A Multi-center Evaluation of Aldafermin in a Randomized, Double-blind, Placebo-controlled Study in Subjects With Primary Sclerosing Cholangitis.

Phase 2

Withdrawn

• Conditions: Primary Sclerosing Cholangitis
• Interventions: Drug: aldafermin; Drug: Placebo

• Registration Number: NCT06654726
• Lead Sponsor: NGM Biopharmaceuticals, Inc

Brief Summary

A multi-center evaluation of aldafermin in a randomized, double-blind, placebo-controlled study in subjects with Primary Sclerosing Cholangitis.

Detailed Description

Evaluation of Efficacy, Safety and Tolerability of Aldafermin in a Phase 2b/3, Randomized, Double-blind, Placebo-controlled, Multi-center Study in Subjects with Primary Sclerosing Cholangitis (ALPINE-PSC)

Study Timeline

• Study Start: 2024-10-21
• Study First Submitted: 2024-10-22
• Study First Submitted QC: 2024-10-22
• Study First Posted: 2024-10-23
• Primary Completion: 2024-12-05
• Study Completion: 2025-03-18
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-03
• Last Update Posted: 2025-05-07

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

1. Able and willing to give informed consent

2. Confirmed diagnosis of PSC based on either:

   1. Abnormal cholangiography consistent with PSC as measured by MRCP with contrast
   2. Liver biopsy consistent with PSC

3. LSM by VCTE, indicative of at least F2 liver fibrosis stage

4. Laboratory parameters:

   1. TBL ≤2.5 mg/dL unless known for Gilbert's Syndrome
   2. ALT and ALT <5x ULN
   3. MELD score ≤12

Exclusion Criteria

1. Clinically significant acute or chronic liver disease of an etiology other than PSC
2. Alternate causes of sclerosing cholangitis including IgG4 related sclerosing cholangitis
3. Subjects with moderate to severe hepatic impairment
4. Subjects with moderate to severe renal impairment
5. Placement of bile duct stent or percutaneous bile duct drain within 12 weeks of Screening

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
aldafermin	aldafermin	Aldafermin 3mg given subcutaneously, daily, for up to 6 years
placebo	Placebo	Placebo given subcutaneously, daily, for up to 6 years

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Enhanced Liver Fibrosis Score at Week 96	96 Weeks	Enhanced Liver Fibrosis (ELF) score is a non-invasive blood test derived from the measurement of hyaluronic acid (HA), amino terminal propeptide of type III procollagen (PIIINP), and tissue inhibitor of metalloprotease 1 (TIMP1) using a proprietary algorithm (Siemens). ELF score is a laboratory test, is unitless, and is used as a continuous variable. The minimal ELF score is zero, the maximal ELF score is unknown. The higher the ELF score, the worse the disease outcome.; ELF is a score on a scale of severity assessment against biopsy-proven fibrosis. A score of \<7.7 is none to mild, \> 7.7-9.8 is moderate, \> 9.8 is severe.

Trial Locations

Locations (1)

NGM Clinical Study Site; 🇺🇸 Seattle, Washington, United States