Clinical trials: Eye Diseases, Hereditary
Retinitis Pigmentosa
BS01 in Patients With Retinitis Pigmentosa
A Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAG-ChronosFP) in Patients with Retinitis Pigmentosa
- Trial name
- NCT04278131
- Testing
- BS01
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Bionic Sight LLC
Neuroblastoma, Brain Tumor
Efficacy of Prophylactic Itraconazole in High-Dose Chemotherapy and Autologous Hematopoietic Stem Cell Transplantation
The purpose of this study is to investigate whether the prophylactic use of itraconazole is a better option than empirical use of itraconazole in the management (prevention and treatment) of fungal infection associated with…
- Conditions
- Neuroblastoma, Brain Tumor, Retinoblastoma, Wilms Tumor
- Trial name
- NCT00336531
- Testing
- itraconazole
- Where
- South Korea
- Volunteers
- 100 people
- Sponsor
- Samsung Medical Center
Bardet-Biedl Syndrome (BBS), Obesity
An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
An open-label, single-arm, multicenter, Expanded Access Protocol [EAP] designed to provide treatment access to setmelanotide (3 mg, administered subcutaneously [SC], once daily) for eligible patients with BBS who have no…
- Trial name
- NCT05183802
- Testing
- Setmelanotide, administered subcutaneously [SC], once daily.
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Fetal Malformation, Pregnancy Related
Realization of Sequencing of All Known Human Genes in Case of Detection of Cerebral, Renal or Ophthalmological Fetal Malformations During Pregnancy in Order to Make an Etiological Diagnosis and to Precise the Fetal Prognosis
Congenital malformations concern 3% of pregnancies; most of them can be seen during pregnancy. For some malformations, an invasive sample (trophoblast biopsy or amniocentesis) is proposed to search a chromosomal abnormality by…
- Conditions
- Fetal Malformation, Pregnancy Related, Rare Genetic Disease
- Trial name
- PRENATEX (NCT04406480)
- Testing
- CGH-array and exome sequencing
- Where
- 12 sites in France
- Volunteers
- 90 people
- Sponsor
- University Hospital, Strasbourg, France
Retinitis Pigmentosa, Retinal Degeneration
Safety and Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind Patients
Patients suffering from hereditary retinal degeneration receive a retinal implant to restore sight. Subretinal implant "ON" results in significant visual acuity improvement, when compared to "OFF" condition.
- Trial name
- NCT01024803
- Testing
- Device name: "Retina Implant model Alpha". Surgical implantation of medical device into eye
- Where
- 6 sites in Germany, Hungary and 1 more
- Volunteers
- 39 people
- Sponsor
- Retina Implant AG
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
Rare Diseases, Albinism
Implementation of Long-read Sequencing for the Diagnosis of Rare Diseases.
Following on from the third national plan for rare diseases (PNMR3), the main objectives of the PNMR4 are to reduce diagnostic uncertainty and dead ends and to strengthen translational research to promote diagnosis and the…
- Conditions
- Rare Diseases, Albinism, Intellectual Disability
- Trial name
- LongEpi (NCT07400913)
- Testing
- Long-read sequencing
- Where
- France
- Volunteers
- 150 people
- Sponsor
- University Hospital, Bordeaux
Brain and Central Nervous System Tumors, Leukemia
Decision Making About Participating in Pharmacokinetic Studies in Patients Enrolled in a Phase I Treatment Clinical Trial
RATIONALE: Gathering information about why patients or their guardians agree or decline to participate in the optional pharmacokinetic portion of a phase I treatment study may help doctors plan clinical trials in the future.
- Conditions
- Brain and Central Nervous System Tumors, Leukemia, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00890435
- Testing
- questionnaire administration, psychosocial assessment and care
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Children's Oncology Group
Retinoblastoma
Carboplatin Periocular Injection for Retinoblastoma
This study will evaluate the clinical efficacy of periocular injections of carboplatin together with chemotherapy in the treatment of Retinoblastoma as compared to chemotherapy alone.
- Trial name
- NCT02137928
- Testing
- carboplatin periocular injection, chemotherapy
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Sun Yat-sen University
Macular Corneal Dystrophy
Fresh Corneal Lenticule Implantation in Macular Corneal Distrophy With Relex Smile Surgery
The aim of the study is to examine the effect of fresh corneal lenticule implantation as allogenic graft taken from myopic patients to implanted in patients with macular corneal dystrophy using Visumax Femtosecond Laser Smile…
- Trial name
- NCT04642729
- Testing
- Fresh Corneal Lenticule Implantation using Relex-Smile Surgery
- Where
- Kosovo
- Volunteers
- 15 people
- Sponsor
- Eye Hospital Pristina Kosovo
Retinoblastoma
Chemotherapy in Treating Patients With Retinoblastoma
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
- Trial name
- NCT00002675
- Testing
- filgrastim, carboplatin, cisplatin
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Healthy Subjects
Effect of Oat Compounds on Postprandial Glucose Response
In this project we will investigate synergistic effects of oat polar lipids and beta glucans on postprandial glucose tolerance
- Trial name
- NCT05177172
- Testing
- High beta glucans, Low beta glucans, Low beta glucans + low polar lipids
- Where
- Sweden
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Lund University
Eye Disease, Achromatopsia
CNTF Implants for CNGB3 Achromatopsia
Background: Achromatopsia is an inherited condition that causes vision loss because cells in the retina do not work properly. It causes loss of acuity, sensitivity to light, and loss of color vision.
