NCT01567787

Brief Summary

The purpose of this study is to determine if Proton Therapy can provide effective and safe treatment for Malignant Peripheral Nerve Sheath Tumors of the spine and Neurofibromas of the spine.

Trial Health

15
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Jun 2013

Typical duration for not_applicable

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 13, 2012

Completed
17 days until next milestone

First Posted

Study publicly available on registry

March 30, 2012

Completed
1.2 years until next milestone

Study Start

First participant enrolled

June 1, 2013

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2015

Completed
Last Updated

February 10, 2017

Status Verified

February 1, 2017

Enrollment Period

2.2 years

First QC Date

March 13, 2012

Last Update Submit

February 9, 2017

Conditions

Nerve Sheath TumorsNeurofibroma

Keywords

Proton Radiation TherapySpinal tumorsNeurofibroma(NF1)Malignant peripheral nerve sheath tumor(MPNST)

Outcome Measures

Primary Outcomes (1)

  • Local Control

    7 years after completion of RT

Secondary Outcomes (5)

  • Progression or palliation of pain, numbness, or weakness

    At 3, 6, 12, 24 and 60 months after RT

  • Number of adverse events

    At 3, 6, 12, 24, 60 months and 15 years after RT

  • Quality of Life

    3, 6, 12, 24 and 60 months after RT

  • Rate of malignant transformation within the high-dose volume

    15 years after RT

  • Rate of second malignant primaries outside of high dose volume, but located in tissues exposed to radiation within the beam path

    15 years after RT

Study Arms (2)

Proton Radiation for MPNST

EXPERIMENTAL

Proton radiation 30 cobalt gray equivalent(CGE)at 6 CGE per fraction

Radiation: Proton Radiation for MPNST

Proton Radiation for neurofibromas

EXPERIMENTAL

Proton radiation 25 cobalt gray equivalent(CGE) at 5 CGE per fraction

Radiation: Proton Radiation for neurofibromas

Interventions

Proton Radiation for MPNSTRADIATION

30 CGE at 6 CGE/Fx

Proton Radiation for MPNST
Proton Radiation for neurofibromasRADIATION

25 CGE at 5 CGE/Fx

Proton Radiation for neurofibromas

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • MPNSTs that are biopsy-positive and resected, subtotally resected or unresectable.
  • Symptomatic (pain, numbness, or weakness) NF1 neurofibromas that are biopsy-positive and unresectable or subtotally resected.
  • Symptomatic NF1 neurofibromas that are unbiopsied and PET-negative, if the patient refuses biopsy/surgery or is medically inoperable.
  • Asymptomatic NF1 neurofibromas with radiologic progression after surgery.
  • PET-positive, unbiopsied lesions in NF1 patients who refuse biopsy/surgery or are medically inoperable.

You may not qualify if:

  • Spinal instability.
  • Metal stabilization hardware within the target area.
  • Previously irradiated at this disease site.
  • Spinal cord compression with complete loss of function.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (26)

  • Hottinger AF, Khakoo Y. Neuro-oncology of Neurofibromatosis Type 1. Curr Treat Options Neurol. 2009 Jul;11(4):306-14. doi: 10.1007/s11940-009-0034-4.

    PMID: 19523356BACKGROUND

  • Friedman JM, Birch PH. Type 1 neurofibromatosis: a descriptive analysis of the disorder in 1,728 patients. Am J Med Genet. 1997 May 16;70(2):138-43. doi: 10.1002/(sici)1096-8628(19970516)70:23.0.co;2-u.

    PMID: 9128932BACKGROUND

  • Cnossen MH, de Goede-Bolder A, van den Broek KM, Waasdorp CM, Oranje AP, Stroink H, Simonsz HJ, van den Ouweland AM, Halley DJ, Niermeijer MF. A prospective 10 year follow up study of patients with neurofibromatosis type 1. Arch Dis Child. 1998 May;78(5):408-12. doi: 10.1136/adc.78.5.408.

    PMID: 9659085BACKGROUND

  • Mautner VF, Asuagbor FA, Dombi E, Funsterer 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.

    PMID: 18559970BACKGROUND

  • Seppala MT, Haltia MJ, Sankila RJ, Jaaskelainen JE, Heiskanen O. Long-term outcome after removal of spinal neurofibroma. J Neurosurg. 1995 Apr;82(4):572-7. doi: 10.3171/jns.1995.82.4.0572.

    PMID: 7897516BACKGROUND

  • Thakkar SD, Feigen U, Mautner VF. Spinal tumours in neurofibromatosis type 1: an MRI study of frequency, multiplicity and variety. Neuroradiology. 1999 Sep;41(9):625-9. doi: 10.1007/s002340050814.

