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BELGIUM
VLAAMS BRABANT
LEUVEN
NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - BE
University Hospitals Leuven - Gasthuisberg
Department of Human Genetics
FRANCE
AUVERGNE-RHONE-ALPES
BRON
Efficacy of methylphenidate on hyperactivity and attention deficit disorders in children with neurofibromatosis type 1 : a randomized double blind study methylphenidate versus placebo (Phase IV)
CHU de Lyon HCL - GH Est
Service de neurologie pédiatrique
FRANCE
ILE-DE-FRANCE
CRÉTEIL
Multicenter phase IIa open trial evaluating RAD001 as monotherapy of neurofibromatosis type 1 patients, in the treatment of inoperable internal plexiform neurofibromas.
CHU Henri Mondor
Service de dermatologie
FRANCE
PAYS DE LA LOIRE
NANTES
Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1 - FR
CHU de Nantes
CHU Nantes
GERMANY
Baden-Württemberg
TÜBINGEN
RD-Cure Gene Therapy Trial: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients With CNGA3-linked Achromatopsia Investigated in an Exploratory, Dose-escalation Trial (Phase I/II) - DE
Department für Augenheilkunde Tübingen
Universitäts-Augenklinik
NETHERLANDS
Zuid-Holland
ROTTERDAM
NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - NL
Erasmus MC - Erasmus Medisch Centrum
Afdeling Kinderneurologie
NETHERLANDS
Zuid-Holland
ROTTERDAM
NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - NL
Erasmus MC - Erasmus Medisch Centrum
Afdeling Neurowetenschappen
NETHERLANDS
Zuid-Holland
ROTTERDAM
NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - BE
Erasmus MC - Erasmus Medisch Centrum
SPAIN
Cataluña
ESPLUGUES DE LLOBREGAT
NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - ES
Hospital Sant Joan de Déu Barcelona
Servicio de Oncología y Hematología
UNITED KINGDOM
Greater London
LONDON
A Paediatric Phase I/II Study Of Intermittent Dosing Of The Mek-1 Inhibitor Selumetinib In Children With Neurofibromatosis Type-1 And Inoperable Plexiform Neurofibroma And/Or Progressive Optic Pathway Glioma
GOSH NHS Foundatin Trust
Great Ormond Street Hospital
UNITED KINGDOM
Greater London
LONDON
UNITED KINGDOM
Greater London
LONDON
A clinical trial of AAV - CNGA3 retinal gene therapy for patients with achromatopsia -GB
Moorfield's Eye Hospital
UNITED KINGDOM
Greater London
LONDON
Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3
University College London - UCL Darwin Building
University College London
FRANCE
ILE-DE-FRANCE
PARIS
AOA1 : Evolution of Albumin on AOA1 Patients Supplemented With Coenzyme Q10 (Phase III)
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Institut du Cerveau et de la Moëlle épinière - INSERM U1127
AUSTRIA
STEIERMARK
GRAZ
IRIS 2: Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy - AT
Medizinische Universität Graz
Universitäts-Augenklinik
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BIIB092 in Participants With Progressive Supranuclear Palsy (Phase 2) - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
WIEN
A Randomized, Double-masked, Placebo Controlled Study of the Beneficial Effects of Eplerenone on Central Serous Chorioretinopathy (Phase 2-3)
Hanusch Krankenhaus
VIROS - Vienna Institute for Research in Ocular Surgery
CANADA
Alberta
EDMONTON
An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein-1 (REP1) (Phase I)
University of Alberta
CHM
CANADA
Alberta
EDMONTON
A Repeated Measures Study of Simvastatin on Choroideremia: Simvastatin Intervention and Reversal in Choroideremia Patients and Age-matched Controls (Phase I-II)
University of Alberta
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety and Efficacy of Davunetide for the Treatment of Progressive Supranuclear Palsy - FR
Institution: Information not provided - FR
FRANCE
ILE-DE-FRANCE
PARIS
Effect of Spironolactone in Treating Chronic Non-resolutive Central Serous Chorioretinitis (Phase I-II)
CHU Paris Centre - Hôpital Hôtel-Dieu
Service d'Ophtalmologie
FRANCE
ILE-DE-FRANCE
PARIS
Study of the Distractibility Syndrome in Patients With Progressive Supranuclear Palsy (Phase II)
CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
Neurologie et thérapeutique expérimentale
FRANCE
PAYS DE LA LOIRE
NANTES
Randomized Placebo-Controlled Trial of Valproic Acid in Patients With Progressive Supranuclear Palsy (Phase II)
