MicroRNAs in Patients With Neurofibromatosis Type 1

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Brief Title

MicroRNAs in Patients With Neurofibromatosis Type 1

Official Title

MicroRNAs as Disease Markers for Central Nervous System Tumors in Patients With Neurofibromatosis Type 1

Brief Summary

      MicroRNAs are small molecules which have recently been discovered in cells. They are known to
      be responsible for the normal development of cells and when they are disrupted can contribute
      to the development of cancer. Many previous studies have been done evaluating the expression
      of microRNAs in normal tissues as well as in a wide variety of cancers.

      Recently, microRNAs from tumor cells have been detected circulating in the blood of patients
      with cancer. This presents a novel opportunity to assess the utility of microRNAs in the
      blood as an early predictor of cancer as well as a marker of response to therapy. No previous
      studies have been performed evaluating microRNAs in archived tumor tissue and blood of
      patients with Neurofibromatosis type 1 (NF-1). The investigators propose a feasibility study
      to evaluate the presence of microRNAs in archived tumor tissue and the blood of patients with
      NF-1. If the investigators are able to identify circulating microRNAs in this population of
      pediatric patients, they will build upon this data in proposing a future study.
    



Study Type

Observational


Primary Outcome

Evaluate miRNA expression patterns in tissue of low grade gliomas

Secondary Outcome

 Evaluated miRNA expression patterns between patients with and without imaging findings of gliomas

Condition

Glioma


Study Arms / Comparison Groups

 NF-1 without evidence of glioma
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

9

Start Date

February 2012

Completion Date

July 2015

Primary Completion Date

July 2015

Eligibility Criteria

        Inclusion Criteria:

          -  Patients ages 2 years to 21 years.

          -  Patients with NF-1 being followed in the Neurofibromatosis Clinic.

          -  Patients have had MRI imaging in the 24 months prior to enrollment on the study.

          -  Patients may have known concurrent malignancies such as plexiform neurofibroma.

          -  Patients and/or parents/legal guardians must have signed an informed consent and
             assent when applicable.

        Exclusion Criteria:

          -  Patients who have had prior tumor-directed therapy (including chemotherapy and/or
             radiation)

          -  Patients with a prior or current diagnosis of a malignant peripheral nerve sheath
             tumor.

          -  Patients who are considered too ill to participate as determined by their treating
             physician

          -  Patients who are pregnant or lactating
      

Gender

All

Ages

2 Years - 21 Years

Accepts Healthy Volunteers

No

Contacts

Rishi Lulla, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01595139

Organization ID

2012-14927


Responsible Party

Principal Investigator

Study Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago


Study Sponsor

Rishi Lulla, MD, Principal Investigator, Ann & Robert H Lurie Children's Hospital of Chicago


Verification Date

February 2016