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Ensaio (s) clínico (s) nacional (is)

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

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - BE UZ Leuven - Campus Gasthuisberg Department of Human Genetics

Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1 - FR CHU de Nantes CHU Nantes

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

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

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

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

An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/8-hCARp.hCNGB3) for gene therapy of adults and children with achromatopsia owing to defects in CNGB3 - Gene Therapy for Achromatopsia: CNGB3 MeiraGTx UK II Limited

A clinical trial of AAV - CNGA3 retinal gene therapy for patients with achromatopsia -GB Moorfield's Eye Hospital

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

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

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

IRIS 2: Compensation for Blindness with the Intelligent Retinal Implant System (IRIS V2) in Patients with Retinal Dystrophy - DE Universitäts-Augenklinik Bonn

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

HCQ4Surfdefect: Hydroxychloroquine (HCQ) in pediatric ILD (interstitial lung disease) - AT Allgemeines Krankenhaus der Stadt Wien Klinische Abteilung für Pädiatrische Pulmologie, Allergologie und Endokrinologie

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

ELIKIDS: Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 (Phase III) - ES Hospital Quirónsalud Zaragoza Servicio de Hematología

ELIKIDS: Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 (Phase III) - ES Hospital Universitario Cruces Servicio de Pediatría

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.

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

SPECTRA Trial: Study on Half-dose Photodynamic Therapy Versus Eplerenone in Chronic Central Serous Chorioretinopathy (phase IV) LUMC - Leids Universitair Medisch Centrum Afdeling Oogheelkunde

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

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

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

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 4) - AT Institution: Information not provided - AT

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

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

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

REFLECT: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year (Phase III) - ES Hospital Universitario Ramón y Cajal Servicio de Oftalmología

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

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

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

Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity University Hospital Birmingham Marshfield Clinic

OPA1: Raxone® treatment for patients with dominant optic atrophy due to OPA1 gene mutation Medizinische Universität Graz Universitäts-Augenklinik

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 -BE Ghent University Hospital - UZ Gent Department of Ophthalmology

The ADVISE Study: Advanced Visualization In Corneal Surgery Evaluation Intra-operative Optical Coherence Tomography in Posterior Lamellar Keratoplasty - BE UZ Leuven - Campus Gasthuisberg Dienst oogziekten/Ophthalmology

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

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

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

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

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)

An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA) Moorfield's Eye Hospital

Long-term Follow-up Study of Participants Following 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 Owing to Defects in RPE65 (LCA2) Moorfield's Eye Hospital

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

XIRIUS Study: A Dose Escalation, Phase 1/2 Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR) NightstaRx Ltd

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

Ensaio (s) clínico (s) multinacional (ais)

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

ELIKIDS: Open Label, Two Cohort (With and Without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients With Gaucher Disease Type 1 and Type 3 Genzyme Corporation

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 Biogen Idec Limited

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

A Participant-Blind, Investigator-Blind, Placebo-Controlled, Phase 1b Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of UCB0107 in Study Participants With Progressive Supranuclear Palsy (PSP) UCB Biopharma SPRL

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

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) Santhera Pharmaceuticals

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 ProQR Therapeutics N.V.

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 ProQR Therapeutics N.V.

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

NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial - BE
UZ Leuven - Campus Gasthuisberg

Department of Human Genetics

Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1 - FR
CHU de Nantes

CHU Nantes

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

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

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

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

An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/8-hCARp.hCNGB3) for gene therapy of adults and children with achromatopsia owing to defects in CNGB3 - Gene Therapy for Achromatopsia: CNGB3
MeiraGTx UK II Limited

A clinical trial of AAV - CNGA3 retinal gene therapy for patients with achromatopsia -GB
Moorfield's Eye Hospital

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

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

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

IRIS 2: Compensation for Blindness with the Intelligent Retinal Implant System (IRIS V2) in Patients with Retinal Dystrophy - DE
Universitäts-Augenklinik Bonn

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

HCQ4Surfdefect: Hydroxychloroquine (HCQ) in pediatric ILD (interstitial lung disease) - AT
Allgemeines Krankenhaus der Stadt Wien

Klinische Abteilung für Pädiatrische Pulmologie, Allergologie und Endokrinologie

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

ELIKIDS: Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 (Phase III) - ES
Hospital Quirónsalud Zaragoza

Servicio de Hematología

ELIKIDS: Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 (Phase III) - ES
Hospital Universitario Cruces

Servicio de Pediatría

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.

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

SPECTRA Trial: Study on Half-dose Photodynamic Therapy Versus Eplerenone in Chronic Central Serous Chorioretinopathy (phase IV)
LUMC - Leids Universitair Medisch Centrum

Afdeling Oogheelkunde

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

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

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

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 4) - AT
Institution: Information not provided - AT

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

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

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

REFLECT: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year (Phase III) - ES
Hospital Universitario Ramón y Cajal

Servicio de Oftalmología

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

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

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

Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
University Hospital Birmingham

Marshfield Clinic

OPA1: Raxone® treatment for patients with dominant optic atrophy due to OPA1 gene mutation
Medizinische Universität Graz

Universitäts-Augenklinik

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 -BE
Ghent University Hospital - UZ Gent

Department of Ophthalmology

The ADVISE Study: Advanced Visualization In Corneal Surgery Evaluation Intra-operative Optical Coherence Tomography in Posterior Lamellar Keratoplasty - BE
UZ Leuven - Campus Gasthuisberg

Dienst oogziekten/Ophthalmology

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

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

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

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

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)

An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)
Moorfield's Eye Hospital

Long-term Follow-up Study of Participants Following 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 Owing to Defects in RPE65 (LCA2)
Moorfield's Eye Hospital

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

XIRIUS Study: A Dose Escalation, Phase 1/2 Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)
NightstaRx Ltd

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

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

ELIKIDS: Open Label, Two Cohort (With and Without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients With Gaucher Disease Type 1 and Type 3
Genzyme Corporation

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
Biogen Idec Limited

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

A Participant-Blind, Investigator-Blind, Placebo-Controlled, Phase 1b Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of UCB0107 in Study Participants With Progressive Supranuclear Palsy (PSP)
UCB Biopharma SPRL

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

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)
Santhera Pharmaceuticals

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
ProQR Therapeutics N.V.

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
ProQR Therapeutics N.V.