Testing the Combination of Pevonedistat With Chemotherapy for Bile Duct Cancer of the Liver
Active, enrolledPhase 2Results postedLast updated 17 November 2025
What this trial tests
Phase 2 trial testing Carboplatin in Metastatic Cholangiocarcinoma in 40 participants. Participants enrolled and being followed up; not accepting new ones.
18 and older, any sex, with Metastatic Cholangiocarcinoma or Metastatic Hepatocellular Carcinoma. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Objective ResponsePrimary· Every 3 months for the first year and every 6 months for years 2-3
Response was evaluated using the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1). Objective response includes complete response (CR) and partial response (PR).
CR is defined as disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \< 10 mm.
PR is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.
Group
Value
95% CI
Arm A (Pevonedistat)
0
Arm B (Pevonedistat, Paclitaxel, Carboplatin)
0
Arm A (Pevonedistat)
17
Arm B (Pevonedistat, Paclitaxel, Carboplatin)
17
Clinical BenefitSecondary· Every 3 months for the first year and every 6 months for years 2-3
Clinical benefit was evaluated using the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1), including complete response (CR), partial response (PR) and stable disease (SD) that lasted greater than or equal to 24 weeks.
CR is defined as disappearance of all target and non-target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \< 10 mm.
PR is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.
SD is defined as neither suffi
Group
Value
95% CI
Arm A (Pevonedistat)
0
Arm B (Pevonedistat, Paclitaxel, Carboplatin)
1
Arm A (Pevonedistat)
17
Arm B (Pevonedistat, Paclitaxel, Carboplatin)
16
Progression-free Survival (PFS)Secondary· Every 3 months for the first year and every 6 months for years 2-3
PFS was defined as the time from randomization to progression or death, whichever came first; patients alive without evidence of disease progression were censored at the date of last disease assessment. Patients who died without documented progression and the death occurred \> 6 weeks of last disease assessment will be censored at the date of last disease assessment that showed progression-free.
Progression was evaluated using the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1), defined as at least a 20% increase in the sum of the diameters of target lesi
Group
Value
95% CI
Arm A (Pevonedistat)
1.54
1.41 – 2.10
Arm B (Pevonedistat, Paclitaxel, Carboplatin)
2.92
1.71 – 5.55
Overall Survival (OS)Secondary· Every 3 months for the first year and every 6 months for years 2-3
OS was defined as the time from randomization to death from any cause, and patients still living were censored at the date last known alive.
Group
Value
95% CI
Arm A (Pevonedistat)
4.80
4.04 – 12.50
Arm B (Pevonedistat, Paclitaxel, Carboplatin)
6.54
4.20 – 14.80
Adverse events — posted to ClinicalTrials.gov
Time frame: Assessed every 3 weeks while on treatment and for 30 days after the end of treatment, up to 3 years.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Arm A (Pevonedistat)
Serious: 8/17 (47%)
Deaths: 17/17
Arm B (Pevonedistat, Paclitaxel, Carboplatin)
Serious: 12/17 (71%)
Deaths: 15/17
Serious adverse events (41 terms)
Reaction
System
Arm A (Pevonedistat)
Arm B (Pevonedistat, Pacli…
Febrile neutropenia
Blood and lymphatic system disorders
—
—
Fever
General disorders
—
—
Abdominal pain
Gastrointestinal disorders
—
—
Nausea
Gastrointestinal disorders
—
—
Cholecystitis
Hepatobiliary disorders
—
—
Sepsis
Infections and infestations
—
—
Acute kidney injury
Renal and urinary disorders
—
—
Hypertension
Vascular disorders
—
—
Anemia
Blood and lymphatic system disorders
—
—
Death NOS
General disorders
—
—
Fatigue
General disorders
—
—
Disease progression
General disorders
—
—
Abdominal distension
Gastrointestinal disorders
—
—
Ascites
Gastrointestinal disorders
—
—
Colonic perforation
Gastrointestinal disorders
—
—
Constipation
Gastrointestinal disorders
—
—
Gastroparesis
Gastrointestinal disorders
—
—
Ileus
Gastrointestinal disorders
—
—
Vomiting
Gastrointestinal disorders
—
—
Hepatobiliary disorders - Other, specify
Hepatobiliary disorders
—
—
Biliary tract infection
Infections and infestations
—
—
Hepatic infection
Infections and infestations
—
—
Infections and infestations - Other, specify
Infections and infestations
—
—
INR increased
Investigations
—
—
Neutrophil count decreased
Investigations
—
—
Other adverse events (135 terms — click to expand)
This phase II trial studies how well pevonedistat alone or in combination with chemotherapy (paclitaxel and carboplatin) works in treating patients with bile duct cancer of the liver. Pevonedistat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as paclitaxel and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This study may help the study doctors find out how well pevonedistat shrinks bile duct cancer of the liver when given alone and when in combination with paclitaxel and carboplatin.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04832438 — 9-ING-41 Plus Carboplatin in Patients With Advanced, Metastatic Salivary Gland Carcinoma
· Phase 2
· withdrawn
NCT07229339 — Zipalertinib With Carboplatin and Pemetrexed for the Treatment of Resectable, Stage II-IIIB, Non-Small Cell Lung Cancer
· Phase 2
· not yet recruiting
NCT07346196 — A Trial of Locoregionally Advanced Squamous Cell Carcinoma of The Head and Neck
· Phase 2
· not yet recruiting
NCT07441681 — Comparing Radiation Plus Cetuximab to Radiation Plus Chemotherapy in People With Head and Neck Cancer Who Cannot Receive
· Phase 3
· not yet recruiting
NCT07281417 — Testing the Addition of Cemiplimab (REGN2810) to Chemotherapy Treatment Given Prior to Surgery in Patients With Sinonasa
· Phase 2
· recruiting
Other recruiting trials for Metastatic Cholangiocarcinoma
Currently open trials in the same condition.
NCT06178588 — Sacituzumab Govitecan for the Treatment for Patients With Locally Advanced, Recurrent, or Metastatic Cholangiocarcinoma
· Phase 2
· active not recruiting
NCT04068194 — Testing the Combination of New Anti-cancer Drug Peposertib With Avelumab and Radiation Therapy for Advanced/Metastatic S
· Phase 1, PHASE2
· active not recruiting
Other National Cancer Institute (NCI) trials
Trials by the same sponsor.
NCT07147231 — Testing the Effectiveness of the Anti-cancer Drug Pidnarulex (CX-5461), in Combination With Another Anti-cancer Drug Cem
· Phase 1, PHASE2
· recruiting
NCT07572123 — Evaluating the Addition of Maintenance Immunotherapy Compared to the Usual Treatment of Chemotherapy and Autologous Stem
· Phase 2, PHASE3
· not yet recruiting
NCT07281417 — Testing the Addition of Cemiplimab (REGN2810) to Chemotherapy Treatment Given Prior to Surgery in Patients With Sinonasa
· Phase 2
· recruiting
NCT07012044 — A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After
· Phase 1
· not yet recruiting
NCT07437950 — Comparing Different Treatment Lengths for Venetoclax in Older People With Newly Diagnosed Acute Myeloid Leukemia (A Myel
· Phase 2
· not yet recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Cancer Institute (NCI)
Last refreshed: 17 November 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04175912.