Clinical trials: Inherited Ophthalmic Diseases
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
Central Nervous System/Leptomeningeal Neoplasms
131I-omburtamab for the Treatment of Central Nervous System/Leptomeningeal Neoplasms in Children and Young Adults
The researchers are doing this study to provide access to treatment with 131I-omburtamab for children and young adults who have CNS/leptomeningeal neoplasms.
- Trial name
- NCT05064306
- Testing
- 131I-omburtamab
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
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.
Retinitis Pigmentosa, Cystoid Macular Edema
Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema
This study aims at investigating the therapeutic potential of recombinant human Nerve Growth Factor ( rhNGF ) eye drops treatment in patients with Retinitis Pigmentosa (RP) associated with cystoid macular edema (CME) in a phase…
- Trial name
- NEMO (NCT02609165)
- Testing
- rhNGF 180 µg/ml eye drops solution, vehicle eye drops
- Where
- 2 sites in Italy
- Volunteers
- 45 people
- Sponsor
- Ospedale San Raffaele
Retinopathy of Prematurity
PhaseI/II Investigator-Initiated Trial to Investigate Safety and Efficacy of Ripasudil in Patients With Retinopathy of Prematurity
This is an open-label, single-arm phase I/II trial to evaluate the safety and efficacy of Rho-associated protein kinase(ROCK) inhibitor Ripasudil eye drops for preterm infants with Retinopathy of Prematurity(ROP).
- Trial name
- NCT04621136
- Testing
- Ripasudil ophthalmic solution 0.4%
- Where
- 3 sites in Japan
- Volunteers
- 24 people
- Sponsor
- Kyushu University
Retinitis Pigmentosa (RP)
Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa
This study evaluates the safety and potential activity of a single dose of live human retinal progenitor cells (jCell) administered to adults with retinitis pigmentosa.
- Trial name
- NCT02320812
- Testing
- human retinal progenitor cells
- Where
- 2 sites in United States
- Volunteers
- 28 people
- Sponsor
- jCyte, Inc
Thyroid Eye Disease
The Effect of a New Antioxidant Combination (ASTED) on Mild Thyroid Eye Disease (TED)
This randomized clinical trial is designed to evaluate the effect of selected antioxidant vitamins and minerals supplement named as ASTED: 1. β- Carotene (30 mg) 2. Vit C (100 mg) 3. Vit E (Alpha-Tocopherol Acetate): 60-200 IU 4.
- Trial name
- NCT02393183
- Testing
- ASTED, Selenium
- Where
- Iran
- Sponsor
- Iran University of Medical Sciences
Leukemia, Lymphoma
DT2219ARL for Relapsed or Refractory CD19 (+), CD 22 (+) B-Lineage Leukemia Or Lymphoma
This is a phase I dose escalation study of DT2219ARL for the treatment of relapsed or refractory B-lineage leukemia and lymphoma. Patients will receive a single course of DT2219ARL as a 4 hour infusion on days 1, 3, 5, and 8.
- Trial name
- NCT00889408
- Testing
- DT2219ARL
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Alström Syndrome (ALMS)
Clinical Study of a Single Ciliopathy: Alström Syndrome
The aim of the study is to characterize the clinical manifestations of ALMS within the ciliopathies to prevent complications and determine preventive and therapeutic targets.
- Trial name
- NCT02890550
- Where
- 14 sites in France
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Thyroid Associated Ophthalmopathy
Optic Disc, Macula, and Retinal Nerve Fiber Layer Measurements Obtained by OCT in Thyroid Associated Ophthalmopathy
The aim of the study is to compare the measurements of retinal nerve fiber layer (RNFL), macula and optical disc parameters obtained by optical coherence tomography (OCT), and intraocular pressure (IOP) between the patients with…
- Trial name
- NCT02766660
- Testing
- Optical Coherence Tomography
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Neon Hospital
Retinitis Pigmentosa
Biomarkers in Retinitis Pigmentosa
The objective of this study is to discover biomarkers that demonstrate a correlation between the severity of retinitis pigmentosa (RP) and the thickness of the retinal pigment epithelium (RPE).
- Trial name
- RPMARKER (NCT06306690)
- Testing
- OCT, OCT angiography, flicker ERG and Ultra Wide Field retinography and autofluorescence.
- Where
- Italy
- Volunteers
- 35 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Neuromuscular Diseases, Eye Diseases
Precision Diagnosis and Therapy for Rare Diseases by Interpreting Non-coding Genomes
PARADIGM study, funded by the PNRR research grant, will focus on Eye Diseases (ED) and Neuro-Muscular Diseases (NMD) as groups of genetically heterogeneous diseases which are extensively studied by the Partners partecipating in…
- Conditions
- Neuromuscular Diseases, Eye Diseases, Genetic Disease
- Trial name
- NCT06775561
- Testing
- PARADIGM study aims to streamline the process from genomic characterization of RGD patients with ED/NMD to identification of the suitable personalized therapy.
