Clinical trials: Inherited Ophthalmic Diseases
Retinal Degeneration, ABCA4-Related Retinopathies
Natural History of Eye Diseases Related to ABCA4 Mutations
Background: The ABCA4 gene contains a blueprint for the ABCA4 protein. When this protein is absent or faulty (such as in Stargardt s disease), waste material from dead cells collects in the eye.
- Trial name
- NCT01736293
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- National Eye Institute (NEI)
Behcet Syndrome, Behcet Disease
Low Dose Interferon Alpha Treatment for Oral Ulcers of Behcet's Disease
The purpose of this study is to determine the safety and efficacy of natural human interferon alpha (IFN alpha) on size, number, incidence and pain of oral ulcers associated with Behçet's Disease (BD).
- Conditions
- Behcet Syndrome, Behcet Disease, Mucocutaneous Ulceration
- Trial name
- NCT00483184
- Testing
- Veldona,
- Where
- 4 sites in Turkey (Türkiye)
- Volunteers
- 84 people
- Sponsor
- Nobel Pharmaceuticals
Thyroid Associated Ophthalmopathies
The Safety and Efficacy of Sequential Hormone Therapy and IBI311 Therapy in Patients With Active Moderate to Severe TAO in the Initial Treatment.
Thyroid-associated ophthalmopathy (TAO) is an organ-specific autoimmune disease closely related to thyroid disease, which leads the incidence of orbital disease in adults and is the most common cause of diffuse toxic goiter…
- Trial name
- NCT07152340
- Testing
- IBI311, Glucocorticoids
- Where
- 2 sites in China
- Volunteers
- 64 people
- Sponsor
- Shanghai Changzheng Hospital
Retinopathy of Prematurity
Real World Study of the Effectiveness and Safety of Conbercept Ophthalmic Injection in the Treatment of Retinopathy of Prematurity - Multicenter, Retrospective and Observational Study Based on Real World Data
This study is a retrospective, multi-center real world study. The real world data comes from the electronic medical record system and disease database of the research centers .The patient's demographic information, disease…
- Trial name
- NCT04985448
- Where
- 2 sites in China
- Volunteers
- 1,000 people
- Sponsor
- Chengdu Kanghong Biotech Co., Ltd.
Neuroblastoma, Ewing's Sarcoma
Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
This study will be carried out in children with diagnosis of cancer with tumors known to express N-glycolylated gangliosides. The disease must be resistant to conventional therapy.
- Conditions
- Neuroblastoma, Ewing's Sarcoma, Wilm's Tumor, Retinoblastoma
- Trial name
- NCT01598454
- Testing
- racotumomab
- Where
- Argentina
- Volunteers
- 15 people
- Sponsor
- Laboratorio Elea Phoenix S.A.
Retinitis Pigmentosa, Cone Rod Dystrophy
Restoring Vision With the Intelligent Retinal Implant System (IRIS V1)in Patients With Retinal Dystrophy
This study evaluates the safety and effectiveness of the Intelligent Retinal Implants System (IRIS V1)
- Conditions
- Retinitis Pigmentosa, Cone Rod Dystrophy, Choroideremia
- Trial name
- IRIS-1 (NCT01864486)
- Testing
- Intelligent Retinal Implant System
- Where
- 5 sites in Austria, France and 1 more
- Volunteers
- 20 people
- Sponsor
- Science Corporation
Retinitis Pigmentosa, Cone-Rod Dystrophy
IRIS PILOT - Extended Pilot Study With a Retinal Implant System
Investigate whether blind subjects that fulfil the patient criteria provided with a Retinal Implant are able to differentiate between simple patterns like horizontal bar, vertical bar and cross.
- Conditions
- Retinitis Pigmentosa, Cone-Rod Dystrophy, Choroideremia
- Trial name
- NCT00427180
- Testing
- Retinal Implant System (IRIS)
- Volunteers
- 20 people
- Sponsor
- Intelligent Medical Implants GmbH
Retinitis Pigmentosa
Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
The purpose of this study is to evaluate the short-term safety of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa.
