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Pembrolizumab and GM-CSF in Biliary Cancer
Phase 2 trial testing Pembrolizumab in Biliary Cancer in 42 participants. Completed in 31 December 2020.
| Lead sponsor | Robin Kate Kelley |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 42 |
| Start date | 6 May 2016 |
| Primary completion | 31 December 2020 |
| Estimated completion | 31 December 2020 |
| Sites | 1 location across United States |
Robin Kate Kelley
18 and older, any sex, with Biliary Cancer. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Proportion of subjects with measurable disease at study entry who obtained either a complete response (CR) or partial response (PR) (confirmed + unconfirmed) using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 at any time during the course of treatment.
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 0.12 | 0.04 – 0.26 |
Safety events will be summarized based on proportion of total subjects, by preferred term. Only treatment-related \>=grade 3 Adverse Events (AE)s will be reported.
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 0.095 | 0.03 – 0.23 |
PD-L1 expression will be measured by immunohistochemistry (IHC) and classified as positive or negative by central laboratory testing (QualTek Laboratories) using pre-specified cut-points; will be reported along with 95% confidence interval (CI)
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 0.36 | 0.19 – 0.56 |
Proportion of participants with PFS Time from date of first dose of protocol therapy to date of first documented radiographic and/or clinical disease progression per RECIST version 1.1 or death from any cause
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 0.26 | 0.14 – 0.42 |
Time from first documented evidence of CR or PR until the first documented sign of disease progression or death
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 294 | 144 – 1379 |
Time from first documented evidence of CR or PR until the first documented sign of disease progression or death stratified by sub-type of biliary cancer
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 294 | 144 – 1379 |
Time from date of first dose of protocol therapy to date of first documented radiographic and/or clinical disease progression per RECIST version 1.1 or death from any cause
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 63 | 55 – 125 |
Time from date of first dose of protocol therapy to date of first documented radiographic and/or clinical disease progression per RECIST version 1.1 or death from any cause stratified by sub-type of biliary cancer
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 63 | 53 – 175 |
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 58 | 53 – 151 |
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 121 | 57 – 132 |
Time from first dose of protocol therapy to the date of death due to any cause
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 393 | 243 – 573 |
Time from first dose of protocol therapy to the date of death due to any cause stratified by sub-type of biliary cancer.
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 424 | 295 – 1033 |
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 286 | 108 – 623 |
| Group | Value | 95% CI |
|---|---|---|
| Pembrolizumab and GM-CSF | 165 | 156 – 393 |
Time frame: Up to 4 years. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Pembrolizumab and GM-CSF |
|---|---|---|
| Biliary tract infection | Infections and infestations | — |
| Fever | General disorders | — |
| Sepsis | Infections and infestations | — |
| Lung Infection | Infections and infestations | — |
| Hyperglycemia | Metabolism and nutrition disorders | — |
| Hypotension | Vascular disorders | — |
| Enterocolitis | Gastrointestinal disorders | — |
| Delirium | Psychiatric disorders | — |
| Aspartate aminotransferase increased | Investigations | — |
| Alanine aminotransferase increased | Investigations | — |
| Hepatic Hemorrhage | Hepatobiliary disorders | — |
| Ascities | Gastrointestinal disorders | — |
| Bile duct stenosis | Hepatobiliary disorders | — |
| Cholangitis | Hepatobiliary disorders | — |
| Nausea | Gastrointestinal disorders | — |
| Colitis | Gastrointestinal disorders | — |
| Abdominal Distension | Gastrointestinal disorders | — |
| Dehydration | Metabolism and nutrition disorders | — |
| Abdominal Pain | Gastrointestinal disorders | — |
| Hepatobiliary disorders - Other | Hepatobiliary disorders | — |
| Hyperthyroidism | Endocrine disorders | — |
| Extraocular muscle paresis | Eye disorders | — |
| Reaction | System | Pembrolizumab and GM-CSF |
|---|---|---|
| Leukocytosis | Blood and lymphatic system disorders | — |
| Fatigue | General disorders | — |
| Diarrhea | Gastrointestinal disorders | — |
| Abdominal pain | Gastrointestinal disorders | — |
| Hypoalbuminemia | Metabolism and nutrition disorders | — |
| Fever | General disorders | — |
| Injection site reaction | General disorders | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — |
| Pruritus | Skin and subcutaneous tissue disorders | — |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — |
| Nausea | Gastrointestinal disorders | — |
| Chills | General disorders | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — |
| Ascites | Gastrointestinal disorders | — |
| Weight loss | Investigations | — |
| Anorexia | Metabolism and nutrition disorders | — |
| Upper respiratory infection | Infections and infestations | — |
| Constipation | Gastrointestinal disorders | — |
| Edema limbs | General disorders | — |
| Back pain | Musculoskeletal and connective tissue disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Bone pain | Musculoskeletal and connective tissue disorders | — |
| Hypothyroidism | Endocrine disorders | — |
| Aspartate aminotransferase increased | Investigations | — |
| Peripheral sensory neuropathy | Nervous system disorders | — |
| Blood bilirubin increased | Investigations | — |
| Creatinine increased | Investigations | — |
| Platelet count decreased | Investigations | — |
| Dry mouth | Gastrointestinal disorders | — |
| Gastrointestinal disorders - Other | Gastrointestinal disorders | — |
| General disorders and administration site conditions - Other | General disorders | — |
| Non-cardiac chest pain | General disorders | — |
| Pain in extremity | Musculoskeletal and connective tissue disorders | — |
| Dry eye | Eye disorders | — |
| Abdominal distension | Gastrointestinal disorders | — |
| Bloating | Gastrointestinal disorders | — |
| Dyspepsia | Gastrointestinal disorders | — |
| Anemia | Blood and lymphatic system disorders | — |
| Flank pain | Musculoskeletal and connective tissue disorders | — |
Most-reported serious reactions: Biliary tract infection, Fever, Sepsis, Lung Infection, Hyperglycemia, Hypotension, Enterocolitis, Delirium.
Data from ClinicalTrials.gov NCT02703714 adverse events section.
This is an open label phase II trial to examine efficacy and safety of a novel combination of pembrolizumab plus induction GM-CSF in patients with advanced biliary cancers treated at University of California, San Francisco (UCSF). This phase II study will examine the efficacy and safety of the novel combination of pembrolizumab plus induction GM-CSF in advanced biliary cancer patients with the hypotheses that the combination may increase proportion of patients with overall response compared to contemporary historical controls, with acceptable safety.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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