Neurofibromatosis 1 Clinical Trial
Official title:
Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1 and High Plexiform Neurofibroma Tumor Burden
Verified date | February 2021 |
Source | Children's National Research Institute |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This study is being conducted to determine if Whole Body MRI (WBMRI) can be used to identify Atypical Neurofibromas (ANF) in Neurofibromatosis Type 1 (NF1) patients with high tumor burden. Each enrolled participant will have two (2) WBMRIs without sedation during the study period. Eligible participants must be Male or Female between the ages of 8-30 with diagnosed NF1; with one or more PN greater than 3cm in diameter and willing to comply with study procedures.
Status | Active, not recruiting |
Enrollment | 10 |
Est. completion date | August 1, 2022 |
Est. primary completion date | August 1, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 8 Years to 30 Years |
Eligibility | Inclusion Criteria: 1. Males or females between 8-30 years of age 2. Confirmed diagnosis of NF1 3. Stated willingness to comply with all study procedures and availability for the duration of the study 4. Prior MRI documentation confirming >=1 PN that is >3cm in diameter Exclusion Criteria: 1. Unable to undergo MRI without sedation 2. Presence of metal or other devices that are contraindicated for MRI |
Country | Name | City | State |
---|---|---|---|
United States | Children's National Health System | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Children's National Research Institute |
United States,
Ahlawat S, Fayad LM, Khan MS, Bredella MA, Harris GJ, Evans DG, Farschtschi S, Jacobs MA, Chhabra A, Salamon JM, Wenzel R, Mautner VF, Dombi E, Cai W, Plotkin SR, Blakeley JO; Whole Body MRI Committee for the REiNS International Collaboration; REiNS International Collaboration Members 2016. Current whole-body MRI applications in the neurofibromatoses: NF1, NF2, and schwannomatosis. Neurology. 2016 Aug 16;87(7 Suppl 1):S31-9. doi: 10.1212/WNL.0000000000002929. — View Citation
Beert E, Brems H, Daniëls B, De Wever I, Van Calenbergh F, Schoenaers J, Debiec-Rychter M, Gevaert O, De Raedt T, Van Den Bruel A, de Ravel T, Cichowski K, Kluwe L, Mautner V, Sciot R, Legius E. Atypical neurofibromas in neurofibromatosis type 1 are premalignant tumors. Genes Chromosomes Cancer. 2011 Dec;50(12):1021-32. doi: 10.1002/gcc.20921. Epub 2011 Aug 24. — View Citation
Cai W, Kassarjian A, Bredella MA, Harris GJ, Yoshida H, Mautner VF, Wenzel R, Plotkin SR. Tumor burden in patients with neurofibromatosis types 1 and 2 and schwannomatosis: determination on whole-body MR images. Radiology. 2009 Mar;250(3):665-73. doi: 10.1148/radiol.2503080700. — View Citation
Domínguez-Vigil IG, Moreno-Martínez AK, Wang JY, Roehrl MHA, Barrera-Saldaña HA. The dawn of the liquid biopsy in the fight against cancer. Oncotarget. 2017 Dec 8;9(2):2912-2922. doi: 10.18632/oncotarget.23131. eCollection 2018 Jan 5. Review. — View Citation
Higham CS, Dombi E, Rogiers A, Bhaumik S, Pans S, Connor SEJ, Miettinen M, Sciot R, Tirabosco R, Brems H, Baldwin A, Legius E, Widemann BC, Ferner RE. The characteristics of 76 atypical neurofibromas as precursors to neurofibromatosis 1 associated malignant peripheral nerve sheath tumors. Neuro Oncol. 2018 May 18;20(6):818-825. doi: 10.1093/neuonc/noy013. — View Citation
Kim A, Stewart DR, Reilly KM, Viskochil D, Miettinen MM, Widemann BC. Malignant Peripheral Nerve Sheath Tumors State of the Science: Leveraging Clinical and Biological Insights into Effective Therapies. Sarcoma. 2017;2017:7429697. doi: 10.1155/2017/7429697. Epub 2017 May 16. Review. — View Citation
