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Nationale klinische studie(s)
SPANJE
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
SPANJE
Madrid
MADRID
A Phase 3 trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and AlstrC6m syndrome (AS) Patients with Moderate to Severe Obesity - ES
Hospital Infantil Universitario Niño Jesús
Servicio de Endocrinología, Crecimiento y Metabolismo
VERENIGD KONINKRIJK
West Midlands
BIRMINGHAM
Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
University Hospital Birmingham
Marshfield Clinic
BELGIË
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
BELGIË
OOST-VLAANDEREN
GENT
INSIGHT: Een open-label, extensieonderzoek om de veiligheid, verdraagbaarheid, werkzaamheid en farmacokinetiek van QR-110 te evalueren bij personen met Leber congenitale amaurose (LCA) vanwege de c.2991 + 1655A> G-mutatie (p.Cys998X) in het CEP290-gen - BE
Ghent University Hospital - UZ Gent
Department of Ophthalmology
BELGIË
OOST-VLAANDEREN
GENT
ILLUMINATE: Dubbel gemaskeerd, gerandomiseerd, gecontroleerd onderzoek met meerdere doses om de werkzaamheid, veiligheid, verdraagbaarheid en systemisch blootstelling van QR-110 te evalueren in Leber congenitale amaurose (LCA) als gevolg van c.2991 + 1655A> G-mutatie (p.Cys998X) in het CEP290-gen - BE
Ghent University Hospital - UZ Gent
Department of Ophthalmology
BELGIË
OOST-VLAANDEREN
GENT
STELLAR: Een studie om de veiligheid en verdraagbaarheid van QR-421a te evalueren voor de eerste maal bij personen met retinitis pigmentosa (RP) als gevolg van mutaties in Exon 13 van het USH2A-gen -BE
Ghent University Hospital - UZ Gent
Department of Ophthalmology
DUITSLAND
Baden-Württemberg
TÜBINGEN
Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients With CNGA3-linked Achromatopsia Investigated in an Exploratory, Dose-escalation Trial (RD-Cure Gene Therapy Trial)
Department für Augenheilkunde Tübingen
Universitäts-Augenklinik
DUITSLAND
Baden-Württemberg
TÜBINGEN
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
Department für Augenheilkunde Tübingen
Universitäts-Augenklinik
DUITSLAND
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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)
FRANKRIJK
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
ITALIË
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
SPANJE
Cataluña
BARCELONA
A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura (Complement C5 Inhibitor) Compared to Sham in Subjects with Autosomal Recessive Stargardt Disease - ES
Centro Médico Teknon - Grupo Quirónsalud
Servicio de Oftalmología
SPANJE
Madrid
MADRID
The SeaSTAR Study: A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease - ES
Hospital Universitario Fundación Jiménez Díaz
Servicio de Genética Clínica
USA
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
VERENIGD KONINKRIJK
Greater London
LONDON
VERENIGD KONINKRIJK
Greater London
LONDON
A clinical trial of AAV - CNGA3 retinal gene therapy for patients with achromatopsia -GB
Moorfield's Eye Hospital
VERENIGD KONINKRIJK
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
Multinationale klinische studie(s)
NEDERLAND
Zuid-Holland
LEIDEN
NEDERLAND
Zuid-Holland
LEIDEN