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TerminatedNCT03407170Updated Jul 15, 2020Results posted

Immunologic Determinants of Response to Pembrolizumab (MK-3475) in Advanced Melanoma (MK-3475-161/KEYNOTE-161)

A Phase 2 interventional study of pembrolizumab in Advanced Melanoma, sponsored by Merck Sharp & Dohme LLC. Terminated at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-07-15.

Sponsored by Merck Sharp & Dohme LLC · Phase 2, Interventional, and Treatment

Why this study was terminated
Lack of Accrual
Phase
Phase 2
Study type
Interventional
Enrollment
1
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

In this study, participants with advanced melanoma will be treated with pembrolizumab (MK-3475) and their tumors and blood will be analyzed for changes related to pembrolizumab therapy.

The primary hypotheses are that participants who respond to pembrolizumab have:

  1. a higher fraction of cytotoxic tumor-infiltrating T-lymphocytes (FCT) at baseline compared to those who do not respond to pembrolizumab
  2. a higher fold-increase in FCT compared to baseline than those who do not respond to pembrolizumab
  3. a higher Average Specific Cytotoxic T-lymphocyte Frequency Ratio (ASCTFR) compared to those who do not respond to pembrolizumab
02

Conditions studied

  • Advanced Melanoma

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Keywords

  • Programmed Cell Death-1 (PD-1)
  • PD1
  • Programmed Cell Death-Ligand 1 (PD-L1)
  • Programmed Cell Death-Ligand 2 (PD-L2)
  • PDL1
  • PDL2
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Has a histologically confirmed diagnosis of unresectable stage III or metastatic melanoma not amenable to local therapy
  • Has testing for a BRAF mutation prior to study entry
  • Has measurable disease per RECIST 1.1 as assessed by the investigator/radiology
  • Has resolution of toxic effect(s) of the most recent prior chemotherapy to Grade 1 or less (except alopecia). If the participant received major surgery or radiation therapy of >30 Gray units, they must have recovered from the toxicity and/or complications from the intervention
  • Has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 within 10 days of study start
  • A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies: is not a woman of childbearing potential (WOCBP) OR is a WOCBP and agrees to use a contraceptive method, consistent with local regulations regarding the methods of contraception for those participating in clinical studies, during the treatment period and for at least 120 days after the last dose of study treatment
  • A male participant is eligible to participate if he agrees not to donate sperm PLUS to be abstinent from heterosexual intercourse as their preferred and usual lifestyle OR agree to use contraception, consistent with local regulations regarding the methods of contraception for those participating in clinical studies, during the treatment period and for at least 120 days after the last dose of study treatment

Exclusion criteria

Exclusion Criteria:

  • Has disease that is suitable for local therapy administered with curative intent
  • Has a history of interstitial lung disease
  • Has a positive pregnancy test within 72 hours before the first dose of study therapy
  • Has received prior therapy with an anti-Programmed Cell Death Protein 1 (PD-1), anti-Programmed Cell Death-Ligand 1 (PD-L1), anti-Programmed Cell Death-Ligand 2 (PD-L2) agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (eg, cytotoxic T-lymphocyte-associated protein 4 [CTLA-4], OX-40, CD137)
  • Has received prior systemic anti-cancer therapy including investigational agents within 4 weeks prior to study start
  • Has received prior radiotherapy within 2 weeks of start of study therapy
  • Has received a live vaccine within 30 days prior to the first dose of study therapy
  • Has received transfusion of blood products (including platelets or red blood cells) or administration of colony stimulating factors (including granulocyte colony stimulating factor [G-CSF], granulocyte macrophage colony stimulating factor [GMCSF] or recombinant erythropoietin) within 4 weeks prior to study start
  • Is currently participating in or has participated in a study of an investigational agent or has used an investigational device within 4 weeks prior to the first dose of study therapy
  • Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior the first dose of study therapy
  • Has a known additional malignancy that is progressing or has required active treatment within the past 3 years with the exception of basal cell and squamous cell carcinoma of the skin, transitional cell carcinoma of urothelial cancer, or carcinoma in situ (eg, breast carcinoma, cervical cancer in situ) that have undergone potentially curative therapy
  • Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis
  • Has severe hypersensitivity (≥Grade 3) to pembrolizumab and/or any of its excipients
  • Has an active autoimmune disease that has required systemic treatment in past 2 years
  • Has a history of (non-infectious) pneumonitis that required steroids or has current pneumonitis
  • Has an active infection requiring systemic therapy
  • Has a known history of human immunodeficiency virus (HIV) infection
  • Has a known history of Hepatitis B (defined as Hepatitis B surface antigen [HBsAg] reactive) or known active Hepatitis C virus (defined as HCV RNA [qualitative] is detected) infection
  • Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the participant's participation for the full duration of the study, or is not in the best interest of the participant to participate, in the opinion of the treating investigator
  • Has known psychiatric or substance abuse disorders that would interfere with cooperating with the requirements of the study
  • Is pregnant or breastfeeding or expecting to conceive or father children starting with the screening visit through 120 days after the last dose of study therapy
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
1 participant (actual)

Study arms

  • Experimental
    Pembrolizumab

    Participants receive pembrolizumab 200 mg by intravenous (IV) infusion every 3 weeks (Q3W) for up to 24 months

    Biological: pembrolizumab

Interventions

  • Biologicalpembrolizumab

    200 mg by IV infusion Q3W for up to 24 months

    Also known as: KEYTRUDA®, MK-3475

05

What researchers measure

Primary outcomes

  1. Mean Fraction of Cytotoxic T-lymphocytes (FCT) for Participants With Response Versus Participants With Progression

    FCT is defined as the fraction of CD8+ T-cells expressing a predefined single-cell ribonucleic acid (RNA) gene signature to the total tumor infiltrating CD8+T-cells isolated from tumor biopsies.

    Time frame: Up to approximately 59 weeks

  2. Average Specific Cytotoxic T-lymphocyte Frequency Ratio (ASCTFR) for Participants With Response Versus Participants With Progression

    ASCTFR is defined as the arithmetic average of the log10 ratio of the frequency of individual specific cytotoxic T-Cell Receptor (TCR) clones of on-treatment to pre-treatment.

    Time frame: Up to approximately 59 weeks

  3. Change in Baseline of Fraction of Cytotoxic T-lymphocytes (FCT) for Participants With Response Versus Participants With Progression

    The fold change from baseline in FCT. FCT is defined as the fraction of CD8+ T-cells expressing a predefined single-cell RNA gene signature to the total tumor infiltrating CD8+T-cells isolated from tumor biopsies.

    Time frame: Up to approximately 59 weeks

Secondary outcomes

  1. Number of Participants Who Experienced an Adverse Event (AE)

    An AE is any untoward medical occurrence in a participant temporally associated with the use of study treatment, whether or not considered related to the study treatment. The number of participants who experienced an AE is presented.

    Time frame: Up to approximately 59 weeks

  2. Number of Participants Who Discontinued Any Study Drug Due to an Adverse Event (AE)

    The number of participants who discontinued any study treatment due to an AE is presented.

    Time frame: Up to approximately 59 weeks

  3. Overall Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

    ORR is defined as the percentage of participants in the analysis population who have a Complete Response (CR: Disappearance of all target lesions) or a Partial Response (PR: ≥30% decrease in the sum of diameters of target lesions) per RECIST Version 1.1 as assessed by the investigator.

    Time frame: Up to approximately 59 weeks

  4. Progression-free Survival (PFS) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

    PFS is defined as the time from start of treatment to the first documented progressive disease (PD) or death due to any cause, whichever occurs first, per RECIST Version 1.1 as assessed by the investigator.

    Time frame: Up to approximately 59 weeks

  5. Overall Survival (OS)

    OS is defined as the time from the start of treatment to death due to any cause.

    Time frame: Up to approximately 59 weeks

  6. Neoepitope Burden

    Neoepitope sequencing will be generated based on single cell ribonucleic acid (RNA) sequencing (scRNAseq), whole exome sequencing, and an epitope prediction algorithm to obtain neoepitope burden.

    Time frame: Up to approximately 59 weeks

  7. Antigenic Determinants of Highly-functional CD8 + T-cell Clones

    T-cell receptors (TCRs) from CD8+ T-cell clones will be identified by single cell ribonucleic acid (RNA) sequencing (scRNAseq) and their killing function will be confirmed by TCR-transduced T-cells recognizing autologous tumor-derived cell lines.

    Time frame: Up to approximately 59 weeks

06

Results

Posted Jul 15, 2020
Limitations and caveats
Study was stopped early due to lack of accrual.

Participant flow

Participant flow — Overall Study
MilestonePembrolizumab
Started1
Completed0
Not completed1
Withdrew: Adverse event1

Outcome measures

PrimaryMean Fraction of Cytotoxic T-lymphocytes (FCT) for Participants With Response Versus Participants With Progression

FCT is defined as the fraction of CD8+ T-cells expressing a predefined single-cell ribonucleic acid (RNA) gene signature to the total tumor infiltrating CD8+T-cells isolated from tumor biopsies.

Time frame:
Up to approximately 59 weeks

No measurements were reported for this outcome.

