Clinical trial NCT06060665: Intended to Determine the Effects of Seladelpar on Normalization of Alkaline Phosphatase Levels in Subjects With Primary Biliary Cholangitis (PBC)
Official title: IDEAL: A 52-week, Double-blind, Placebo-controlled, Randomized, Phase 3 Study Intended to Determine the Effects of Seladelpar on Normalization of Alkaline Phosphatase Levels in Subjects With Primary Biliary Cholangitis (PBC) and an Incomplete Response or Intolerance to Ursodeoxycholic Acid (UDCA)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Primary Biliary Cholangitis
Keywords: Primary Biliary Cholangitis (PBC)
PBC
Lead sponsor: Gilead Sciences
Sponsor class: INDUSTRY
First posted: 2023-09-29
Enrollment: 90
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To Determine the Effects of Seladelpar on Normalization of Alkaline Phosphatase Levels in Subjects with Primary Biliary Cholangitis (PBC) and an Incomplete Response or Intolerance to Ursodeoxycholic Acid (UDCA).

The primary objective is to evaluate the effect of seladelpar treatment at Week 52 compared to placebo based on normalization of alkaline phosphatase (ALP) defined by a composite endpoint of ALP ≤ 1.0× upper limit of normal (ULN) and ≥ 15% decrease from baseline in PBC participants with an ALP value greater than ULN but less than 1.67× ULN.


Eligibility criteria: Key Inclusion Criteria:

Individuals must meet the following criteria to be eligible for study participation:

1. Male or female with a diagnosis of primary biliary cholangitis (PBC) based on history.
2. Ursodeoxycholic acid (UDCA) for the 12 months prior to screening (with stable dose for \> 3 months prior to screening) OR intolerant to UDCA (last dose of UDCA \> 3 months prior to screening).
3. ALP \> 1× ULN and \< 1.67× ULN.
4. Females of reproductive potential must use at least 1 barrier contraceptive and a second effective birth control method during the study and for at least 90 days after the last dose. Male participants who are sexually active with female partners of reproductive potential must use barrier contraception, and their female partners must use a second effective birth control method during the study and for at least 90 days after the last dose.

Key Exclusion Criteria:

