Primary Sclerosing CholangitisPhase 2/3ALPINE-PSCNCT06654726
A Multi-center Evaluation of Aldafermin in a Randomized, Double-blind, Placebo-controlled Study in Subjects With Primary Sclerosing Cholangitis.
A Phase 2/3 study testing aldafermin in people with Primary Sclerosing Cholangitis, run by NGM Biopharmaceuticals, Inc.
Trial summary
A multi-center evaluation of aldafermin in a randomized, double-blind, placebo-controlled study in subjects with Primary Sclerosing Cholangitis. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Sclerosing Cholangitis
- What is being tested?
- aldafermin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 21 Oct 2024to 18 Mar 2025
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
Show all 9Show fewer
- TBL ≤2.5 mg/dL unless known for Gilbert's Syndrome
- ALT and ALT <5x ULN
- MELD score ≤12
Who cannot join
- 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
About this study
Official title: 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).
A multi-center evaluation of aldafermin in a randomized, double-blind, placebo-controlled study in subjects with Primary Sclerosing Cholangitis.
Read the 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)
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- aldafermin, Placebo
- Keywords
- PSC
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
aldaferminDrug
aldafermin
PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| aldafermin | Experimental | Aldafermin 3mg given subcutaneously, daily, for up to 6 years Receives: Drug: aldafermin |
| placebo | Placebo comparator | Placebo given subcutaneously, daily, for up to 6 years Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 96 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2024
- Study start21 Oct 2024
- Primary completion5 Dec 2024
- Study completion18 Mar 2025
Why it stopped: Study was terminated by the Sponsor because the FDA advised a trial design change which made it infeasible to continue the study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Sclerosing CholangitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- NGM Biopharmaceuticals, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06654726
- Sponsor's protocol ID
- 282-PS-231
- Acronym
- ALPINE-PSC
- Status last verified
- May 2025
- First submitted
- 22 Oct 2024
- Last updated
- 7 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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).
- ClinicalTrials.gov ID
- NCT06654726
- Acronym / sponsor ID
- ALPINE-PSC / 282-PS-231
- Conditions
- Primary Sclerosing Cholangitis
- Keywords
- PSC
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study was terminated by the Sponsor because the FDA advised a trial design change which made it infeasible to continue the study.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- NGM Biopharmaceuticals, IncIndustry (pharma, biotech, devices)
- Study start
- 21 Oct 2024 (actual)
- Primary completion
- 5 Dec 2024 (actual)
- Study completion
- 18 Mar 2025 (actual)
- First posted
- 23 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| aldafermin | Experimental | Drug: aldaferminAldafermin 3mg given subcutaneously, daily, for up to 6 years |
| placebo | Placebo comparator | Drug: PlaceboPlacebo given subcutaneously, daily, for up to 6 years |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Able and willing to give informed consent
- Confirmed diagnosis of PSC based on either:
- LSM by VCTE, indicative of at least F2 liver fibrosis stage
- Laboratory parameters:
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| aldaferminaldafermin | Drug | aldafermin |
| Placeboplacebo | Drug | placebo |
Primary outcome
Change From Baseline in Enhanced Liver Fibrosis Score at Week 96
Time frame: 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.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing aldafermin in people with Primary Sclerosing Cholangitis, run by NGM Biopharmaceuticals, Inc.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 7 May 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Enhanced Liver Fibrosis Score at Week 96 (time frame: 96 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2025.