Primary Sclerosing CholangitisPhase 2NCT02704364

A Study of NGM282 in Patients With Primary Sclerosing Cholangitis

A Phase 2 study testing NGM282 in people with Primary Sclerosing Cholangitis, run by NGM Biopharmaceuticals, Inc at 38 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study is to determine the safety, tolerability, and activity of NGM282 in patients 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?: NGM282
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 62 participants
Where: 38 sitesin 4 countries
Study dates: Mar 2016to Jun 2017
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Confirmed diagnosis of PSC

Who cannot join

Clinically significant acute or chronic liver disease of an etiology other than PSC
Secondary or IgG4 related sclerosing cholangitis

Trial locations and contacts

38 sites in 4 countries.

Country

City, postcode or hospital

38 sites in 4 countries

United States26 sites

NGM Clinical Study Site 106
Aurora, Colorado
NGM Clinical Study Site 111
Charlottesville, Virginia
NGM Clinical Study Site 120
Cincinnati, Ohio
NGM Clinical Study Site 113
Dallas, Texas
NGM Clinical Study Site 117
Dallas, Texas
NGM Clinical Study Site 104
Detroit, Michigan
NGM Clinical Study Site 116
Durham, North Carolina
NGM Clinical Study Site 110
Gainesville, Florida
NGM Clinical Study Site 119
Houston, Texas
NGM Clinical Study Site 109
Indianapolis, Indiana
NGM Clinical Study Site 102
Kansas City, Missouri
NGM Clinical Study Site 124
Lakewood Rch, Florida
NGM Clinical Study Site 105
Miami, Florida
NGM Clinical Study Site 114
Nashville, Tennessee
NGM Clinical Study Site 103
New York, New York
NGM Clinical Study Site 122
Newport News, Virginia
NGM Clinical Study Site 101
Norfolk, Virginia
NGM Clinical Study Site 121
Richmond, Virginia
NGM Clinical Study Site 118
Sacramento, California
NGM Clinical Study Site 112
San Clemente, California
NGM Clinical Study Site 127
San Francisco, California
NGM Clinical Study Site 108
Seattle, Washington
NGM Clinical Study Site 126
Seattle, Washington
NGM Clinical Study Site 125
Southlake, Texas
NGM Clinical Study Site 107
St Louis, Missouri
NGM Clinical Study Site 115
Washington D.C., District of Columbia

Other trials in United States

Netherlands6 sites

NGM Clinical Study Site 402
Amsterdam
NGM Clinical Study Site 407
Amsterdam
NGM Clinical Study Site 406
Leiden
NGM Clinical Study Site 401
Nijmegen
NGM Clinical Study Site 404
Rotterdam
NGM Clinical Study Site 405
Utrecht

Other trials in Netherlands

United Kingdom5 sites

NGM Clinical Study Site 505
Birmingham
NGM Clinical Study Site 504
Liverpool
NGM Clinical Study Site 502
London
NGM Clinical Study Site 501
Newcastle
NGM Clinical Study Site 503
Norwich

Other trials in United Kingdom

France1 site

NGM Clinical Study Site 301
Paris

Other trials in France

About this study

Official title: A Phase 2, Randomized, Double Blind, Placebo Controlled, Parallel Group, Multiple Center Study to Evaluate the Safety, Tolerability, and Efficacy of NGM282 Administered for 12 Weeks in Patients With Primary Sclerosing Cholangitis

The purpose of this study is to determine the safety, tolerability, and activity of NGM282 in patients with Primary Sclerosing Cholangitis.

Conditions: Primary Sclerosing Cholangitis
Being tested: NGM282, Placebo
Medical subject headings: Cholangitis, Sclerosing

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NGM282Biological
PlaceboOther

Study groups in NCT02704364
Study group	Role	What they receive
NGM282 Dose 1	Experimental	NGM282 Dose 1 Receives: Biological: NGM282
NGM282 Dose 2	Experimental	NGM282 Dose 2 Receives: Biological: NGM282
Placebo	Active comparator	Placebo Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Mean Change From Baseline in Alkaline Phosphatase in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in alkaline phosphatase levels.
Measured: Baseline to Week 12

Secondary outcomes (17)