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Bardet-Biedl Syndrome
Clinical Registry Investigating Bardet-Biedl Syndrome
Bardet-Biedl Syndrome (BBS) is a rare genetic disorder associated with a vast array of symptoms. The features of BBS are highly variable, even between siblings, making long-term follow-up and centralization of information vital…
- Trial name
- CRIBBS (NCT02329210)
- Where
- United States
- Volunteers
- 1,200 people
- Sponsor
- Marshfield Clinic Research Foundation
Graves' Orbitopathy
A Randomized Clinical Trial to Evaluate the Effects of Atorvastatin on Graves' Orbitopathy (GO): the STAGO-2 Study
Graves' orbitopathy (GO) is the most common extra-thyroidal manifestation of Graves' disease (GD). Based on its clinical signs and symptoms, GO is graded as mild, moderate-to-severe, or severe, and active or inactive, the latter…
- Trial name
- STAGO-2 (NCT05049603)
- Testing
- Atorvastatin 20mg
- Where
- Italy
- Volunteers
- 102 people
- Sponsor
- University of Pisa
Retinal Detachment, Epiretinal Membrane
PIONEER: Intraoperative and Perioperative OCT Study
OCT provides high-resolution information regarding the anatomic structure of the tissues of the eye in a 2-dimensional and 3-dimensional view. Much of this information is not able to be recognized by a clinician.
- Conditions
- Retinal Detachment, Epiretinal Membrane, Macular Hole, Fuchs Endothelial Dystrophy
- Trial name
- PIONEER (NCT02423161)
- Where
- United States
- Volunteers
- 750 people, healthy volunteers welcome
- Sponsor
- The Cleveland Clinic
Behcet Disease, Antineutrophil Cytoplasmic Antibody (ANCA) Positive Vasculitis
Armenian NAtionwide REGistry of Systemic Autoimmune and Autoinflammatory Diseases
Longitudinal prospective multicenter Armenian registry of systemic autoimmune, autoinflammatory diseases with constitution of bio-banking.
- Conditions
- Behcet Disease, Antineutrophil Cytoplasmic Antibody (ANCA) Positive Vasculitis, Takayasu Arteritis, Giant Cell Arteritis
- Trial name
- NAREG (NCT05904301)
- Testing
- Usual medical management of patients, additional blood and stool samples for biobanking
- Where
- 6 sites in Armenia
- Volunteers
- 800 people
- Sponsor
- Santé Arménie French-Armenian Research Center
Pseudophakic Bullous Keratopathy, Fuchs' Endothelial Dystrophy
The OPTIMISE Study
Rationale: The cornea is the most transplanted tissue in the Netherlands, with more than 1,500 procedures performed each year. A minimally invasive technique called Descemet Membrane Endothelial Keratoplasty (DMEK) has become the…
- Conditions
- Pseudophakic Bullous Keratopathy, Fuchs' Endothelial Dystrophy, Intraocular Pressure
- Trial name
- NCT05716945
- Testing
- Fluorometholone 0.1% Oph Susp, Dexamethasone 0.1% ophthalmic suspension, Stop steroids
- Where
- 8 sites in Netherlands
- Volunteers
- 342 people
- Sponsor
- Maastricht University Medical Center
Retinitis Pigmentosa
Study of the Correlation Between Macular Thickness Analyzed by Optical Coherence Tomography and the Visual Functions in Retinitis Pigmemtosa
Purpose: To study the associations between visual functions and the retinal tomography detected by optical coherence tomography (OCT) in patients with retinitis pigmentosa (RP).
- Trial name
- NCT00784901
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- National Taiwan University Hospital
Retinopathy of Prematurity (ROP)
Comparing Alternative Ranibizumab Dosages for Safety and Efficacy in Retinopathy of Prematurity
This study is designed as an exploratory study to assess safety and efficacy of two different doses of the anti-VEGF agent ranibizumab (0.12 mg vs. 0.20 mg) in the treatment of infants with retinopathy of prematurity.
- Trial name
- CARE-ROP (NCT02134457)
- Testing
- ranibizumab
- Where
- 9 sites in Germany
- Volunteers
- 19 people
- Sponsor
- University Hospital Freiburg
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors
This pilot trial studies different high-dose chemotherapy regimens with or without total-body irradiation (TBI) to compare how well they work when given before autologous stem cell transplant (ASCT) in treating patients with…
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT00536601
- Testing
- etoposide, cyclophosphamide, carmustine
- Where
- United States
- Volunteers
- 174 people
- Sponsor
- Roswell Park Cancer Institute
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