Clinical trials: Inherited Ophthalmic Diseases
Retinopathy of Prematurity
Synergistic Pharmacologic Intervention for Prevention of ROP (SPIPROP Study)
Phase 2, open-label, randomized, multi-center studies in infants and premature infants are necessary to determine treatment and preventative strategies for ROP.
- Trial name
- SPIPROP (NCT02344225)
- Testing
- Caffeine citrate, Ibuprofen, Ketorolac
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- State University of New York - Downstate Medical Center
Low-grade Glioma, Plexiform Neurofibroma
Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway.
This is a phase 2, open-label, interventional clinical trial that will study the response rate of pediatric glioma and plexiform neurofibroma (PN) to oral administration of trametinib.
- Conditions
- Low-grade Glioma, Plexiform Neurofibroma, Central Nervous System Glioma
- Trial name
- NCT03363217
- Testing
- Trametinib
- Where
- 7 sites in Canada
- Volunteers
- 114 people
- Sponsor
- St. Justine's Hospital
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Wet Age-related Macular Degeneration, Neovascular Age-related Macular Degeneration
ADVM-022 Intravitreal Gene Therapy for Wet AMD
ADVM-022 (AAV.7m8-aflibercept) is a gene therapy product developed for the treatment of neovascular (wet) age-related macular degeneration (wet AMD).
- Trial name
- OPTIC (NCT03748784)
- Testing
- ADVM-022
- Where
- 11 sites in United States
- Volunteers
- 30 people
- Sponsor
- Adverum Biotechnologies, Inc.
Retinitis Pigmentosa
A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa
The Goal of this study is to evaluate the safety, tolerability, and clinical responses following single dose of DSP-3077. Study enrolls both male and female patients in 3 cohorts with each cohort defined by visual acuity (VA)…
- Trial name
- NCT06891885
- Testing
- DSP-3077 Retinal Sheet Cohort 1, DSP-3077 Retinal Sheet Cohort 2, DSP-3077 Retinal Sheet Cohort 3
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sumitomo Pharma America, Inc.
Stargardt Disease
Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
- Trial name
- STARLIGHT (NCT05417126)
- Testing
- Gene Therapy-vMCO-010
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Nanoscope Therapeutics Inc.
Retinoblastoma
Ocular Conservative Treatment for Retinoblastoma : Efficacy of the New Management Strategies and Visual Outcome
This protocol includes 2 independent studies. Both studies are multicenter studies, evaluating different therapeutic approaches in two different populations of patients: Study 1, a randomized phase-II study evaluating the…
- Trial name
- RETINO 2018 (NCT04681417)
- Testing
- Melphalan or Melphalan + Topotecan, etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy, Carboplatin administered on Day 1
- Where
- 28 sites in France
- Volunteers
- 225 people
- Sponsor
- Institut Curie
Retinitis Pigmentosa 11, Retinal Degeneration
MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)
A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy
- Conditions
- Retinitis Pigmentosa 11, Retinal Degeneration, Eye Diseases, Retinal Disease
- Trial name
- NCT06455826
- Testing
- VP-001
- Where
- 5 sites in United States
- Volunteers
- 6 people
- Sponsor
- PYC Therapeutics
Sjogren-Larsson Syndrome
ADX-629 Therapy for Sjogren-Larsson Syndrome
This research study will determine whether orally administered ADX-629 is safe and has biochemical efficacy in participants with Sjögren-Larsson syndrome (SLS), a rare inherited disorder of fatty aldehyde metabolism The disease…
- Trial name
- NCT05443685
- Testing
- ADX-629
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Nebraska
Graves Ophthalmopathy, Graves Disease
Doxycycline Treatment in Mild Thyroid-Associated Ophthalmopathy
The aim of this study is to evaluate the effects of subantimicrobial dose doxycycline (50 mg/d), administered for 12 weeks, on patients with mild Thyroid-Associated Ophthalmopathy (TAO).
- Conditions
- Graves Ophthalmopathy, Graves Disease, Thyroid-associated Ophthalmopathy, Thyroid Diseases
- Trial name
- NCT02203682
- Testing
- Doxycycline hyclate
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Sun Yat-sen University
Thyroid Associated Ophthalmopathies
Efficacy and Safety of Sequential Hormone Therapy and Tetuzumab Therapy in Patients With Moderate to Severe TAO in the Active Stage After Glucocorticoid 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
- NCT07152366
- Testing
- IBI311, Glucocorticoids
- Where
- China
- Volunteers
- 96 people
- Sponsor
- Shanghai Changzheng Hospital
Thyroid Eye Disease
An Open-label, Rollover Trial to Evaluate the Efficacy and Safety of AMG 732 in Participants With Thyroid Eye Disease
The main objective of this trial is to assess the efficacy of AMG 732 in participants with thyroid eye disease (TED) who are defined as primary nonresponders or relapsed during the safety follow-up in the parent trial…
- Trial name
- HAZEL 401 (NCT07438405)
- Testing
- AMG 732
- Where
- 8 sites in United States, Poland and 2 more
- Volunteers
- 30 people
- Sponsor
- Amgen
Fuch's Endothelial Dystrophy
Comparison of Two Techniques in Achieving Corneal Graft
The cornea is a transparent membrane, half a millimeter thick, embedded as a window in front of the eye. When the eye becomes opaque, the eye is not seeing and the only way to restore the visual acuity is to replace the cornea by…
- Trial name
- NCT01610973
- Testing
- Endothelial descemet membrane graft
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Rouen
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
ABT-751 in Treating Young Patients With Refractory Solid Tumors
RATIONALE: Drugs used in chemotherapy, such as ABT-751, work in different ways to stop tumor cells from dividing so they stop growing or die.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00036959
- Testing
- ABT-751
- Where
- 3 sites in United States
- Volunteers
- 90 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Chronic Pain, Depression Disorders
Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World Study
What is the purpose of this study? The use of medicinal cannabis has grown significantly in Brazil. However, because many patients use these products through "compassionate use" (when traditional treatments haven't worked), there…
- Conditions
- Chronic Pain, Depression Disorders, Anxiety Disorders, Quality of Life
- Trial name
- CANAREAL (NCT07362407)
- Testing
- Participants are followed while using cannabis-based products (such as CBD or THC oils, extracts, or isolates) as prescribed by their own physicians for therapeutic purposes.
- Where
- Brazil
- Volunteers
- 384 people
- Sponsor
- Santa Catarina Federal University
Behcet Disease
Role of Regulatory B Cells in Behçet's Disease
There are limited data about the role of regulatory B cells in Behcet Disease. In this study we aimed to identify the proportions of total B lymphocytes and their regulatory subset in different Behcet Disease phenotype and…
- Trial name
- NCT04376411
- Testing
- Flow-cytometric assay, 2D Echocardiographic analysis, Two-Dimensional speckle tracking echocardiography
- Where
- Egypt
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Assiut University
Retinitis Pigmentosa
Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene
The study is a Phase I/II, monocentric, open-label, dose-ranging safety and efficacy gene therapy intervention by subretinal administration of AAV2/5-hPDE6B.
- Trial name
- NCT03328130
- Testing
- AAV2/5-hPDE6B
- Where
- France
- Volunteers
- 19 people
- Sponsor
- eyeDNA Therapeutics
Cataract, Glaucoma
Potential Research Participants for Future Studies of Inherited Eye Diseases
This study will evaluate potential candidates for future clinical research studies related to diagnosed or undiagnosed genetic eye disorders or diseases.
- Conditions
- Cataract, Glaucoma, Retinitis Pigmentosa, Macular Degeneration
- Trial name
- NCT00559234
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- National Eye Institute (NEI)
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
- Trial name
- NCT06275620
- Testing
- AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics
Retinitis Pigmentosa
Clinical Implication of Retinitis Pigmentosa Molecular Diagnostic Using High Throughput Sequencing.
The retinitis pigmentosa (RP) are genetic conditions that cause retinal degeneration leading to severe low vision and is the leading cause of consultation in reference centers dedicated to the ophthalmic genetics.
- Trial name
- RP-SEQ-HD (NCT02860520)
- Where
- 4 sites in France
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
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