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Unresectable MelanomaPhase 2NCT04703426

Sargramostim (GM-CSF) + PD-1

A Phase 2 study testing Sargramostim (GM-CSF) and Pembrolizumab (anti-PD-1) in people with Unresectable Melanoma and Metastatic Melanoma, run by Dana-Farber Cancer Institute.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This research study is testing the combination of two drugs, sargramostim and pembrolizumab. The study is designed to see if the combination of these study drugs would improve the control of unresectable or metastatic melanoma cancer when compared to use of these drugs alone. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Sargramostim (GM-CSF) and Pembrolizumab (anti-PD-1)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
16 Apr 2021to 9 Jun 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologic or cytologic diagnosis of metastatic or unresectable stage III or IV cutaneous melanoma
  • Prior treatment with immunotherapy
  • Age ≥ 18 years.
  • ECOG performance status ≤2 (Karnofsky ≥60%, see Appendix A)
  • Participants must have normal organ and marrow function as defined below:
  • leukocytes ≥3,000/mcL
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  • absolute neutrophil count ≥1,500/mcL
  • platelets ≥100,000/mcL
  • total bilirubin within normal institutional limits
  • AST(SGOT)/ALT(SGPT) ≤2.5 × institutional upper limit of normal
  • creatinine within normal institutional limits OR creatinine clearance ≥60 mL/min/1.73 m2 for participants with creatinine levels above institutional normal.
  • Measurable disease (by CT, PET/CT or MRI)
  • The effects of GMCSF and PD-1 inhibition on the developing human fetus are unknown.
  • For this reason women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 7 months after completion of study drug administration.
  • Ability to understand and the willingness to sign a written informed consent document.

Who cannot join

  • Participants who have had chemotherapy or radiotherapy within 4 weeks prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier.
  • Participants who are receiving any other investigational agents.
  • Participants with known brain metastases must have documented stability over a four week interval and not be requiring active treatment for these. Prior radiation, surgery and stereotactic radiosurgery are allowed but must be completed four weeks prior to initiating therapy.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to Pembrolizumab or sargramostim.
  • Need for systemic steroids at the time of enrollment. Physiologic replacement at a dose of less than 10mg daily prednisone equivalent is allowed.
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
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  • Participants who are considered Women of Child-Bearing Potential (WOCBP) must have a negative serum pregnancy test in order to be eligible. Pregnant women are excluded from this study because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Pembrolizumab, breastfeeding should be discontinued if the mother is treated with Pembrolizumab. These potential risks may also apply to other agents used in this study.
  • Known active HIV, Hepatitis B or Hepatitis C patients. HIV-positive participants on combination antiretroviral therapy are ineligible because of the potential for an immunologic effect with the therapy. Appropriate studies will be undertaken in participants receiving combination antiretroviral therapy when indicated.
  • Autoimmune disease that requires treatment at the time of enrollment.

About this study

Official title: Copy of A Phase II Trial of Pembrolizumab (Anti PD-1) Therapy Combined With Sargramostim (GM-CSF) in Unresectable or Metastatic Melanoma

This research study is testing the combination of two drugs, sargramostim and pembrolizumab. The study is designed to see if the combination of these study drugs would improve the control of unresectable or metastatic melanoma cancer when compared to use of these drugs alone.

The names of the study drugs involved in this study are:

  • Pembrolizumab
  • Sargramostim (GM-CSF)
Read the detailed description

This is an open-label phase II study looking at safety and efficacy of the combination of pembrolizumab (PD-1 inhibition and sargramostim (GMCSF) in people with unresectable stage III or IV melanoma who may have received prior immunotherapy in the metastatic setting.

The U.S. Food and Drug Administration (FDA) has not approved sargramostim as a treatment option for people with stage III or IV melanoma The U.S. Food and Drug Administration (FDA) has approved pembrolizumab as a treatment option for people with stage III or IV melanoma who have received prior immunotherapy in the metastatic setting.

The research study procedures include screening for eligibility and study treatment including evaluations and follow up visits.

Participants will receive the study drug(s) for as long as they do not have serious side effects and their disease does not get worse and will be followed for safety 30 days after the last dose of study drug(s). Participants may also be followed for long term follow-up every 12 weeks from the last dose of study drug(s).

It is expected that about 30 people will take part in this research study.

Being tested
Sargramostim (GM-CSF), Pembrolizumab (anti-PD-1)
Keywords
Unresectable Melanoma, Metastatic Melanoma, Stage III Melanoma, Stage IV Melanoma
Medical subject headings
Melanoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Sargramostim (GM-CSF)Drug

    Also called Leukine

    Drug that binds to the protein PD-1 to help immune cells kill cancer cells better, it is given as an intravenous injection through a vein.

  • Pembrolizumab (anti-PD-1)Drug

    Also called Keytruda

    Drug that stimulates blood cells that may help support the immune system during cancer treatment, given as intravenous infusion

Study groups in NCT04703426
Study groupRoleWhat they receive
Sargramostim (GM-CSF) + Pembrolizumab (anti-PD-1)ExperimentalParticipants will receive 12 weeks of sargramostim (GM-CSF) and pembrolizumab (anti-PD-1). Participants may be pre-medicated with drugs to reduce the chance of having a sensitivity reaction to the study treatment of pembrolizumab (anti-PD-1) and sargramostim (GM-CSF). Study cycles are 21 days in length: * Pembrolizumab (anti-PD-1) will be given by intravenous infusion once on day 1 of every 21 day cycle * Sargramostim (GM-CSF) will be self-administered by participants via a subcutaneous (below the skin) injection daily for days 1 - 14 of each 21- day cycle. Participants will be assessed at 12 weeks for disease response/progression and further study treatment.
Receives: Drug: Sargramostim (GM-CSF); Drug: Pembrolizumab (anti-PD-1)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Response Rate (ORR)

    The primary study endpoint is response rate per RECIST criteria.

    Measured: 12 weeks

Secondary outcomes (5)

  1. Number of Participants With Treatment-Related Adverse Events

    Number and proportion of adverse events, graded as defined by CTCAE version 5.0

    Measured: 12 weeks

  2. Overall Survival Rate

    Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) will be used.

    Measured: 12 weeks

  3. Progression Free Survival Rate

    Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) will be used.

    Measured: 12 weeks

  4. Overall Response Rate (ORR)-irRC

    To evaluate the overall response rate in advanced melanoma to combination anti- PD-1 therapy and sargramostim by irRC criteria.

    Measured: 12 weeks

  5. CD4+ ICOS T cell changes

    Evaluate changes in CD4+ ICOS T cells from biopsies (pre-treatment, on-treatment, post-treatment) and correlate using Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1)

    Measured: 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Jan 2021
  2. Study start16 Apr 2021
  3. Primary completion10 Jun 2024 (estimated)
  4. Study completion9 Jun 2025 (estimated)

Why it stopped: Study terminated by sponsor

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04703426
Sponsor's protocol ID
20-370
Status last verified
May 2023
First submitted
7 Jan 2021
Last updated
22 May 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sargramostim (GM-CSF) and Pembrolizumab (anti-PD-1) in people with Unresectable Melanoma and Metastatic Melanoma, run by Dana-Farber Cancer Institute.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 22 May 2023. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (ORR) (time frame: 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04703426.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2023.

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