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MicroRNAs in Patients With Neurofibromatosis Type 1

This study has been completed.
Information provided by (Responsible Party):
Rishi Lulla, Ann & Robert H Lurie Children's Hospital of Chicago Identifier:
First received: May 8, 2012
Last updated: February 3, 2016
Last verified: February 2016

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.

Glioma Neurofibromatosis Type 1

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: MicroRNAs as Disease Markers for Central Nervous System Tumors in Patients With Neurofibromatosis Type 1

Resource links provided by NLM:

Further study details as provided by Rishi Lulla, Ann & Robert H Lurie Children's Hospital of Chicago:

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

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

Enrollment: 9
Study Start Date: February 2012
Study Completion Date: July 2015
Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
NF-1 without evidence of glioma
NF-1 with evidence of glioma


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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01595139

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

J.D. Storey, A direct approach to false discovery rates. JRSS B 64 (2002) 479-498.

Responsible Party: Rishi Lulla, Principal Investigator, Ann & Robert H Lurie Children's Hospital of Chicago Identifier: NCT01595139     History of Changes
Other Study ID Numbers: 2012-14927
Study First Received: May 8, 2012
Last Updated: February 3, 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
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 Neoplasms
Nervous System Neoplasms
Peripheral Nervous System Diseases
Neuromuscular Diseases processed this record on September 21, 2017