Clinical trials: Optic Neuropathy
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Acute Demylelinating Optic Neuritis
Fingolimod (FTY720) in Acute Demyelinating Optic Neuritis (ADON)
To evaluate the efficacy and safety of fingolimod 0.5mg versus placebo in patients with suspected acute demyelinating optic neuritis (ADON) receiving standard steroid treatment
- Trial name
- NCT01757691
- Testing
- Fingolimod 0.5mg/daily
- Where
- 2 sites in United States, Spain
- Volunteers
- 2 people
- Sponsor
- Novartis Pharmaceuticals
Open Angle Glaucoma (OAG), NAION( Non-arteritic Anterior Ischemic Optic Neuropathy)
Evaluating ER-100 for Safety in People With Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy (Optic Nerve Conditions)
The goal of this clinical trial is to evaluate the safety and tolerability of a single dose of ER-100 in adults with optic nerve conditions, specifically Open Angle Glaucoma (OAG) and Non-Arteritic Anterior Ischemic Optic…
- Trial name
- NCT07290244
- Testing
- ER-100 epigenetic therapy
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Life Biosciences Inc.
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- REVERSE (NCT02652780)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 37 people
- Sponsor
- GenSight Biologics
Non-arteritic Ischemic Optic Neuropathy, Optic Nerve Injuries
Safety and Efficacy Study of RPh201 Treatment of Ischemic Optic Neuropathy (ION).
The study objectives are to assess any changes in visual acuity and visual field observed following the administration of RPh201 during an overall treatment period of at least 13 consecutive weeks with an option to extended the…
- Trial name
- NCT02045212
- Testing
- RPh201
- Where
- Israel
- Volunteers
- 22 people
- Sponsor
- Regenera Pharma Ltd
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Leber Hereditary Optic Neuropathy
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
- Trial name
- NCT02064569
- Testing
- GS010
- Where
- France
- Volunteers
- 19 people
- Sponsor
- GenSight Biologics
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
This pivotal, phase 3 study is designed to confirm the efficacy and safety of setmelanotide, a potent melanocortin receptor type 4 (MC4R) agonist, for the treatment of obesity and hyperphagia in participants with Bardet Biedl…
- Trial name
- NCT03746522
- Testing
- Setmelanotide
- Where
- 12 sites in United States, Canada and 4 more
- Volunteers
- 52 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
- Trial name
- NCT04201262
- Testing
- Ravulizumab
- Where
- 41 sites in United States, Australia and 11 more
- Volunteers
- 58 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Optic Nerve Disease, Macular Disease
Implementation of the NEDS EyeCTester App
Group 4a and 4b was an FDA trial that showed that patients who have an Amsler grid abnormality on paper, have a similar abnormality on the app.
- Conditions
- Optic Nerve Disease, Macular Disease, Visual Pathway Disorder
- Trial name
- NCT04607369
- Testing
- EyeCTester
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Neuro-Eye Diagnostic Systems, LLC
Neuromyelitis Optica Spectrum Disorder
Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
A Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible pediatric participants 2 to < 18 years of age with recently active neuromyelitis optica…
- Trial name
- NCT05549258
- Testing
- Inebilizumab
- Where
- 19 sites in United States, Argentina and 9 more
- Volunteers
- 15 people
- Sponsor
- Amgen