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Cell-Based Approaches For Modeling and Treating Ataxia-Telangiectasia

NCT02246491 · Status: TERMINATED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2019-03-19

No results posted yet for this study

Summary

This research is being done to better understand the causes of the disease Ataxia-Telangiectasia and, in the longer-term, develop new therapies for the disease using stem cells.

Induced pluripotent stem cells (iPSC) are a type of cells that can be made in the laboratory from cells in your body, such as blood cells or skin cells (fibroblasts). These stem cells can then be used for research purposes. For example, stem cells can be used to investigate how the mutation in ATM causes the actual symptoms of Ataxia-Telangiectasia. In addition, the stem cells can be used to screen for drugs that could be helpful to treat the disease or to develop new laboratory techniques to correct the mutation that causes Ataxia-Telangiectasia. where the mutation that causes the disease is corrected by the investigators. The stem cells generated in this study will not be used directly for patient therapy and therefore this research does not have a direct benefit to you. However, it will help advance our understanding of the disease and develop future therapies.

Patients who enroll in this study will get all of the standard therapy they would get for their tumor whether or not they participate in this study. There is no extra or different therapy given. The study involves a one-time procedure (either blood collection or skin biopsy).

Conditions

  • Ataxia-Telangiectasia (A-T)

Interventions

OTHER

A-T iPS cell line

Reprogramming A-T patients iPS cell line

OTHER

Carrier patients iPS cell line

Reprogramming iPS cell line from carrier patients

Sponsors & Collaborators

Principal Investigators

  • Sonia Franco, M.D. · SKCCC at Johns Hopkins

Study Design

Allocation
NON_RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-02-03
Primary Completion
2018-07-05
Completion
2018-07-05

Countries

  • United States

Study Locations

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Entities

Companies

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 NCT02246491 on ClinicalTrials.gov