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Neoadjuvant Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in Cutaneous Stage L-lll Melanoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02451488
Recruitment Status : Completed
First Posted : May 22, 2015
Results First Posted : February 10, 2020
Last Update Posted : February 10, 2020
Information provided by (Responsible Party):
James W. Jakub, Mayo Clinic

Brief Summary:
Randomized trial to determine if neo-adjuvant subcutaneous GM-CSF restores the host regional lymph node immunity

Condition or disease Intervention/treatment Phase
Melanoma Drug: GM-CSF Other: Standard of Care Phase 4

Detailed Description:
The sentinel lymph nodes in patients with melanoma are immunosuppressed and the investigators have shown this occurs early in the disease process. This regional nodal immunosuppression precedes nodal metastasis and may be required for nodal spread. Administration of GM-CSF has been used to alter the immune response to metastatic melanoma. The investigators propose to assess whether administration of a short course of GM-CSF preoperatively to patients about to undergo wide local excisions and sentinel lymph node dissection can alter the immune environment of the sentinel lymph node and restore an immune surveillance profile in the sentinel lymph node.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Neoadjuvant GM-CSF Treatment and Modulation of Immune Cell Profile of the SLN in Melanoma
Study Start Date : May 2015
Actual Primary Completion Date : November 4, 2016
Actual Study Completion Date : November 4, 2016

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Melanoma
MedlinePlus related topics: Melanoma

Arm Intervention/treatment
Experimental: GM-CSF
Patients will be treated with 14 days of GM-CSF self-administered subcutaneously daily for 14 days in a dose of 125 µg/m2 beginning on the day of enrollment. Patients will be instructed in the self-administration of GM-CSF and after they have demonstrated competency with the procedure, they will self-administer the treatment at home. Patients will undergo surgery within 1 day to 5 days after cessation of the GM-CSF therapy.
Drug: GM-CSF
14 days in a dose of 125 µg/m^2
Other Name: Leukine

Standard of Care
no neo-adjuvant therapy prior to surgical intervention
Other: Standard of Care
No neo-adjuvant therapy prior to surgical intervention

Primary Outcome Measures :
  1. Th1/Th2 Normalized Gene Expression [ Time Frame: 14 days post treatment ]
    The Th1/Th2/Th3 Reverse transcription polymerase chain reaction (RT-qPCR) arrays will be used to quantify RNA expression of Th1 and Th2 messenger ribonucleic acids (mRNAs). Normalized gene expression was calculated from qRT-PCR results comparing GM-CSF and control subjects for both Th1-associated gene T-bet and Th2-associated gene GATA3 respectively. Fold change values are calculated by taking the normalized gene expression in GM-CSF treated group samples divided by the normalized gene expression in the control group samples.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria

To be eligible for the study, patients must satisfy the following criteria:

  • Histologically confirmed primary cutaneous malignant melanoma
  • 1-4mm Breslow depth
  • Scheduled for sentinel lymph node biopsy as part of their standard surgical management
  • Man or woman, age >/= 18 years
  • Women of childbearing potential (WOCBP) must be using an adequate method of contraception to avoid pregnancy throughout the study and for up to 2 weeks after the study in such a manner that the risk of pregnancy is minimized. WOCBP include any female who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy or bilateral oophorectomy) or is not postmenopausal. Sexually active WOCBP must use an effective method of birth control during the course of the study, in a manner such that risk of failure is minimized. All WOCBP must have a negative pregnancy test prior to first receiving GM-CSF.
  • Men must agree to use and utilize an adequate method of contraception throughout treatment and for at least 2 weeks after study drug is stopped
  • All patients must be willing and able to give written informed consent.

Exclusion Criteria Subjects meeting any of the following criteria are ineligible for study entry

  • Clinical stage III or IV disease
  • Autoimmune disease: Patients with a history of inflammatory bowel disease are excluded from this study as are patients with a history of immunologic disease (e.g. rheumatoid arthritis, scleroderma, systemic lupus erythematosus, autoimmune vasculitis, motor neuropathy considered of autoimmune origin)
  • Any underlying medical conditions which, in the opinion of the investigator, will make the administration of GM-CSF hazardous or obscure the interpretation of adverse events; such as, psychological, familial, sociological or geographical conditions potentially hampering compliance with the study protocol and followup schedule
  • Any vaccination therapy within 4 weeks prior to GM-CSF administration
  • Concomitant therapy with any of the following within the past 3 months: GM-CSF, interferon, other non-study immunotherapy regimes; cytotoxic chemotherapy
  • Immunosuppressive mediations (steroids, tumor necrosis factor (TNF)-inhibitors, azathioprine, etc.) within the past 6 weeks
  • Active or chronic infection with HIV, hepatitis B or hepatitis C
  • WOCBP who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and 2 weeks after cessation of the study drug.
  • Prisoners or subjects who are compulsorily detained

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02451488

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United States, Minnesota
Mayo Clinic in Rochester
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
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Principal Investigator: James Jakub, MD Mayo Clinic
  Study Documents (Full-Text)

Documents provided by James W. Jakub, Mayo Clinic:
Additional Information:
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Responsible Party: James W. Jakub, PI, Mayo Clinic Identifier: NCT02451488    
Other Study ID Numbers: 14-005510
First Posted: May 22, 2015    Key Record Dates
Results First Posted: February 10, 2020
Last Update Posted: February 10, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: data will not be shared

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Nevi and Melanomas
Immunologic Factors
Physiological Effects of Drugs