302. Leber hereditary optic neuropathy
[
8 clinical trials,
10 drugs（DrugBank:
3 drugs),
5 target genes / 30 target pathways ]
Searched query = "Leber hereditary optic neuropathy"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2017-002187-40-BE | 15/03/2018 | 28 February 2019 | Efficacy and Safety of Bilateral Intravitreal Injection of GS010 | Efficacy 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 - REFLECT | Leber 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 BIOLOGICS | Authorised | Female: yes Male: yes | 90 | Phase 3 | France;Spain;Belgium;Netherlands;Italy;United Kingdom | |||
| 2 | NCT03153293 | December 27, 2017 | 4 November 2019 | Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic Neuropathy | Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic Neuropathy | Leber Hereditary Optic Neuropathy | Drug: rAAV2-ND4 | Huazhong University of Science and Technology | Shiyan Taihe Hospital | Not recruiting | 10 Years | 65 Years | All | 159 | Phase 2/Phase 3 | China |
| 3 | EUCTR2015-001265-11-DE | 21/01/2016 | 3 April 2017 | A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or Less | A 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 Gene | Leber 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-BIOLOGICS | Authorised | Female: yes Male: yes | 40 | Phase 3 | France;United States;Germany;Italy;United Kingdom | |||
| 4 | EUCTR2015-001266-26-DE | 21/01/2016 | 30 April 2019 | A Phase III gene therapy clinical trial in LHON Subjects Affected for more than 7months or more | A 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 Gene | Leber 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-BIOLOGICS | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | France;United States;Germany;Italy;United Kingdom | |||
| 5 | EUCTR2013-001405-90-FR | 26/12/2013 | 10 March 2014 | A Phase I/IIa gene therapy clinical trial in LHON patients | A 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 gene | Leber 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-BIOLOGICS | Authorised | Female: yes Male: yes | Phase 1/2a | France | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | JPRN-UMIN000017939 | 2013/10/01 | 22 July 2019 | 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 neuropathy - Clinical trial of Idebenone in patients with LHON | 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 | Hyogo College of Medicine | Kitasato University Jikei University School of Medicine Tokyo Medical University | Not Recruiting | 10years-old | 80years-old | Male and Female | 50 | Phase 1,2 | Japan |
| 7 | NCT02176733 | July 2011 | 19 February 2015 | Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy | Leber Hereditary Optic Neuropathy | Drug: cyclosporine | University Hospital, Angers | Recruiting | 18 Years | N/A | Both | 12 | Phase 2 | France | ||
| 8 | NCT01267422 | April 2011 | 19 October 2017 | Safety 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 Neuropathy | Leber Hereditary Optic Neuropathy | Drug: rAAV2-ND4 | Bin Li | Huazhong University of Science and Technology | Not recruiting | 8 Years | 60 Years | All | 9 | N/A | China |