VA
( DrugBank: - / KEGG DRUG: - )
2 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 | 24 |
告示番号 | 疾患名(ページ内リンク) | 臨床試験数 |
---|---|---|
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 : 147 / 薬物数 : 176 - (DrugBank : 43) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 110
Showing 1 to 10 of 24 trials
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 |
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1 | NCT05537220 (ClinicalTrials.gov) | June 202320230600 | 8/9/202220220908 | Oral N-acetylcysteine for Retinitis Pigmentosa | NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis Pigmentosa NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study ... | Retinitis Pigmentosa | Drug: N-acetylcysteine;Drug: Placebo | Johns Hopkins University | National Eye Institute (NEI);Duke University;Emory University;Massachusetts Eye and Ear Infirmary;Mayo Clinic;Medical College of Wisconsin;Retina Foundation of the Southwest;Stanford University;University of California, Davis;University of Florida;University of Illinois at Chicago;University of Iowa;University of Miami;University of Michigan;University of Minnesota;University of Oklahoma;University of Southern California;University of Utah;University of Washington;University of Wisconsin, Madison;Vanderbilt University;Vitreo Retinal Associates, PA;University of Houston;Medical University of Graz;McGill University;Universität Tübingen;Centro Medico ABC;Radboud University Medical Center;University of Amsterdam;University Hospital, Basel, Switzerland;University College London Hospitals;Northwestern University National Eye Institute (NEI);Duke University;Emory University;Massachusetts Eye and Ear Infirmary;Ma ... | Not yet recruiting | 18 Years | 65 Years | All | 438 | Phase 3 | United States;Austria;Canada;Germany;Mexico;Netherlands;Switzerland;United Kingdom |
2 | EUCTR2020-002873-88-IE (EUCTR) | 19/07/202220220719 | 15/10/202120211015 | 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: Botaretigene Sparoparvovec Product Code: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: Botaretigene Sparoparvovec Product Code: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: Botaretigene Sparoparvovec Product Code: AAV5-hRKp.RPGR INN or Proposed INN: botaretigen ... | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Phase 3 | United States;Spain;Ireland;Israel;Switzerland;Italy;United Kingdom;France;Canada;Belgium;Denmark;Netherlands;Germany United States;Spain;Ireland;Israel;Switzerland;Italy;United Kingdom;France;Canada;Belgium;Denmark;Ne ... | ||
3 | EUCTR2020-002255-37-IE (EUCTR) | 26/05/202220220526 | 15/10/202120211015 | 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 ... | ||
4 | EUCTR2020-002255-37-ES (EUCTR) | 28/03/202220220328 | 02/11/202120211102 | 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 | EUCTR2020-002873-88-ES (EUCTR) | 28/03/202220220328 | 02/11/202120211102 | 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 ... | ||
6 | EUCTR2020-002255-37-NL (EUCTR) | 14/03/202220220314 | 22/10/202120211022 | 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 ... | ||
7 | EUCTR2020-002873-88-NL (EUCTR) | 14/03/202220220314 | 22/10/202120211022 | 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 ... | ||
8 | EUCTR2020-002255-37-DK (EUCTR) | 01/03/202220220301 | 08/11/202120211108 | 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 ... | ||
9 | EUCTR2020-002873-88-DK (EUCTR) | 01/03/202220220301 | 08/11/202120211108 | 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 ... | ||
10 | NCT04850118 (ClinicalTrials.gov) | August 202120210800 | 5/4/202120210405 | 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 |
302. レーベル遺伝性視神経症
臨床試験数 : 23 / 薬物数 : 15 - (DrugBank : 5) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 33
Showing 1 to 3 of 3 trials
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 |
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1 | EUCTR2015-001265-11-IT (EUCTR) | 18/03/201620160318 | 04/11/202020201104 | 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 | ||
2 | EUCTR2015-001266-26-IT (EUCTR) | 18/03/201620160318 | 10/05/201920190510 | 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 | ||
3 | JPRN-UMIN000017939 | 2013/10/0120131001 | 20/06/201520150620 | 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 |