Back to results

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

Primary Purpose

Melanoma

Status

Completed

Phase

Phase 4

Locations

United States

Study Type

Interventional

Intervention

GM-CSF

Standard of Care

About this trial

This is an interventional treatment trial for Melanoma

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Mayo Clinic in Rochester

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Other

Arm Label

GM-CSF

Standard of Care

Arm 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.

no neo-adjuvant therapy prior to surgical intervention

Outcomes

Primary Outcome Measures

Th1/Th2 Normalized Gene Expression

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT02451488

First Posted

May 18, 2015

Last Updated

January 29, 2020

Sponsor

Mayo Clinic

1. Study Identification

Unique Protocol Identification Number

NCT02451488

Brief Title

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

Official Title

Neoadjuvant GM-CSF Treatment and Modulation of Immune Cell Profile of the SLN in Melanoma

Study Type

Interventional

2. Study Status

Record Verification Date

January 2020

Overall Recruitment Status

Completed

Study Start Date

May 2015 (undefined)

Primary Completion Date

November 4, 2016 (Actual)

Study Completion Date

November 4, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Mayo Clinic

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Melanoma

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

8 (Actual)

8. Arms, Groups, and Interventions

Arm Title

GM-CSF

Arm Type

Experimental

Arm Description

Arm Title

Standard of Care

Arm Type

Other

Arm Description

no neo-adjuvant therapy prior to surgical intervention

Intervention Type

Drug

Intervention Name(s)

GM-CSF

Other Intervention Name(s)

Leukine

Intervention Description

14 days in a dose of 125 µg/m^2

Intervention Type

Other

Intervention Name(s)

Standard of Care

Intervention Description

No neo-adjuvant therapy prior to surgical intervention

Primary Outcome Measure Information:

Title

Th1/Th2 Normalized Gene Expression

Description

Time Frame

14 days post treatment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

James Jakub, MD

Organizational Affiliation

Mayo Clinic

Official's Role

Principal Investigator

Facility Information:

Facility Name

Mayo Clinic in Rochester

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing Plan Description

data will not be shared

Links:

URL

https://www.mayo.edu/research/clinical-trials

Description

Mayo Clinic Clinical Trials

Learn more about this trial

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

We'll reach out to this number within 24 hrs