ILLUMINATE-A, a phase 3 study of lumasiran, an investigational RNAi therapeutic, in children and adults with primary hyperoxaluria type 1 (PH1)

被引：0

作者：

Garrelfs, Sander ^{[1
]}

Frishberg, Yaacov ^{[2
]}

Hulton, Sally ^{[3
]}

Koren, Michael ^{[4
]}

O'Riordan, William ^{[5
]}

Cochat, Pierre ^{[6
,7
,8
]}

Deschenes, Georges ^{[9
]}

Shasha-Lavsky, Hadas ^{[10
]}

Saland, Jeffrey ^{[11
]}

van't Hoff, William ^{[12
]}

Fuster, Daniel G. ^{[13
]}

Magen, Daniella ^{[14
]}

Moochhala, Shabbir ^{[15
]}

Schalk, Gesa ^{[16
]}

Simkova, Eva ^{[17
]}

Groothoff, Jaap ^{[1
]}

Sas, David ^{[18
]}

Meliambro, Kristin ^{[11
]}

Lu, Jiandong ^{[19
]}

Garg, Pushkal ^{[19
]}

Gansner, John ^{[19
]}

McGregor, Tracy ^{[19
]}

Lieske, John ^{[18
]}

机构：

[1] Amsterdam UMC, Dept Pediat Nephrol, Amsterdam, Netherlands

[2] Shaare Zedek Med Ctr, Div Pediat Nephrol, Jerusalem, Israel

[3] Birmingham Childrens Hosp, Dept Nephrol, Birmingham, W Midlands, England

[4] Jacksonville Ctr Clin Res, Jacksonville, FL USA

[5] EStudySite, San Diego, CA USA

[6] Hosp Civils Lyon, Lyon, France

[7] Univ Lyon, Ctr Rare Renal Dis, Lyon, France

[8] INSERM, Pediat Clin Invest Ctr, Lyon, France

[9] Hosp Robert Debre, Dept Pediat Nephrol, Paris, France

[10] Galilee Med Ctr, Nahariyya, Israel

[11] Icahn Sch Med, New York, NY USA

[12] Great Ormond St Hosp Sick Children, Dept Pediat Nephrol, London, England

[13] Univ Bern, Div Nephrol & Hypertens, Inselspital, Univ Hosp Bern, Bern, Switzerland

[14] Rambam Hlth Care Campus, Dept Pediat Nephrol, Haifa, Israel

[15] Royal Free Hosp, London, England

[16] Univ Bonn, Bonn, Germany

[17] Al Jalila Childrens Hosp, Dubai, U Arab Emirates

[18] Mayo Clin, Rochester, MN USA

[19] Alnylam Pharmaceut, Cambridge, MA USA

来源：

SWISS MEDICAL WEEKLY | 2020年

关键词：

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

OC 15

引用

页码：8S / 8S

页数：1

共 50 条

[31] GENETIC-ANALYSIS OF PATIENTS WITH PRIMARY HYPEROXALURIA TYPE-1 (PH1) [J].

FRISHBERG, Y ;

REINER, O .

JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 1994, 5 (03) :621-621

[32] Lumasiran for Patients With Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 12-Month Analysis of the Phase 3 ILLUMINATE-C Trial [J].

Frishberg, Yaacov ;

Michael, Mini ;

Groothoff, Jaap ;

Shasha-Lavsky, Hadas ;

Lieske, John C. ;

Simkova, Eva ;

Sellier-Leclerc, Anne-Laure A. ;

Devresse, Arnaud ;

Egziabher, Fitsum Guebre ;

Bakkaloglu, Sevcan A. ;

Mourani, Chebl ;

Saqan, Rola ;

Singer, Richard F. ;

Willey, Richard G. ;

Gansner, John M. ;

Magen, Daniella .

JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2022, 33 (11) :416-416

[33] IN VIVO GENOME EDITING THERAPY FOR PRIMARY HYPEROXALURIA TYPE I (PH1) [J].