PrimaryAverage Specific Cytotoxic T-lymphocyte Frequency Ratio (ASCTFR) for Participants With Response Versus Participants With Progression

ASCTFR is defined as the arithmetic average of the log10 ratio of the frequency of individual specific cytotoxic T-Cell Receptor (TCR) clones of on-treatment to pre-treatment.

Time frame:
Up to approximately 59 weeks

No measurements were reported for this outcome.

PrimaryChange in Baseline of Fraction of Cytotoxic T-lymphocytes (FCT) for Participants With Response Versus Participants With Progression

The fold change from baseline in FCT. FCT is defined as the fraction of CD8+ T-cells expressing a predefined single-cell RNA gene signature to the total tumor infiltrating CD8+T-cells isolated from tumor biopsies.

Time frame:
Up to approximately 59 weeks

No measurements were reported for this outcome.

SecondaryNumber of Participants Who Experienced an Adverse Event (AE)

An AE is any untoward medical occurrence in a participant temporally associated with the use of study treatment, whether or not considered related to the study treatment. The number of participants who experienced an AE is presented.

Time frame:
Up to approximately 59 weeks
Reported as:
Count of participants · Participants
Number of Participants Who Experienced an Adverse Event (AE)
ParticipantsPembrolizumab
Number of Participants Who Experienced an Adverse Event (AE)1
SecondaryNumber of Participants Who Discontinued Any Study Drug Due to an Adverse Event (AE)

The number of participants who discontinued any study treatment due to an AE is presented.

Time frame:
Up to approximately 59 weeks
Reported as:
Count of participants · Participants
Number of Participants Who Discontinued Any Study Drug Due to an Adverse Event (AE)
ParticipantsPembrolizumab
Number of Participants Who Discontinued Any Study Drug Due to an Adverse Event (AE)1
SecondaryOverall Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

ORR is defined as the percentage of participants in the analysis population who have a Complete Response (CR: Disappearance of all target lesions) or a Partial Response (PR: ≥30% decrease in the sum of diameters of target lesions) per RECIST Version 1.1 as assessed by the investigator.

Time frame:
Up to approximately 59 weeks

No measurements were reported for this outcome.

SecondaryProgression-free Survival (PFS) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

PFS is defined as the time from start of treatment to the first documented progressive disease (PD) or death due to any cause, whichever occurs first, per RECIST Version 1.1 as assessed by the investigator.

Time frame:
Up to approximately 59 weeks

No measurements were reported for this outcome.

SecondaryOverall Survival (OS)

OS is defined as the time from the start of treatment to death due to any cause.

Time frame:
Up to approximately 59 weeks

No measurements were reported for this outcome.

SecondaryNeoepitope Burden

Neoepitope sequencing will be generated based on single cell ribonucleic acid (RNA) sequencing (scRNAseq), whole exome sequencing, and an epitope prediction algorithm to obtain neoepitope burden.

Time frame:
Up to approximately 59 weeks

No measurements were reported for this outcome.

SecondaryAntigenic Determinants of Highly-functional CD8 + T-cell Clones

T-cell receptors (TCRs) from CD8+ T-cell clones will be identified by single cell ribonucleic acid (RNA) sequencing (scRNAseq) and their killing function will be confirmed by TCR-transduced T-cells recognizing autologous tumor-derived cell lines.

Time frame:
Up to approximately 59 weeks

No measurements were reported for this outcome.

Adverse events

Collected over Up to approximately 59 weeks. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Pembrolizumab———

Baseline characteristics

Zero participants are reported due to the risk of identification of a person.

Age, Categorical
Age, Categorical(Participants)Pembrolizumab
<=18 years—
Between 18 and 65 years—
>=65 years—
Sex: Female, Male
Sex: Female, Male(Participants)Pembrolizumab
Female—
Male—
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Pembrolizumab
Hispanic or Latino—
Not Hispanic or Latino—
Unknown or Not Reported—
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Pembrolizumab
American Indian or Alaska Native—
Asian—
Native Hawaiian or Other Pacific Islander—
Black or African American—
White—
More than one race—
Unknown or Not Reported—
07

Study locations

2 sites
  • Massachusetts General Hospital ( Site 0102)
    Boston, Massachusetts 02114, United States
  • Dana Farber Cancer Institute ( Site 0101)
    Boston, Massachusetts 02215, United States
08

References and documents

Study documents

  • Protocol and statistical analysis plan · Jun 14, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf

09

Registry details

Key details

Study ID
NCT03407170
Lead sponsor
Merck Sharp & Dohme LLC
Responsible party
Sponsor
First posted
Jan 23, 2018
Start date
Jun 28, 2018
Primary completion
Aug 14, 2019
Completion
Aug 14, 2019
Results posted
Jul 15, 2020
Last update
Jul 15, 2020

Study contacts

Medical Director
study director · Merck Sharp & Dohme LLC

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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