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Va    (DrugBank: -)

3 diseases
IDDisease name (Link within this page)Number of trials
90Retinitis pigmentosa5
301Macular dystrophy2
302Leber hereditary optic neuropathy3

90. Retinitis pigmentosa    [ 103 clinical trials,   158 drugs,   (DrugBank: 42 drugs),   52 drug target genes,   107 drug target pathways]
Searched query = "Retinitis pigmentosa", "Rod dystrophy", "Rod-Cone Dystrophy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
5 / 103 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1JPRN-UMIN000023618
2016/09/0101/09/2016Comparison of posterior capsule opacification between 2 intraocular lenses in cataract patients associated with retinitis pigmentsa Cataract associated with patients with retinitis pigmentosaCataract surgery and implantation of 1P intraocular lens (PCB00V)
Cataract surgery and implantation of 3P intraocular lens (VA-70AD)		Kyushu University HospitalNULLComplete: follow-up complete40years-oldNot applicableMale and Female40Not selectedJapan
2NCT04356716
(ClinicalTrials.gov)		November 11, 201420/4/2020Sildenafil for Treatment of Choroidal IschemiaSildenafil for Treatment of Choroidal IschemiaChoroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis PigmentosaDrug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA)Columbia UniversityNULLRecruiting18 YearsN/AAll25Phase 2United States
3NCT01399515
(ClinicalTrials.gov)		March 20113/5/2011Efficacy and Safety of Oral Valproic Acid for Retinitis PigmentosaRetinitis Pigmentosa;Retinal Diseases;Eye Diseases;Eye Disease, Hereditary;Retinal DegenerationDrug: Valproic AcidSeoul National University HospitalNULLCompleted18 YearsN/ABoth200Phase 2Korea, Republic of
4JPRN-JMA-IIA00053
24/12/201022/11/2010Clinical Trial of Therapeutic Effect of Valproic Acid in Retinitis PigmentosaClinical Trial of Therapeutic Effect of Valproic Acid in Retinitis Pigmentosa Retinitis PigmentosaIntervention type:DRUG. Intervention1:Sodium valproate, Dose form:TABLET, Route of administration:ORAL.Yasuhiko HiramiMasayo Takahashi, Yasuo KurimotoCompleted>=20 YEARSNo LimitBOTH30NOT APPLICABLEJapan
5NCT01233609
(ClinicalTrials.gov)		November 20101/11/2010Trial of Oral Valproic Acid for Retinitis PigmentosaA Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Autosomal Dominant Retinitis PigmentosaRetinitis PigmentosaDrug: Valproic Acid;Drug: PlaceboFoundation Fighting Blindness Clinical Research InstituteUnited States Department of DefenseCompleted18 YearsN/AAll90Phase 2United States

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301. Macular dystrophy    [ 41 clinical trials,   47 drugs,   (DrugBank: 12 drugs),   12 drug target genes,   66 drug target pathways]
Searched query = "Macular dystrophy", "Best disease", "Stargardt disease", "Cone dystrophy", "Cone rod dystrophy", "X-linked juvenile retinoschisis", "Central areolar choroidal dystrophy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 41 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT04356716
(ClinicalTrials.gov)		November 11, 201420/4/2020Sildenafil for Treatment of Choroidal IschemiaSildenafil for Treatment of Choroidal IschemiaChoroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis PigmentosaDrug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA)Columbia UniversityNULLRecruiting18 YearsN/AAll25Phase 2United States
2NCT00470977
(ClinicalTrials.gov)		May 20074/5/2007Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related MaculopathyTreatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy With Intravitreal Injection of Lucentis (Ranibizumab Injection)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)Drug: ranibizumab injection (0.5 mg)Manhattan Eye, Ear & Throat HospitalGenentech, Inc.Completed18 YearsN/ABoth18Phase 1;Phase 2United States

302. Leber hereditary optic neuropathy    [ 20 clinical trials,   14 drugs,   (DrugBank: 4 drugs),   5 drug target genes,   32 drug target pathways]
Searched query = "Leber hereditary optic neuropathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 20 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2015-001265-11-IT
(EUCTR)		18/03/201604/11/2020A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or LessSperimentazione 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 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]		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		GENSIGHT BIOLOGICSNULLNot RecruitingFemale: yes
Male: yes		40Phase 3United States;France;Germany;United Kingdom;Italy
2EUCTR2015-001266-26-IT
(EUCTR)		18/03/201610/05/2019A Phase III gene therapy clinical trial in LHON Subjects Affected for more 7 monthsSperimentazione 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 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]		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		GENSIGHT BIOLOGICSNULLNot RecruitingFemale: yes
Male: yes		40Phase 3France;United States;Germany;United Kingdom;Italy
3JPRN-UMIN000017939
2013/10/0120/06/2015Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathyMulticenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy - Clinical trial of Idebenone in patients with LHON Leber hereditary optic neuropathy1. 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		Hyogo College of MedicineKitasato UniversityJikei University School of MedicineTokyo Medical UniversityComplete: follow-up complete10years-old80years-oldMale and Female50Phase 1;Phase 2Japan

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