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

NCT03332030 · Status: WITHDRAWN · Type: OBSERVATIONAL

Last updated 2024-02-07

No results posted yet for this study

Summary

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.

Conditions

  • Neurofibromatosis Type 1
  • Tumors of the Central Nervous System

Interventions

DIAGNOSTIC_TEST

Collection of Stem Cells

One time collection of a 20 ml blood sample

Sponsors & Collaborators

  • Roger Packer

    lead OTHER

Principal Investigators

  • Roger Packer, MD · Children's National Research Institute

Eligibility

Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2015-11-27
Primary Completion
2024-02-05
Completion
2025-07-01

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03332030 on ClinicalTrials.gov