Primary Sclerosing Cholangitis (PSC)Phase 1NCT05082779
Study in Healthy Volunteers to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of CS0159
A Phase 1 study testing CS0159 in people with Primary Sclerosing Cholangitis (PSC), run by Cascade Pharmaceuticals, Inc at 1 site in United States.
Trial summary
The whole study includes 2 parts. Both the SAD study and MAD study are randomized, double-blinded, and placebo-controlled studies, conducted in healthy subjects, to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics profiles of CS0159. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Primary Sclerosing Cholangitis (PSC)
- What is being tested?
- CS0159
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 79 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Oct 2021to 12 Oct 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy male and non-pregnant female volunteers
- In good health, determined by having no clinically significant findings from medical history, physical examination, 12-lead ECG, vital signs measurements, and clinical laboratory evaluations
Who cannot join
- Subjects with special dietary requirements and cannot follow a uniform diet.
- Pregnant or nursing females or females who have pregnancy plans during the trial or within 3 months after the trial.
- Any subject with SARS-CoV-2 infection, based on a positive polymerase chain reaction for SARS-CoV-2.
- History or evidence of clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to subject safety or interfere with the study evaluations, procedures, or completion.
Trial locations and contacts
1 site in 1 country.
United States1 site
Labcorp Clinical Research Unit, Inc.
Daytona Beach, Florida, 32117
About this study
Official title: A Phase I, Randomized, Double-Blind, Placebo-Contralled, Single Asending Dose / Multiple Ascending Dose Study of CS0159 to to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics, and Effect Food in Healthy Subject
The whole study includes 2 parts. Both the SAD study and MAD study are randomized, double-blinded, and placebo-controlled studies, conducted in healthy subjects, to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics profiles of CS0159. The SAD part also involves a pilot food effect (FE) study, designed to assess the food effect on single-dose PK profile in healthy subjects.
Read the detailed description
A total of 48 healthy subjects will be allocated to 1 of 6 cohorts (cohort A1~A6) in the SAD study, each cohort including 8 subjects (6 subjects will receive investigational new drug (IND) product and 2 receive placebo). Each subject in fasted state will be randomly assigned to receive a single oral dose of CS0159 or placebo.To ensure the safety for all SAD cohorts (including A3 in both treatment periods).
The MAD study will enroll 32 healthy subjects, allocated to 1 of 4 cohorts (cohort B1~B4) and each cohort including 8 participants (6 subjects will receive IND products and 2 receive placebo). Subjects will be randomly assigned to orally receive the IND product or placebo.
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Being tested
- CS0159
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CS0159Drug
Also called Placebo
Tablets administered orally
| Study group | Role | What they receive |
|---|---|---|
| Cohort A1: 0.2 mg | Experimental | Participants in fasted state will receive CS0159 0.2 mg or placebo once on Day 1. Receives: Drug: CS0159 |
| Cohort A2: 0.6 mg | Experimental | Participants in fasted state will receive CS0159 0.6 mg or placebo once on Day 1. Receives: Drug: CS0159 |
| Cohort A3: 1 mg | Experimental | Participants in fasted state will receive CS0159 1 mg or placebo once on Day 1 followed by a 7-day washout period then given in 1 mg tablet (in fed state) on Day 8. Receives: Drug: CS0159 |
| Cohort A4: 2 mg | Experimental | Participants in fasted state will receive CS0159 2 mg or placebo once on Day 1. Receives: Drug: CS0159 |
| Cohort A5: 4 mg | Experimental | Participants in fasted state will receive CS0159 4 mg or placebo once on Day 1. Receives: Drug: CS0159 |
| Cohort A6: 8 mg | Experimental | Participants in fasted state will receive CS0159 8 mg or placebo once on Day 1. Receives: Drug: CS0159 |
| Cohort B1: 0.4 mg | Experimental | Participants in fasted state will receive CS0159 0.4 mg or placebo once daily for a consecutive 14 days. Receives: Drug: CS0159 |
| Cohort B2: 1 mg | Experimental | Participants in fasted state will receive CS0159 1 mg or placebo once daily for a consecutive 14 days. Receives: Drug: CS0159 |