- Trial name
- CNTF-CNGB3-1 (NCT01648452)
- Testing
- NT-501 CNTF-releasing implant
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Eye Institute (NEI)
Choroideremia
A Study to Assess Choroideremia (CHM) Health Outcomes
The primary objectives of this study are to describe disease progression and severity by age in participants with CHM, to assess health-related quality-of-life, resource utilization and work productivity, and to assess…
- Trial name
- NCT04750785
- Testing
- No Intervention
- Where
- 8 sites in United States
- Volunteers
- 46 people
- Sponsor
- Biogen
Type 1 Diabetes, Diabetes Insipidus
Tracking Neurodegeneration in Early Wolfram Syndrome
The goal of this study is to determine the pattern of early neurodegenerative changes in WFS (Wolfram Syndrome). The investigator will perform cross-sectional and longitudinal assessments of youth with WFS, targeting sensitive…
- Conditions
- Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
- Trial name
- TRACK (NCT02455414)
- Where
- United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Graves Ophthalmopathy, Thyroid Associated Ophthalmopathy
Treatment of Graves´ophthalmopathy with Simvastatin (GO-S)
In an investigator initiated multicenter trial (Malmö, Odense, Århus) the investigators aim at evaluating activity of Graves´ophthalmopathy (GO) and progress to severe GO in patients with mild to moderate Graves´ ophthalmopathy…
- Conditions
- Graves Ophthalmopathy, Thyroid Associated Ophthalmopathy, Thyroid Associated Orbitopathy
- Trial name
- NCT03131726
- Testing
- Simvastatin 40mg
- Where
- Sweden
- Volunteers
- 80 people
- Sponsor
- Lund University
Primary Congenital Glaucoma, Pediatric Glaucoma
Subscleral Trabeculectomy With Inverted T-Shaped Deep Sclerectomy for Primary Congenital Glaucoma
Primary congenital glaucoma (PCG) is a vision-threatening disorder of childhood characterized by elevated intraocular pressure (IOP) resulting from developmental abnormalities of the anterior chamber angle.
- Conditions
- Primary Congenital Glaucoma, Pediatric Glaucoma, Intraocular Pressure
- Trial name
- TDS-PCG (NCT07701278)
- Testing
- Subscleral Trabeculectomy Combined With Inverted T-Shaped Deep Sclerectomy
- Where
- Egypt
- Volunteers
- 34 people
- Sponsor
- Benha University
Choroidal Ischemia, Vitelliform Macular Dystrophy
Sildenafil for Treatment of Choroidal Ischemia
The hypothesis of this study is to determine if there is a benefit afforded by the use of systemic Sildenafil to patients with choroidal and retinal degenerations and dystrophies, such as vitelliform degeneration, dry and…
- Conditions
- Choroidal Ischemia, Vitelliform Macular Dystrophy, Age-related Macular Degeneration, Central Serous Retinopathy
- Trial name
- NCT04356716
- Testing
- Sildenafil, Standard of Care Sildenafil, Ocular Coherence Tomography-Angiography (OCT-A)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Columbia University
Behçet's Disease, Juvenile Psoriatic Arthritis
A Study of Apremilast in Children With Oral Ulcers Associated With Behçet's Disease or Juvenile Psoriatic Arthritis
The primary objective of this study is to evaluate the long-term safety of apremilast in subjects 2 years of age or older with oral ulcers associated with Behçets disease or 5 years of age or older with active juvenile psoriatic…
- Trial name
- NCT05767047
- Testing
- Apremilast
- Where
- 7 sites in Greece, Israel and 2 more
- Volunteers
- 48 people
- Sponsor
- Amgen
RP2-associated Retinitis Pigmentosa, X-Linked Retinitis Pigmentosa (XLRP)
InsightRP2 Registry
InsightRP2 is a secure online patient registry specific to RP2-associated retinitis pigmentosa (RP). It is our goal to further the scientific understanding of this rare disease and to support research in to a gene therapy for…
- Conditions
- RP2-associated Retinitis Pigmentosa, X-Linked Retinitis Pigmentosa (XLRP), Retinitis Pigmentosa 2
- Trial name
- NCT06982417
- Where
- Germany
- Volunteers
- 200 people
- Sponsor
- University of Göttingen
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