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MicroRNAs in Patients With Neurofibromatosis Type 1

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01595139
Recruitment Status : Completed
First Posted : May 9, 2012
Last Update Posted : February 5, 2016
Information provided by (Responsible Party):
Rishi Lulla, Ann & Robert H Lurie Children's Hospital of Chicago

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.

Condition or disease
Glioma Neurofibromatosis Type 1

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Study Type : Observational
Actual Enrollment : 9 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: MicroRNAs as Disease Markers for Central Nervous System Tumors in Patients With Neurofibromatosis Type 1
Study Start Date : February 2012
Actual Primary Completion Date : July 2015
Actual Study Completion Date : July 2015

NF-1 without evidence of glioma
NF-1 with evidence of glioma

Primary Outcome Measures :
  1. Evaluate miRNA expression patterns in tissue of low grade gliomas [ Time Frame: 2 years ]

Secondary Outcome Measures :
  1. Evaluated miRNA expression patterns between patients with and without imaging findings of gliomas [ Time Frame: 2 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participation in the study will be offered to patient's ages 2 years to 21 years seen for a routine visit in the Neurofibromatosis Clinic at Children's Memorial Hospital and Lurie Children's Hospital in Chicago.

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01595139

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United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
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Principal Investigator: Rishi Lulla, MD Ann & Robert H Lurie Children's Hospital of Chicago
J.D. Storey, A direct approach to false discovery rates. JRSS B 64 (2002) 479-498.

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Responsible Party: Rishi Lulla, Principal Investigator, Ann & Robert H Lurie Children's Hospital of Chicago Identifier: NCT01595139    
Other Study ID Numbers: 2012-14927
First Posted: May 9, 2012    Key Record Dates
Last Update Posted: February 5, 2016
Last Verified: February 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Additional relevant MeSH terms:
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Neurofibromatosis 1
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms