Clinical trials: Optic Atrophies, Hereditary
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
Leber's Hereditary Optic Neuropathy
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy
This is a Phase 2, prospective, randomized, double-masked, vehicle controlled, single-center study in approximately 12 subjects with LHON to evaluate safety, tolerability and efficacy of elamipretide (MTP-131) topical ophthalmic…
- Trial name
- NCT02693119
- Testing
- elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Stealth BioTherapeutics Inc.
Clinical and Nutritional Safety.
Oat Products in the Treatment of Coeliac Disease in Children
In many Northern European countries oat-based products have been used in the dieto-therapy of coeliac disease for many years. The purpose of this study is to evaluate clinical tolerance and liking of gluten-free products…
- Trial name
- NCT00808301
- Testing
- Gluten-free products
- Where
- 7 sites in Italy
- Volunteers
- 300 people
- Sponsor
- Heinz Italia SpA
Multiple Sclerosis, Tomography, Optical Coherence
Retinal Atrophy and Neurofilament Light Chain in People With Multiple Sclerosis Taking Ofatumumab
This study aims to assess whether, and the degree to which, ofatumumab modulates or reduces rates of retinal atrophy in people with relapsing-remitting MS (RMS), according to baseline serum neurofilament light chain (sNfL) levels.
- Conditions
- Multiple Sclerosis, Tomography, Optical Coherence, Retinal Degeneration, Visual Acuity
- Trial name
- NCT06167642
- Testing
- Ofatumumab
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Johns Hopkins University
Stargardt Disease
Safety and Efficacy of Emixustat in Stargardt Disease
The purpose of this study is to determine if emixustat hydrochloride reduces the rate of progression of macular atrophy compared to placebo in subjects with Stargardt disease. Funding Source -- FDA OOPD
- Trial name
- SeaSTAR (NCT03772665)
- Testing
- Emixustat
- Where
- 29 sites in United States, Brazil and 9 more
- Volunteers
- 194 people
- Sponsor
- Kubota Vision Inc.
Gestational Diabetes Mellitus in Pregnancy
Impact of DHA/Oat on Metabolic Health in Gestational Diabetes Mellitus
The randomized controlled trial (RCT) recruits pregnant women with de novo diagnosis of gestational diabetes. Women bearing a singleton pregnancy are randomized into four arms: DHA, oat, oat plus DHA, and placebo.
- Trial name
- NCT03569501
- Testing
- DHA, oat grains
- Where
- China
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
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
Cardiovascular Diseases, Coronary Heart Disease
Meta-analysis of Oat Fiber and Cardiovascular Risk Reduction
Oat fibre has been shown to lower cholesterol and may have cardioprotective effects. However, whether this translates to actual cardiovascular risk reduction is unclear, as there is a lack of controlled human trials.
- Trial name
- NCT05171283
- Testing
- Oats or Oat Fibre
- Where
- Canada
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- University of Toronto
Leber's Hereditary Optic Neuropathy (LHON)
Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
The purpose of this survey is to collect visual acuity data from patients with LHON in order to establish the clinical course (natural history) and visual acuity outcomes in patients with a genetically confirmed diagnosis of…
- Trial name
- NCT02796274
- Where
- 21 sites in Belgium, France and 5 more
- Volunteers
- 219 people
- Sponsor
- Santhera Pharmaceuticals
OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA.
- Conditions
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
- Trial name
- PYC-001-CL-102 (NCT06970106)
- Testing
- PYC-001
- Where
- 3 sites in Australia, New Zealand
- Volunteers
- 24 people
- Sponsor
- PYC Therapeutics
Stargardt Disease
An Observational Study in Children and Adults With Stargardt Disease
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease…
- Trial name
- NCT06591806
- Where
- 2 sites in United States, Norway
- Volunteers
- 80 people
- Sponsor
- AAVantgarde Bio Srl
Retinitis Pigmentosa, Choroideremia
Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
The Vision Research and Assessment Institute (VRAI) was established with the purpose of serving as a testing facility for efficacy endpoints for patients with Low Vision.
- Conditions
- Retinitis Pigmentosa, Choroideremia, Stargardt Macular Dystrophy, Stargardt Disease
- Trial name
- NCT06375239
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Ray Therapeutics, Inc.
Retinal Degeneration, Retinal Diseases
SGLT2 Inhibitors in Geographic Atrophy
AMD is a leading cause of blindness in individuals over 50 years old, with dry AMD being the most common form. Geographic atrophy (GA) is an advanced stage of dry AMD characterized by progressive retinal cell degeneration.
- Conditions
- Retinal Degeneration, Retinal Diseases, Eye Diseases, Geographic Atrophy
- Trial name
- NCT07174687
- Testing
- Dapagliflozin
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Washington University School of Medicine
Macular Degeneration Advanced, Macular Degeneration, Senile
Visual Rehabilitation Through Acoustic Biofeedback With Retimax Vision Trainer in Patients With Advanced Age-related Macular Degeneration
At present there are no real therapies able to improve visual performance in patients with age-related macular degeneration, atrophic type.
- Conditions
- Macular Degeneration Advanced, Macular Degeneration, Senile, Macular Degeneration, Dry, Macular Scar
- Trial name
- NCT04084587
- Testing
- Retimax Vision Trainer
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- University of Modena and Reggio Emilia
Leber Congenital Amaurosis 10, Blindness
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- ILLUMINATE (NCT03913143)
- Testing
- sepofarsen
- Where
- 14 sites in United States, Belgium and 7 more
- Volunteers
- 36 people
- Sponsor
- ProQR Therapeutics
Optic Pathway Glioma
Safety and Efficacy of the PAINLESS Nerve Growth Factor CHF6467 in Optic Pathway Glioma (OPG)
Infantile optic pathway glioma (OPG) is generally benign and slow-growing, but due to infiltration and compression of sensitive neuronal structures in the optical pathways, progressive visual loss is a frequent and highly…
- Trial name
- NGF-OPG (NCT05733572)
- Testing
- CHF6467
- Where
- Italy
- Volunteers
- 36 people
- Sponsor
- Benedetto Falsini
Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON)
Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy
Leber Hereditary Optic Neuropathy (LHON) is a rare genetic disease that causes sudden and severe vision loss, usually in young adults.
- Conditions
- Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON), Mitochondrial Disease, Optic Nerve Disease
- Trial name
- NICOLHON (NCT07258667)
- Testing
- Nicotinamide treatment
- Where
- France
- Volunteers
- 13 people
- Sponsor
- University Hospital, Angers
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
OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
- Conditions
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
- Trial name
- NCT06461286
- Testing
- PYC-001
- Where
- 2 sites in Australia
- Volunteers
- 18 people
- Sponsor
- PYC Therapeutics
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