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] IN VIVO GENOME EDITING THERAPY FOR PRIMARY HYPEROXALURIA TYPE I (PH1)
Wang, Zi Jun
AMERICAN JOURNAL OF KIDNEY DISEASES, 2025, 85 (04)
[32] Molecular analysis of the AGXT gene in Italian patients with primary hyperoxaluria type 1 (PH1)
Ferrettini, C
Pirulli, D
Cosseddu, D
Marangella, M
Petrarulo, M
Mazzola, G
Vatta, S
Amoroso, A
JOURNAL OF NEPHROLOGY, 1998, 11 : 18 - 22
[33] 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
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
[34] DEVELOPMENT OF A PHARMACOKINETIC/PHARMACODYNAMIC MODEL OF ALN-GO1, AN INVESTIGATIONAL RNAi THERAPEUTIC FOR PRIMARY HYPEROXALURIA
Attarwala, H.
Goel, V.
McGregor, T.
Erbe, D.
Haslett, P.
Robbie, G.
CLINICAL PHARMACOLOGY & THERAPEUTICS, 2018, 103 : S24 - S24
[35] Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1 A Placebo-Controlled Randomized Clinical Trial
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
[36] New Gene Therapy Approach for the Treatment of Primary Hyperoxaluria Type 1 (PH1) Patients. Proof of Concept in a Mouse Model of PH1
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
[37] Pre-emptive liver transplantation for primary hyperoxaluria type 1(PH1): A case report
Wang Jun
Qu Wei
Zeng Zhigui
Sun Liying
Zhu Zhijun
PEDIATRIC TRANSPLANTATION, 2019, 23
[38] LUMASIRAN FOR PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1 WITH IMPAIRED KIDNEY FUNCTION: DATA FROM THE 6-MONTH ANALYSIS OF THE PHASE 3 ILLUMINATE-C TRIAL
Groothoff, Jaap
Michael, Mini
Shasha-Lavsky, Hadas
Lieske, John
Frishberg, Yaacov
Simkova, Eva
Sellier-Leclerc, Anne-Laure
Arnaud, Devresse
Guebre-Egziabher, Fitsum
Bakkaloglu, Sevcan Azime
Mourani, Chebl
Saqan, Rola
Singer, Richard
Willey, Richard
Habtemariam, Bahru
Bhan, Ishir
Gansner, John
Magen, Daniella
NEPHROLOGY DIALYSIS TRANSPLANTATION, 2022, 37 : I837 - I837
[39] Deleterious AGXT Missense Variant Associated with Type 1 Primary Hyperoxaluria (PH1) in Zwartbles Sheep
Letko, Anna
Dijkman, Reinie
Strugnell, Ben
Hafliger, Irene M.
Paris, Julia M.
Henderson, Katrina
Geraghty, Tim
Orr, Hannah
Scholes, Sandra
Drogemuller, Cord
GENES, 2020, 11 (10) : 1 - 9
[40] DEVELOPMENT OF AN RNAI THERAPEUTIC TARGETING GLYCOLATE OXIDASE FOR THE TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1
Liebow, Abigail
Erbe, David
Li, Xingsheng
Fargue, Sonia
Milstein, Stuart
Kuchimanchi, Satya
Fitzgerald, Kevin
Querbes, William
Holmes, Ross
Knight, John
Meyers, Rachel
PEDIATRIC NEPHROLOGY, 2015, 30 (09) : 1564 - 1564

← 1 2 3 4 5 →