    PMID: 10525761BACKGROUND

  • Tonsgard JH. Clinical manifestations and management of neurofibromatosis type 1. Semin Pediatr Neurol. 2006 Mar;13(1):2-7. doi: 10.1016/j.spen.2006.01.005.

    PMID: 16818170BACKGROUND

  • Needle MN, Cnaan A, Dattilo J, Chatten J, Phillips PC, Shochat S, Sutton LN, Vaughan SN, Zackai EH, Zhao H, Molloy PT. Prognostic signs in the surgical management of plexiform neurofibroma: the Children's Hospital of Philadelphia experience, 1974-1994. J Pediatr. 1997 Nov;131(5):678-82. doi: 10.1016/s0022-3476(97)70092-1.

    PMID: 9403645BACKGROUND

  • Ducatman BS, Scheithauer BW, Piepgras DG, Reiman HM, Ilstrup DM. Malignant peripheral nerve sheath tumors. A clinicopathologic study of 120 cases. Cancer. 1986 May 15;57(10):2006-21. doi: 10.1002/1097-0142(19860515)57:103.0.co;2-6.

    PMID: 3082508BACKGROUND

  • Evans DG, Baser ME, McGaughran J, Sharif S, Howard E, Moran A. Malignant peripheral nerve sheath tumours in neurofibromatosis 1. J Med Genet. 2002 May;39(5):311-4. doi: 10.1136/jmg.39.5.311.

    PMID: 12011145BACKGROUND

  • Ferner RE, Gutmann DH. International consensus statement on malignant peripheral nerve sheath tumors in neurofibromatosis. Cancer Res. 2002 Mar 1;62(5):1573-7.

    PMID: 11894862BACKGROUND

  • Chao RC, Pyzel U, Fridlyand J, Kuo YM, Teel L, Haaga J, Borowsky A, Horvai A, Kogan SC, Bonifas J, Huey B, Jacks TE, Albertson DG, Shannon KM. Therapy-induced malignant neoplasms in Nf1 mutant mice. Cancer Cell. 2005 Oct;8(4):337-48. doi: 10.1016/j.ccr.2005.08.011.

    PMID: 16226708BACKGROUND

  • Stiller CA, Chessells JM, Fitchett M. Neurofibromatosis and childhood leukaemia/lymphoma: a population-based UKCCSG study. Br J Cancer. 1994 Nov;70(5):969-72. doi: 10.1038/bjc.1994.431.

    PMID: 7947106BACKGROUND

  • Huson SM, Compston DA, Harper PS. A genetic study of von Recklinghausen neurofibromatosis in south east Wales. II. Guidelines for genetic counselling. J Med Genet. 1989 Nov;26(11):712-21. doi: 10.1136/jmg.26.11.712.

    PMID: 2511319BACKGROUND

  • Sharif S, Ferner R, Birch JM, Gillespie JE, Gattamaneni HR, Baser ME, Evans DG. Second primary tumors in neurofibromatosis 1 patients treated for optic glioma: substantial risks after radiotherapy. J Clin Oncol. 2006 Jun 1;24(16):2570-5. doi: 10.1200/JCO.2005.03.8349.

    PMID: 16735710BACKGROUND

  • Papageorgio C, Seiter K, Feldman EJ. Therapy-related myelodysplastic syndrome in adults with neurofibromatosis. Leuk Lymphoma. 1999 Feb;32(5-6):605-8. doi: 10.3109/10428199909058420.

    PMID: 10048435BACKGROUND

  • Gerszten PC, Burton SA, Ozhasoglu C, McCue KJ, Quinn AE. Radiosurgery for benign intradural spinal tumors. Neurosurgery. 2008 Apr;62(4):887-95; discussion 895-6. doi: 10.1227/01.neu.0000318174.28461.fc.

    PMID: 18496194BACKGROUND

  • Karabatsou K, Kiehl TR, Wilson DM, Hendler A, Guha A. Potential role of 18fluorodeoxyglucose-positron emission tomography/computed tomography in differentiating benign neurofibroma from malignant peripheral nerve sheath tumor associated with neurofibromatosis 1. Neurosurgery. 2009 Oct;65(4 Suppl):A160-70. doi: 10.1227/01.NEU.0000337597.18599.D3.

