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

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[21] LUMASIRAN LOWERED URINARY OXALATE IN PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1 IRRESPECTIVE OF PYRIDOXINE USE, HYDRATION STATUS, AND GENOTYPE IN THE PHASE 3 CLINICAL TRIAL ILLUMINATE-A [J].

Saland, Jeffrey ;

Ngo, Taylor ;

Gansner, John ;

McGregor, Tracy ;

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AMERICAN JOURNAL OF KIDNEY DISEASES, 2021, 77 (04) :644-644

[22] DIGESTIVE MANIFESTATIONS OF PRIMARY HYPEROXALURIA TYPE1(PH1) IN CHILDREN [J].

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Tfifha, Meniar ;

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[23] THE CHALLENGE DIAGNOSTIC AND THERAPEUTIC OF PRIMARY HYPEROXALURIA TYPE 1 (PH1): A CASE REPORT [J].

Malgieri, Gabriele ;

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[24] LONG-TERM EFFICACYAND SAFETY IN THE PHASE 3 ILLUMINATE-B TRIAL OF LUMASIRAN FOR PRIMARY HYPEROXALURIA TYPE 1 IN INFANTS AND YOUNG CHILDREN [J].

Frishberg, Yaacov ;

Hayes, Wesley ;

Ben-Shalom, Efrat ;

Shasha-Lavsky, Hadas ;

Sas, David J. ;

Michael, Mini ;

Sellier-Leclerc, Anne-Laure ;

Hogan, Julien ;

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Murphy, Desmond ;

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AMERICAN JOURNAL OF KIDNEY DISEASES, 2025, 85 (05) :668-669

[25] Identification of new mutations in primary hyperoxaluria type 1 (PH1) [J].

von Schnakenburg, C ;

Rumsby, G .

JOURNAL OF NEPHROLOGY, 1998, 11 :15-17

[26] LUMASIRAN DEMONSTRATED COMPARABLE OXALATE REDUCTION AND SAFETY IN CHILDREN AND ADULTS WITH PRIMARY HYPEROXALURIA TYPE 1 [J].

Shasha-Lavsky, Hadas ;

Garrelfs, Sander ;

Sas, David ;

Lieske, John ;

Ngo, Taylor ;

Makarova, Nune ;

Gansner, John ;

McGregor, Tracy ;

Frishberg, Yaacov .

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[27] Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial [J].

Hayes, Wesley ;

Sas, David J. ;

Magen, Daniella ;

Shasha-Lavsky, Hadas ;

Michael, Mini ;

Sellier-Leclerc, Anne-Laure ;

Hogan, Julien ;

Ngo, Taylor ;

Sweetser, Marianne T. ;

Gansner, John M. ;

McGregor, Tracy L. ;

Frishberg, Yaacov .

PEDIATRIC NEPHROLOGY, 2023, 38 (04) :1075-1086

[28] Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial [J].

Wesley Hayes ;

David J. Sas ;

Daniella Magen ;

Hadas Shasha-Lavsky ;

Mini Michael ;

Anne-Laure Sellier-Leclerc ;

Julien Hogan ;

Taylor Ngo ;

Marianne T. Sweetser ;

John M. Gansner ;

Tracy L. McGregor ;

Yaacov Frishberg .

Pediatric Nephrology, 2023, 38 :1075-1086

[29] Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial [J].

Frishberg, Yaacov ;

Hayes, Wesley ;

Shasha-Lavsky, Hadas ;

Sas, David J. ;

Michael, Mini ;

Sellier-Leclerc, Anne-Laure ;

Hogan, Julien ;

Willey, Richard ;

Gansner, John M. ;

Magen, Daniella .

FRONTIERS IN PEDIATRICS, 2024, 12

[30] Lumasiran for Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 24-Month Analysis of the Phase 3 ILLUMINATE-C Trial [J].

Lieske, John C. ;

Magen, Daniella ;

Sellier-Leclerc, Anne-Laure A. ;

Shasha-Lavsky, Hadas ;

Simkova, Eva ;

Devresse, Arnaud ;

Michael, Mini ;

Frishberg, Yaacov ;

Bakkaloglu, Sevcan A. ;

Mourani, Chebl ;

Saqan, Rola ;

Singer, Richard F. ;

Guzzo, Isabella ;

Makarova, Nune ;

Willey, Richard G. ;

Kaspar, Cristin ;

Gansner, John M. ;

Groothoff, Jaap .

JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2023, 34 (11) :566-566

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