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

Phase 4

Completed

• Conditions: Melanoma
• Interventions: Other: Standard of Care; Drug: GM-CSF

• Registration Number: NCT02451488
• Lead Sponsor: Mayo Clinic

Brief Summary

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

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.

Study Timeline

• Study Start: 2015-05
• Study First Submitted: 2015-05-18
• Study First Submitted QC: 2015-05-19
• Study First Posted: 2015-05-22
• Primary Completion: 2016-11-04
• Study Completion: 2016-11-04
• Results First Submitted: 2019-12-15
• Status Verified: 2020-01
• Results First Submitted QC: 2020-01-29
• Last Update Submitted: 2020-01-29
• Results First Posted: 2020-02-10
• Last Update Posted: 2020-02-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Standard of Care	Standard of Care	no neo-adjuvant therapy prior to surgical intervention
GM-CSF	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.

Primary Outcome Measures

Name	Time	Method
Th1/Th2 Normalized Gene Expression	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.

Trial Locations

Locations (1)

Mayo Clinic in Rochester; 🇺🇸 Rochester, Minnesota, United States