CHU de Nantes - Hôpital Nord Guillaume et René Laënnec
Clinique neurologique
FRANCE
PAYS DE LA LOIRE
NANTES
IRIS 2: Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy - FR
CHU de Nantes - Hôtel Dieu
Service d'ophtalmologie
GERMANY
Baden-Württemberg
TÜBINGEN
Treatment Retina Implant Trial - DE
Department für Augenheilkunde Tübingen
Forschungsinstitut für Augenheilkunde
GERMANY
Baden-Württemberg
TÜBINGEN
THOR: Tübingen Choroideremia Gene Therapy Trial Open Label Phase 2 Clinical Trial Using an Adeno-associated Viral Vector Encoding Rab-escort Protein 1 (This study is enrolling participants by invitation only) - DE
Department für Augenheilkunde Tübingen
Universitäts-Augenklinik
GERMANY
Baden-Württemberg
TÜBINGEN
GEMINI Study: An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia with Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1) -DE-
Department für Augenheilkunde Tübingen
Universitäts-Augenklinik
GERMANY
Baden-Württemberg
TÜBINGEN
STAR: A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial Of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)
Department für Augenheilkunde Tübingen
Universitäts-Augenklinik
GERMANY
Baden-Württemberg
TÜBINGEN
Rivastigmine (Exelon®) for treatment of dementia in patient with Progressive Supranuclear Paresis - DE
Hertie-Institut für klinische Hirnforschung (HIH)
Abteilung Neurologie mit Schwerpunkt neurodegenerative Erkrankungen
GERMANY
Baden-Württemberg
ULM
A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety and Efficacy of Davunetide for the Treatment of Progressive Supranuclear Palsy -DE- Completed
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie
GERMANY
Bayern
HAUSHAM
PROSPERA: A randomized, double-Blind, placebo-controlled, parallel group study to assess the efficacy, safety and tolerability of Rasagiline in subjects with Progressive Supranuclear Palsy (Phase III) - DE - Prematurely Ended
Krankenhaus Agatharied GmbH
Abteilung für Neurologie
GERMANY
Berlin
ADDRESS: NOT PROVIDED - DE
TAUROS: A double-blind, placebo-controlled, randomized, parallel-group phase II study evaluating the safety, tolerability, and efficacy of two different oral doses of NP031112, a GSK-3 Inhibitor, versus Placebo in the treatment of patients with mild-to-moderate Progressive Supranuclear Palsy DE
Institution: Information not provided - DE
GERMANY
Hessen
MARBURG
Monocentre, prospective, double-blind, placebo-controlled, randomized clinical phase IIa trial to assess the safety, tolerability, and immediate biological effects of Coenzyme Q10 in Progressive Supranuclear Palsy - DE
Universitätsklinikum Gießen und Marburg GmbH, Standort Marburg
Klinik und Poliklinik für Neurologie
GERMANY
Nordrhein-Westfalen
BONN
IRIS 2: Compensation for Blindness with the Intelligent Retinal Implant System (IRIS V2) in Patients with Retinal Dystrophy - DE
Universitäts-Augenklinik Bonn
ITALY
LOMBARDIA
MILANO
Autologous Mesenchymal Stem Cell Therapy in Progressive Supranuclear Palsy: a Randomized, Double-blind, Controlled Clinical Trial
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Centro Trasfusionale
ITALY
TOSCANA
FIRENZE
A Historically-Controlled Phase II/III study to Evaluate Efficacy and Safety of Kedrion Human Plasminogen Eye Drop Preparation in Patients Diagnosed with Ligneous Conjunctivitis
Azienda Ospedaliera Universitaria Anna Meyer
S.O.C. Oftalmologia Pediatrica
NETHERLANDS
Zuid-Holland
LEIDEN
SPECTRA Trial: Study on Half-dose Photodynamic Therapy Versus Eplerenone in Chronic Central Serous Chorioretinopathy (phase IV)
LUMC - Leids Universitair Medisch Centrum
Afdeling Oogheelkunde
SPAIN
Andalucía
SEVILLA
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Hospital Universitario Virgen del Rocío
SPAIN
Aragón
ZARAGOZA
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease (Phase III) - ES (completed)
Hospital Universitario Miguel Servet
Servicio de Hematología y Hemoterapia
SPAIN
Aragón
ZARAGOZA
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease (Phase III) (completed)
Hospital Universitario Miguel Servet
Servicio de Hematología y Hemoterapia
SPAIN
Cataluña
BARCELONA
