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A Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of Progression

Phase 3
Recruiting
Conditions
Primary IgA Nephropathy
Interventions
Drug: sefaxersen (RO7434656)
Drug: Placebo
Registration Number
NCT05797610
Lead Sponsor
Hoffmann-La Roche
Brief Summary

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of sefaxersen (RO7434656), a novel Antisense Oligonucleotide (ASO) therapy in participants with primary IgA nephropathy (IgAN) who are at high risk of progressive kidney disease despite optimized supportive care.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
428
Inclusion Criteria
  • Primary IgAN, as evidenced by a kidney biopsy performed within 10 years prior to or during screening, without known secondary cause
  • Treatment with maximum tolerated doses of angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs) for at least 90 days immediately prior to screening, except for interruptions due to illness (not greater than 7 consecutive days), unless the potential participant is intolerant to these medications
  • Urine Protein-to-Creatinine Ratio (UPCR) ≥ 1 gram per gram (g/g) or urine protein excretion ≥ 1 gram per day (g/day) (with UPCR ≥ 0.8 g/g), all measured from a 24-hour urine collection during screening
  • eGFR ≥ 20 mL/min/1.73 m^2, as calculated by the 2021 Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation (Inker et al. 2021a)
  • Vaccination against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae according to national vaccination recommendations
  • Female participants of childbearing potential must use adequate contraception
Exclusion Criteria
  • Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 12 weeks after the final dose of sefaxersen
  • Histopathologic or other evidence of another autoimmune glomerular disease
  • Presence of ≥ 50% crescents on kidney biopsy, sustained doubling of serum creatinine within 3 months prior to screening, or rapidly progressive glomerulonephritis in the opinion of the investigator
  • History of kidney transplantation
  • Glycated Hemoglobin (HbA1c) ≥ 6.5% or a clinical diagnosis of diabetes mellitus of any type
  • Systolic blood pressure >140 mmHg or diastolic blood pressure >90 mmHg from the average of two measurements performed at least 1 minute apart during screening
  • Initiation of SGLT2 inhibitors within 16 weeks prior to screening or during screening
  • Initiation of endothelin receptor antagonists within 90 days prior to screening or during screening
  • Initiation of mineralocorticoid receptor antagonists or non-dihydropyridine calcium channel blockers within 90 days prior to screening or during screening
  • Use of herbal therapies within 90 days prior to or during screening
  • Treatment with investigational therapy within 28 days prior to screening or 5.5 drug-elimination half-lives of that investigational product prior to screening
  • Treatment with an investigational therapy planned during the treatment period
  • Previous treatment with sefaxersen
  • Treatment with oral or intravenous (IV) corticosteroids with a dose equivalent to ≥ 7.5 milligrams per day (mg/day) of prednisone for 7 days or equivalent to ≥ 5 mg/day of prednisone for 14 days within 90 days prior to screening
  • Treatment with corticosteroids with systemic effects during screening
  • Treatment with a systemic calcineurin inhibitor within 2 months prior to screening or during screening
  • Treatment with anti-CD20 therapy within 9 months of screening or during screening
  • Treatment with other systemic immunosuppressive agents within 6 months of randomization including, but not limited to, complement inhibitors, alkylating agents (e.g., cyclophosphamide or chlorambucil), azathioprine, or mycophenolate
  • Planned major procedure or major surgery during screening or the study
  • Substance abuse within 12 months prior to screening or during screening
  • Any serious medical condition or abnormality in clinical laboratory tests that precludes an individual's safe participation in and completion of the study
  • History of malignancy within < 5 years prior to screening, with the exception of malignancies with a negligible risk of metastasis or death
  • Usage of GLP-1-based therapy (i.e., GLP-1 mono-agonists, GLP-1/GIP dual agonists, etc.) within 90 days prior to screening or during screening, or intent to initiate during the study period

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
sefaxersen (RO7434656)sefaxersen (RO7434656)Participants will receive subcutaneous (SC) doses of sefaxersen (RO7434656) on Days 1, 15, and 29 followed by once every 4 weeks until Week 105. Participants may be eligible to switch to open-label treatment after Week 105 at the investigator's discretion until up to 1 year after the common-close timepoint, the date when the last participant completes the Week 105 assessment, withdraws, or is discontinued from the study.
PlaceboPlaceboParticipants will receive SC doses of sefaxersen (RO7434656) matching placebo on Days 1, 15, and 29 followed by once every 4 weeks until Week 105. Participants may be eligible to switch to open-label treatment after Week 105 at the investigator's discretion until up to 1 year after the common-close timepoint, the date when the last participant completes the Week 105 assessment, withdraws, or is discontinued from the study.
Primary Outcome Measures
NameTimeMethod
Change From Baseline in the Urine Protein-to-Creatinine Ratio (UPCR) at Week 37Baseline, Week 37

UPCR will be assessed in urine sampled over 24 hours.