| Cohort B3: 2 mg | Experimental | Participants in fasted state will receive CS0159 2 mg or placebo once daily for a consecutive 14 days. Receives: Drug: CS0159 |
| Cohort B4: 4mg | Experimental | Participants in fasted state will receive CS0159 4 mg or placebo once daily for a consecutive 14 days. Receives: Drug: CS0159 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Single-Dose Pharmacokinetic (PK) Parameter
Area under the concentration-time curve (AUC) from time zero to infinity (AUC0-∞)
Measured: Day 1 after dosing
Single-Dose Pharmacokinetic (PK) Parameter: (AUC0-last)
AUC from time zero to the time of the last measured concentration
Measured: Day 1 after dosing
Single-Dose Pharmacokinetic (PK) Parameter: (Cmax)
Maximum observed plasma concentration
Measured: Day 1 after dosing
Single-Dose Pharmacokinetic (PK) Parameter: (Tmax)
Time of the maximum observed plasma concentration
Measured: Day 1 after dosing
Multiple-Dose PK Parameter
Maximum concentration during a dosing interval Ct_max
Measured: Day 1 after dosing; day 14
Multiple-Dose PK Parameter: (Ct_min, Day 14)
Minimum concentration during a dosing interval
Measured: Day 1 after dosing; day 14
Multiple-Dose PK Parameter: (AUCtau)
AUC over one dosing interval
Measured: Day 1 after dosing; day 14
To characterize the safety and tolerability of single dose of CS0159
Incidence and severity of adverse events
Measured: up to Day 31
To characterize the safety and tolerability of multiple doses of CS0159
Incidence and severity of adverse events
Measured: up to Day 44
Secondary outcomes (2)
Pharmacodynamic (PD) Parameter: FGF19
fibroblast growth factor 19
Measured: Day -1; day 1
Pharmacodynamic (PD) Parameter: C4
serum concentration
Measured: Day -1; day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2021
- Study start26 Oct 2021
- Primary completion16 Sep 2022
- Study completion12 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Sclerosing Cholangitis (PSC)Studies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 79 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cascade Pharmaceuticals, Inc
- Sponsor type
- University, hospital or other
- Collaborators
- Covance
- Responsible party
- Sponsor
- Investigators
- Kathleen Doisy, MD, Principal investigator, Labcorp Clinical Research Unit, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05082779
- Sponsor's protocol ID
- CS0159-001
- Status last verified
- Oct 2021
- First submitted
- 25 Sep 2021
- Last updated
- 18 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Zang Y, Shi J, Zhao G, Tang B, Liu M, Yao B, Wang G, Pan H, Yang S, Deng R, Zhao Y, Zhang Z, Guo HR, Sun DD, Wang H, Gao L, Yu J, Diao X, Li Y, Li J, Xu HE. A first-in-class pulsatile FXR agonist for bile-acid-related liver diseases. Nature. 2026 Jul;655(8123):758-768. doi: 10.1038/s41586-026-10633-1. Epub 2026 Jun 10.
PubMed 42271063
Study at a glance
From the registry record, last updated by the sponsor on 18 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Randomized, Double-Blind, Placebo-Contralled, Single Asending Dose / Multiple Ascending Dose Study of CS0159 to to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics, and Effect Food in Healthy Subject
- ClinicalTrials.gov ID
- NCT05082779
- Acronym / sponsor ID
- CS0159-001
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 79 participants (actual)
- Lead sponsor
- Cascade Pharmaceuticals, IncUniversity, hospital or other
- Collaborators
- Covance
- Study start
- 26 Oct 2021 (actual)
- Primary completion
- 16 Sep 2022 (actual)
- Study completion
- 12 Oct 2022 (actual)
- First posted
- 19 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The whole study includes 2 parts. Both the SAD study and MAD study are randomized, double-blinded, and placebo-controlled studies, conducted in healthy subjects, to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics profiles of CS0159. The SAD part also involves a pilot food effect (FE) study, designed to assess the food effect on single-dose PK profile in healthy subjects.
Detailed description
A total of 48 healthy subjects will be allocated to 1 of 6 cohorts (cohort A1~A6) in the SAD study, each cohort including 8 subjects (6 subjects will receive investigational new drug (IND) product and 2 receive placebo). Each subject in fasted state will be randomly assigned to receive a single oral dose of CS0159 or placebo.To ensure the safety for all SAD cohorts (including A3 in both treatment periods).