Mean Percent Change From Baseline in Alkaline Phosphatase in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in alkaline phosphatase levels.
Measured: Baseline to Week 12
Mean Rate of Change in Alkaline Phosphatase in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess rate of change in alkaline phosphatase levels. Serum levels of alkaline phosphatase was measured in a central laboratory using standard enzymatic method. The unit of measure is U/L.
Measured: Baseline to Week 12
Mean Change From Baseline in Aspartate Aminotransferase and Alanine Aminotransferase in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in aspartate aminotransferase and alanine aminotransferase levels.
Measured: Baseline to Week 12
Mean Percent Change From Baseline in Aspartate Aminotransferase and Alanine Aminotransferase in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in aspartate aminotransferase and alanine aminotransferase levels.
Measured: Baseline to Week 12
Mean Change From Baseline in Bilirubin (Direct and Total) in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in bilirubin (direct and total) levels.
Measured: Baseline to Week 12
Mean Percent Change From Baseline in Bilirubin (Direct and Total) in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in bilirubin (direct and total) levels.
Measured: Baseline to Week 12
Mean Change From Baseline in Gamma Glutamyl Transferase in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in gamma glutamyl transferase levels.
Measured: Baseline to Week 12
Mean Percent Change From Baseline in Gamma Glutamyl Transferase in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in gamma glutamyl transferase levels.
Measured: Baseline to Week 12
Mean Change From Baseline in Cholesterol Levels in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in cholesterol levels.
Measured: Baseline to Week 12
Mean Percent Change From Baseline in Cholesterol Levels in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in cholesterol levels.
Measured: Baseline to Week 12
Mean Change From Baseline in 25-Hydroxyvitamin D in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in 25-Hydroxyvitamin D levels.
Measured: Baseline to Week 12
Mean Percent Change From Baseline in 25-Hydroxyvitamin D in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in 25-Hydroxyvitamin D levels.
Measured: Baseline to Week 12
Mean Change From Baseline in Prothrombin International Normalized Ratio in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in Prothrombin levels.
Measured: Baseline to Week 12
Mean Percent Change From Baseline in Prothrombin International Normalized Ratio in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in Prothrombin levels.
Measured: Baseline to Week 12
Mean Change From Baseline in Calprotectin in Patients With Primary Sclerosing Cholangitis
Blood samples were collected to assess changes in Calprotectin levels.
Measured: Baseline to Week 12
Mean Change From Baseline in Enhanced Liver Fibrosis Score in Patients With Primary Sclerosing Cholangitis
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: Baseline to Week 12
Severity of Inflammatory Bowel Disease-Associated Intestinal Symptoms in Patients With Primary Sclerosing Cholangitis
Severity of inflammatory bowel disease (IBD)-associated symptoms and acute cholangitis are summarized with number and percentage. The partial Mayo IBD scale assesses stool frequency, rectal bleeding, and there is a Physician's global assessment. Each section is summed and remission is defined as a total score of 0-1, mild disease 2-4, moderate disease 5-6, and severe disease 7-9. Higher total scores indicate more severe disease. The number of participants are being presented based on the severity of their IBD symptoms.
Measured: Baseline to Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Mar 2016Jun 2017

First posted10 Mar 2016 (estimated)
Study startMar 2016
Primary completionJun 2017
Study completionJun 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
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
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 62 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02704364
Sponsor's protocol ID: 15-0106
Other IDs: 2015-003392-30, 2015
Status last verified: Mar 2025
First submitted: 29 Feb 2016
Last updated: 17 Jun 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Sanyal AJ, Ling L, Beuers U, DePaoli AM, Lieu HD, Harrison SA, Hirschfield GM. Potent suppression of hydrophobic bile acids by aldafermin, an FGF19 analogue, across metabolic and cholestatic liver diseases. JHEP Rep. 2021 Feb 19;3(3):100255. doi: 10.1016/j.jhepr.2021.100255. eCollection 2021 Jun.
PubMed 33898959

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach
Primary Sclerosing Cholangitis
Not yet recruitingPhase 2
FEAST and HOPE: Redox PSC Pilot Study
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RecruitingPhase 3
A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis
Primary Sclerosing Cholangitis
Active, not recruitingPhase 2
A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.
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Active, not recruitingPhase 2
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
Primary Sclerosing Cholangitis

All Primary Sclerosing Cholangitis trials

Study at a glance

From the registry record, last updated by the sponsor on 17 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Randomized, Double Blind, Placebo Controlled, Parallel Group, Multiple Center Study to Evaluate the Safety, Tolerability, and Efficacy of NGM282 Administered for 12 Weeks in Patients With Primary Sclerosing Cholangitis
ClinicalTrials.gov ID: NCT02704364
Acronym / sponsor ID: 15-0106
Conditions: Primary Sclerosing Cholangitis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 62 participants (actual)
Lead sponsor: NGM Biopharmaceuticals, IncIndustry (pharma, biotech, devices)
Study start: Mar 2016 (actual)
Primary completion: Jun 2017 (actual)
Study completion: Jun 2017 (actual)
First posted: 10 Mar 2016 (estimated)
Results: Posted 17 Jun 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of this study is to determine the safety, tolerability, and activity of NGM282 in patients with Primary Sclerosing Cholangitis.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 3