- Trial name
- NCT01068561
- Testing
- intravitreal injection of autologous bone marrow stem cells
- Where
- Brazil
- Volunteers
- 5 people
- Sponsor
- University of Sao Paulo
Childhood Soft Tissue Sarcoma, Childhood Liver Cancer
Liposomal Doxorubicin in Treating Children With Refractory Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of liposomal doxorubicin in treating children who have…
- Conditions
- Childhood Soft Tissue Sarcoma, Childhood Liver Cancer, Bone Cancer, Brain Tumor
- Trial name
- NCT00019630
- Testing
- doxorubicin HCl liposome
- Where
- 2 sites in United States
- Sponsor
- National Cancer Institute (NCI)
Extensive Macular Atrophy With Pseudodrusen (EMAP), Retinitis Pigmentosa (RP)
Subtenon Autologous Platelet-Rich Plasma in Inherited and Degenerative Retinal Diseases
his prospective, comparative pilot study investigates the safety and functional outcomes of subtenon autologous platelet-rich plasma (PRP) in patients with Retinitis Pigmentosa (RP) and Extensive Macular Atrophy with…
- Trial name
- RETINA-PRP (NCT07341919)
- Testing
- Subtenon Injection of Autologous Platelet-Rich Plasma
- Where
- Brazil
- Volunteers
- 30 people
- Sponsor
- Rubens Camargo Siqueira
Retinopathy of Prematurity
Effect of Vitamin E for Prevention of Retinopathy of Prematurity: A Randomized Clinical Trial.
The retinopathy of prematurity (ROP) is a public health problem, the main causes of ROP are prematurity, use of oxygen, malnutrition and oxidative stress.
- Trial name
- NCT03274596
- Testing
- Vitamin E
- Volunteers
- 90 people
- Sponsor
- Instituto Nacional de Perinatologia Isidro Espinosa de los Reyes
Retinitis Pigmentosa
A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
This integrated Ph1/2 clinical study is to assess the safety, tolerability and pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of OCT-980 in healthy volunteers and participants with RHO-adRP.
- Trial name
- NCT07408232
- Testing
- Phase 1b/2: Cohort 1 - OCT-980 Tablet, Phase 1b/2: Cohort 2 - OCT-980 Tablet, Phase 1b/2: Cohort 3 - OCT-980 Tablet
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Octant, Inc.
Retinitis Pigmentosa
A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)
The primary objective of the study is to assess the safety and tolerability of two dose regimens of recombinant human nerve growth factor (rhNGF) eye drops solution administered over 6 months versus a vehicle control in patients…
- Trial name
- Lumos (NCT02110225)
- Testing
- rhNGF 60 µg/ml eye drops solution, rhNGF 180 µg/ml eye drops solution
- Where
- 5 sites in Italy
- Volunteers
- 50 people
- Sponsor
- Dompé Farmaceutici S.p.A
AMD, nAMD
Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD
ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD or nAMD).
- Conditions
- AMD, nAMD, Wet Age-related Macular Degeneration, wAMD
- Trial name
- ATMOSPHERE (NCT04704921)
- Testing
- ABBV-RGX-314, Ranibizumab (LUCENTIS®)
- Where
- 89 sites in United States
- Volunteers
- 671 people
- Sponsor
- AbbVie
Gaucher Disease
OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
Gaucher disease is an inherited functional deficiency of glucocerebrosidase. This enzyme breaks down a fatty substance (lipid) called glucocerebroside, which is present in all cells of the body.
- Trial name
- NCT00041535
- Testing
- OGT 918
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 30 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Oculocutaneous Albinism
Peripheral Serotonin and Albinism
Serotonin (5-HT or 5-hydroxytryptamine) is a monoamine primarily known for its role as a neurotransmitter in the central nervous system (CNS).
- Trial name
- SEPIAs (NCT06138509)
- Testing
- Dosage of serotonin and metabolites, Blood parameters
- Where
- France
- Volunteers
- 160 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Leber Hereditary Optic Neuropathy
A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy
This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA.
- Trial name
- NCT03153293
- Testing
- rAAV2-ND4
- Where
- China
- Volunteers
- 159 people
- Sponsor
- Huazhong University of Science and Technology
Anesthesia, Intraocular Pressure
Establishment of an Anaesthetic Protocol for Examinations for Children With Glaucoma or Suspected Glaucoma
Correct measurement of the intraocular pressure of children with glaucoma or suspected glaucoma is essential for diagnosis and therapy.
- Conditions
- Anesthesia, Intraocular Pressure, Glaucoma, Suspect, Pediatric Glaucoma
- Trial name
- eyeBIS (NCT03972852)
- Where
- Germany
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Johannes Gutenberg University Mainz
Retinitis Pigmentosa
Prospective Exploratory Cohort Study on Ganglion Cell Degeneration in Retinitis Pigmentosa Patients
The aim of this study is an explorative prospective observation of retinal ganglion cell degeneration and/or inner retina degeneration in Retinitis pigmentosa patients.
- Trial name
- NCT07056738
- Testing
- imaging
- Where
- Germany
- Volunteers
- 130 people
- Sponsor
- University Medical Center Goettingen
Choroideremia
Choroideremia Gene Therapy Clinical Trial
Phase II gene therapy study, involving a total of 6 male patients with choroideremia. The study will be conducted at the Bascom Palmer Eye Institute, University of Miami.
- Trial name
- NCT02553135
- Testing
- Injection of AAV2-REP1 (10e11 vg)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Byron Lam
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