Advanced Malignancies and Hepatic ImpairmentPhase 1NCT07422610
Pharmacokinetics and Safety Study of Pelabresib in Patients With Advanced Malignancies and Hepatic Impairment
A Phase 1 study testing pelabresib in people with Advanced Malignancies and Hepatic Impairment, run by Novartis Pharmaceuticals at 10 sites in 4 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Advanced Malignancies and Hepatic Impairment
- What is being tested?
- pelabresib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 10 sitesin 4 countries
- Study dates
- 19 May 2026to 2 Aug 2028
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- Platelet count ≥ 150 × 109 /L in the absence of thrombopoietic factors or transfusions within 2 weeks of the screening assessment
- Absolute neutrophil count (ANC) ≥ 1 × 109 /L in the absence of granulocyte growth factors
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- Adequate renal function (creatinine clearance of ≥30 mL/min, calculated using Cockcroft-Gault formula)
- 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:
- 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
- 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
Who cannot join
- 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.
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- 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.
Trial locations and contacts
10 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
10 sites in 4 countries
Spain4 sites, 4 recruiting
- Recruiting
Novartis Investigative Site
Barcelona, 08023
- Recruiting
Novartis Investigative Site
Madrid, 28040
- Recruiting
Novartis Investigative Site
Madrid, 28050
- Recruiting
Novartis Investigative Site
Pozuelo de Alarcón, Madrid, 28223
Italy3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
Candiolo, TO, 10060
- Recruiting
Novartis Investigative Site
Milan, MI, 20132
- Recruiting
Novartis Investigative Site
Milan, MI, 20141
France2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Bordeaux, 33075
- Recruiting
Novartis Investigative Site
Saint-Herblain, 44805
United Kingdom1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
London, W12 0HS
About this study
Official title: 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
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.
Read the detailed description
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:
- Group 1 includes participants with normal hepatic function
- Group 2 includes participants with moderate or severe HI
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.
- Being tested
- pelabresib
- Keywords
- Hepatic impairment (HI), Moderate or severe hepatic impairment, Advanced malignancies, Pharmacokinetics (PK), Bromodomain and extraterminal (BET)
- Medical subject headings
- Lymphoma, Follicular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pelabresibDrug
Also called DAK539 (formerly CPI-0610)
pelabresib 125 mg orally (PO) once daily (QD) for 14 days, followed by a 7-day break
| Study group | Role | What they receive |
|---|---|---|
| Group 1 (normal hepatic function) | Experimental | 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. Receives: Drug: pelabresib |
| Group 2 (moderate or severe HI) | Experimental | 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. Receives: Drug: pelabresib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Cycle 1 Day 14 (1 cycle = 21 days)
Secondary outcomes (11)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Through study completion, an average of 28 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2026
- Study start19 May 2026
- Primary completion28 Jun 2028 (estimated)
- Study completion2 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Malignancies and Hepatic ImpairmentStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07422610
- Sponsor's protocol ID
- CDAK539A12103
- Other IDs
- 2025-521001-40-00, 2025
- Status last verified
- Sep 2026
- First submitted
- 22 Jan 2026
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No