A Double-Blind, Placebo-Controlled, Randomized, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of Two Different Oral Doses of NP031112, a GSK-3 Inhibitor, Versus Placebo in the Treatment of Patients With Mild-to-Moderate Progressive Supranuclear Palsy (Phase II) - ES (completed)
Hospital Clínic de Barcelona
Servicio de Neurología
SPAIN
Cataluña
BARCELONA
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Hospital de la Santa Creu i Sant Pau
SPAIN
Cataluña
SANT CUGAT DEL VALLÉS
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Hospital Universitari General de Catalunya
SPAIN
Cataluña
TERRASSA
A Double-Blind, Placebo-Controlled, Randomized, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of Two Different Oral Doses of NP031112, a GSK-3 Inhibitor, Versus Placebo in the Treatment of Patients With Mild-to-Moderate Progressive Supranuclear Palsy (Phase II) - ES (completed)
Hospital Universitari Mutua de Terrassa
Servicio de Neurología
SPAIN
Comunidad Valenciana
VALENCIA
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Hospital Universitario y Politécnico La Fe
SPAIN
Madrid
MADRID
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Complejo hospitalario Ruber Juan Bravo
SPAIN
Madrid
MADRID
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Hospital General Universitario Gregorio Marañón
SPAIN
Madrid
MADRID
A Double-Blind, Placebo-Controlled, Randomized, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of Two Different Oral Doses of NP031112, a GSK-3 Inhibitor, Versus Placebo in the Treatment of Patients With Mild-to-Moderate Progressive Supranuclear Palsy (Phase II) - ES (completed)
Hospital Universitario La Paz
Servicio de Neurología
SPAIN
Madrid
MADRID
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Hospital Universitario Ramón y Cajal
SPAIN
Madrid
MADRID
A Double-Blind, Placebo-Controlled, Randomized, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of Two Different Oral Doses of NP031112, a GSK-3 Inhibitor, Versus Placebo in the Treatment of Patients With Mild-to-Moderate Progressive Supranuclear Palsy (Phase II) - ES (completed)
Hospital Universitario Ramón y Cajal
Servicio de Neurología
SPAIN
Navarra
PAMPLONA
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Clínica Universidad de Navarra
SPAIN
País Vasco
BARAKALDO
PASSPORT: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Intravenously Administered BMS-986168 in Participants with Progressive Supranuclear Palsy (Phase II) - ES
Hospital Universitario Cruces - Osakidetza
SPAIN
País Vasco
BARAKALDO
A Double-Blind, Placebo-Controlled, Randomized, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of Two Different Oral Doses of NP031112, a GSK-3 Inhibitor, Versus Placebo in the Treatment of Patients With Mild-to-Moderate Progressive Supranuclear Palsy (Phase II) - ES (completed)
Hospital Universitario Cruces - Osakidetza
Servicio de Neurología
SPAIN
País Vasco
SAN SEBASTIÁN
A Double-Blind, Placebo-Controlled, Randomized, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of Two Different Oral Doses of NP031112, a GSK-3 Inhibitor, Versus Placebo in the Treatment of Patients With Mild-to-Moderate Progressive Supranuclear Palsy (Phase II) - ES (completed)
Hospital Universitario Donostia
Servicio de Neurología
UNITED KINGDOM
Greater London
LONDON
An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
Moorfield's Eye Hospital
UNITED KINGDOM
Greater Manchester
ADDRESS: NOT PROVIDED - UK
AL-108-231: A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety and Efficacy of Davunetide for the Treatment of Progressive Supranuclear Palsy - UK
Institution: Information not provided - UK
UNITED KINGDOM
Greater Manchester
ADDRESS: NOT PROVIDED - UK
TAUROS: A double-blind, placebo-controlled, randomized, parallel-group phase II study evaluating the safety, tolerability, and efficacy of two different oral doses of NP031112, a GSK-3 Inhibitor, versus Placebo in the treatment of patients with mild-to-moderate Progressive Supranuclear Palsy - UK
Institution: Information not provided - UK
UNITED KINGDOM
Oxfordshire
OXFORD
An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
Le Gros Clark Building - University of Oxford
Neuroscience
UNITED STATES
New Jersey
SOUTH PLAINFIELD
STAR: A Phase 2, Multicenter, Randomized, Double Masked, Placebo Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia - US
PTC Therapeutics Inc.
