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

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).

Status

Withdrawn

Phases

Phase 2Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06654726

Acronym

ALPINE-PSC

Enrollment

Registered

2024-10-23

Start date

2024-10-21

Completion date

2025-03-18

Last updated

2025-05-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary Sclerosing Cholangitis

Keywords

PSC

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)

Interventions

DRUGaldafermin

aldafermin

DRUGPlacebo

placebo

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to 75 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026