Clinical trial NCT06095986: A Study Evaluating the Safety, Tolerability, and Efficacy of Aramchol Meglumine in Primary Sclerosing Cholangitis
Official title: A Proof of Concept, Prospective, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, and Efficacy of Aramchol Meglumine in Patients With Primary Sclerosing Cholangitis
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Primary Sclerosing Cholangitis
Lead sponsor: Galmed Pharmaceuticals Ltd
Sponsor class: INDUSTRY
Collaborators: name: Virginia Commonwealth University
First posted: 2023-10-23
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Parallel group analysis
Primary purpose: TREATMENT

Brief summary: The objectives of this study is to Evaluate the Safety, Tolerability, and Efficacy of Aramchol Meglumine in Patients with Primary Sclerosing Cholangitis

Detailed description: The objectives of this study are to:

* Establish the safety and tolerability of once daily (QD) Aramchol meglumine in patients with PSC
* Examine whether once daily (QD) Aramchol meglumine has any effect on serum alkaline phosphatase
* Provide a comprehensive readout of clinical efficacy following once daily (QD) Aramchol meglumine administration

Eligibility criteria: Inclusion Criteria:

1. Male or female age 18 years and above (inclusive at first screening visit)
2. Established diagnosis of large duct PSC based on abnormal cholangiography as assessed by magnetic resonance cholangiopancreatography (MRCP) or Endoscopic retrograde cholangiopancreatography (ERCP)
3. Alkaline phosphatase \> 150 IU/l
4. Stable inflammatory bowel disease therapy \> 3months for IBD patients
5. If receiving treatment with Ursodeoxycholic acid (UDCA; ursodiol), therapy is at a dose of \<20 mg/kg/day, has been stable for at least 6 months before screening
6. Ability to understand the nature of the study and to sign a written informed consent form (ICF)

Exclusion Criteria:

1. Other causes of liver disease, including secondary sclerosing cholangitis or viral, metabolic, or alcoholic liver disease, as assessed clinically
2. Active Crohn's disease (CDAI \> 40) or ulcerative colitis (Mayo IBD score \> 4) or active non-hemorrhoidal rectal bleeding
3. Small bowel resection \> 100 cm
4. Cirrhosis (clinically evident or by biopsy)
5. Prior hepatic decompensation event
6. Recent (\< 6 weeks) acute cholangitis or hospitalization for PSC or IBD
7. Bleeding diathesis or other contraindication for liver biopsy
8. Known GI or hepatobiliary malignancy
9. Prior liver transplantation
10. Prior exposure to study drug
11. Active untreated viral hepatitis or other concomitant liver disease

Primary outcomes:
measure: Change from Baseline in serum alkaline phosphatase (ALP)
timeFrame: 48 weeks
Secondary outcomes:
measure: Change from Baseline in hepatobiliary fibrosis using the Nakanuma staging scale
timeFrame: 48 weeks
measure: Change from Baseline in Enhanced liver fibrosis (ELF)
timeFrame: 48 weeks
measure: Change from Baseline in MRCP
timeFrame: 48 weeks
measure: Change from Baseline in quantitative liver function using Gadoxetate clearance
timeFrame: 48 weeks
measure: Change from Baseline in 5D-itch scale
timeFrame: 48 weeks
measure: Change from Baseline to Week 48 in Patient-Reported Outcomes Measurement Information System (PROMIS)-19 score
timeFrame: 48 weeks
measure: Change from Baseline in the Mayo IBD symptom severity score
timeFrame: 48 weeks
measure: Change from Baseline in the revised Mayo risk score (rMRS)
timeFrame: 48 weeks
measure: Change from Baseline in the UK-PSC score
timeFrame: 48 weeks
measure: Change from Baseline in the PSC risk estimate tool (PREsTo)
timeFrame: 48 weeks
Other outcomes:
Locations:
city: Richmond
country: United States
facility: The Sanyal Institute for Liver Disease &amp; Metabolic Health at VCU
state: Virginia
Study officials:
affiliation: The Sanyal Institute for Liver Disease & Metabolic Health at VCU
name: Arun Sanyal, MD
role: PRINCIPAL_INVESTIGATOR
References: