MicroRNAs in Patients With Neurofibromatosis Type 1

• ClinicalTrials.gov Identifier: NCT01595139
• Recruitment Status: Completed
• First Posted: May 9, 2012
• Last Update Posted: February 5, 2016
• Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
• Information provided by (Responsible Party): Rishi Lulla, Ann & Robert H Lurie Children's Hospital of Chicago

Tracking Information

• First Submitted Date: May 8, 2012
• First Posted Date: May 9, 2012
• Last Update Posted Date: February 5, 2016
• Study Start Date: February 2012
• Actual Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: May 8, 2012): Evaluate miRNA expression patterns in tissue of low grade gliomas [ Time Frame: 2 years ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures (submitted: May 8, 2012): Evaluated miRNA expression patterns between patients with and without imaging findings of gliomas [ Time Frame: 2 years ]
• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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.

• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Case-Only; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.

Condition

• Glioma
• Neurofibromatosis Type 1

• Intervention: Not Provided

Study Groups/Cohorts

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

Publications *

• Fernandez-L A, Northcott PA, Taylor MD, Kenney AM. Normal and oncogenic roles for microRNAs in the developing brain. Cell Cycle. 2009 Dec 15;8(24):4049-54. Epub 2009 Dec 5.
• Taylor DD, Gercel-Taylor C. MicroRNA signatures of tumor-derived exosomes as diagnostic biomarkers of ovarian cancer. Gynecol Oncol. 2008 Jul;110(1):13-21. doi: 10.1016/j.ygyno.2008.04.033. Erratum in: Gynecol Oncol. 2010 Jan;116(1):153.
• Lawrie CH, Gal S, Dunlop HM, Pushkaran B, Liggins AP, Pulford K, Banham AH, Pezzella F, Boultwood J, Wainscoat JS, Hatton CS, Harris AL. Detection of elevated levels of tumour-associated microRNAs in serum of patients with diffuse large B-cell lymphoma. Br J Haematol. 2008 May;141(5):672-5. doi: 10.1111/j.1365-2141.2008.07077.x. Epub 2008 Mar 3.
• Mitchell PS, Parkin RK, Kroh EM, Fritz BR, Wyman SK, Pogosova-Agadjanyan EL, Peterson A, Noteboom J, O'Briant KC, Allen A, Lin DW, Urban N, Drescher CW, Knudsen BS, Stirewalt DL, Gentleman R, Vessella RL, Nelson PS, Martin DB, Tewari M. Circulating microRNAs as stable blood-based markers for cancer detection. Proc Natl Acad Sci U S A. 2008 Jul 29;105(30):10513-8. doi: 10.1073/pnas.0804549105. Epub 2008 Jul 28.
• Huang Z, Huang D, Ni S, Peng Z, Sheng W, Du X. Plasma microRNAs are promising novel biomarkers for early detection of colorectal cancer. Int J Cancer. 2010 Jul 1;127(1):118-26. doi: 10.1002/ijc.25007.
• Ng EK, Chong WW, Jin H, Lam EK, Shin VY, Yu J, Poon TC, Ng SS, Sung JJ. Differential expression of microRNAs in plasma of patients with colorectal cancer: a potential marker for colorectal cancer screening. Gut. 2009 Oct;58(10):1375-81. doi: 10.1136/gut.2008.167817. Epub 2009 Feb 6.
• Heneghan HM, Miller N, Lowery AJ, Sweeney KJ, Kerin MJ. MicroRNAs as Novel Biomarkers for Breast Cancer. J Oncol. 2009;2009:950201. doi: 10.1155/2010/950201. Epub 2009 Jul 20.
• Zhu W, Qin W, Atasoy U, Sauter ER. Circulating microRNAs in breast cancer and healthy subjects. BMC Res Notes. 2009 May 19;2:89. doi: 10.1186/1756-0500-2-89.
• Albers AC, Gutmann DH. Gliomas in patients with neurofibromatosis type 1. Expert Rev Neurother. 2009 Apr;9(4):535-9. doi: 10.1586/ern.09.4. Review.
• Chai G, Liu N, Ma J, Li H, Oblinger JL, Prahalad AK, Gong M, Chang LS, Wallace M, Muir D, Guha A, Phipps RJ, Hock JM, Yu X. MicroRNA-10b regulates tumorigenesis in neurofibromatosis type 1. Cancer Sci. 2010 Sep;101(9):1997-2004. doi: 10.1111/j.1349-7006.2010.01616.x.
• Smyth GK. Linear models and empirical bayes methods for assessing differential expression in microarray experiments. Stat Appl Genet Mol Biol. 2004;3:Article3. Epub 2004 Feb 12.
• J.D. Storey, A direct approach to false discovery rates. JRSS B 64 (2002) 479-498.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: February 3, 2016): 9
• Original Estimated Enrollment (submitted: May 8, 2012): 40
• Actual Study Completion Date: July 2015
• Actual Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 21 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01595139
• Other Study ID Numbers: 2012-14927
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Responsible Party: Rishi Lulla, Ann & Robert H Lurie Children's Hospital of Chicago
• Study Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
• Collaborators: Not Provided

Investigators

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

• PRS Account: Ann & Robert H Lurie Children's Hospital of Chicago
• Verification Date: February 2016