Improving Clinical Trials for Anticomplement Therapies in Complement-Mediated Glomerulopathies: Report of a Scientific Workshop Sponsored by the National Kidney Foundation
Andrew Bomback
(1)
,
Gerald Appel
(2)
,
Debbie Gipson
(3)
,
Michelle Hladunewich
(4)
,
Richard Lafayette
(5)
,
Carla Nester
(6)
,
Samir Parikh
(7)
,
Richard Smith
(6)
,
Howard Trachtman
(2)
,
Peter Heeger
(8)
,
Sanjay Ram
(9)
,
Brad Rovin
(10)
,
Shadab Ali
(11)
,
Nicole Arceneaux
(12)
,
Isa Ashoor
(13)
,
Laura Bailey-Wickins
(14)
,
Jonathan Barratt
(15)
,
Laurence Beck
(16)
,
Daniel Cattran
(17)
,
Paolo Cravedi
(8)
,
Elif Erkan
(18)
,
Fernando Fervenza
(19)
,
Ashley Frazer-Abel
(20)
,
Veronique Fremeaux-Bacchi
(21)
,
Lindsey Fuller
,
Rasheed Gbadegesin
(22)
,
Jonathan Hogan
(23)
,
Krzysztof Kiryluk
(1)
,
Moglie Le Quintrec-Donnette
(24)
,
Christoph Licht
(17)
,
John Mahan
(10)
,
Matthew Pickering
(25)
,
Richard Quigg
(3)
,
Michelle Rheault
(26)
,
Pierre Ronco
(27)
,
Minnie Sarwal
(28)
,
Christine Sethna
(29)
,
Cathie Spino
(3)
,
Mark Stegall
(19)
,
Marina Vivarelli
(30)
,
David Feldman
,
Joshua Thurman
(20)
1
CUIMC -
Columbia University Irving Medical Center
2 NYU Langone Medical Center - New York University Langone Medical Center
3 University of Michigan Medical School [Ann Arbor]
4 Sunnybrook - Sunnybrook Health Sciences Centre [Toronto]
5 Stanford School of Medicine [Stanford]
6 University of Iowa [Iowa City]
7 College of Medicine and Public Health [Colombus]
8 MSSM - Icahn School of Medicine at Mount Sinai [New York]
9 UMASS - University of Massachusetts Medical School [Worcester]
10 OSU - The Ohio State University [Columbus]
11 NKF - National Kidney Foundation
12 NKF - National Kidney Foundation
13 LSU - Louisiana State University
14 NKF - National Kidney Foundation
15 University of Leicester
16 BUSM - Boston University School of Medicine
17 University of Toronto
18 Cincinnati Children's Hospital Medical Center
19 Mayo Clinic [Rochester]
20 University of Colorado Anschutz Medical Campus [Aurora]
21 Service de Néphrologie et Hémodialyse [CHU HEGP]
22 Duke University [Durham]
23 HUP - Hospital of the University of Pennsylvania
24 CHRU Montpellier - Centre Hospitalier Régional Universitaire [Montpellier]
25 Imperial College London
26 University of Minnesota Medical School
27 SU FM - Sorbonne Université - Faculté de Médecine
28 UC San Francisco - University of California [San Francisco]
29 Donald and Barbara Zucker School of Medicine at Hofstra/Northwell
30 IRCCS Ospedale Pediatrico Bambino Gesù = Bambino Gesù Children’s Hospital
2 NYU Langone Medical Center - New York University Langone Medical Center
3 University of Michigan Medical School [Ann Arbor]
4 Sunnybrook - Sunnybrook Health Sciences Centre [Toronto]
5 Stanford School of Medicine [Stanford]
6 University of Iowa [Iowa City]
7 College of Medicine and Public Health [Colombus]
8 MSSM - Icahn School of Medicine at Mount Sinai [New York]
9 UMASS - University of Massachusetts Medical School [Worcester]
10 OSU - The Ohio State University [Columbus]
11 NKF - National Kidney Foundation
12 NKF - National Kidney Foundation
13 LSU - Louisiana State University
14 NKF - National Kidney Foundation
15 University of Leicester
16 BUSM - Boston University School of Medicine
17 University of Toronto
18 Cincinnati Children's Hospital Medical Center
19 Mayo Clinic [Rochester]
20 University of Colorado Anschutz Medical Campus [Aurora]
21 Service de Néphrologie et Hémodialyse [CHU HEGP]
22 Duke University [Durham]
23 HUP - Hospital of the University of Pennsylvania
24 CHRU Montpellier - Centre Hospitalier Régional Universitaire [Montpellier]
25 Imperial College London
26 University of Minnesota Medical School
27 SU FM - Sorbonne Université - Faculté de Médecine
28 UC San Francisco - University of California [San Francisco]
29 Donald and Barbara Zucker School of Medicine at Hofstra/Northwell
30 IRCCS Ospedale Pediatrico Bambino Gesù = Bambino Gesù Children’s Hospital
Richard Smith
- Fonction : Auteur
- PersonId : 770007
- ORCID : 0000-0003-1201-6731
- IdRef : 067404715
Lindsey Fuller
- Fonction : Auteur
David Feldman
- Fonction : Auteur
Résumé
Blocking the complement system as a therapeutic strategy has been proposed for numerous glomerular diseases but presents myriad questions and challenges, not the least of which is demonstrating efficacy and safety. In light of these potential issues and because there are an increasing number of anticomplement therapy trials either planned or under way, the National Kidney Foundation facilitated an all-virtual scientific workshop entitled "Improving Clinical Trials for Anti-Complement Therapies in Complement-Mediated Glomerulopathies." Attended by patient representatives and experts in glomerular diseases, complement physiology, and clinical trial design, the aim of this workshop was to develop standards applicable for designing and conducting clinical trials for anticomplement therapies across a wide spectrum of complement-mediated glomerulopathies. Discussions focused on study design, participant risk assessment and mitigation, laboratory measurements and biomarkers to support these studies, and identification of optimal outcome measures to detect benefit, specifically for trials in complement-mediated diseases. This report summarizes the discussions from this workshop and outlines consensus recommendations.
Mots clés
Glomerular disease
Clinical trial design
Complement biomarkers
Complement blockade
Complement inhibition
Complement-mediated glomerulopathy
Drug approval pathway
Drug safety
Glomerulonephritides
Infectious risk
Patient-reported outcome (PRO)
Rare diseases
Research priorities
Surrogate outcome
Trial end points
Glomerular disease