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 302. レーベル遺伝性視神経症 [臨床試験数:8,薬物数:10(DrugBank:3),標的遺伝子数:5,標的パスウェイ数:7] 

Searched query = "Leber hereditary optic neuropathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-002187-40-BE15/03/201828 February 2019Efficacy and Safety of Bilateral Intravitreal Injection of GS010Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year - REFLECTLeber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene ;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
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: lenadogene nolparvovec
CAS Number: 1640969-63-6
Current Sponsor code: GS010
Other descriptive name: Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4)
Concentration unit: vector genomes (vg)/mL
Concentration type: not less then
Concentration number: 1E12-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Intravitreal use
GENSIGHT BIOLOGICSAuthorised Female: yes
Male: yes
90Phase 3France;Spain;Belgium;Netherlands;Italy;United Kingdom
2NCT03153293December 27, 20174 November 2019Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic NeuropathySafety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic NeuropathyLeber Hereditary Optic NeuropathyDrug: rAAV2-ND4Huazhong University of Science and TechnologyShiyan Taihe HospitalNot recruiting10 Years65 YearsAll159Phase 2/Phase 3China
3EUCTR2015-001265-11-DE21/01/20163 April 2017A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or LessA Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 (rAAV2/2-ND4) in Subjects Affected for 6 Months or Less by Leber Hereditary Optic Neuropathy Due to the G11778A Mutation in the Mitochondrial NADH Dehydrogenase 4 GeneLeber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;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
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: pending
CAS Number: 1640969-63-6
Current Sponsor code: rAAV2/2-ND4
Other descriptive name: RAAV2/2-ND4 VECTOR
Concentration unit: titre titre
Concentration type: equal
Concentration number: 1E12-
GENSIGHT-BIOLOGICSAuthorisedFemale: yes
Male: yes
40Phase 3France;United States;Germany;Italy;United Kingdom
4EUCTR2015-001266-26-DE21/01/201630 April 2019A Phase III gene therapy clinical trial in LHON Subjects Affected for more than 7months or moreA Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 (rAAV2/2-ND4) in Subjects Affected for more than 6 months and to 12 mounths by Leber Hereditary Optic Neuropathy Due to the G11778A Mutation in the Mitochondrial NADH Dehydrogenase 4 GeneLeber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;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
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: pending
CAS Number: 1640969-63-6
Current Sponsor code: rAAv2/2-ND4
Other descriptive name: RAAV2/2-ND4 VECTOR
Concentration unit: titre titre
Concentration type: equal
Concentration number: 1E12-
GENSIGHT-BIOLOGICSNot Recruiting Female: yes
Male: yes
40Phase 3France;United States;Germany;Italy;United Kingdom
5EUCTR2013-001405-90-FR26/12/201310 March 2014A Phase I/IIa gene therapy clinical trial in LHON patientsA phase I/IIa, non randomized, escalating dose, open-label study to evaluate safety and efficacy of GS010 (rAAV2/2-ND4) in patients suffering from Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 geneLeber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
MedDRA version: 16.1 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
Pharmaceutical Form: Suspension for injection
Other descriptive name: RAAV2/2-ND4 VECTOR
Concentration unit: titre titre
Concentration type: range
Concentration number: 6.3E11-2.0E13
GENSIGHT-BIOLOGICSAuthorisedFemale: yes
Male: yes
Phase 1/2aFrance
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6JPRN-UMIN0000179392013/10/0122 July 2019Multicenter 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 LHONLeber 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 University Jikei University School of Medicine Tokyo Medical UniversityNot Recruiting10years-old80years-oldMale and Female50Phase 1,2Japan
7NCT02176733July 201119 February 2015Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic NeuropathyLeber Hereditary Optic NeuropathyDrug: cyclosporineUniversity Hospital, AngersRecruiting18 YearsN/ABoth12Phase 2France
8NCT01267422April 201119 October 2017Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic NeuropathyLeber Hereditary Optic NeuropathyDrug: rAAV2-ND4Bin LiHuazhong University of Science and TechnologyNot recruiting8 Years60 YearsAll9N/AChina

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