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

NCT06654726 — Phase 2 / Phase 3, Withdrawn, NGM Biopharmaceuticals, Inc

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A Phase 2b/3, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Safety, and Tolerability of Aldafermin in Subjects With Primary Sclerosing Cholangitis (ALPINE-PSC).

Registry	NCT06654726, acronym ALPINE-PSC
Status	Withdrawn - Study was terminated by the Sponsor because the FDA advised a trial design change which made it infeasible to continue the study.
Phase	Phase 2 / Phase 3
Study type	Interventional; randomized, parallel, masking: quadruple, treatment
Sponsor	NGM Biopharmaceuticals, Inc (Industry)
Conditions	Primary Sclerosing Cholangitis
Interventions	aldafermin, Placebo (Drug)
Dates	start 2024-10-21, primary completion 2024-12-05, completion 2025-03-18, first posted 2024-10-23, last updated 2025-05-07

-Summary

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

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+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)

+Arms and interventions

Arm	Type	Interventions	Description
aldafermin	Experimental	Drug: aldafermin	Aldafermin 3mg given subcutaneously, daily, for up to 6 years
placebo	Placebo comparator	Drug: Placebo	Placebo given subcutaneously, daily, for up to 6 years

aldafermin (Drug): aldafermin
Placebo (Drug): placebo

+Outcome measures

Primary outcomes

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.

+Eligibility

Sex: All, ages: 18 Years to 75 Years

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