UNITED STATES
New York
NEW YORK
Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia (Phase III) - US
New York University School of Medicine
UNITED STATES
Texas
DALLAS
LEAP: A 52-week Two-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 (Phase II) - US
Baylor University Medical Center
Institute of Metabolic Disease
FRANCE
ILE-DE-FRANCE
PARIS
RESCUE: A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene (Phase III) - FR
Centre hospitalier national d'ophtalmologie des Quinze-Vingts
Centre de Référence REFERET
FRANCE
ILE-DE-FRANCE
PARIS
REVERSE: Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for More Than 6 Months and To 12 Months by LHON Due to the G11778A Mutation in the ND4 Gene (Phase III) - FR
Centre hospitalier national d'ophtalmologie des Quinze-Vingts
Centre de Référence REFERET
FRANCE
ILE-DE-FRANCE
PARIS
An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene (Phase I-II)
Institut de la Vision
Centre d'Investigation Clinique (CIC) de l'hôpital des Quinze-Vingts
FRANCE
PAYS DE LA LOIRE
ANGERS
CICLO-NOHL : Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy (Phase II)
CHU d'Angers
Service d'Ophtalmologie
FRANCE
PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
RIVA-PSP : Efficacy of Rivastigmine on Motor, Cognitive and Behavioural Impairment in Progressive Supranuclear Palsy: A Randomised Double Blind Placebo-controlled Clinical Trial (Phase III)
CHU de Marseille - Hôpital de la Timone
Service de Neurologie, pathologie du mouvement
GERMANY
Bayern
MÜNCHEN
RESCUE: A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene (Phase III) -DE-
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik
GERMANY
Bayern
MÜNCHEN
RESCUE/REVERSE: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials -DE-
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik
GERMANY
Bayern
MÜNCHEN
A double-blind, randomised, placebo-controlled study of the efficacy, safety and tolerability of idebenone in the treatment of patients with Leber's Hereditary Optic Neuropathy (LHON) (Phase II) - DE
LMU Klinikum der Universität München - Campus Großhadern
Arbeitsgruppe Neurogenetik
NETHERLANDS
Gelderland
NIJMEGEN
The KHENERGY Study: An Exploratory, Double-blind, Randomized, Placebo-controlled, Single-center, Two-way Cross-over Study With KH176 in Patients With the Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation and Clinical Signs of Mitochondrial Disease
Radboudumc - Radboud universitair medisch centrum
Radboud Centrum voor Mitochondriële Geneeskunde
SPAIN
Madrid
MADRID
Setmelanotide (RM-493): Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity - ES
Hospital Infantil Universitario Niño Jesús
Servicio de Endocrinología, Crecimiento y Metabolismo
SPAIN
Madrid
MADRID
LEROS: External Natural History Controlled, open-Label Intervention Study to Assess the Efficacy and Safety of Long-Term Treatment with Raxone® in Leber's Hereditary Optic Neuropathy (LHON) (Phase IV) - ES
Hospital Universitario Ramón y Cajal
Servicio de Oftalmología
UNITED KINGDOM
Greater London
LONDON
RESCUE/REVERSE: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials-GB
Moorfield's Eye Hospital
UNITED KINGDOM
Tyne & Wear
NEWCASTLE UPON TYNE
A Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Patients With Leber's Hereditary Optic Neuropathy (Phase II)
Newcastle University Institute for Ageing (NUIA), Newcastle Biomedicine
Wellcome Trust Centre for Mitochondrial Research (Institute of Neuroscience)
UNITED KINGDOM
West Midlands
BIRMINGHAM
Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
University Hospital Birmingham
Marshfield Clinic
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene -GB
Institution: Information not provided - US
BELGIUM
OOST-VLAANDEREN
GENT
Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-CBSB-hRPE65) Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations (Clinical Trials of Gene Therapy for Leber Congenital Amaurosis).