Secondary Outcome Measures
NameTimeMethod
Estimated Glomerular Filtration Rate (eGFR) Slope at Week 105 from BaselineBaseline, Week 105

eGFR will be calculated using the 2021 Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation.

Time to the Composite Kidney Failure EndpointUp to approximately 7 years

Time to the composite kidney failure endpoint is defined as receipt of kidney transplantation, need for kidney replacement therapy, or a sustained decline in eGFR of ≥ 30% or a sustained eGFR \<15 mL/min/1.73m\^ 2 over at least 4 weeks (both eGFR criteria requires two consecutive central laboratory eGFR values meeting criteria ≥ 4 weeks apart), whichever occurs first, without the receipt of other immunosuppressive or background therapies for the treatment of IgAN.

Change From Baseline in Fatigue at Week 105Baseline, Week 105

Fatigue will be assessed with the Functional Assessment of Chronic Illness Therapy-Fatigue subscale (FACIT-F). The FACIT-F Scale is a 13-item scale used to measure self-reported fatigue. Items are assessed on a 5-point Likert scale, with responses ranging from 0 for "not at all" to 4 for "very much". The total raw score is the sum of the values of each scored question and ranges from 0 to 52. A higher score indicates less fatigue.

Percentage of Participants with Treatment-Emergent Adverse Events (TEAEs)Up to approximately 7 years
Plasma Concentration of sefaxersenUp to approximately 7 years

Trial Locations

Locations (177)

