Pelabresib and liver function in adults with advanced cancer
A Phase 1b, Open-Label, Multicenter Study to Evaluate the Pharmacokinetic Profile of Pelabresib (DAK539/CPI-0610) in Patients With Advanced Malignancies and Hepatic Impairment
This tests how pelabresib is processed in the body at steady state in adults with advanced cancer who have normal or moderate-to-severe liver impairment.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | Novartis Industry-sponsored |
| Drugs / interventions | chemotherapy |
| Locations | 1 site (London) |
| Trial ID | NCT07422610 on ClinicalTrials.gov |
What this trial studies
The study compares pelabresib pharmacokinetics at steady state between participants with normal hepatic function and those with moderate or severe hepatic impairment. Part 1 enrolls matched groups to measure drug exposure and safety without requiring treatment-free washout periods. After Part 1, participants who show clinical benefit may continue pelabresib in Part 2 until discontinuation criteria or end of study. Key eligibility includes adults 18–75 with advanced malignancies lacking standard curative options and adequate laboratory values.
Who should consider this trial
Good fit: Adults aged 18–75 with advanced malignancies who have either normal liver function or moderate-to-severe hepatic impairment and who meet the study's lab and clinical entry criteria are ideal candidates.
Not a fit: Patients who have available standard curative treatments, who do not meet the laboratory or performance criteria, or who have comorbidities that preclude pelabresib use are unlikely to benefit from participation.
Why it matters
Potential benefit: If successful, the results could guide safer, evidence-based dosing of pelabresib for patients with liver impairment.
How similar studies have performed: Other pharmacokinetic studies of BET inhibitors and earlier pelabresib trials have characterized exposure changes across patient groups, but steady-state hepatic impairment data for pelabresib are limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria:
* Is at least 18 and not older than 75 years of age at the time of signing the informed consent.
* Group 1 only: There is a matching participant in Group 2 and an enrollment slot is available for the Group 1 participant.
* Has a confirmed documented diagnosis of an advanced malignancy for which no standard and/or curative treatment options are available.
* Has the following acceptable laboratory assessments prior to the first dose of study treatment:
1. Platelet count ≥ 150 × 109 /L in the absence of thrombopoietic factors or transfusions within 2 weeks of the screening assessment
2. Absolute neutrophil count (ANC) ≥ 1 × 109 /L in the absence of granulocyte growth factors
3. Adequate renal function (creatinine clearance of ≥30 mL/min, calculated using Cockcroft-Gault formula)
4. Peripheral blood blast count \< 5%. Assessment of blasts in bone marrow is not mandatory at screening, however, blasts must be \<5% if the assessment is performed.
* Has a life expectancy ≥3 months.
* Has fully recovered from major surgery and from the acute toxic effects of prior chemotherapy and radiotherapy .
* Hepatic function:
1. Is in Group 1 and is classified as having normal hepatic function based on NCI-ODWG criteria (i.e., total bilirubin ≤ upper limit of normal (ULN) and aspartate aminotransferase (AST) ≤ ULN); or
2. Is in Group 2 and has stable moderate or severe HI as defined by NCI ODWG criteria:
* moderate HI: total bilirubin \>1.5 × to 3 × ULN, and any AST value
* severe HI: total bilirubin \> 3 × ULN, and any AST value
Key Exclusion Criteria:
* Has a history of hypersensitivity to the study treatment or its excipients or to drugs of similar chemical class.
* Has any other medical condition which, in the investigator's opinion, makes the participant unsuitable for the study.
* Is a female participant who is pregnant (confirmed by a pregnancy test at screening) or is breastfeeding.
* Is a woman of childbearing potential (WOCBP) who does not agree to follow the contraceptive guidance during the treatment period and for at least 184 days after the last dose of study treatment, and who does not agree to refrain from donating eggs during this period.
* Has esophageal variceal bleeding within the past 2 months prior to the first dose of pelabresib.
* Has an active clinically significant infection.
* Has impaired cardiac function or clinically significant cardiac diseases
* Has a GI tumor, impaired GI function, GI disease, or significant resection of stomach or other portion of the GI tract that could alter the absorption of pelabresib, including any unresolved nausea, vomiting, or diarrhea.
Other protocol-defined inclusion/exclusion criteria may apply.
Where this trial is running
London
- Novartis Investigative Site — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Novartis Pharmaceuticals
- Email: novartis.email@novartis.com
- Phone: +41613241111
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.