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

NCT06654726 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: 282-PS-231
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Primary Sclerosing Cholangitis

Keywords

PSC

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Enhanced Liver Fibrosis Score at Week 96

  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.

  96 Weeks

Study Arms (2)

aldafermin

EXPERIMENTAL

Aldafermin 3mg given subcutaneously, daily, for up to 6 years

Drug: aldafermin

placebo

PLACEBO COMPARATOR

Placebo given subcutaneously, daily, for up to 6 years

Drug: Placebo

Interventions

aldafermin DRUG

aldafermin

aldafermin

Placebo DRUG

placebo

placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able and willing to give informed consent
• Confirmed diagnosis of PSC based on either:
• Abnormal cholangiography consistent with PSC as measured by MRCP with contrast
• Liver biopsy consistent with PSC
• LSM by VCTE, indicative of at least F2 liver fibrosis stage
• Laboratory parameters:
• TBL ≤2.5 mg/dL unless known for Gilbert's Syndrome
• ALT and ALT \<5x ULN
• MELD score ≤12

You may not qualify if:

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

Sponsors & Collaborators

• NGM Biopharmaceuticals, Inc: lead

MeSH Terms

Conditions

Cholangitis, Sclerosing

Interventions

aldafermin

Condition Hierarchy (Ancestors)

Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 22, 2024
• First Posted: October 23, 2024
• Study Start: October 21, 2024
• Primary Completion: December 5, 2024
• Study Completion: March 18, 2025
• Last Updated: May 7, 2025

Record last verified: 2025-05