Hopital Bichat - Claude Bernard AP-HP

🇫🇷

Paris, France

Hôpital de Rangueil

🇫🇷

Toulouse, France

Hospital Universitario La Paz - PPDS

🇪🇸

Madrid, Spain

Universitatsklinikum der RWTH Aachen

🇩🇪

Aachen, Germany

Klinikum Koln-Merheim

🇩🇪

Köln, Germany

Hopital Henri Mondor

🇫🇷

Creteil, France

UAB Nephrology Research Clinic

🇺🇸

Birmingham, Alabama, United States

Sunrise Medical Management LLC

🇺🇸

Surprise, Arizona, United States

Tucson Neuroscience Research - M3 WR

🇺🇸

Tucson, Arizona, United States

Southern California Medical Research Center

🇺🇸

La Palma, California, United States

Academic Medical Research Institute - Los Angeles

🇺🇸

Los Angeles, California, United States

UCLA University of California Los Angeles

🇺🇸

Los Angeles, California, United States

North America Research Institute-San Dimas

🇺🇸

San Dimas, California, United States

Hope Clinic & Research Center

🇺🇸

Hialeah, Florida, United States

LCC Medical Research - Miami - ClinEdge - PPDS

🇺🇸

Miami, Florida, United States

L&C Professional Medical Research Institute

🇺🇸

W. Miami, Florida, United States

Cowry Medical Group LLC

🇺🇸

Acworth, Georgia, United States

Care Institute Idaho Kidney Institute

🇺🇸

Chubbuck, Idaho, United States

Nephrology Associates of Northern Illinois

🇺🇸

Hinsdale, Illinois, United States

University of Iowa

🇺🇸

Iowa City, Iowa, United States

Massachussets General Hospital

🇺🇸

Boston, Massachusetts, United States

University of Mississippi Medical Center

🇺🇸

Jackson, Mississippi, United States

Sierra Nevada Nephrology Consultants

🇺🇸

Reno, Nevada, United States

NYU Langone Nephrology Associates - Mineola

🇺🇸

Mineola, New York, United States

North Carolina Nephrology, PA

🇺🇸

Raleigh, North Carolina, United States

Ohio State University Wexner Medical Center - Outpatient Care East

🇺🇸

Columbus, Ohio, United States

Kidney and Hypertension Center

🇺🇸

Roseburg, Oregon, United States

Global Medical Research - M3 WR

🇺🇸

Dallas, Texas, United States

Texas Kidney Institute - Dallas

🇺🇸

Dallas, Texas, United States

Pioneer Research Solutions

🇺🇸

Houston, Texas, United States

Prolato Clinical Research Center

🇺🇸

Houston, Texas, United States

R & H Clinical Research

🇺🇸

Katy, Texas, United States

Revival Research Corporation - Sherman - ClinEdge - PPDS

🇺🇸

Sherman, Texas, United States

Nephrology Associates of Northern Virginia Inc

🇺🇸

Fairfax, Virginia, United States

Virginia Commonwealth University

🇺🇸

Richmond, Virginia, United States

Milwaukee Nephrologists, Sc

🇺🇸

Wauwatosa, Wisconsin, United States

Centro Medico Dra. Laura Maffei- Investigacion Clinica Aplicada

🇦🇷

Ciudad Autonoma Buenos Aires, Argentina

Consultorios Médicos Dr. Doreski

🇦🇷

Ciudad Autonoma Buenos Aires, Argentina

Sanatorio Mayo Privado

🇦🇷

Cordoba, Argentina

Sanatorio Allende

🇦🇷

Cordoba, Argentina

Instituto Medico de la Fundacion Estudios Clinicos

🇦🇷

Rosario, Argentina

Nepean Hospital

🇦🇺

Kingswood, New South Wales, Australia

St George Hospital

🇦🇺

Kogarah, New South Wales, Australia

Liverpool Hospital

🇦🇺

Liverpool, New South Wales, Australia

Princess Alexandra Hospital

🇦🇺

Woolloongabba, Queensland, Australia

Royal Adelaide Hospital

🇦🇺

Adelaide, South Australia, Australia

Royal Melbourne Hospital

🇦🇺

Parkville, Victoria, Australia

Sunshine Hospital

🇦🇺

St Albans, Victoria, Australia

Santa Casa de Misericordia de Belo Horizonte - PPDS

🇧🇷

Belo Horizonte, Minas Gerais, Brazil

Freire Pesquisa Clinica

🇧🇷

Belo Horizonte, Minas Gerais, Brazil

Hospital de Clinicas de Porto Alegre HCPA PPDS

🇧🇷

Porto Alegre, Rio Grande Do Sul, Brazil

Centro de Pesquisa Clinica da Fundação Pró Rim

🇧🇷

Joinville, Santa Catarina, Brazil

Faculdade de Medicina da Universidade de Sao Paulo

🇧🇷

Sao Paulo, São Paulo, Brazil

Hospital Do Rim E Hipertensao Fundacao Oswaldo Ramos

🇧🇷

Sao Paulo, São Paulo, Brazil

Instituto D?Or Pesquisa e Ensino - Hospital Gloria D?Or

