Clinical trials: Inherited Retinal Diseases
Retinitis Pigmentosa (RP)
Retrospective Natural History Study of Retinitis Pigmentosa
This is natural history study of rods and cones degenerations in patients with Retinitis Pigmentosa (RP) caused by pathogenic mutations in RHO, PDE6A or PDE6B gene mutations.
- Trial name
- PHENOROD1 (NCT03975543)
- Where
- France
- Volunteers
- 113 people
- Sponsor
- SparingVision
Zellweger Spectrum Disorder
Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder
The goal of this observational study is to define the course of the retinal degeneration in a ZSD patient cohort. The objective of this study is to gather information so the investigators can: 1.
- Trial name
- ZSDvision (NCT06190626)
- Where
- 3 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Cytomegalovirus Retinitis, HIV Infections
A Randomized Study Comparing the Safety and Efficacy of Two Regimens of Oral Ganciclovir to Intravenous Ganciclovir Maintenance Therapy for Cytomegalovirus Retinitis in People With AIDS Who Have Received Prior Ganciclovir Therapy
To compare the safety and tolerance of oral ganciclovir at a double dose 3 times/day or a single dose 6 times/day to IV ganciclovir given for 20 weeks of maintenance therapy.
- Trial name
- NCT00002247
- Testing
- Ganciclovir
- Where
- 20 sites in United States, Canada
- Volunteers
- 225 people
- Sponsor
- Hoffmann-La Roche
Pediatric Solid Tumor, Germ Cell Tumor
EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
This is a phase I, open-label, non-randomized study that will enroll pediatric and young adult research participants with relapsed or refractory non-CNS solid tumors to evaluate the safety, feasibility, and efficacy of…
- Conditions
- Pediatric Solid Tumor, Germ Cell Tumor, Retinoblastoma, Hepatoblastoma
- Trial name
- NCT03618381
- Testing
- second generation 4-1BBζ EGFR806-EGFRt, second generation 4-1BBζ EGFR806-EGFRt and a second generation 4 1BBζ CD19-Her2tG
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Seattle Children's Hospital
Stargardt's Disease
Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration
Primary Objective: To evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt's Macular Degeneration. Secondary Objective: To assess: Safety Biological activity
- Trial name
- NCT01736592
- Testing
- Long term follow up in all patients who received SAR422459 in previous study TDU13583
- Where
- 2 sites in United States, France
- Volunteers
- 27 people
- Sponsor
- Sanofi
Retinoblastoma
Chemotherapy With or Without Radiation Therapy or Observation in Treating Young Patients With Advanced Retinoblastoma Who Have Undergone Surgery to Remove the Eye
RATIONALE: Drugs used in chemotherapy, such as carboplatin, vincristine, etoposide, and cytarabine, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00360750
- Testing
- carboplatin, cytarabine, etoposide
- Where
- 13 sites in United Kingdom
- Sponsor
- Children's Cancer and Leukaemia Group
Inherited Retinal Dystrophy, Retinitis Pigmentosa
Mechanisms of Inherited Retinal Dystrophies Using Whole Genome Sequencing and in Vitro and in Vivo Models
Inherited retinal dystrophies (IRDs), a large group of heterogeneous and rare disorders, may result in irreversible bilateral visual loss and blindness.
- Conditions
- Inherited Retinal Dystrophy, Retinitis Pigmentosa, Macular Dystrophy
- Trial name
- NCT05793515
- Testing
- whole genome sequencing
- Where
- Italy
- Volunteers
- 120 people
- Sponsor
- Istituto Superiore di Sanità
Deafness, Kidney Diseases
Endolymphatic Sac Tumors in a Population of Patients With Von Hippel-Lindau Disease:The Natural History and Pathobiology, and a Prospective Non-Randomized Clinical Trial of Hearing Preservation Surgery in Patients With Early Stage Endolymphatic Sac Tumors
The von Hippel Lindau (VHL) gene has recently been identified as the genetic defect resulting in a syndrome of multiple neoplasias.
- Conditions
- Deafness, Kidney Diseases, Kidney Neoplasms, Neoplasms
- Trial name
- NCT00001668
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)