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Withdrawn No phase listed Observational Accepts healthy volunteers

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

ClinicalTrials.gov ID: NCT03332030

Public ClinicalTrials.gov record NCT03332030. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Nov 11, 2026, 12:27 AM EST

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Official title

Development of Stem Cell Lines in Children With Neurofibromatosis Type 1 and Tumors of the Central Nervous System

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT03332030
Recruitment status
Withdrawn
Study type
Observational
Phase
Not listed
Lead sponsor
Roger Packer
Other
Enrollment
Not listed

Conditions and interventions

Eligibility (public fields only)

Age range
Not listed
Sex
All
Healthy volunteers
Accepts healthy volunteers

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Nov 27, 2015 (Actual)
Primary completion
Feb 5, 2024 (Actual)
Completion
Jul 1, 2025 (Estimated)
Last update posted
Feb 7, 2024

2015 – 2025

United States locations

U.S. sites
1
U.S. states
1
U.S. cities
1
Facility City State ZIP Site status
Children's National Medical Center Washington D.C. District of Columbia 20010 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT03332030, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Feb 7, 2024 · Synced Nov 11, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT03332030 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →