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Essai(s) clinique(s) national(aux)

Berlin
ADDRESS: NOT PROVIDED - DE

PHYOX 7: A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis -DE
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE
ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1) -DE
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE

PHYOX 7: A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis -DE
Institution: Information not provided - DE

Nordrhein-Westfalen
BONN

ePHex OLE: An open-label single-arm treatment extension study to evaluate the long-term efficacy and safety of Oxabact® for patients with primary hyperoxaluria who completed study OC5-DB-02 -DE
Universitäts-Kinderklinik Bonn
Abteilung für allgemeine Pädiatrie und Poliklinik

Nordrhein-Westfalen
BONN
A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 - DE
Universitätsklinikum Bonn (AöR)
Universitätsklinikum Bonn

Nordrhein-Westfalen
BONN
ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 -DE-
Universitätsklinikum Bonn (AöR)
Universitätsklinikum Bonn

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
ILLUMINATE-C: une étude à bras unique visant à évaluer l'efficacité, l'innocuité, la pharmacocinétique et la pharmacodynamique du Lumasiran chez des patients atteints d'hyperoxalurie primaire avancée de type 1 - BE
Cliniques universitaires Saint-Luc - UCLouvain
Centre de Référence des Maladies Rénales Rares

LIEGE
LIEGE
ePHex OLE: Étude d'extension, à un seul bras, ouverte visant à évaluer l'efficacité et l'innocuité à long terme d'Oxabact® chez des patients atteints d'hyperoxalurie primaire ayant terminé l'étude OC5-DB-02 - BE
CHU de Liège - Site du Sart Tilman
Néphrologie

Madrid
ADDRESS: NOT PROVIDED - ES
PHYOX 2: A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyperoxaluria - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

PHYOX 7: A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis - ES
Institution: Information not provided - ES

Washington
ADDRESS: NOT PROVIDED - US
A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-TermAdministration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1- GB
Institution: Information not provided - US

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Étude d'extension de phase II, multicentrique, en ouvert, visant à évaluer l'administration à long terme d'ALN-GO1 chez des patients atteints d'hyperoxalurie primitive de type 1 - FR
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
PHYOX8: Etude multicentrique de phase 2 en ouvert visant à évaluer la tolérance, les propriétés pharmacocinétiques et l'efficacité de nedosiran chez les patients pédiatriques âgés de 5 ans ou moins présentant une hyperoxalurie primaire et une fonction rénale relativement intacte -FR
Institution: Information not provided - FR

Greater London
ADDRESS: NOT PROVIDED - GB
PHYOX8: A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients From Birth to 5 Years of Age With Primary Hyperoxaluria and Relatively Intact Renal Function -GB
Institution: Information not provided - GB

Greater London
LONDON

ePHex OLE: An Open-label Single-arm Treatment Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact for Patients With Primary Hyperoxaluria Who Completed Study OC5-DB-02-GB
Royal Free London Hospital
University College London, Centre for Nephrology

West Midlands
BIRMINGHAM
ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 -GB
Birmingham Children's Hospital NHS Foundation Trust

Suisse Alémanique
BERN
ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1) - CH
Universitätsspital Inselspital
Universitätsklinik für Nephrologie und Hypertonie

Sachsen
LEIPZIG
ADVANCE: A randomized, double-blind, placebo-controlled, multinational, multicenter study with open-label treatment extension to assess the effect of MIN-102 on the progression of adrenomyeloneuropathy in male patients with X-linked adrenoleukodystrophy (Phase II-III) -DE-
Universitätsklinikum Leipzig AöR
Klinik und Poliklinik für Neurologie

Cataluña
BARCELONA
ADVANCE: A randomized, double-blind, placebo-controlled, multinational, multicenter study with open-label treatment extension to assess the effect of MIN-102 on the progression of adrenomyeloneuropathy in male patients with X-linked adrenoleukodystrophy - ES
Hospital Universitari Vall d'Hebron
Servicio de Neurología

LAZIO
ROMA

ALD-104 - A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects 0 to 17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) - IT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
U.O. di Oncoematologia

Hamburg
HAMBURG

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) -DE
UKE - Universitätsklinikum Hamburg-Eppendorf
Arbeitsgruppe Degenerative Gehirnkrankheiten

Niedersachsen
GÖTTINGEN

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) -DE
Universitätsmedizin Göttingen
ZSEG - Zentrum für seltene Erkrankungen Göttingen

Cataluña
ESPLUGUES DE LLOBREGAT

NEXUS: An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions - ES
Hospital Sant Joan de Déu Barcelona

Cataluña
L'HOSPITALET DE LLOBREGAT
Effect of plasma exchange by albumin replacement in Adrenomyeloneuropathy: unicentric, single arm, proof of concept trial - ES
IDIBELL - Instituto de Investigación Biomédica de Bellvitge
Laboratorio de Enfermedades Neurometabólicas
Essai(s) clinique(s) multinational(aux)

Massachusetts
CAMBRIDGE
ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Alnylam Pharmaceuticals, Inc.

Washington
ADDRESS: NOT PROVIDED - US
A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-TermAdministration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
PHYOX8: A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients From Birth to 5 Years of Age With Primary Hyperoxaluria and Relatively Intact Renal Function.
Institution: Information not provided - US

Region Stockholm
ADDRESS: NOT PROVIDED - SE
ePHex OLE: An Open-label Single-arm Treatment Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact for Patients With Primary Hyperoxaluria Who Completed Study OC5-DB-02
Institution: Information not provided - SE

Madrid
ADDRESS: NOT PROVIDED - ES
ADVANCE: A randomized, double-blind, placebo-controlled, multinational, multicenter study with open-label treatment extension to assess the effect of MIN-102 on the progression of adrenomyeloneuropathy in male patients with X-linked adrenoleukodystrophy
Institution: Information not provided - ES

Washington
ADDRESS: NOT PROVIDED - US