MicroRNAs in Patients With Neurofibromatosis Type 1

NCT ID: NCT01595139

Last Updated: 2024-12-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

9 participants

Study Classification

OBSERVATIONAL

Study Start Date

2012-02-29

Study Completion Date

2015-07-31

Brief Summary

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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

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Conditions

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Glioma Neurofibromatosis Type 1

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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NF-1 without evidence of glioma

No interventions assigned to this group

NF-1 with evidence of glioma

No interventions assigned to this group

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ann & Robert H Lurie Children's Hospital of Chicago

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Rishi Lulla, MD

Role: PRINCIPAL_INVESTIGATOR

Ann & Robert H Lurie Children's Hospital of Chicago

Locations

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Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Countries

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United States

References

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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. doi: 10.4161/cc.8.24.10243. Epub 2009 Dec 5.

Reference Type BACKGROUND
PMID: 19901543 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18589210 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18318758 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18663219 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 19876917 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 19201770 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 19639033 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 19454029 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 19344304 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 20550523 (View on PubMed)

Smyth GK. Linear models and empirical bayes methods for assessing differential expression in microarray experiments. Stat Appl Genet Mol Biol. 2004;3:Article3. doi: 10.2202/1544-6115.1027. Epub 2004 Feb 12.

Reference Type BACKGROUND
PMID: 16646809 (View on PubMed)

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

Reference Type BACKGROUND

Other Identifiers

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2012-14927

Identifier Type: -

Identifier Source: org_study_id