A Phase 1 interventional study of pelabresib in Advanced Malignancies and Hepatic Impairment, sponsored by Novartis Pharmaceuticals. Recruiting at 10 sites in 4 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-18.
Sponsored by Novartis Pharmaceuticals · Phase 1, Interventional, and Treatment
The primary purpose of this study is to evaluate the impact of hepatic function on the pharmacokinetic (PK) profile of pelabresib in participants with advanced malignancies who have either hepatic impairment (HI) or normal liver function. To reduce participant burden and maximize benefit, the PK of pelabresib will be assessed at steady-state rather than after a single dose, avoiding treatment-free washout periods.
This study consists of 2 main parts: Part 1 will assess the PK characteristics of pelabresib in participants with hepatic impairment versus normal hepatic function, and during Part 2, extended treatment with pelabresib may be offered in case of clinical benefit, as assessed by the investigator.
A participant is considered to have started the study and entered the screening period upon signing the informed consent form (ICF). Enrollment occurs when participant receive their first dose of study treatment via the IRT system. A participant is considered to have completed the study if they have completed all phases of the study including the end of treatment (EOT) and 30-day safety follow-up visits. Participants with hematological malignancies will be followed up every 3 months beyond EOT.
Part 1: Impact of hepatic impairment on pelabresib PK
In Part 1, the PK characteristics of pelabresib will be investigated in participants with advanced malignancies comprising 2 different study groups according to their hepatic function:
Following completion of Part 1, participants can directly proceed to Part 2
Part 2: Continued treatment After completing Part 1, in case of clinical benefit, participants can continue with pelabresib treatment in Part 2 until the end of study (EOS) is reached, until the participant meets discontinuation criteria, or until they receive pelabresib treatment via alternative means of access, whichever occurs first. Clinical benefit is determined by the investigator.
End of treatment visit An EOT visit is required for all participants within 7 days of the last dose of pelabresib treatment (or within 7 days of the decision to discontinue treatment, if the decision was made > 7 days after the last dose).
30-day safety follow-up All participants will be followed up for AEs and serious AEs (SAEs) for 30 days (±3 days) following the last dose of pelabresib. If the participant starts another anticancer treatment or switches to pelabresib treatment via an alternative source (e.g., in an extension study or commercially available pelabresib), the safety follow-up will end at the time of the first administration of the respective treatment.
Leukemic transformation follow-up for participants with hematological malignancies After the EOT visit and the 30-Day safety follow-up visit, participants with hematological malignancies will be followed up every 3 months for leukemic transformation until the overall end of the study, confirmation of AML, withdrawal of consent, loss to follow-up, or death, whichever occurs first.
931 studies on the registry are indexed under Lymphoma, Follicular; 237 are open to participants now.
This study's planned enrollment of 24 is below the median of 48 across 797 interventional studies indexed under Lymphoma, Follicular.
Browse Lymphoma, Follicular studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Has the following acceptable laboratory assessments prior to the first dose of study treatment:
Hepatic function:
Is in Group 2 and has stable moderate or severe HI as defined by NCI ODWG criteria:
Key Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
Part 1: Participants receive pelabresib 125 mg orally (PO) once daily (QD) for 14 days, followed by a 7-day break (1 cycle = 21 days). If clinical benefit is observed, treatment continues in Part 2 until end of study (EOS), discontinuation criteria, or alternative access.
Drug: pelabresib
Part 1: Participants receive pelabresib 125 mg orally (PO) once daily (QD) for 14 days, followed by a 7-day break (1 cycle = 21 days). If clinical benefit is observed, treatment continues in Part 2 until end of study (EOS), discontinuation criteria, or alternative access.
Drug: pelabresib
pelabresib 125 mg orally (PO) once daily (QD) for 14 days, followed by a 7-day break
Also known as: DAK539 (formerly CPI-0610)
Area Under the Curve from 0 to 24 hours on Day 14 (AUC₀-24h,D14) of pelabresib at steady state per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. AUC₀-24h,D14 of pelabresib at steady state per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14: 0, 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (1 cycle = 21 days)
Maximum Plasma Concentration on Day 14 (Cmax,D14) of pelabresib at steady state per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. Cmax,D14 of pelabresib at steady state per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (1 cycle = 21 days)
Apparent Clearance (CL/F) of pelabresib at steady state per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. CL/F of pelabresib at steady state per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (1 cycle = 21 days)
Apparent Volume of Distribution (V/F) of pelabresib at steady state per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. V/F of pelabresib at steady state per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (1 cycle = 21 days)
Terminal Half-Life (T½) of pelabresib at steady state per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. T½ of pelabresib at steady state per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14 (1 cycle = 21 days)
Maximum Plasma Concentration on Day 1 (Cmax,D1) of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. Cmax,D1 of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 1 (1 cycle = 21 days)
Area Under the Curve from 0 to 24 hours on Day 1 (AUC₀-24h,D1) of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. AUC₀-24h,D1 of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 1: 0, 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (1 cycle = 21 days)
Trough Concentration on Day 14 (Ctrough,D14) of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. Ctrough,D14 of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14: predose (1 cycle = 21 days)
Accumulation Ratio (Rac) of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. Rac of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1: Day 14 compared to Day 1 (1 cycle = 21 days)
Time to Maximum Concentration (Tmax) of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. Tmax of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1: Day 1 and Day 14 (1 cycle = 21 days)
Terminal Half-Life (T½) of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. T½ of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 1 (1 cycle = 21 days)
Fraction Unbound (fu) of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. fu of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 14: 0, 2, and 8 hours postdose (1 cycle = 21 days)
fu-adjusted AUC of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. fu-adjusted AUC of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1: Day 1 and Day 14 (1 cycle = 21 days)
fu-adjusted Cmax of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. fu-adjusted Cmax of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1: Day 1 and Day 14 (1 cycle = 21 days)
fu-adjusted Ctrough of pelabresib per study group
Venous whole blood samples will be collected for pharmacokinetics characterization. fu-adjusted Ctrough of pelabresib per study group will be listed and summarized using descriptive statistics.
Time frame: Cycle 1 Day 1 and Day 14: predose (1 cycle = 21 days)
Number of Participants with adverse events (AEs), serious AEs (SAEs)
Incidence of adverse events by type, frequency, and severity, as graded by the NCI CTCAE version 5.0.
Time frame: Through study completion, an average of 28 months
Plan to share: No
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Novartis Pharmaceuticals