🇧🇷

Rio de Janeiro, Brazil

Vancouver General Hospital

🇨🇦

Vancouver, British Columbia, Canada

Cape Breton Regional Hospital

🇨🇦

Sydney, Nova Scotia, Canada

Hopital Tenon

🇫🇷

Paris, France

London Health Sciences Centre · Victoria Hospital

🇨🇦

London, Ontario, Canada

Sunnybrook Health Sciences Centre

🇨🇦

Toronto, Ontario, Canada

Centre Hospitalier Universitaire de Quebec

🇨🇦

Quebec, Canada

Beijing Friendship Hospital, Capital Medical University - PPDS

🇨🇳

Beijing, Beijing, China

Peking University First Hospital

🇨🇳

Beijing City, China

Peking University People's Hospital

🇨🇳

Beijing, China

Changzhou First People's Hospital

🇨🇳

Changzhou, China

Sichuan Academy of Medical Sciences and Sichuan Provincial Peoples Hospital

🇨🇳

Chengdu City, China

Guangdong Provincial People's Hospital

🇨🇳

Guangzhou City, China

Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University

🇨🇳

Hangzhou City, China

Zhejiang Provincial People?s Hospital

🇨🇳

Hangzhou, China

The Second Affiliated Hospital of Nanchang University

🇨🇳

Nanchang City, China

The Second Affiliated Hospital of Nanjing Medical University

🇨🇳

Nanjing, China

Ningbo No.2 Hospital

🇨🇳

Ningbo City, China

Ruijin Hospital Shanghai Jiaotong University School of Medicine

🇨🇳

Shanghai City, China

Xinhua Hospital Affiliated To Shanghai Jiaotong University School of Medicine

🇨🇳

Shanghai, China

Shenzhen People's Hospital

🇨🇳

Shenzhen City, China

The University of Hong Kong - Shenzhen Hospital

🇨🇳

Shenzhen City, China

The Second Affiliated Hospital of Soochow University

🇨🇳

Suzhou, China

Renmin Hospital of Wuhan University

🇨🇳

Wuhan, China

The First Affiliated Hospital of Xian Jiao Tong University

🇨🇳

Xi'an City, China

General Hospital of Ningxia Medical University

🇨🇳

Yinchuan, China

Affiliated Hospital of Jiangsu University

🇨🇳

Zhenjiang, China

Fakultni nemocnice Hradec Kralove

🇨🇿

Hradec Kralove, Czechia

Vseobecna fakultni nemocnice v Praze

🇨🇿

Praha 2, Czechia

Centre Hospitalier

🇫🇷

Annonay, France

CHU Boulogne sur Mer

🇫🇷

Boulogne Sur Mer, France

Universitätsmedizin der Johannes Gutenberg-Universität Mainz

🇩🇪

Mainz, Germany

University General Hospital of Heraklion

🇬🇷

Heraklion, Greece

Venizeleio General Hospital of Heraklion

🇬🇷

Irakleio Kritis, Greece

University General Hospital of Patras

🇬🇷

Patra, Greece

Ippokratio General Hospital of Thessaloniki

🇬🇷

Thessaloniki, Greece

Queen Mary Hospital

🇭🇰

Hong Kong, Hong Kong

Azienda Ospedaliera Universitaria Federico II

🇮🇹

Napoli, Campania, Italy

Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi-Via Massarenti

🇮🇹

Bologna, Emilia-Romagna, Italy

Fondazione Policlinico Universitario A Gemelli

🇮🇹

Roma, Lazio, Italy

Ospedale Policlinico San Martino

🇮🇹

Genova, Liguria, Italy

IRCCS Pavia - Istituti Clinici Scientifici Maugeri Spa ? Società Benefit

🇮🇹

Pavia, Lombardia, Italy

Ospedale San Giovanni Bosco

🇮🇹

Torino, Piemonte, Italy

Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari

🇮🇹

Bari, Puglia, Italy

Fujita Health University Hospital

🇯🇵

Aichi, Japan

Hirosaki University Hospital

🇯🇵

Aomori, Japan

Juntendo University Urayasu Hospital

🇯🇵

Chiba, Japan

University of Yamanashi Hospital

🇯🇵

Chuo, Japan

Matsuyama Red Cross Hospital

🇯🇵

Ehime, Japan

Japanese Red Cross Fukuoka Hospital

🇯🇵

Fukuoka, Japan

Hiroshima University Hospital

🇯🇵

Hiroshima, Japan

Hokkaido University Hospital

🇯🇵

Hokkaido, Japan

Tokyo Medical University Ibaraki Medical Center

🇯🇵

Inashiki-Gun, Japan

Kanazawa Medical University Hospital

🇯🇵

Ishikawa, Japan

Iwate Medical University Hospital

🇯🇵

Iwate, Japan

Nara Medical University Hospital

🇯🇵

Kashihara, Japan

Kumamoto University Hospital

🇯🇵

Kumamoto, Japan

Tohoku University Hospital

🇯🇵

Miyagi, Japan

Kyoto City Hospital

🇯🇵

Nakagyo-ku, Japan

Nara Prefecture General Medical Center

🇯🇵

Nara, Japan

