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

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ClinicalTrials.gov Identifier: NCT02451488
Recruitment Status : Completed
First Posted : May 22, 2015
Results First Posted : February 10, 2020
Last Update Posted : February 10, 2020
Sponsor:
Information provided by (Responsible Party):
James W. Jakub, Mayo Clinic

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Melanoma
Interventions Drug: GM-CSF
Other: Standard of Care
Enrollment 8
Recruitment Details  
Pre-assignment Details  
Arm/Group Title GM-CSF Standard of Care
Hide Arm/Group Description

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.

GM-CSF: 14 days in a dose of 125 µg/m^2

no neo-adjuvant therapy prior to surgical intervention

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

Period Title: Overall Study
Started 4 4
Completed 4 4
Not Completed 0 0
Arm/Group Title GM-CSF Standard of Care Total
Hide Arm/Group Description

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.

GM-CSF: 14 days in a dose of 125 µg/m^2

no neo-adjuvant therapy prior to surgical intervention

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

Total of all reporting groups
Overall Number of Baseline Participants 4 4 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 4 participants 8 participants
54.5  (14.3) 62.7  (18.8) 58.6  (16.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 8 participants
Female
2
  50.0%
2
  50.0%
4
  50.0%
Male
2
  50.0%
2
  50.0%
4
  50.0%
Race and Ethnicity Not Collected   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 0 participants 0 participants
0
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 4 participants 4 participants 8 participants
4 4 8
1.Primary Outcome
Title Th1/Th2 Normalized Gene Expression
Hide Description 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.
Time Frame 14 days post treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Three subjects were in in the GM-CSF group and three subjects were in the Standard of Care group. One subject in the GM-CSF group received GM-CSF but did not have tissue sample collected for research analysis. One subject in the Standard of Care group did not have a large enough tissue sample collected to be analyzed.
Arm/Group Title GM-CSF Standard of Care
Hide Arm/Group Description:

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.

GM-CSF: 14 days in a dose of 125 µg/m^2

no neo-adjuvant therapy prior to surgical intervention Standard of Care: No neo-adjuvant therapy prior to surgical intervention
Overall Number of Participants Analyzed 3 3
Mean (95% Confidence Interval)
Unit of Measure: fold change
T-bet (Th1)
1.029
(0.999 to 1.059)
0.119
(-1.506 to 1.654)
GATA3 (Th2)
0.074
(-0.041 to 0.279)
0.192
(0.068 to 0.316)
Time Frame 14 days
Adverse Event Reporting Description Subjects receiving intervention will have adverse events (AEs) recorded during the 14 day course of self-administrated GM-CSF. Subjects will be provided a diary to record any AEs. Subjects will be provided a list of AEs and to call immediately for any grade 3 or high AEs. Grade I-II AEs will be logged into their diary. Diary will be collected the day of surgery. All-Cause Mortality, Serious, and Other (Not Including Serious) AEs were not monitored/assessed for the Standard of Care Arm/Group.
 
Arm/Group Title GM-CSF Standard of Care
Hide Arm/Group Description

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.

GM-CSF: 14 days in a dose of 125 µg/m^2

no neo-adjuvant therapy prior to surgical intervention

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

All-Cause Mortality
GM-CSF Standard of Care
Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/0 
Hide Serious Adverse Events
GM-CSF Standard of Care
Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/0 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
GM-CSF Standard of Care
Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/0 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. James Jakub
Organization: Mayo Clinic
Phone: 507-284-3629
EMail: Jakub.James@mayo.edu
Layout table for additonal information
Responsible Party: James W. Jakub, Mayo Clinic
ClinicalTrials.gov Identifier: NCT02451488    
Other Study ID Numbers: 14-005510
First Submitted: May 18, 2015
First Posted: May 22, 2015
Results First Submitted: December 15, 2019
Results First Posted: February 10, 2020
Last Update Posted: February 10, 2020