Clinical trials: Leber Hereditary Optic Neuropathy
Leber's Hereditary Optic Neuropathy (LHON)
Evaluation of Impact of Disease and Visual Disability on Quality of Life and Loss of Independence of Patients Living in France With Leber's Hereditary Optic Neuropathy (LHON) Through Qualitative and Quantitative Data Collection
Leber's hereditary optic neuropathy (LHON) is a rare mitochondrial genetic disorder characterized by optic nerve atrophy due to the degeneration of retinal ganglion cells, which leads to acute visual loss.
- Trial name
- NCT05555784
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Argo Sante
Leber's Hereditary Optic Neuropathy
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
The study is a dose-escalation study, phase 1. The objective of this proposed clinical trial is to evaluate the safety of mitochondrially targeted ND4 gene therapy with the adeno-associated viral vector in appropriate LHON…
- Trial name
- LHON (NCT02161380)
- Testing
- injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Byron Lam
Leber's Hereditary Optic Neuropathy (LHON)
Study to Assess the Efficacy and Safety of Raxone in LHON Patients
LEROS is an open-label interventional Phase IV study, designed to further assess the efficacy and safety of Raxone® in the long-term treatment of LHON patients.
- Trial name
- LEROS (NCT02774005)
- Testing
- Idebenone
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 199 people
- Sponsor
- Santhera Pharmaceuticals
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Leber's Hereditary Optic Neuropathy (LHON)
Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)
The overall objective of the proposed research is to test the hypothesis that Near-infrared Light-emitting Diode (NIR-LED) therapy will stimulate mitochondrial function, attenuate oxidative stress, and improve cell survival and…
- Trial name
- LHON (NCT01389817)
- Testing
- Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))
- Where
- 3 sites in United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Medical College of Wisconsin
Optic Atrophy, Hereditary, Leber
A Randomized, Double-blind, Placebo-controlled Trial of Curcumin in Leber's Hereditary Optic Neuropathy (LHON)
Background Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited ocular disorder associated with a mutation in mtDNA .
- Trial name
- NCT00528151
- Testing
- curcumin
- Where
- Thailand
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Mahidol University
Leber Congenital Amaurosis 10, Blindness
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders
- Trial name
- HYPERION (NCT06891443)
- Testing
- sepofarsen
- Where
- 18 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Laboratoires Thea
Leber Hereditary Optic Neuropathy (LHON)
Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations
The objective of this clinical study is to evaluate the safety and efficacy of NR082 in the treatment of LHON caused by mitochondrial ND4 gene mutation.
- Trial name
- NCT05293626
- Testing
- NR082 Injection, Injection needle
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Neurophth Therapeutics Inc
Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
EAP_GS010_single Patient
Expanded Access Use for a single patient of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected with G11778A ND4 Leber Hereditary Optic Neuropathy
- Trial name
- NCT03672968
- Testing
- GS010
- Sponsor
- GenSight Biologics
Leber Hereditary Optic Neuropathy
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.
- Trial name
- rAAV2-ND4 (NCT01267422)
- Testing
- rAAV2-ND4
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Bin Li
Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion
New Non-invasive Modalities for Assessing Retinal Structure and Function
This study investigates a new technology to assess the structure and function inside the eye. Retinal imaging of subjects with inner and outer retinal defects to detect areas of abnormal structure and function compared to other…
- Conditions
- Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy
- Trial name
- NCT03475173
- Testing
- LSFG-NAVI
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Randy Kardon
Neuro-Ophthalmology, Optic Neuropathies
Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study
OCT-A and eco-colordoppler are technologies which enable optic nerve head (ONH) blood perfusion detection. The aim of the study is to assess ONH vascular and structural components in optic neuropathies with and without ONH edema…
- Conditions
- Neuro-Ophthalmology, Optic Neuropathies, Optic Atrophy, Hereditary, Leber, Optic Neuropathy, Anterior Ischemic
- Trial name
- FlowONH (NCT07075887)
- Volunteers
- 70 people
- Sponsor
- Barboni
Leber Hereditary Optic Neuropathy (LHON)
Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations
The objective of this clinical study is to evaluate the safety, tolerability and preliminary efficacy of NFS-02 in the treatment of LHON caused by mitochondrial ND1 gene mutation.
- Trial name
- NCT05820152
- Testing
- NFS-02 Injection
- Where
- 4 sites in United States, China
- Volunteers
- 11 people
- Sponsor
- Neurophth Therapeutics Inc
Leber's Hereditary Optic Neuropathy
Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy
This study is meant to assess the effectiveness of idebenone on visual function measures in patients with Leber's Hereditary Optic Neuropathy over a 6 months period.
- Trial name
- RHODOS (NCT00747487)
- Testing
- Idebenone
- Where
- 3 sites in Canada, Germany and 1 more
- Volunteers
- 85 people
- Sponsor
- Santhera Pharmaceuticals
Safety Issues, Efficacy, Self
Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Study of the efficiency of Béfizal® 200 mg in 14 adult patients with a LHON that occurred for less than 5 years. Patient must have certain specific mutations
- Trial name
- Béfinohl (NCT04561466)
- Testing
- Béfizal
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Hôpital Necker-Enfants Malades
Healthy Subjects, Leber Hereditary Optic Neuropathy
Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT)
Leber hereditary optic neuropathy (LHON), due to mitochondrial DNA (mtDNA) mutations, is responsible for profound visual impairment. However, there is evidence that optic nerve damage begins before vision declines.
- Trial name
- MétabOCT (NCT06682819)
- Testing
- Optical coherent tomography
- Where
- France
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Hôpital Necker-Enfants Malades
Leber's Hereditary Optic Neuropathy (LHON)
Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
The objective of this clinical study is to select the optimal dose and evaluate the safety and efficacy of NR082 in treatment of LHON caused by mitochondrial ND4 gene mutation.
- Trial name
- GOLD (NCT04912843)
- Testing
- NR082 injection
- Where
- China
- Volunteers
- 102 people
- Sponsor
- Wuhan Neurophth Biotechnology Limited Company
Leber's Hereditary Optic Neuropathy
Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON)
The objective of the study is to determine whether administration of idebenone can shorten the time to improvement of visual acuity in patients with chronic vision loss due to LHON.
- Trial name
- NCT01495715
- Testing
- Idebenone
- Sponsor
- Santhera Pharmaceuticals
Leber Hereditary Optic Neuropathy
REALITY LHON Registry
This study is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on retrospective subjects' medical chart abstractions and cross-sectional administration of patient-reported…
- Trial name
- REALITY (NCT03295071)
- Testing
- Patient-reported outcomes (PROs)
- Where
- 11 sites in United States, France and 3 more
- Volunteers
- 44 people
- Sponsor
- GenSight Biologics
Leber's Hereditary Optic Neuropathy
Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
Expanded access Protocol to treat LHON subjects with EPI743
- Trial name
- NCT02300753
- Testing
- EPI-743
- Where
- United States
- Sponsor
- Edison Pharmaceuticals Inc