The MAD study will enroll 32 healthy subjects, allocated to 1 of 4 cohorts (cohort B1~B4) and each cohort including 8 participants (6 subjects will receive IND products and 2 receive placebo). Subjects will be randomly assigned to orally receive the IND product or placebo.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 10
| Arm | Type | Receives |
|---|---|---|
| Cohort A1: 0.2 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 0.2 mg or placebo once on Day 1. |
| Cohort A2: 0.6 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 0.6 mg or placebo once on Day 1. |
| Cohort A3: 1 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 1 mg or placebo once on Day 1 followed by a 7-day washout period then given in 1 mg tablet (in fed state) on Day 8. |
| Cohort A4: 2 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 2 mg or placebo once on Day 1. |
| Cohort A5: 4 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 4 mg or placebo once on Day 1. |
| Cohort A6: 8 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 8 mg or placebo once on Day 1. |
| Cohort B1: 0.4 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 0.4 mg or placebo once daily for a consecutive 14 days. |
| Cohort B2: 1 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 1 mg or placebo once daily for a consecutive 14 days. |
| Cohort B3: 2 mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 2 mg or placebo once daily for a consecutive 14 days. |
| Cohort B4: 4mg | Experimental | Drug: CS0159Participants in fasted state will receive CS0159 4 mg or placebo once daily for a consecutive 14 days. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (2 listed)
- Healthy male and non-pregnant female volunteers
- In good health, determined by having no clinically significant findings from medical history, physical examination, 12-lead ECG, vital signs measurements, and clinical laboratory evaluations
Key exclusion criteria (4 listed)
- Subjects with special dietary requirements and cannot follow a uniform diet.
- Pregnant or nursing females or females who have pregnancy plans during the trial or within 3 months after the trial.
- Any subject with SARS-CoV-2 infection, based on a positive polymerase chain reaction for SARS-CoV-2.
- History or evidence of clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to subject safety or interfere with the study evaluations, procedures, or completion.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CS0159Also: PlaceboTablets administered orally | Drug | Cohort A1: 0.2 mg, Cohort A2: 0.6 mg, Cohort A3: 1 mg, Cohort A4: 2 mg, Cohort A5: 4 mg, Cohort A6: 8 mg, Cohort B1: 0.4 mg, Cohort B2: 1 mg, Cohort B3: 2 mg, Cohort B4: 4mg |
Primary outcome
Single-Dose Pharmacokinetic (PK) Parameter
Time frame: Day 1 after dosing
Area under the concentration-time curve (AUC) from time zero to infinity (AUC0-∞)
Single-Dose Pharmacokinetic (PK) Parameter: (AUC0-last)
Time frame: Day 1 after dosing
AUC from time zero to the time of the last measured concentration
Single-Dose Pharmacokinetic (PK) Parameter: (Cmax)
Time frame: Day 1 after dosing
Maximum observed plasma concentration
Single-Dose Pharmacokinetic (PK) Parameter: (Tmax)
Time frame: Day 1 after dosing
Time of the maximum observed plasma concentration
Multiple-Dose PK Parameter
Time frame: Day 1 after dosing; day 14
Maximum concentration during a dosing interval Ct_max
Multiple-Dose PK Parameter: (Ct_min, Day 14)
Time frame: Day 1 after dosing; day 14
Minimum concentration during a dosing interval
Multiple-Dose PK Parameter: (AUCtau)
Time frame: Day 1 after dosing; day 14
AUC over one dosing interval
To characterize the safety and tolerability of single dose of CS0159
Time frame: up to Day 31
Incidence and severity of adverse events
To characterize the safety and tolerability of multiple doses of CS0159
Time frame: up to Day 44
Incidence and severity of adverse events
2 secondary outcomes
Pharmacodynamic (PD) Parameter: FGF19
Time frame: Day -1; day 1
Pharmacodynamic (PD) Parameter: C4
Time frame: Day -1; day 1
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2022.
- Derived from this study: PubMed 42271063Zang Y, Shi J, Zhao G, Tang B, Liu M, Yao B, Wang G, Pan H, Yang S, Deng R, Zhao Y, Zhang Z, Guo HR, Sun DD, Wang H, Gao L, Yu J, Diao X, Li Y, Li J, Xu HE. A first-in-class pulsatile FXR agonist for bile-acid-related liver diseases. Nature. 2026 Jul;655(8123):758-768. doi: 10.1038/s41586-026-10633-1. Epub 2026 Jun 10.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kathleen Doisy, MD principal investigator
Labcorp Clinical Research Unit, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CS0159 in people with Primary Sclerosing Cholangitis (PSC), run by Cascade Pharmaceuticals, Inc at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Single-Dose Pharmacokinetic (PK) Parameter (time frame: Day 1 after dosing). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05082779, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Oct 2022.