## Protocol Section
### Identification Module
**NCT ID:** NCT05082779

**Brief Title:** Study in Healthy Volunteers to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of CS0159

**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

#### Organization Study ID Info
**ID:** CS0159-001
#### Organization
**Class:** OTHER
**Full Name:** Cascade Pharmaceuticals, Inc
### Status Module
#### Completion Date
**Date:** 2022-10-12

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2022-10-18

**Type:** ACTUAL

**Last Update Submit Date:** 2022-10-15

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2022-09-16

**Type:** ACTUAL

#### Start Date
**Date:** 2021-10-26

**Type:** ACTUAL

**Status Verified Date:** 2021-10

#### Study First Post Date
**Date:** 2021-10-19
**Type:** ACTUAL
**Study First Submit Date:** 2021-09-25
**Study First Submit QC Date:** 2021-10-05
### Sponsor Collaborators Module

#### Collaborators

**Class:** INDUSTRY

**Name:** Covance

#### Lead Sponsor

**Class:** OTHER

**Name:** Cascade Pharmaceuticals, Inc

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** False

### Description Module

**Brief 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. 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.

### Conditions Module

**Conditions:**

- Primary Sclerosing Cholangitis (PSC)

### Design Module

#### Design Info

**Allocation:** RANDOMIZED

**Intervention Model:** PARALLEL

##### Masking Info

**Masking:** DOUBLE

**Who Masked:**

- PARTICIPANT

- INVESTIGATOR

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 79

**Type:** ACTUAL

**Phases:**

- PHASE1

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Participants in fasted state will receive CS0159 0.2 mg or placebo once on Day 1.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort A1: 0.2 mg

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** Participants in fasted state will receive CS0159 0.6 mg or placebo once on Day 1.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort A2: 0.6 mg

**Type:** EXPERIMENTAL

#### Arm Group 3

**Description:** 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.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort A3: 1 mg

**Type:** EXPERIMENTAL

#### Arm Group 4

**Description:** Participants in fasted state will receive CS0159 2 mg or placebo once on Day 1.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort A4: 2 mg

**Type:** EXPERIMENTAL

#### Arm Group 5

**Description:** Participants in fasted state will receive CS0159 4 mg or placebo once on Day 1.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort A5: 4 mg

**Type:** EXPERIMENTAL

#### Arm Group 6

**Description:** Participants in fasted state will receive CS0159 8 mg or placebo once on Day 1.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort A6: 8 mg

**Type:** EXPERIMENTAL

#### Arm Group 7

**Description:** Participants in fasted state will receive CS0159 0.4 mg or placebo once daily for a consecutive 14 days.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort B1: 0.4 mg

**Type:** EXPERIMENTAL

#### Arm Group 8

**Description:** Participants in fasted state will receive CS0159 1 mg or placebo once daily for a consecutive 14 days.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort B2: 1 mg

**Type:** EXPERIMENTAL

#### Arm Group 9

**Description:** Participants in fasted state will receive CS0159 2 mg or placebo once daily for a consecutive 14 days.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort B3: 2 mg

**Type:** EXPERIMENTAL

#### Arm Group 10

**Description:** Participants in fasted state will receive CS0159 4 mg or placebo once daily for a consecutive 14 days.

**Intervention Names:**

- Drug: CS0159

**Label:** Cohort B4: 4mg

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- 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

**Description:** Tablets administered orally

**Name:** CS0159

**Other Names:**

- Placebo

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** Area under the concentration-time curve (AUC) from time zero to infinity (AUC0-∞)

**Measure:** Single-Dose Pharmacokinetic (PK) Parameter

**Time Frame:** Day 1 after dosing

**Description:** AUC from time zero to the time of the last measured concentration

**Measure:** Single-Dose Pharmacokinetic (PK) Parameter: (AUC0-last)

**Time Frame:** Day 1 after dosing

**Description:** Maximum observed plasma concentration

**Measure:** Single-Dose Pharmacokinetic (PK) Parameter: (Cmax)

**Time Frame:** Day 1 after dosing

**Description:** Time of the maximum observed plasma concentration

**Measure:** Single-Dose Pharmacokinetic (PK) Parameter: (Tmax)

**Time Frame:** Day 1 after dosing

**Description:** Maximum concentration during a dosing interval Ct_max

**Measure:** Multiple-Dose PK Parameter

**Time Frame:** Day 1 after dosing; day 14

**Description:** Minimum concentration during a dosing interval

**Measure:** Multiple-Dose PK Parameter: (Ct_min, Day 14)

**Time Frame:** Day 1 after dosing; day 14

**Description:** AUC over one dosing interval

**Measure:** Multiple-Dose PK Parameter: (AUCtau)

**Time Frame:** Day 1 after dosing; day 14

**Description:** Incidence and severity of adverse events

**Measure:** To characterize the safety and tolerability of single dose of CS0159

**Time Frame:** up to Day 31

**Description:** Incidence and severity of adverse events

**Measure:** To characterize the safety and tolerability of multiple doses of CS0159

**Time Frame:** up to Day 44

#### Secondary Outcomes

**Description:** fibroblast growth factor 19

**Measure:** Pharmacodynamic (PD) Parameter: FGF19

**Time Frame:** Day -1; day 1

**Description:** serum concentration

**Measure:** Pharmacodynamic (PD) Parameter: C4

**Time Frame:** Day -1; day 1

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

1. Healthy male and non-pregnant female volunteers
2. 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

Exclusion Criteria:

1. Subjects with special dietary requirements and cannot follow a uniform diet.
2. Pregnant or nursing females or females who have pregnancy plans during the trial or within 3 months after the trial.
3. Any subject with SARS-CoV-2 infection, based on a positive polymerase chain reaction for SARS-CoV-2.
4. 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.

**Healthy Volunteers:** True

**Maximum Age:** 55 Years

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Daytona Beach

**Country:** United States

**Facility:** Labcorp Clinical Research Unit, Inc.

**State:** Florida

**Zip:** 32117

#### Overall Officials

**Official 1:**

**Affiliation:** Labcorp Clinical Research Unit, Inc.

**Name:** Kathleen Doisy, MD

**Role:** PRINCIPAL_INVESTIGATOR

### IPD Sharing Statement Module

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000001649

- Term: Bile Duct Diseases

- ID: D000001660

- Term: Biliary Tract Diseases

- ID: D000004066

- Term: Digestive System Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC06

- Name: Digestive System Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M6002

- Name: Cholangitis

- Relevance: HIGH

- As Found: Cholangitis

- ID: M17914

- Name: Cholangitis, Sclerosing

- Relevance: HIGH

- As Found: Primary Sclerosing Cholangitis

- ID: M4935

- Name: Bile Duct Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M4946

- Name: Biliary Tract Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M7255

- Name: Digestive System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M8883

- Name: Gastrointestinal Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T4712

- Name: Primary Sclerosing Cholangitis

- Relevance: HIGH

- As Found: Primary Sclerosing Cholangitis

### Condition Browse Module - Meshes

- ID: D000002761

- Term: Cholangitis

- ID: D000015209

- Term: Cholangitis, Sclerosing

### Misc Info Module

- Version Holder: 2024-05-24