Wang, Zi Jun .

AMERICAN JOURNAL OF KIDNEY DISEASES, 2025, 85 (04)

[34] Treatment preferences among individuals with primary hyperoxaluria type 1 (PH1): a real-world study [J].

Goldfarb, David S. ;

Chen, Jing Voon ;

Zincavage, Rebekah ;

Padilla, Brad ;

Sussman, Matthew ;

Salem, Sandra ;

Modersitzki, Frank .

ORPHANET JOURNAL OF RARE DISEASES, 2025, 20 (01)

[35] Molecular analysis of the AGXT gene in Italian patients with primary hyperoxaluria type 1 (PH1) [J].

Ferrettini, C ;

Pirulli, D ;

Cosseddu, D ;

Marangella, M ;

Petrarulo, M ;

Mazzola, G ;

Vatta, S ;

Amoroso, A .

JOURNAL OF NEPHROLOGY, 1998, 11 :18-22

[36] DEVELOPMENT OF A PHARMACOKINETIC/PHARMACODYNAMIC MODEL OF ALN-GO1, AN INVESTIGATIONAL RNAi THERAPEUTIC FOR PRIMARY HYPEROXALURIA [J].

Attarwala, H. ;

Goel, V. ;

McGregor, T. ;

Erbe, D. ;

Haslett, P. ;

Robbie, G. .

CLINICAL PHARMACOLOGY & THERAPEUTICS, 2018, 103 :S24-S24

[37] ILLUMINATE-C, a Single-Arm, Phase 3 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 and CKD Stages 3b-5, Including Those on Hemodialysis [J].

Michael, Mini ;

Groothoff, Jaap ;

Shasha-Lavsky, Hadas ;

Lieske, John C. ;

Frishberg, Yaacov ;

Simkova, Eva ;

Sellier-Leclerc, Anne-Laure A. ;

Devresse, Arnaud ;

Egziabher, Fitsum Guebre ;

Bakkaloglu, Sevcan A. ;

Mourani, Chebl ;

Saqan, Rola ;

Singer, Richard F. ;

Willey, Richard G. ;

Habtemarian, Bahru A. ;

Bhan, Ishir ;

McGregor, Tracy ;

Magen, Daniella .

JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2021, 32 (10) :B2-B2

[38] Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1 A Placebo-Controlled Randomized Clinical Trial [J].

Frishberg, Yaacov ;

Deschenes, Georges ;

Groothoff, Jaap W. ;

Hulton, Sally-Anne ;

Magen, Daniella ;

Harambat, Jerome ;

van't Hoff, William G. ;

Lorch, Ulrike ;

Milliner, Dawn S. ;

Lieske, John C. ;

Haslett, Patrick ;

Garg, Pushkal P. ;

Vaishnaw, Akshay K. ;

Talamudupula, Sandeep ;

Lu, Jiandong ;

Habtemariam, Bahru A. ;

Erbe, David, V ;

McGregor, Tracy L. ;

Cochat, Pierre .

CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2021, 16 (07) :1025-1036

[39] New Gene Therapy Approach for the Treatment of Primary Hyperoxaluria Type 1 (PH1) Patients. Proof of Concept in a Mouse Model of PH1 [J].

Molinos-Vicente, Andrea ;

Garcia-Torralba, Aida ;

Nieto-Romero, Virginia ;

Rodriguez-Diaz, Saray ;

Cantore, Alessio ;

Roberto Rodriguez-Madoz, Juan ;

Salido, Eduardo ;

Carlos Segovia, Jose ;

Garcia-Bravo, Maria .

MOLECULAR THERAPY, 2021, 29 (04) :246-246

[40] Pre-emptive liver transplantation for primary hyperoxaluria type 1(PH1): A case report [J].

Wang Jun ;

Qu Wei ;

Zeng Zhigui ;

Sun Liying ;

Zhu Zhijun .

PEDIATRIC TRANSPLANTATION, 2019, 23

← 1 2 3 4 5 →