Clinical trials: Inherited Ophthalmic Diseases
Retinopathy of Prematurity, Newborns
A Study to Collect Data on the Use of Eylea in Babies Born Too Early Who Have a Condition of the Eye Where Blood Vessels Grow Abnormally in the Retina (Retinopathy of Prematurity)
This is an observational study to collect data from Japanese babies with retinopathy of prematurity (ROP) who will be treated with Eylea.
- Conditions
- Retinopathy of Prematurity, Newborns, Infants
- Trial name
- NCT05705258
- Testing
- Aflibercept (Eylea, BAY86-5321)
- Where
- Japan
- Volunteers
- 75 people
- Sponsor
- Bayer
Retinoblastoma
Retinoblastoma Phase II Expanded Access Clinical Trial
In this research study investigators want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all…
- Trial name
- NCT06679634
- Testing
- Chemoplaque also referred to as Episcleral Topotecan
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Targeted Therapy Technologies, LLC
Rheumatoid Arthritis, Osteoarthritis
Regulatory B Cells in Inflammatory Rheumatisms and Biomarkers of Response to Biologic Treatments
B cells are known to play an important role in auto-immune diseases by activating T cells, secreting inflammatory cytokines and autoreactive antibodies.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Systemic Lupus Erythematosus, Sjogren's Syndrome
- Trial name
- NCT01642706
- Where
- France
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Retinitis Pigmentosa, Choroideremia
Longitudinal Study of a Bionic Eye
This is a longitudinal observational study with participants who have been implanted with the suprachoroidal retinal prosthesis.
- Trial name
- NCT05158049
- Where
- Australia
- Volunteers
- 7 people
- Sponsor
- Center for Eye Research Australia
Retinitis Pigmentosa, Usher Syndrome Type 2
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with…
- Trial name
- HELIA (NCT05085964)
- Testing
- RNA antisense oligonucleotide for intravitreal injection
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Laboratoires Thea
Gyrate Atrophy, Gyrata of Choroid and Retina; Atrophy
Gyrate Atrophy Ocular and Systemic Study
The Gyrate Atrophy Ocular and Systemic Study characterizes the natural history of ornithine levels and retinal degeneration (RD) associated with disease-causing OAT variants in the presence of standard care dietary treatment…
- Conditions
- Gyrate Atrophy, Gyrata of Choroid and Retina; Atrophy, Ornithine-δ-aminotransferase, OAT
- Trial name
- GYROS (NCT05312736)
- Where
- 11 sites in United States, Brazil and 6 more
- Volunteers
- 46 people
- Sponsor
- Jaeb Center for Health Research
Stargardt Disease
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021.
- Trial name
- AURORA (NCT07439887)
- Testing
- RTx-021
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- Ray Therapeutics, Inc.
Myelofibrosis, Gaucher Disease
Clinical and Pathophysiological Investigations Into Erdheim Chester Disease
Background: Erdheim Chester Disease (ECD) is a very rare disease in which abnormal white blood cells start growing and affect the bones, kidneys, skin, and brain.
- Conditions
- Myelofibrosis, Gaucher Disease, Pulmonary Fibrosis, Hermansky-Pudlak Syndrome (HPS)
- Trial name
- NCT01417520
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Cutaneous Neurofibroma, Neurofibromatosis Type 1
Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma
This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas.
- Conditions
- Cutaneous Neurofibroma, Neurofibromatosis Type 1, Optic Nerve Glioma
- Trial name
- NCT02839720
- Testing
- Laboratory Biomarker Analysis, Selumetinib Sulfate
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- National Cancer Institute (NCI)
Ataxia Telangiectasia, Dysmetria
Videoocular Assessment of Eye Movement Activity in an Ataxia Telangiectasia
Ataxia-telangiectasia (A-T) is a multisystem auto-somal recessive disorder linked to the A-T mutated gene (ATM) on chromosome 11q22-23, and characterized by progressive neural degeneration, immunodeficiency, and progressive…
- Trial name
- NCT05471310
- Where
- Russia
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Behçet's Syndrome (BS)
Prospective Multiethnic Validation of the Behçet's Syndrome Overall Damage Index (BODI)
PROBE is an observational multicenter cohort study, consisting of a cross-sectional and a 5-years prospective phase, designed to target the major study objectives, such as testing the comprehensiveness of the Behçet's syndrome…
- Trial name
- PROBE (NCT07073261)
- Where
- Italy
- Volunteers
- 970 people
- Sponsor
- University of Cagliari
Graves Ophthalmopathy
A Safety and Tolerability Study of Otelixizumab in Thyroid Eye Disease
The purpose of this study is to investigate the safety and tolerability of otelixizumab in patients with Graves' ophthalmopathy (thyroid eye disease).
- Trial name
- NCT01114503
- Testing
- Otelixizumab - low dose, Otelixizumab - medium low dose, Otelixizumab - medium high dose
- Where
- United Kingdom
- Volunteers
- 2 people
- Sponsor
- GlaxoSmithKline
Retinopathy of Prematurity (ROP)
The Effect of Two Non-Pharmacological Methods on Pain During Retinopathy Examination
The goal of this randomized controlled trial is to determine the effect of reverse kangaroo care position and ROP (Retinopathy of Prematurity) position applied during ROP examination in premature infants on pain.
- Trial name
- NCT06694103
- Testing
- Reverse Kangaroo Care Position, ROP Position
- Where
- Turkey (Türkiye)
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- Sinem Basdemir
Retinopathy of Prematurity
The Incidence of Treatment-requiring Retinopathy of Prematurity in Greece
A prospective observational study of the incidence of treatment-requiring ROP (TR-ROP), at a national level, in Greece.
- Trial name
- GR-ROP (NCT05099588)
- Testing
- Other: observational study
- Where
- Greece
- Volunteers
- 43 people
- Sponsor
- Aristotle University Of Thessaloniki
Retinitis Pigmentosa
Management of Retinitis Pigmentosa Via Combination of Wharton's Jelly-derived Mesenchymal Stem Cells and Magnovision
Purpose To investigate whether the natural progression rate of retinitis pigmentosa (RP) can be decreased with subtenon umbilical cord Wharton's jelly derived mesenchymal stemcell (WJ-MSC) application alone or combination with…
- Trial name
- NCT05800301
- Testing
- Wharton's jelly derived mesenchymal stemcells, Magnovision
- Where
- 3 sites in Turkey (Türkiye)
- Volunteers
- 80 people
- Sponsor
- Ankara Universitesi Teknokent
Rheumatic Diseases, Adult Onset Still Disease
Rheumatology Patient Registry and Biorepository
To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.
- Conditions
- Rheumatic Diseases, Adult Onset Still Disease, Ankylosing Spondylitis, Psoriatic Arthritis
- Trial name
- NCT04402086
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Yale University
Eye Diseases, Macular Degeneration
Study Evaluating the Safety, Pharmacokinetics and Pharmacodynamics of OPT-302 With or Without Lucentis™ in Patients With Wet AMD
The purpose of this first-in-human study is to evaluate the safety, pharmacokinetics (PK) and pharmacodynamics of OPT-302 administered as monthly intravitreal injections for 3 months with and without Lucentis™ in patients with…
- Conditions
- Eye Diseases, Macular Degeneration, Retinal Diseases, Retinal Degeneration
- Trial name
- NCT02543229
- Testing
- OPT-302, Lucentis™
- Where
- 14 sites in United States
- Volunteers
- 51 people
- Sponsor
- Opthea Limited
Leber Congenital Amaurosis 10, Blindness
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- BRIGHTEN (NCT04855045)
- Testing
- sepofarsen
- Where
- 9 sites in Belgium, Brazil and 5 more
- Volunteers
- 15 people
- Sponsor
- ProQR Therapeutics
Psychosis
Culturally Adapted Cognitive Behavior Therapy for Psychosis in Pakistan
Aim: To assess the feasibility of application of culturally-adapted CBT (CaCBTp) for use in Pakistan. Design: Randomized Control Trial Setting: Psychiatry Departments of General Hospitals in Karachi.
- Trial name
- NCT02202694
- Testing
- Culturally Adapted Cognitive behavior therapy
- Where
- Pakistan
- Volunteers
- 36 people
- Sponsor
- Pakistan Institute of Living and Learning
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