Last reviewed · How we verify
NCT02193724
Feasibility of Generating Pluripotent Stem Cells From Patients With Familial Retinoblastoma
trial testing Skin Biopsy in Retinoblastoma in 15 participants. Completed in 23 August 2019.
23 August 2019
Quick facts
| Lead sponsor | St. Jude Children's Research Hospital |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 15 |
| Start date | 4 November 2014 |
| Primary completion | 23 August 2019 |
| Estimated completion | 23 August 2019 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Skin Biopsy
- Blood Draw — full drug profile →
Conditions studied
- Retinoblastoma — all drugs for Retinoblastoma →
Sponsor
St. Jude Children's Research Hospital
Who can join
Eligibility, any sex, with Retinoblastoma. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
5 peer-reviewed publications reference this trial (live from Europe PMC):
-
Ethical and Safety Issues of Stem Cell-Based Therapy.
Volarevic V, Markovic BS, Gazdic M, Volarevic A, et al · · 2018 · cited 552× · PMID 29333086 · DOI 10.7150/ijms.21666 -
Retinoblastoma from human stem cell-derived retinal organoids.
Norrie JL, Nityanandam A, Lai K, Chen X, et al · · 2021 · cited 81× · PMID 34315877 · DOI 10.1038/s41467-021-24781-7 -
Cell therapy in acute respiratory distress syndrome.
Horie S, Gonzalez HE, Laffey JG, Masterson CH. · · 2018 · cited 44× · PMID 30416812 · DOI 10.21037/jtd.2018.08.28 -
Cell Transdifferentiation and Reprogramming in Disease Modeling: Insights into the Neuronal and Cardiac Disease Models and Current Translational Strategies.
Kalra RS, Dhanjal JK, Das M, Singh B, et al · · 2021 · cited 16× · PMID 34685537 · DOI 10.3390/cells10102558 -
Human Stem Cell Transplantation for Retinal Degenerative Diseases: Where Are We Now?
Alcalde I, Sánchez-Fernández C, Martín C, De Pablo N, et al · · 2022 · cited 7× · PMID 35056410 · DOI 10.3390/medicina58010102
Verify or expand the search:
- PubMed search for NCT02193724
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Skin Biopsy
Trials testing the same drug.
- NCT07349771 — Axatilimab Plus Standard of Care Therapy for the Prevention of Graft Versus Host Disease Following Allogeneic Hematopoie · Phase 2 · not yet recruiting
- NCT07011810 — Axatilimab for Sclerotic Chronic Graft-versus-Host Disease · Phase 2 · recruiting
- NCT05515692 — Electron Beam Radiotherapy for the Treatment of Refractory Sclerodermatous Chronic Graft Versus Host Disease · EARLY_PHASE1 · withdrawn
- NCT05739149 — A Study to Evaluate Microvessel Ultrasound Imaging of Wound Healing in Patients With Chronic Ulcers · NA · recruiting
- NCT05993871 — Diabetic Small Fiber Neuropathy: Clinical, Electrophysiological and Neurosonographic Study · completed
Other recruiting trials for Retinoblastoma
Currently open trials in the same condition.
- NCT06725173 — Detailed Phenotypic and Genotype Study to Correlate RB1 Mutations Relating to Primary Ocular Tumors and Secondary Extra- · recruiting
- NCT06972602 — High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed · EARLY_PHASE1 · recruiting
- NCT06424301 — Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma · EARLY_PHASE1 · recruiting
- NCT06367530 — Trilateral Retinoblastoma: Incidence and Outcomes · recruiting
- NCT05650749 — GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma · Phase 1 · recruiting
Other St. Jude Children's Research Hospital trials
Trials by the same sponsor.
- NCT07222735 — Hypofractionated Radiation in Combination With B7-H3-CAR T Cells for Pediatric Patients With Relapsed/Refractory Sarcoma · Phase 1 · recruiting
- NCT07085338 — A Phase II Study With a Safety Run-In of the Addition of N-803 to a Chemoimmunotherapy Backbone for the Treatment of Pat · Phase 2 · recruiting
- NCT05664113 — Feasibility, Safety, and Potential Efficacy of Fecal Microbiota Transplantation (FMT) for Gastrointestinal Dysfunction i · Phase 1 · recruiting
- NCT06993688 — Revealing Information Genuinely & Honestly Across Time - Communication Preferences Visit · NA · recruiting
- NCT06326463 — CAR T-cell Therapy Directed to CD70 for Pediatric Patients With Hematological Malignancies · Phase 1 · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT02193724 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by St. Jude Children's Research Hospital
- Last refreshed: 23 October 2020
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02193724.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing