A Study Evaluating the Safety, Tolerability, and Efficacy of Aramchol Meglumine in Primary Sclerosing Cholangitis
- Conditions
- Primary Sclerosing Cholangitis
- Interventions
- Registration Number
- NCT06095986
- Lead Sponsor
- Galmed Research and Development, Ltd.
- 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
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 24
- Male or female age 18 years and above (inclusive at first screening visit)
- Established diagnosis of large duct PSC based on abnormal cholangiography as assessed by magnetic resonance cholangiopancreatography (MRCP) or Endoscopic retrograde cholangiopancreatography (ERCP)
- Alkaline phosphatase > 150 IU/l
- Stable inflammatory bowel disease therapy > 3months for IBD patients
- 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
- Ability to understand the nature of the study and to sign a written informed consent form (ICF)
- Other causes of liver disease, including secondary sclerosing cholangitis or viral, metabolic, or alcoholic liver disease, as assessed clinically
- Active Crohn's disease (CDAI > 40) or ulcerative colitis (Mayo IBD score > 4) or active non-hemorrhoidal rectal bleeding
- Small bowel resection > 100 cm
- Cirrhosis (clinically evident or by biopsy)
- Prior hepatic decompensation event
- Recent (< 6 weeks) acute cholangitis or hospitalization for PSC or IBD
- Bleeding diathesis or other contraindication for liver biopsy
- Known GI or hepatobiliary malignancy
- Prior liver transplantation
- Prior exposure to study drug
- Active untreated viral hepatitis or other concomitant liver disease
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description PSC patients administered with Aramchol meglumine Aramchol meglumine Adult subjects with clinically diagnosed PSC that are administered with Aramchol meglumine PSC patients administered with placebo Aramchol meglumine Adult subjects with clinically diagnosed PSC that are administered with matching placebo
- Primary Outcome Measures
Name Time Method Change from Baseline in serum alkaline phosphatase (ALP) 48 weeks The change from Baseline to Week 48 in ALP levels
- Secondary Outcome Measures
Name Time Method Change from Baseline in hepatobiliary fibrosis using the Nakanuma staging scale 48 weeks Change from Baseline to Week 48 in liver histology using the Nakanuma stage classification. A score of 0 is classified as stage 1 (no or minimal disease progression), while 1 score 5 or 6 is classified as stage 4 (advanced disease progression).
Change from Baseline in Enhanced liver fibrosis (ELF) 48 weeks Change from Baseline to Week 48 in Enhanced liver fibrosis (ELF) test. Score below 7.7 indicate no to mild fibrosis, and score higher than 11.3 indicate cirrhosis.
Change from Baseline in MRCP 48 weeks Change from Baseline to Week 48 in magnetic resonance cholangiopancreatography (MRCP)
Change from Baseline in quantitative liver function using Gadoxetate clearance 48 weeks Change from Baseline to Week 48 in quantitative liver function using Gadoxetate clearance
Change from Baseline in 5D-itch scale 48 weeks Change from Baseline to Week 48 in the 5 dimension itch scale (5d-itch scale) measuring pruritus (the 5 dimensions are degree, duration, direction, disability and distribution). Higher degree mean worse outcome \<8 on rating scale mean no pruritus, and \>22 on rating scale indicate severe pruritus
Change from Baseline to Week 48 in Patient-Reported Outcomes Measurement Information System (PROMIS)-19 score 48 weeks Change from Baseline to Week 48 in the Patient-Reported Outcomes Measurement Information System (PROMIS)-19 questionnaire score. The average score for Physical Function is 50, with a score of 40 considered below average and a score of 60 considered above average
Change from Baseline in the Mayo IBD symptom severity score 48 weeks Change from Baseline to Week 48 in the Mayo Inflammatory bowel disease (IBD) symptom severity score. A score of 3 to 5 points indicates mildly active disease and a score of 11 to 12 points indicates severely active bowel disease
Change from Baseline in the revised Mayo risk score (rMRS) 48 weeks Change from Baseline to Week 48 in the revised Mayo risk score (rMRS). The score provide the estimated probability of survival (%) based on age, bilirubin, AST, and history of bleeding
Change from Baseline in the UK-PSC score 48 weeks Change from Baseline to Week 48 in the united kingdom primary sclerosing cholangitis (UK-PSC) score. The score provide the estimated probability of survival (%) based on age, bilirubin, albumin, platelets, hemoglobin and ALP
Change from Baseline in the PSC risk estimate tool (PREsTo) 48 weeks Change from Baseline to Week 48 in the PSC risk estimate tool (PREsTo). The tool consists of bilirubin, albumin, ALP, platelets, AST, hemoglobin, sodium, patient age and the number of years since PSC was diagnosed, and it predicts short-term and long-term need for liver transplantation or death.
Trial Locations
- Locations (1)
The Sanyal Institute for Liver Disease & Metabolic Health at VCU
🇺🇸Richmond, Virginia, United States