facility: Umberto I Policlinico di Roma
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario A Gemelli IRCCS
city: Torino
country: Italy
facility: Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino
city: Vicenza
country: Italy
facility: Azienda Ulss 8 Berica- Ospedale Di Vicenza
city: Fukushima
country: Japan
facility: Fukushima Medical University Hospital
city: Iruma-gun
country: Japan
facility: Saitama Medical University Hospital
city: Kanagawa
country: Japan
facility: St Marianna University Hospital
city: Kitakyusyu-Shi
country: Japan
facility: Japan Community Health care Organization Kyushu Hospital
city: Kofu-Shi
country: Japan
facility: Yamanashi Prefectural Central Hospital
city: Ogaki
country: Japan
facility: Ogaki Municipal Hospital
city: Sapporo
country: Japan
facility: Sapporo Medical University Hospital
city: Sendai
country: Japan
facility: Tohoku University Hospital
city: Suita
country: Japan
facility: Osaka University Hospital
city: Toyama
country: Japan
facility: Toyama Prefectural Central Hospital
city: Wakayama
country: Japan
facility: Wakayama Medical University Hospital
city: Yonago
country: Japan
facility: Tottori University Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Hospital
city: Daejeon
country: Korea, Republic of
facility: Chungnam National University Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Ampang
country: Malaysia
facility: Hospital Ampang
city: Johor Bahru
country: Malaysia
facility: Hospital Sultanah Aminah
city: Kota Kinabalu
country: Malaysia
facility: Hospital Queen Elizabeth
city: Kuala Lumpur
country: Malaysia
facility: University Malaya Medical Centre
city: Kuantan
country: Malaysia
facility: Hospital Tengku Ampuan Afzan
city: Kuching
country: Malaysia
facility: Hospital Umum Sarawak
city: Petaling Jaya
country: Malaysia
facility: Sunway Medical Centre
city: Amsterdam
country: Netherlands
facility: Amsterdam Universitair Medische Centra Locatie Vrije Universiteit Medisch Centrum
city: Amsterdam
country: Netherlands
facility: Academic Medical Centre Amsterdam
city: Maastricht
country: Netherlands
facility: Maastricht Universitair Medisch Centrum
city: Bialystok
country: Poland
facility: Interhem
city: Gdansk
country: Poland
facility: Klinika Hematologii i Transplantologii, UCK
city: Katowice
country: Poland
facility: Pratia Onkologia Katowice
city: Lodz
country: Poland
facility: Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im M Kopernika w Lodzi
city: Opole
country: Poland
facility: Szpital Wojewodzki w Opolu
city: Warszawa
country: Poland
facility: MTZ Clinical Research Powered by Pratia
city: Alcalá de Henares
country: Spain
facility: Hosp. Univ. Principe de Asturias
city: Badalona
country: Spain
facility: Hosp. Univ. Germans Trias I Pujol
city: Barcelona
country: Spain
facility: Hosp Clinic de Barcelona
city: Burgos
country: Spain
facility: Complejo Asistencial Univ. de Burgos
city: Córdoba
country: Spain
facility: Hosp Reina Sofia
city: Las Palmas de Gran Canaria
country: Spain
facility: Hosp. Univ. de Gran Canaria Dr. Negrin
city: Madrid
country: Spain
facility: Hosp. Univ. Infanta Leonor
city: Madrid
country: Spain
facility: Hosp. Univ. Ramon Y Cajal
city: Madrid
country: Spain
facility: Hospital Clinico San Carlos
city: Madrid
country: Spain
facility: Hosp. Univ. 12 de Octubre
city: Madrid
country: Spain
facility: Hosp. Univ. La Paz
city: Majadahonda
country: Spain
facility: Hospital Universitario Puerta de Hierro Majadahonda
city: Málaga
country: Spain
facility: Hosp Regional Univ de Malaga
city: Málaga
country: Spain
facility: Hosp Virgen de La Victoria
city: Málaga
country: Spain
facility: Hosp. Virgen Del Rocio
city: Palma de Mallorca
country: Spain
facility: Hosp. Son Llatzer
city: Pamplona
country: Spain
facility: Clinica Univ. de Navarra
city: Pozuelo de Alarcon
country: Spain
facility: Hosp. Quiron Madrid Pozuelo
city: Valencia
country: Spain
facility: Hosp. Univ. I Politecni La Fe
city: Zaragoza
country: Spain
facility: Hosp. Univ. Miguel Servet
city: Cherkassy
country: Ukraine
facility: Cherkassy Regional Oncology Dispensary, Department of Hematology
city: Dnipro
country: Ukraine
facility: Dnipropetrovsk State Medical Academy, Dnipropetrovsk City Multifield Clinical Hospital # 4
city: Kyiv
country: Ukraine
facility: Medical Center 'Ok Clinic' of LLC 'International Institute of Clinical Studies'
city: Kyiv
country: Ukraine
facility: Kyiv City Clinical Hospital #9, Department of infectious diseases
city: Ternopil
country: Ukraine
facility: Ternopil University Hospital of Ternopil Regional Council
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospitals NHS Trust
city: Liverpool
country: United Kingdom
facility: Liverpool University Hospitals NHS Foundation Trust
city: London
country: United Kingdom
facility: Barts Health NHS Trust
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Foundation Trust
city: Plymouth
country: United Kingdom
facility: University Hospitals Plymouth NHS Trust
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References:
citation: Liu AP, Cheuk DK. Disease-modifying treatments for primary autoimmune haemolytic anaemia. Cochrane Database Syst Rev. 2021 Mar 26;3(3):CD012493. doi: 10.1002/14651858.CD012493.pub2.