Clinical trials: Inherited Ophthalmic Diseases
Retinopathy of Prematurity
Pan-VEGF Blockade for the Treatment of Retinopathy of Prematurity (BLOCK-ROP)
The purpose of this study is to determine whether a single intravitreal (into the gel of the eye) injection of Avastin 0.625mg or 0.75mg is equivalent (non-inferior) to treatment with standard of care laser in infants with Type I…
- Trial name
- BLOCK-ROP (NCT01232777)
- Testing
- Bevacizumab
- Where
- 19 sites in United States, Canada
- Sponsor
- Vision Research Foundation
Brain and Central Nervous System Tumors, Neuroblastoma
Chemotherapy Followed by Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Brain Tumor
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of…
- Conditions
- Brain and Central Nervous System Tumors, Neuroblastoma, Retinoblastoma, Sarcoma
- Trial name
- NCT00003273
- Testing
- filgrastim, carboplatin, cisplatin
- Where
- 19 sites in United States, Argentina and 1 more
- Sponsor
- NYU Langone Health
Graves Ophthalmopathy
Observing the Relationship of Fibroblast Growth Factor and Fibroblast in Thyroid Eye Disease
Graves' orbitopathy (GO) affects about 50% of patients with Graves' disease (GD), and some cannot be cured by current treatments.
- Trial name
- TED (NCT03324022)
- Where
- Taiwan
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Thyroid Eye Disease
Treatment of Graves' Orbitopathy to Reduce Proptosis With Teprotumumab Infusions in an Open-Label Clinical Extension Study
The overall objective is to evaluate the safety and efficacy of teprotumumab in the treatment of thyroid eye disease (TED) in participants who participated in the lead-in study HZNP-TEP-301 (NCT03298867; OPTIC) and who were…
- Trial name
- OPTIC-X (NCT03461211)
- Testing
- Teprotumumab
- Where
- 13 sites in United States, Germany and 1 more
- Volunteers
- 51 people
- Sponsor
- Amgen
Neuronal Ceroid Lipofuscinosis Type 2
A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 Disease
This is a registrational, first-in-human, open-label, dose escalation and expansion study evaluating the safety, tolerability and effectiveness of TTX-381, administered by injection under the retina, for the treatment of eye…
- Trial name
- NCT05791864
- Testing
- TTX-381
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 26 people
- Sponsor
- Tern Therapeutics, LLC
Premature Retinopathy, Visual Impairment
Evaluation of Visual - Motor Development in Children With Retinopathy of Prematurity
The aim of this study was to investigate the eye structure, visual impairment, visual motor development and motor coordination of children between 2-6 ages that had applied to diod laser photocoagulation and intravitreal anti …
- Conditions
- Premature Retinopathy, Visual Impairment, Motor Delay
- Trial name
- NCT03865134
- Testing
- Snellen Test, Autorefractometer, Titmus Stereo Fly Test
- Where
- Turkey (Türkiye)
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Istanbul Medipol University Hospital
Retinitis Pigmentosa, Eye Diseases
Natural History Study of Patients With EYS-Associated RP
This natural history study of patients with EYS mutations from Russia and former CIS (Commonwealth of Independent States) territories will accelerate the development of outcome measures for clinical trials.
- Trial name
- RUS_EYS (NCT07228793)
- Testing
- Whole exome/genome sequencing
- Where
- Russia
- Volunteers
- 45 people
- Sponsor
- Sensor Technology for Deafblind
Leber Congenital Amaurosis
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD
- Trial name
- NCT00749957
- Testing
- rAAV2-CB-hRPE65
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Beacon Therapeutics
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Wolfram Syndrome
Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
Open label, phase II study non randomized single group assignment of 20 evaluable patients 13 years and older, over 37,5 kg body-weight, with sensorineural hearing loss of at least 20 dB at 8 kHz in high frequency average (HFA)…
- Trial name
- AUDIOWOLF (NCT04940572)
- Testing
- Depakine
- Where
- 2 sites in France, Spain
- Volunteers
- 23 people
- Sponsor
- Centre d'Etude des Cellules Souches
Age Related Maculopathy, Retinal Degeneration
Measuring Reading Rehabilitation Outcomes
The purpose of this study is to measure the effectiveness of a newly-designed oculomotor training program for patients with age-related macular degeneration.