Hyogo Prefectural Nishinomiya Hospital

🇯🇵

Nishinomiya, Japan

Okayama University Hospital

🇯🇵

Okayama-Shi Kita-Ku, Japan

National Hospital Organization Okayama Medical Center

🇯🇵

Okayama, Japan

University of the Ryukyus Hospital

🇯🇵

Okinawa, Japan

Saitama Medical University Hospital

🇯🇵

Saitama, Japan

Osaka University Hospital

🇯🇵

Suita, Japan

Jichi Medical University Hospital

🇯🇵

Tochigi, Japan

Tokushima University Hospital

🇯🇵

Tokushima, Japan

The Jikei University Hospital

🇯🇵

Tokyo, Japan

Juntendo University Hospital

🇯🇵

Tokyo, Japan

Tokyo Women's Medical University Hospital

🇯🇵

Tokyo, Japan

Juntendo University Nerima Hospital, department of renal and hypertension

🇯🇵

Tokyo, Japan

University of Tsukuba Hospital

🇯🇵

Tsukuba, Japan

Yamaguchi University Hospital

🇯🇵

Ube, Japan

Hallym University Sacred Heart Hospital

🇰🇷

Anyang-si, Korea, Republic of

Soon Chun Hyang University Hospital Bucheon

🇰🇷

Bucheon-si,, Korea, Republic of

Chungbuk National University Hospital

🇰🇷

Cheongju si, Korea, Republic of

Ajou University Hospital

🇰🇷

Gyeonggi-do, Korea, Republic of

CHA Bundang Medical Center, CHA University

🇰🇷

Seongnam-si, Korea, Republic of

Seoul National University Bundang Hospital

🇰🇷

Seongnam-si, Korea, Republic of

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

Konkuk University Medical Center

🇰🇷

Seoul, Korea, Republic of

Gangdong Kyung Hee University Hospital

🇰🇷

Seoul, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

SMG-SNU Boramae Medical Center

🇰🇷

Seoul, Korea, Republic of

Hospital Kuala Lumpur

🇲🇾

Kuala Lumpur, FED. Territory OF Kuala Lumpur, Malaysia

University Malaya Medical Centre

🇲🇾

Kuala Lumpur, FED. Territory OF Kuala Lumpur, Malaysia

Hospital Tengku Ampuan Afzan

🇲🇾

Kuantan, Pahang, Malaysia

Hospital Seri Manjung

🇲🇾

Seri Manjong, Pahang, Malaysia

Taiping Hospital

🇲🇾

Taiping, Perak, Malaysia

Hospital Sibu

🇲🇾

Sibu, Sarawak, Malaysia

Hospital Ampang

🇲🇾

Ampang, Malaysia

Miri Hospital

🇲🇾

Miri, Malaysia

Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran

🇲🇽

Barrio Belisario Domínguez Secc XVI, Mexico CITY (federal District), Mexico

Centro de Investigacion Clinica Chapultepec S. A. de C. V.

🇲🇽

Morelia, Michoacan, Mexico

Instituto Mexicano de Trasplantes

🇲🇽

Cuernavaca, Morelos, Mexico

SCM Clinic - Prywatny Szpital Specjalistyczny

🇵🇱

Kraków, Poland

SPZOZ Centralny Szpital Klin

🇵🇱

Lodz, Poland

Szpital Kliniczny im. H.Swiecickiego UM w Poznaniu

🇵🇱

Pozna?, Poland

Szpital Kliniczny Dzieciatka Jezus

🇵🇱

Warszawa, Poland

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu

🇵🇱

Wroc?aw, Poland

National University Hospital

🇸🇬

Singapore, Singapore

Singapore General Hospital

🇸🇬

Singapore, Singapore

Tan Tock Seng Hospital

🇸🇬

Singapore, Singapore

Changi General Hospital

🇸🇬

Singapore, Singapore

Hospital Universitari Arnau de Vilanova

🇪🇸

Lleida, Lerida, Spain

Hospital Universitario Vall d'Hebron - PPDS

🇪🇸

Barcelona, Spain

Hospital Ribera Polusa

🇪🇸

Lugo, Spain

Hospital Universitario 12 De Octubre

🇪🇸

Madrid, Spain

Hospital Universitario Virgen del Rocio - PPDS

🇪🇸

Sevilla, Spain

Changhua Christian Hospital

🇨🇳

Changhua, Taiwan

Chang Gung Medical Foundation

🇨🇳

Kaohsiung, Taiwan

China Medical University Hospital

🇨🇳

Taichung, Taiwan

Far Eastern Memorial Hospital

🇨🇳

Taipei, Taiwan

Chang Gung Memorial Hospital, Linkou

🇨🇳

Taoyuan City, Taiwan

Taichung Veterans General Hospital

🇨🇳

Xitun Dist., Taiwan

Mackay Memorial Hospital-Taipei branch

🇨🇳

Zhongshan Dist., Taiwan

Addenbrookes Hospital

🇬🇧

Cambridge, United Kingdom

Leicester General Hospital

🇬🇧

Leicester, United Kingdom

Guys and St Thomas Hospital

🇬🇧

London, United Kingdom

Kings College Hospital

🇬🇧

London, United Kingdom

Churchill Hospital

🇬🇧

Oxford, United Kingdom

Salford Royal Hospital

🇬🇧

Salford, United Kingdom

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