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NCT02193724

Feasibility of Generating Pluripotent Stem Cells From Patients With Familial Retinoblastoma

Completed Last updated 23 October 2020
What this trial tests

trial testing Skin Biopsy in Retinoblastoma in 15 participants. Completed in 23 August 2019.

Timeline
4 November 2014
Primary endpoint
23 August 2019
23 August 2019

Quick facts

Lead sponsorSt. Jude Children's Research Hospital
StatusCompleted
Study typeOBSERVATIONAL
Enrollment15
Start date4 November 2014
Primary completion23 August 2019
Estimated completion23 August 2019
Sites1 location across United States

Drugs / interventions tested

Conditions studied

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):

  1. 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
  2. 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
  3. 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
  4. 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
  5. 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:

Other trials of Skin Biopsy

Trials testing the same drug.

Other recruiting trials for Retinoblastoma

Currently open trials in the same condition.

Other St. Jude Children's Research Hospital trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

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