Mautner VF, Asuagbor FA, Dombi E, Fünsterer C, Kluwe L, Wenzel R, Widemann BC, Friedman JM. Assessment of benign tumor burden by whole-body MRI in patients with neurofibromatosis 1. Neuro Oncol. 2008 Aug;10(4):593-8. doi: 10.1215/15228517-2008-011. Epub 2008 Jun 17. — View Citation
Nguyen R, Dombi E, Widemann BC, Solomon J, Fuensterer C, Kluwe L, Friedman JM, Mautner VF. Growth dynamics of plexiform neurofibromas: a retrospective cohort study of 201 patients with neurofibromatosis 1. Orphanet J Rare Dis. 2012 Oct 4;7:75. doi: 10.1186/1750-1172-7-75. — View Citation
Plotkin SR, Bredella MA, Cai W, Kassarjian A, Harris GJ, Esparza S, Merker VL, Munn LL, Muzikansky A, Askenazi M, Nguyen R, Wenzel R, Mautner VF. Quantitative assessment of whole-body tumor burden in adult patients with neurofibromatosis. PLoS One. 2012;7(4):e35711. doi: 10.1371/journal.pone.0035711. Epub 2012 Apr 27. — View Citation
Wasa J, Nishida Y, Tsukushi S, Shido Y, Sugiura H, Nakashima H, Ishiguro N. MRI features in the differentiation of malignant peripheral nerve sheath tumors and neurofibromas. AJR Am J Roentgenol. 2010 Jun;194(6):1568-74. doi: 10.2214/AJR.09.2724. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Determine if the cfDNA levels correlate with presence of ANF. | Level of cfDNA in plasma of participants with ANF compared to participants without ANF | 3-4 years | |
Primary | To determine if WBMRI can be used to identify ANF in NF1 patients with high tumor burden. | Number of NF1 participants with high tumor burden who have ANF on WBMRI | 2-3 years | |
Secondary | Determine if clinical signs and symptoms correlate with tumor burden and/or the presence of ANF on WBMRI | Number of patients with high PN tumor burden who have clinical symptoms | 2-3 years |
Status | Clinical Trial | Phase | |
---|---|---|---|
Active, not recruiting |
NCT01362803 -
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
|
Phase 1/Phase 2 | |
Completed |
NCT00352599 -
Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)
|
Phase 1 | |
Completed |
NCT05005845 -
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
|
Phase 2 | |
Completed |
NCT03531814 -
Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials
|
N/A | |
Completed |
NCT00846430 -
Medical Treatment of "High-Risk" Neurofibromas
|
Phase 2 | |
Completed |
NCT03433183 -
SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
|
Phase 2 | |
Recruiting |
NCT05331105 -
HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
|
Phase 2 | |
Completed |
NCT03310996 -
Non-invasive Stimulation in Neurofibromatosis Type 1
|
N/A | |
Recruiting |
NCT03406208 -
Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing
|
N/A | |
Recruiting |
NCT04750928 -
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
|
Phase 1/Phase 2 | |
Not yet recruiting |
NCT06262113 -
A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
|
N/A | |
Active, not recruiting |
NCT04924608 -
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
|
Phase 3 | |
Recruiting |
NCT02544022 -
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
|
||
Recruiting |
NCT04941027 -
Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1
|
||
Completed |
NCT02153931 -
Internet Support Group for Parents of a Child With Neurofibromatosis Type 1
|
||
Completed |
NCT05196854 -
Clinical Hypnosis and Home Blood Pressure Monitoring in Children With Neurofibromatosis Type 1
|
N/A | |
Active, not recruiting |
NCT01650142 -
Modifying Genes in Neurofibromatosis 1
|
N/A | |
Completed |
NCT05377008 -
Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1
|
N/A | |
Completed |
NCT04879160 -
Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial
|
||
Recruiting |
NCT05735717 -
MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
|
Phase 2 |