Retinoblastoma Clinical Trial
Official title:
Feasibility, Validation and Differentiation of Induced Pluripotent Stem Cells Produced From Patients With Heritable Retinoblastoma
The goal of this study is to determine if human RB1-deficient induced pluripotent stem cells (iPSCs) can produce retina, and, furthermore, can give rise to retinoblastoma in culture. This unique opportunity to study the initiation of retinoblastoma in the developing retina will shed light on the cell of origin for retinoblastoma and allow the investigators to study the earliest molecular and cellular events in retinoblastoma tumorigenesis. OBJECTIVES: - To establish the feasibility of producing induced pluripotent stem cells (iPSCs) from retinoblastoma patients with germline RB1 mutations (RB1-deficient iPSCs). - To validate human RB1-deficient iPSCs by confirming karyotype, pluripotency and RB1 mutation. - To differentiate the RB1-deficient iPSCs into retina as a model of the initiation of retinoblastoma in the developing retina.
This is an observational study where a small skin cell sample or peripheral blood sample will be used to produce iPSCs. After RB1-deficient iPSCs are produced, their karyotype and RB1 mutation will be confirmed and their pluripotency will be tested by studying the expression of pluripotent genes and proteins according to standardized guidelines established for human iPSCs. After validation of the RB1-deficient iPSCs, they will be differentiated in the laboratory into retina following established protocols. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04564521 -
Nitroglycerin for Intra-arterial Chemotherapy in Pediatric Retinoblastoma.
|
N/A | |
Recruiting |
NCT02933333 -
G-CSF Alone or Combination With GM-CSF on Prevention and Treatment of Infection in Children With Malignant Tumor
|
Phase 4 | |
Withdrawn |
NCT01151748 -
Intra-arterial Chemotherapy for Advanced Intraocular Retinoblastoma
|
Phase 2 | |
Completed |
NCT01048892 -
Seneca Valley Virus-001 and Cyclophosphamide in Treating Young Patients With Relapsed or Refractory Neuroblastoma, Rhabdomyosarcoma, or Rare Tumors With Neuroendocrine Features
|
Phase 1 | |
Active, not recruiting |
NCT00360750 -
Chemotherapy With or Without Radiation Therapy or Observation in Treating Young Patients With Advanced Retinoblastoma Who Have Undergone Surgery to Remove the Eye
|
N/A | |
Completed |
NCT00003173 -
High-Dose Thiotepa Plus Peripheral Stem Cell Transplantation in Treating Patients With Refractory Solid Tumors
|
Phase 2 | |
Enrolling by invitation |
NCT06227962 -
Cognitive Functioning and Health Related Quality of Life in Retinoblastoma Survivors
|
||
Completed |
NCT01661400 -
Anti-Angiogenic Therapy Post Transplant (ASCR) for Pediatric Solid Tumors
|
Phase 1 | |
Terminated |
NCT02617862 -
PCI Imaging System in Pediatric Ophthalmology
|
N/A | |
Recruiting |
NCT02329002 -
SPT Screening in Irradiated Hereditary Retinoblastoma Survivors
|
N/A | |
Terminated |
NCT01466855 -
A Study of Intra-Ophthalmic Artery Topotecan Infusion for the Treatment of Retinoblastoma
|
Early Phase 1 | |
Completed |
NCT01505569 -
Auto Transplant for High Risk or Relapsed Solid or CNS Tumors
|
N/A | |
Completed |
NCT00002515 -
Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Rare Cancer
|
Phase 2 | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
Completed |
NCT06367569 -
Evaluation of MRI of the Pineal Gland in Retinoblastoma
|
||
Completed |
NCT01884194 -
Morphological Analysis of the Pineal Gland in Pediatric Retinoblastoma Patients Using Magnetic Resonance Imaging
|
N/A | |
Terminated |
NCT01393769 -
Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage
|
Phase 2 | |
Completed |
NCT00006246 -
Busulfan in Treating Children and Adolescents With Refractory CNS Cancer
|
Phase 1 | |
Terminated |
NCT00003926 -
Amifostine to Protect From Side Effects of PSCT in Treating Patients With Solid Tumors
|
Phase 1 | |
Withdrawn |
NCT00003273 -
Chemotherapy Followed by Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Brain Tumor
|
Phase 2 |