Arm	Type	Receives
NGM282 Dose 1	Experimental	Biological: NGM282NGM282 Dose 1
NGM282 Dose 2	Experimental	Biological: NGM282NGM282 Dose 2
Placebo	Active comparator	Other: PlaceboPlacebo

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (1 listed)

Confirmed diagnosis of PSC

Key exclusion criteria (2 listed)

Clinically significant acute or chronic liver disease of an etiology other than PSC
Secondary or IgG4 related sclerosing cholangitis

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
NGM282	Biological	NGM282 Dose 1, NGM282 Dose 2
Placebo	Other	Placebo

Primary outcome

Mean Change From Baseline in Alkaline Phosphatase in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Blood samples were collected to assess changes in alkaline phosphatase levels.

17 secondary outcomes

Mean Percent Change From Baseline in Alkaline Phosphatase in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Rate of Change in Alkaline Phosphatase in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Change From Baseline in Aspartate Aminotransferase and Alanine Aminotransferase in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Percent Change From Baseline in Aspartate Aminotransferase and Alanine Aminotransferase in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Change From Baseline in Bilirubin (Direct and Total) in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Percent Change From Baseline in Bilirubin (Direct and Total) in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Change From Baseline in Gamma Glutamyl Transferase in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Percent Change From Baseline in Gamma Glutamyl Transferase in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Change From Baseline in Cholesterol Levels in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Percent Change From Baseline in Cholesterol Levels in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Change From Baseline in 25-Hydroxyvitamin D in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Percent Change From Baseline in 25-Hydroxyvitamin D in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Change From Baseline in Prothrombin International Normalized Ratio in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Percent Change From Baseline in Prothrombin International Normalized Ratio in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Change From Baseline in Calprotectin in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Mean Change From Baseline in Enhanced Liver Fibrosis Score in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12
Severity of Inflammatory Bowel Disease-Associated Intestinal Symptoms in Patients With Primary Sclerosing Cholangitis
Time frame: Baseline to Week 12

Results and safety

Results posted to the registry on 17 Jun 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
NGM282 1.0 mg	21	19
NGM282 3.0 mg	21	18
Placebo	20	19

Primary outcome: Mean Change From Baseline in Alkaline Phosphatase in Patients With Primary Sclerosing Cholangitis

Time frame: Baseline to Week 12. Mean (Standard Error). Unit: International units/Liter

Measure	NGM282 1.0 mg n = 21	NGM282 3.0 mg n = 20	Placebo n = 20
Value	25.6 (100.3)	-9.8 (101.3)	-0.6 (79.5)

NGM282 1.0 mg vs Placebo: Hodges-Lehmann estimate: -14.0 (95% CI -68.0 to 28.0), p = 0.434, Wilcoxon (Mann-Whitney)
NGM282 3.0 mg vs Placebo: Hodges-Lehmann estimate: 13.0 (95% CI -41.0 to 71.0), p = 0.646, Wilcoxon (Mann-Whitney)

Population analysed: Mean change from baseline in ALP was assessed in participants with available data in the Full Analysis Set.

17 secondary outcomes are reported in the full results.

Adverse events

Time frame: Treatment-emergent adverse events were collected from Day 1 up to Week 16.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
NGM282 1.0 mg	1 of 21 (4.8%)	17 of 21 (81%)	0 of 21
NGM282 3.0 mg	2 of 21 (9.5%)	20 of 21 (95.2%)	0 of 21
Placebo	0 of 20 (0%)	18 of 20 (90%)	0 of 20

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 33898959Sanyal AJ, Ling L, Beuers U, DePaoli AM, Lieu HD, Harrison SA, Hirschfield GM. Potent suppression of hydrophobic bile acids by aldafermin, an FGF19 analogue, across metabolic and cholestatic liver diseases. JHEP Rep. 2021 Feb 19;3(3):100255. doi: 10.1016/j.jhepr.2021.100255. eCollection 2021 Jun.

Related trials for Primary Sclerosing Cholangitis

RecruitingObservational study
Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach
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FEAST and HOPE: Redox PSC Pilot Study
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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.

Stephen J Rossi, PharmD study director

NGM Biopharmaceuticals, Inc

38 sites in 4 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing NGM282 in people with Primary Sclerosing Cholangitis, run by NGM Biopharmaceuticals, Inc at 38 sites in 4 countries.

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 1 inclusion and 2 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: finished, as of the sponsor's last update on 17 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 38 sites in United States, Netherlands, United Kingdom, France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Mean Change From Baseline in Alkaline Phosphatase in Patients With Primary Sclerosing Cholangitis (time frame: Baseline to Week 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02704364, which also lists 1 linked publication.

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 17 Jun 2025.