    PMID: 19927062BACKGROUND

  • Ferner RE, Golding JF, Smith M, Calonje E, Jan W, Sanjayanathan V, O'Doherty M. [18F]2-fluoro-2-deoxy-D-glucose positron emission tomography (FDG PET) as a diagnostic tool for neurofibromatosis 1 (NF1) associated malignant peripheral nerve sheath tumours (MPNSTs): a long-term clinical study. Ann Oncol. 2008 Feb;19(2):390-4. doi: 10.1093/annonc/mdm450. Epub 2007 Oct 11.

    PMID: 17932395BACKGROUND

  • Warbey VS, Ferner RE, Dunn JT, Calonje E, O'Doherty MJ. [18F]FDG PET/CT in the diagnosis of malignant peripheral nerve sheath tumours in neurofibromatosis type-1. Eur J Nucl Med Mol Imaging. 2009 May;36(5):751-7. doi: 10.1007/s00259-008-1038-0. Epub 2009 Jan 14.

    PMID: 19142634BACKGROUND

  • Wentworth S, Pinn M, Bourland JD, Deguzman AF, Ekstrand K, Ellis TL, Glazier SS, McMullen KP, Munley M, Stieber VW, Tatter SB, Shaw EG. Clinical experience with radiation therapy in the management of neurofibromatosis-associated central nervous system tumors. Int J Radiat Oncol Biol Phys. 2009 Jan 1;73(1):208-13. doi: 10.1016/j.ijrobp.2008.03.073. Epub 2008 Aug 5.

    PMID: 18687535BACKGROUND

  • Chopra R, Morris CG, Friedman WA, Mendenhall WM. Radiotherapy and radiosurgery for benign neurofibromas. Am J Clin Oncol. 2005 Jun;28(3):317-20. doi: 10.1097/01.coc.0000156923.52181.3d.

    PMID: 15923807BACKGROUND

  • Sahgal A, Chou D, Ames C, Ma L, Lamborn K, Huang K, Chuang C, Aiken A, Petti P, Weinstein P, Larson D. Image-guided robotic stereotactic body radiotherapy for benign spinal tumors: theUniversity of California San Francisco preliminary experience. Technol Cancer Res Treat. 2007 Dec;6(6):595-604. doi: 10.1177/153303460700600602.

    PMID: 17994789BACKGROUND

  • Dodd RL, Ryu MR, Kamnerdsupaphon P, Gibbs IC, Chang SD Jr, Adler JR Jr. CyberKnife radiosurgery for benign intradural extramedullary spinal tumors. Neurosurgery. 2006 Apr;58(4):674-85; discussion 674-85. doi: 10.1227/01.NEU.0000204128.84742.8F.

    PMID: 16575331BACKGROUND

  • Schadev S, Dodd RL, Chang SD, Soltys SG, Adler JR, Luxton G, Choi CYH, Tupper LA, Gibbs IC. Stereotactic Radiosurgery Yields Long-term Control for Benign Intradural, Extramedullary Spine Tumors. Int J Radiat Oncol Biol Phys 2009; 75:101 (Abstract)

    BACKGROUND

  • Korones DN, Padowski J, Factor BA, Constine LS. Do children with optic pathway tumors have an increased frequency of other central nervous system tumors? Neuro Oncol. 2003 Apr;5(2):116-20. doi: 10.1093/neuonc/5.2.116.

    PMID: 12672283BACKGROUND

MeSH Terms

Conditions

Nerve Sheath NeoplasmsNeurofibromaSpinal Cord NeoplasmsNeurofibrosarcoma

Interventions

Neurofibromin 1

Condition Hierarchy (Ancestors)

Neoplasms, Nerve TissueNeoplasms by Histologic TypeNeoplasmsPeripheral Nervous System NeoplasmsNervous System NeoplasmsNervous System DiseasesPeripheral Nervous System DiseasesNeuromuscular DiseasesCentral Nervous System NeoplasmsNeoplasms by SiteSpinal Cord DiseasesCentral Nervous System DiseasesFibrosarcomaNeoplasms, Fibrous TissueNeoplasms, Connective TissueNeoplasms, Connective and Soft TissueSarcoma

Intervention Hierarchy (Ancestors)

ras GTPase-Activating ProteinsGTPase-Activating ProteinsGTP-Binding Protein RegulatorsIntracellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsTumor Suppressor ProteinsNeoplasm Proteins

Study Officials

  • Randal H Henderson, MD

    University of Florida Proton Therapy Institute

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 13, 2012

First Posted

March 30, 2012

Study Start

June 1, 2013

Primary Completion

August 1, 2015

Study Completion

August 1, 2015

Last Updated

February 10, 2017

Record last verified: 2017-02

Data Sharing

IPD Sharing
Will not share