Clinical trials: Retinal Dystrophy
Retinitis Pigmentosa
Biomarkers In Retinitis Pigmentosa (BIRP)
In this study, markers of oxidative stress will be measured in the aqueous humour and plasma of RP patients compared to controls.
- Trial name
- BIRP (NCT01949623)
- Testing
- Anterior Chamber (AC) tap
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Johns Hopkins University
Retinitis Pigmentosa
Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II
The study will perform UC-MSCs and CM transplantation. There are two groups with different dosages. The first group will be transplanted with 1.5 million cells, meanwhile, the second group is 5 million cells.
- Trial name
- NCT05909488
- Testing
- 1.5 x 10^6 UC-MSC + CM, 5 x 10^6 UC-MSC + CM
- Where
- Indonesia
- Volunteers
- 30 people
- Sponsor
- PT. Prodia Stem Cell Indonesia
Stargardt Disease Type 1 (STGD1)
An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence.
- Trial name
- START (NCT07063251)
- Testing
- HG005
- Where
- China
- Volunteers
- 6 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Retinitis Pigmentosa
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study.
- Trial name
- NCT03374657
- Testing
- CPK850
- Where
- Sweden
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals
Retinitis Pigmentosa
Cone Rescue in Retinitis Pigmentosa by the Treatment of Lycium Barbarum
Objectives: To study the immediate effect and the persistent effect of Lycium barbarum (LB) treatment on retinal functions, especially the cone function, and retinal structure in patients with retinitis pigmentosa (RP) Design…
- Trial name
- NCT02244996
- Testing
- Lycium Barbarum
- Where
- Hong Kong
- Volunteers
- 50 people
- Sponsor
- The Hong Kong Polytechnic University
Retinitis Pigmentosa
Identifying Genes and Mutations Underlying Retinitis Pigmentosa and Allied Diseases
Retinitis Pigmentosa (RP) is the most common form of hereditary retinal degeneration, with a worldwide prevalence of 1:3500. It is one of the most genetically heterogenous conditions in humans, with over 100 causative genes and…
- Trial name
- NCT02309866
- Testing
- Genetic testing
- Volunteers
- 100 people
- Sponsor
- Hillel Yaffe Medical Center
Retinitis Pigmentosa
Retrospective NIS to Evaluate the Patient Benefit of TES
In this retrospective non-interventional study (NIS), the subjective and objective benefit of patients with retinitis pigmentosa (and other dystrophies like Usher, Choroideremia or cone-rod dystrophy), who have been prescribed…
- Trial name
- NCT04983914
- Testing
- Transcorneal Electrostimulation via OkuStim System
- Where
- 4 sites in Germany, Switzerland
- Volunteers
- 49 people
- Sponsor
- Okuvision GmbH
Retinitis Pigmentosa
FIGHT-RP 1 Extension Study
Retinitis Pigmentosa (RP) is a devastating eye disease and at present there are no known treatment options that can alter the rate of vision loss and eventual blindness.
- Trial name
- NCT03999021
- Testing
- NAC effervescent tablets
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Johns Hopkins University
Bietti's Crystalline Dystrophy
Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy
Evaluate the safety and tolerability of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration (BCD)
- Trial name
- NCT06302608
- Testing
- NGGT001
- Where
- China
- Volunteers
- 11 people
- Sponsor
- Xiamen Ophthalmology Center Affiliated to Xiamen University
Retinitis Pigmentosa
In Depth Observational Clinical Trial Of Retinitis Pigmentosa Patients
Typically, clinical research participation favors a specific demographic group, and little research exists on how trial attributes affect participation.
- Trial name
- NCT05849987
- Volunteers
- 500 people
- Sponsor
- Power Life Sciences Inc.
Retinitis Pigmentosa
Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa
The primary objective of the study is to assess the safety of repeat injection of human retinal progenitor cells (jCell) in adult subjects with RP that have previously been treated with jCell.
- Trial name
- NCT04604899
- Testing
- human retinal progenitor cells
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- jCyte, Inc
Bardet-Biedl Syndrome
Characterizing the Genotype and Phenotype in Adults With Bardet-Biedl Syndrome
Bardet-Biedl syndrome (BBS; OMIN #209900) is a rare genetic disorder characterized by six core features: rod-cone dystrophy (retinitis pigmentosa), polydactyly, obesity, genital anomalies, renal anomalies, and learning…
- Trial name
- NCT05400278
- Testing
- No intervention
- Where
- Norway
- Volunteers
- 30 people
- Sponsor
- Oslo University Hospital
Retinitis Pigmentosa, Stargardt Macular Dystrophy
Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)
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, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy From Age-related Macular Degeneration
- Trial name
- DEFINE-IRD (NCT07502664)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Ray Therapeutics, Inc.
Eye Diseases, Retinitis Pigmentosa
Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cell Exosomes for NAION
This trial will study the safety and efficacy of intranasal delivery of cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cell exosomes for the treatment of non-arteritic ischemic optic neuropathy
- Conditions
- Eye Diseases, Retinitis Pigmentosa, Diabetic Retinopathy, Macular Degeneration
- Trial name
- NCT05147701
- Testing
- cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cell exosomes
- Where
- 2 sites in United States, Greece
- Volunteers
- 20 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Macular Edema, Inherited Retinal Dystrophy
Micropulsed Laser in Patients With Macular Oedema in Retinal Dystrophies
The purpose of this study is to investigate whether subthreshold treatment with micropulsed laser can be effective in resolving macular edema in patients with inherited retinal dystrophy.
- Conditions
- Macular Edema, Inherited Retinal Dystrophy, Laser Retinopathy
- Trial name
- NCT05976139
- Testing
- subthreshold treatment with micropulsed laser
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Fondazione G.B. Bietti, IRCCS
Usher Syndromes
Natural History Study of Usher Syndrome ( Light4Deaf )
Clinical centres in the LIGHT4DEAF consortium have developed and will continue to improve a reliable, early molecular diagnosis and protocols for full clinical characterisation of Usher syndrome, which will be valuable for the…
- Trial name
- Light4Deaf (NCT04665726)
- Where
- 4 sites in France
- Volunteers
- 400 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Retinitis Pigmentosa (RP)
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
The purpose of this study is: To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.
- Trial name
- NCT01543906
- Testing
- QLT091001
- Where
- 2 sites in Canada, Ireland
- Volunteers
- 5 people
- Sponsor
- QLT Inc.
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
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
Mitochondrial Myopathies, Mitochondrial DNA Mutation
Genomic Profiling of Mitochondrial Disease - Imaging Analysis for Precise Mitochondrial Medicine
This study is an observational longitudinal study involving the use of MRIs and video recordings taken at home of patients completing basic tasks.
- Conditions
- Mitochondrial Myopathies, Mitochondrial DNA Mutation, Mitochondrial Diseases, Chronic Progressive External Ophthalmoplegia With Myopathy
- Trial name
- NCT05012358
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Mayo Clinic