1. Previous exposure to seladelpar (MBX-8025).
2. A medical condition other than PBC that, in the Investigator's opinion, would preclude full participation in the study (eg, cancer) or confound its results.
3. Advanced PBC as defined by the Rotterdam criteria.
4. Laboratory parameters measured by the Central Laboratory at screening.
5. Clinically important hepatic decompensation.
6. Other chronic liver diseases.
7. Known history of human immunodeficiency virus (HIV) or positive antibody test at screening.
8. Clinically important alcohol consumption, defined as more than 2 drink units per day in women and 3 drink units per day in men, or inability to quantify alcohol intake reliably.
9. History of malignancy diagnosed or treated, active or within 2 years, or ongoing evaluation for malignancy; localized treatment of squamous or noninvasive basal cell skin cancers and cervical carcinoma in situ is allowed if appropriately treated prior to screening.
10. History of drug abuse that would compromise the quality of the clinical study.
11. Treatment with obeticholic acid or fibrates 6 weeks prior to screening.
12. Treatment with colchicine, methotrexate, azathioprine, or long-term systemic corticosteroids within 2 months prior to screening.
13. Initiation or dose adjustment of anti-pruritic drugs indicated for the treatment of pruritus within 1 month prior to screening.
14. Immunosuppressant therapies within 6 months prior to screening.
15. Other medications that affect liver or gastrointestinal functions, as well as the roux-en-y gastric bypass procedure, may be prohibited and should be discussed with the medical monitor on a case by-case basis.
16. Treatment with any other investigational therapy or device within 30 days or within halflives, whichever is longer, prior to screening.
17. Pregnancy or plans to become pregnant, or breastfeeding.
18. Any other condition(s) that would compromise the safety of the individual or compromise the quality of the clinical study, as judged by the investigator.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Percentage of Participants Response defined as Alkaline phosphatase (ALP) ≤ 1.0× Upper Limit of Normal (ULN) AND ≥ 15% Decrease in ALP at Week 52.
timeFrame: 52 weeks
measure: Type, Frequency, and Severity of Treatment-emergent Adverse Events.
timeFrame: 52 weeks
Secondary outcomes:
measure: Percentage of Participant Response Defined as ALP ≤ 1.0× ULN.
timeFrame: 52 weeks
measure: Percentage of Participant Response Defined as ALP ≤ 1.0× ULN AND ≥ 15% Decrease in ALP at 4 Weeks.
timeFrame: 4 weeks
measure: Percentage of Participant Response Defined as ALP ≤ 1.0× ULN AND ≥ 15% Decrease in ALP at 12 Weeks.
timeFrame: 12 weeks
measure: Percentage of Participant Response Defined as ALP ≤ 1.0× ULN AND ≥ 15% Decrease in ALP at 26 Weeks.
timeFrame: 26 weeks
measure: Percentage of Participant Response Defined as ALP ≤ 1.0× ULN AND ≥ 15% Decrease in ALP at 39 Weeks.
timeFrame: 39 weeks
measure: Absolute and Percent Change From Baseline in ALP.
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Chandler
country: United States
facility: The Institute for Liver Health II LLC dba Arizona Clinical Trials; Arizona Liver Health
state: Arizona
city: Pasadena
country: United States
facility: California Liver Research Institute
state: California
city: Sacramento
country: United States
facility: University of California (UC) Davis Medical Center
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado Denver - School of Medicine - Anschutz Medical Campus
state: Colorado
city: Englewood
country: United States
facility: South Denver Gastroenterology
state: Colorado
city: Lakewood Ranch
country: United States
facility: Florida Research Institute
state: Florida
city: Miami
country: United States
facility: University of Miami - Schiff Center for Liver Diseases
state: Florida
city: Miami
country: United States
facility: Gastro Health Research
state: Florida
city: Sarasota
country: United States
facility: Covenant Metabolic Specialists, LLC
state: Florida
city: Tampa
country: United States
facility: Covenant Metabolic Specialists, LLC
state: Florida
city: Atlanta
country: United States
facility: Digestive Healthcare of Georgia
state: Georgia
city: Chicago
country: United States
facility: Northwestern Medicine Clinical Research Unit (CRU)
state: Illinois
city: New Orleans
country: United States
facility: Tulane University Medical Center
state: Louisiana
city: Shreveport
country: United States
facility: Louisiana Research Center, LLC
state: Louisiana
city: Baltimore
country: United States
facility: Mercy Medical Center - Baltimore, Maryland
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Novi
country: United States
facility: Henry Ford Health System
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Kansas City
country: United States
facility: Kansas City Research Institute
state: Missouri
city: Brooklyn
country: United States
facility: New-York-Presbyterian Brooklyn Methodist Hospital
state: New York
city: Manhasset
country: United States
facility: Northwell Health Center for Liver Disease and Transplantation
state: New York
city: New York
country: United States
facility: New York University (NYU) Langone Medical Center
state: New York
city: New York
country: United States
facility: Weill Cornell Medicine
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center - Strong Memorial Hospital
state: New York
city: Liberty Township
country: United States
facility: Gastro Health Research
state: Ohio
city: Philadelphia
country: United States
facility: Perelman Center for Advanced Medicine
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Center for Liver Health
state: Pennsylvania
city: Cordova
country: United States
facility: OGI - Gastro One
state: Tennessee
city: Hixson
country: United States
facility: Galen Hepatology
state: Tennessee
city: Johnson City
country: United States
facility: Gastrointestinal Associates of Northeast Tennessee, P.C.
state: Tennessee
city: Austin
country: United States
facility: American Research Corporation - San Antonio, TX
state: Texas
city: Dallas
country: United States
facility: The Liver Institute at Methodist Dallas Medical Center
state: Texas
city: Dallas
country: United States
facility: Liver Center of Texas
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Denison
country: United States
facility: Soma Clinical Trials, LLC
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine - Baylor Clinic
state: Texas
city: Houston
country: United States
facility: Liver Associates of Texas, P.A.
state: Texas
city: Richmond
country: United States
facility: Bon Secours Liver Institute of Hampton Roads
state: Virginia
city: Seattle
country: United States
facility: Velocity Clinical Research - Seattle
state: Washington
city: Seattle
country: United States
facility: Stanford University
state: Washington
city: Montreal
country: Canada
facility: Centre de Recherche du Centre Hospitallier de I'Universite de Montreal (CRCHUM)
city: Vancouver
country: Canada
facility: (G.I.R.I.) GI Research Institute Foundation
Study officials:
affiliation: Gilead Sciences
name: Gilead Study Director
role: STUDY_DIRECTOR
References: