VA ( DrugBank: - )
3 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 90 | 網膜色素変性症 | 13 |
| 301 | 黄斑ジストロフィー | 2 |
| 302 | レーベル遺伝性視神経症 | 3 |
90. 網膜色素変性症
臨床試験数 : 130 / 薬物数 : 180 - (DrugBank : 41) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 109
Showing 1 to 10 of 13 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2020-002873-88-NL (EUCTR) | 14/03/2022 | 22/10/2021 | Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a ... | Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigme ... | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Cl ... | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: A ... | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Netherlands;Germany United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Ne ... | ||
| 2 | EUCTR2020-002255-37-NL (EUCTR) | 14/03/2022 | 22/10/2021 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive ... | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associa ... | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Cl ... | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: A ... | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | Switzerland;United Kingdom;France;United States;Spain;Ireland;Israel;Italy;Canada;Belgium;Denmark;Netherlands;Germany Switzerland;United Kingdom;France;United States;Spain;Ireland;Israel;Italy;Canada;Belgium;Denmark;Ne ... | ||
| 3 | EUCTR2020-002873-88-DK (EUCTR) | 01/03/2022 | 08/11/2021 | Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a ... | Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigme ... | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Cl ... | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: A ... | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Ge ... | ||
| 4 | EUCTR2020-002255-37-DK (EUCTR) | 01/03/2022 | 08/11/2021 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive ... | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associa ... | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Cl ... | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: A ... | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Ge ... | ||
| 5 | NCT04850118 (ClinicalTrials.gov) | August 2021 | 5/4/2021 | A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in ... | A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and ... | X-Linked Retinitis Pigmentosa | Biological: rAAV2tYF-GRK1-hRPGRco | Applied Genetic Technologies Corp | NULL | Not yet recruiting | 8 Years | 50 Years | Male | 63 | Phase 2/Phase 3 | Israel;Netherlands;United States |
| 6 | NCT04794101 (ClinicalTrials.gov) | March 16, 2021 | 15/12/2020 | Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Vari ... | Follow-up Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Follow-up Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis ... | X-Linked Retinitis Pigmentosa | Biological: Genetic: AAV5-RPGR 4e11;Biological: Genetic: AAV5-RPGR 2e11 | MeiraGTx UK II Ltd | Janssen Research & Development, LLC | Recruiting | 3 Years | N/A | All | 66 | Phase 3 | United States |
| 7 | NCT04671433 (ClinicalTrials.gov) | March 16, 2021 | 5/11/2020 | Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in th ... | Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa ... | X-Linked Retinitis Pigmentosa | Biological: Genetic: AAV5-RPGR | MeiraGTx UK II Ltd | Janssen Research & Development, LLC | Recruiting | 3 Years | N/A | All | 66 | Phase 3 | United States |
| 8 | NCT03316560 (ClinicalTrials.gov) | April 16, 2018 | 10/10/2017 | Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by R ... | A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2t ... | X-Linked Retinitis Pigmentosa | Biological: rAAV2tYF-GRK1-RPGR | Applied Genetic Technologies Corp | NULL | Recruiting | 6 Years | 50 Years | Male | 42 | Phase 1/Phase 2 | United States |
| 9 | JPRN-UMIN000023618 | 2016/09/01 | 01/09/2016 | Comparison of posterior capsule opacification between 2 intraocular lenses in cataract patients associated with retinitis pigmentsa Comparison of posterior capsule opacification between 2 intraocular lenses in cataract patients asso ... | Cataract associated with patients with retinitis pigmentosa | Cataract surgery and implantation of 1P intraocular lens (PCB00V) Cataract surgery and implantation of 3P intraocular lens (VA-70AD) Cataract surgery and implantation of 1P intraocular lens (PCB00V) Cataract surgery and implantation o ... | Kyushu University Hospital | NULL | Complete: follow-up complete | 40years-old | Not applicable | Male and Female | 40 | Not selected | Japan | |
| 10 | NCT04356716 (ClinicalTrials.gov) | November 11, 2014 | 20/4/2020 | Sildenafil for Treatment of Choroidal Ischemia | Sildenafil for Treatment of Choroidal Ischemia | Choroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis Pigmentosa Choroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Ret ... | Drug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA) Drug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Ang ... | Columbia University | NULL | Recruiting | 18 Years | N/A | All | 25 | Phase 2 | United States |
301. 黄斑ジストロフィー
臨床試験数 : 45 / 薬物数 : 46 - (DrugBank : 12) / 標的遺伝子数 : 9 - 標的パスウェイ数 : 67
Showing 1 to 2 of 2 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04356716 (ClinicalTrials.gov) | November 11, 2014 | 20/4/2020 | Sildenafil for Treatment of Choroidal Ischemia | Sildenafil for Treatment of Choroidal Ischemia | Choroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis Pigmentosa Choroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Ret ... | Drug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA) Drug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Ang ... | Columbia University | NULL | Recruiting | 18 Years | N/A | All | 25 | Phase 2 | United States |
| 2 | NCT00470977 (ClinicalTrials.gov) | May 2007 | 4/5/2007 | Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV ... | Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy With Intravitreal Injection of Lucentis (Ranibizumab Injection) Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV ... | Coats' Disease;Idiopathic Retinal Telangiectasia;Retinal Angiomatous Proliferation;Polypoidal Choroidal Vasculopathy;Pseudoxanthoma Elasticum;Pathological Myopia;Multi-focal Choroiditis;Rubeosis Iridis;Von Hippel Lindau Disease;BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder) Coats' Disease;Idiopathic Retinal Telangiectasia;Retinal Angiomatous Proliferation;Polypoidal Choroi ... | Drug: ranibizumab injection (0.5 mg) | Manhattan Eye, Ear & Throat Hospital | Genentech, Inc. | Completed | 18 Years | N/A | Both | 18 | Phase 1/Phase 2 | United States |
302. レーベル遺伝性視神経症
臨床試験数 : 22 / 薬物数 : 16 - (DrugBank : 4) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 33
Showing 1 to 3 of 3 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2015-001266-26-IT (EUCTR) | 18/03/2016 | 10/05/2019 | A Phase III gene therapy clinical trial in LHON Subjects Affected for more 7 months | Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valutare l'efficacia di un'unica iniezione intravitreale di GS010 (rAAV2/2-ND4) in soggetti affetti, per un periodo maggiore di 6 mesi e fino a 12 mesi da neuropatia ottica ereditaria di Leber a causa della mutazione G11778A nel gene mitocondriale NADH deidrogenasi 4 - REVERSE Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valut ... | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene MedDRA version: 20.1;Level: LLT;Classification code 10062951;Term: Leber's hereditary optic atrophy neuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Eye Diseases [C11] Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene Me ... | Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene Product Code: GS010 Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | France;United States;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2015-001265-11-IT (EUCTR) | 18/03/2016 | 04/11/2020 | A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or Less | Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valutare l'efficacia di un'unica iniezione intravitreale di GS010 (rAAV2/2-ND4) in soggetti affetti, per un periodo di 6 mesi o inferiore, da neuropatia ottica ereditaria di Leber a causa della mutazione G11778A nel gene mitocondriale NADH deidrogenasi 4 - RESCUE Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valut ... | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene MedDRA version: 20.1;Level: LLT;Classification code 10062951;Term: Leber's hereditary optic atrophy neuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Eye Diseases [C11] Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene Me ... | Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene Product Code: GS010 INN or Proposed INN: PENDING Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;France;Germany;United Kingdom;Italy | ||
| 3 | JPRN-UMIN000017939 | 2013/10/01 | 20/06/2015 | Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neurop ... | Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy - Clinical trial of Idebenone in patients with LHON Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neurop ... | Leber hereditary optic neuropathy | 1. Clinical trial medicine: Idebenone 900mg/day 2. Objectives: 50 patients with LHON 3. Exclusion criteria: a) A smoker b) A patient with abnormality of hepatic function c) A patient who present seizures, delirium and hallucination d) Pregnancy or Lactation e) A patient who is associated with agranulocytosis f) A patient with chronic renal failure g) A patient with anaphylactic shock against Idebenone 4. Duration of drug administration: 6 months 5. Examinations schedules: Both subjective and objective examinations are performed as following schedules; a) At the base line: Visual acuity (VA), Critical flicker frequency (CFF), Visual field (VF: Humphry 30-2), central retinal thickness (CRT), functional MRI (f-MRI), searching the mitochondrial mutation b) 8 weeks: VA, CFF, VF, CRT, f-MRI c) 16 weeks: VA, CFF, VF, CRT, f-MRI d) 24 weeks: VA, CFF, VF, CRT, f-MRI e) 32 weeks: VA, CFF, VF, CRT, f-MRI f) 40 weeks: VA, CFF, VF, CRT, f-MRI g) 48 weeks: VA, CFF, VF, CRT, f-MRI 1. Clinical trial medicine: Idebenone 900mg/day 2. Objectives: 50 patients with LHON 3. Exclusion cri ... | Hyogo College of Medicine | Kitasato UniversityJikei University School of MedicineTokyo Medical University | Complete: follow-up complete | 10years-old | 80years-old | Male and Female | 50 | Phase 1,2 | Japan |