Ghent University Hospital - UZ Gent
Department of Ophthalmology
BELGIUM
OOST-VLAANDEREN
GENT
An Open-Label, Single Arm, Multiple Dose, Dose Escalation Study to evaluate the safety and tolerability of QR-110 in subjects with Leber's Congenital Amaurosis (LCA) due to c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene
Ghent University Hospital - UZ Gent
Ghent University Hospital
BELGIUM
VLAAMS BRABANT
LEUVEN
The ADVISE Study: Advanced Visualization In Corneal Surgery Evaluation Intra-operative Optical Coherence Tomography in Posterior Lamellar Keratoplasty - BE
University Hospitals Leuven - Gasthuisberg
Dienst oogziekten/Ophthalmology
CANADA
Québec
MONTRÉAL
An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin: Retinol Acyltransferase (LRAT). Extenstion of Study RET IRD 01. (Phase 1b)
Montreal Children's hospital - Hôpital de Montréal pour enfants
Research Institute of McGill University Health Centre
FRANCE
GRAND-EST
STRASBOURG
ILLUMINATE: Double-masked, Randomized, Controlled, Multiple-dose Study to Evaluate Efficacy, Safety, Tolerability and Syst. Exposure of QR-110 in Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene - FR
CHU de Strasbourg - Hôpital Civil
Centre des Affections Rares en Génétique Ophtalmologique - CARGO
FRANCE
ILE-DE-FRANCE
PARIS
A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients With Stargardt's Macular Degeneration - FR
Centre hospitalier national d'ophtalmologie des Quinze-Vingts
Centre de Référence REFERET
FRANCE
ILE-DE-FRANCE
PARIS
ILLUMINATE: Double-masked, Randomized, Controlled, Multiple-dose Study to Evaluate Efficacy, Safety, Tolerability and Syst. Exposure of QR-110 in Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene - FR
Centre hospitalier national d'ophtalmologie des Quinze-Vingts
Centre de Référence REFERET
FRANCE
ILE-DE-FRANCE
PARIS
STELLAR: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene -FR
Centre hospitalier national d'ophtalmologie des Quinze-Vingts
Centre de Référence REFERET
FRANCE
PAYS DE LA LOIRE
NANTES
Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression (Phase I-II)
CHU de Nantes - Hôtel Dieu
Centre d'investigation clinique (CIC)
FRANCE
PAYS DE LA LOIRE
NANTES
Essai de thérapie génique de phase I/II utilisant le vecteur rAAV2/4.hRPE65 pour le traitement de dystrophies rétiniennes liées à des déficiences du gène RPE65
CHU de Nantes - Hôtel Dieu
Service d'ophtalmologie
GERMANY
Bayern
ERLANGEN
Prospective single-centre randomized observer-blind placebo-controlled parallel-group phase IIa clinical trial to investigate the safety and efficacy of ripasudil 0.4% eye drops after descemetorhexis in patients with moderate to advanced Fuchs endothelial corneal dystrophy (FECD)
Universitätsklinikum Erlangen - Kopfkliniken
Augenklinik
GERMANY
Berlin
ADDRESS: NOT PROVIDED - DE
Transcorneal Electrostimulation for Therapy of Retinitis Pigmentosa - DE - Completed
Institution: Information not provided - DE
GERMANY
Sachsen
CHEMNITZ
SFM study: In vitro and clinical comparison of corneal grafts cultured in serum-free versus corneal grafts cultured in serum supplemented culture media in patients with degeneration of the corneal endothelium - DE
Klinikum Chemnitz gGmbH
Klinik für Augenheilkunde
IRELAND
County Dublin
DUBLIN
An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65) - IE
