NCT03332030

Stem Cells in NF1 Patients With Tumors of the Central Nervous System

Withdrawn Last updated 7 February 2024

What this trial tests

trial testing Collection of Stem Cells in Neurofibromatosis Type 1. Withdrawn.

Timeline

27 November 2015

Primary endpoint
5 February 2024

1 July 2025

Quick facts

Lead sponsor	Roger Packer
Status	Withdrawn
Study type	OBSERVATIONAL
Start date	27 November 2015
Primary completion	5 February 2024
Estimated completion	1 July 2025
Sites	1 location across United States

Drugs / interventions tested

• Collection of Stem Cells

Conditions studied

• Neurofibromatosis Type 1 — all drugs for Neurofibromatosis Type 1 →
• Tumors of the Central Nervous System — all drugs for Tumors of the Central Nervous System →

Sponsor

Roger Packer — full company profile →

Who can join

Eligibility, any sex, with Neurofibromatosis Type 1 or Tumors of the Central Nervous System. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Objectives 1. Establish an induced pluripotent stem cell (iPSC) bank for phenotypically well-characterized patients with NF1.

2\. Develop isogenic NF1 wild-type (NF1+/+), NF1 heterozygous (NF1+/-) and NF1 homozygous (NF1-/-) iPSC lines from individual patients using CRISPR/CAS9 technology.

3\. Differentiate and characterize disease-relevant brain cells such as excitatory and inhibitory neurons, astrocytes and oligodendrocytes from patient-specific iPSC lines.

4\. Screen and identify the drug(s) that can reverse or alleviate the disease phenotypes.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects.
   Li H, Yang Y, Hong W, Huang M, et al · · 2020 · cited 733× · PMID 32296011 · DOI 10.1038/s41392-019-0089-y
2. <i>In vivo</i> genome editing in animals using AAV-CRISPR system: applications to translational research of human disease.
   Lau CH, Suh Y. · · 2017 · cited 124× · PMID 29333255 · DOI 10.12688/f1000research.11243.1
3. Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy.
   Rasul MF, Hussen BM, Salihi A, Ismael BS, et al · · 2022 · cited 105× · PMID 35241090 · DOI 10.1186/s12943-021-01487-4
4. Recent advances and applications of CRISPR-Cas9 in cancer immunotherapy.
   Liu Z, Shi M, Ren Y, Xu H, et al · · 2023 · cited 93× · PMID 36797756 · DOI 10.1186/s12943-023-01738-6
5. Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research.
   You L, Tong R, Li M, Liu Y, et al · · 2019 · cited 71× · PMID 30989086 · DOI 10.1016/j.omtm.2019.02.008
6. Electrical Stimulation Promotes Stem Cell Neural Differentiation in Tissue Engineering.
   Cheng H, Huang Y, Yue H, Fan Y. · · 2021 · cited 66× · PMID 33968150 · DOI 10.1155/2021/6697574
7. Viral vectors and extracellular vesicles: innate delivery systems utilized in CRISPR/Cas-mediated cancer therapy.
   Ahmadi SE, Soleymani M, Shahriyary F, Amirzargar MR, et al · · 2023 · cited 55× · PMID 36854897 · DOI 10.1038/s41417-023-00597-z
8. CRISPR/Cas9 - An evolving biological tool kit for cancer biology and oncology.
   Tian X, Gu T, Patel S, Bode AM, et al · · 2019 · cited 53× · PMID 30911676 · DOI 10.1038/s41698-019-0080-7