- Trial name
- NCT00125632
- Testing
- Eccentric Viewing Training for Reading
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- US Department of Veterans Affairs
Mitochondrial Diseases, Kearns-Sayre Syndrome
Global Registry and Natural History Study for Mitochondrial Disorders
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome…
- Conditions
- Mitochondrial Diseases, Kearns-Sayre Syndrome, MIDD, SANDO
- Trial name
- GENOMIT (NCT05554835)
- Where
- 34 sites in Austria, Germany and 1 more
- Volunteers
- 6,000 people
- Sponsor
- LMU Klinikum
Choroideremia
REP1 Gene Replacement Therapy for Choroideremia
The assessment of the efficacy (with respect to preservation of visual function and retinal structure) and safety of a single subretinal injection of AAV2.REP1 in participants with a confirmed diagnosis of choroideremia, as…
- Trial name
- REGENERATE (NCT02407678)
- Testing
- AAV-mediated REP1 gene replacement
- Where
- 2 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- University of Oxford
Thyroid Eye Disease
A Study of IBI311 in Subjects With Inactive or Active Thyroid Eye Disease
This is a multicenter, randomized, double-masked phase II study evaluating the efficacy and safety of IBI311 in subjects with inactive or active thyroid eye disease.
- Trial name
- NCT06525506
- Testing
- IBI311 (20 mg), IBI311 (3-20 mg), IBI311 (10 mg)
- Where
- China
- Volunteers
- 38 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Non-infectious Uveitis
Safety and Efficacy of AIN457 in Noninfectious Uveitis
This study was performed to evaluate the efficacy and safety of AIN457 for patients with active uveitis that requires systemic immunosuppression.
- Trial name
- NCT00685399
- Testing
- AIN457, AIN 457
- Where
- 23 sites in United States, Germany
- Volunteers
- 76 people
- Sponsor
- Novartis Pharmaceuticals
Graft vs Host Disease, Congenital Aniridia
Safety and Performance Study of the ARGOS-IO System in Patients Undergoing Boston Keratoprosthesis Implantation
The ARGOS-IO pressure sensor is intended to be implanted in the human eye in combination with Boston Keratoprosthesis (BKPro) surgery and to remain in place indefinitely.
- Conditions
- Graft vs Host Disease, Congenital Aniridia, Chemical Burns, Stevens-Johnson Syndrome
- Trial name
- NCT02945176
- Testing
- ARGOS-IO system
- Where
- 2 sites in Germany
- Volunteers
- 13 people
- Sponsor
- Implandata Ophthalmic Products GmbH
Suicide Attempt
Treating Suicidality Remotely
Background: Practical and psychological barriers make it difficult for people with Suicidal Thoughts and Behaviors (STBs) to get professional help.
- Trial name
- TREASURE (NCT06370104)
- Testing
- Brief Cognitive Behavioral Therapy for Suicide Prevention, Living under control
- Where
- Netherlands
- Volunteers
- 364 people
- Sponsor
- 113 Suicide Prevention
Healthy Volunteer, Rheumatoid Arthritis
Phenomics in Autoimmune and Inflammatory Diseases
The family of inflammatory/autoimmune systemic diseases (IAD) form a continuum from pure inflammatory diseases to pure autoimmune diseases, encompassing a large panel of inflammatory diseases with some autoimmune components, and…
- Conditions
- Healthy Volunteer, Rheumatoid Arthritis, Ankylosing Spondylitis, Systemic Lupus Erythematosus/Antiphospholipid Syndrome
- Trial name
- TRANSIMMUNOM (NCT02466217)
- Testing
- 1: AID groups, 2: Control groups
- Where
- 2 sites in France
- Volunteers
- 537 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Retinal Degeneration, Primary Open-angle Glaucoma
Intravitreal Mesenchymal Stem Cell Transplantation in Advanced Glaucoma.
Bone marrow-derived mesenchymal stem cells (MSC) therapy is a promising treatment for several degenerative diseases, including retinopathies and glaucoma, however no previous safety study involving humans has been conducted.
- Trial name
- NCT02330978
- Testing
- Intravitreal transplantation of mesenchymal stem cell, Culture and isolation of autologous bone-marrow mesenchymal stem cells
- Where
- Brazil
- Volunteers
- 2 people
- Sponsor
- University of Sao Paulo
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.