Royal Victoria Eye and Ear Hospital
Ophthalmology Research Department
ITALY
CAMPANIA
NAPOLI
A Safety and Efficacy Study in Subjects with Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE): treatment and follow up of 3 Italian patients
AOU Università degli Studi della Campania "Luigi Vanvitelli"- Cappella Cangiani
U.O.C. di Oculistica
ITALY
LAZIO
ROMA
A Novel Therapeutic Strategy Targeting Photoreceptor Oxidative Damage in ABCR-related Retinal Degenerations (Phase I / II )
Policlinico Universitario "A. Gemelli"
Dipartimento per l'assistenza sanitaria di Scienze Oftalmologiche e Otorinolaringoiatriche
ITALY
LOMBARDIA
MILANO
Nerve Growth Factor eye drops as a novel treatment for vision loss in patients with Retinitis Pigmentosa: from preclinical to clinical Phase II trial.
San Raffaele Scientific Institute
Cornea and Ocular Surface Unit
SPAIN
Aragón
ZARAGOZA
Melatonin effects on visual and sleep patterns anomalies in subjects diagnosed with Retinitis Pigmentosa (Phase III) - ES (terminated)
Hospital Clínico Universitario "Lozano Blesa" de Zaragoza
Servicio de Oftalmología
SPAIN
Murcia
EL PALMAR, MURCIA
Phase I Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa - ES (completed)
Hospital Clínico Universitario Virgen de la Arrixaca
Servicio de Oftalmología
UNITED KINGDOM
Greater London
LONDON
UNITED KINGDOM
Greater London
LONDON
UNITED KINGDOM
Greater London
LONDON
An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children with Retinal Dystrophy -GB
Moorfield's Eye Hospital
UNITED KINGDOM
Greater London
LONDON
A Pilot Study Evaluating the Effect of Intravitreal Fluocinolone Acetonide (0.19mg) in Patients with Retinitis Pigmentosa -GB Terminated
Moorfield's Eye Hospital
UNITED KINGDOM
Greater London
LONDON
UNITED KINGDOM
Greater London
LONDON
An Open-Label Dose Escalation Study of an Adeno-Associated Virus Vector (AAV2/2-hRPE65p-hRPE65) for Gene Therapy of Severe Early-Onset Retinal Degeneration (Phase I-II)
UCL Institute of Ophthalmology
Molecular Therapy
UNITED KINGDOM
Greater London
LONDON
An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/5-hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
University College London - UCL Darwin Building
University College London
UNITED STATES
Oregon
PORTLAND
A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing RPE65 (rAAV2-CB-hRPE65) in Patients With Leber Congenital Amaurosis Type 2 - US
OHSU Doernbecher Children's Hospital -Oregon Health & Science University
Casey Eye Institute
Multinational clinical trial(s)
UNITED KINGDOM
Berkshire
MAIDENHEAD
UNITED STATES
California
SAN FRANCISCO
Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Davunetide for the Treatment of Progressive Supranuclear Palsy - coordination - terminated
University of California, San Francisco
Memory and Aging Center
UNITED STATES
Massachusetts
CAMBRIDGE
GERMANY
Baden-Württemberg
LÖRRACH
A double-blind, randomised, placebo-controlled study of the efficacy, safety and tolerability of idebenone in the treatment of patients with Leber's Hereditary Optic Neuropathy (LHON) (Phase II) (coordination)
Santhera Pharmaceuticals (Deutschland) GmbH
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene
Institution: Information not provided - US
NETHERLANDS
Zuid-Holland
LEIDEN
NETHERLANDS
Zuid-Holland
LEIDEN