Primary Sclerosing Cholangitis (PSC)Phase 2NCT03333928
A POC and Dose-Ranging Study of HTD1801 in PSC Patients
A Phase 2 study testing HTD1801 in people with Primary Sclerosing Cholangitis (PSC), run by HighTide Therapeutics (Hong Kong) Limited at 26 sites in 2 countries.
Trial summary
The study was a dose-ranging, 18-week study comparing two doses of HTD1801 (500 mg BID and 1000 mg BID) to placebo in adult subjects with PSC. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Sclerosing Cholangitis (PSC)
- What is being tested?
- HTD1801
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 59 participants
- Where
- 26 sitesin 2 countries
- Study dates
- 9 Feb 2018to 14 Aug 2020
- 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
- Male or female between 18 and 75 years of age;
- Have a clinical diagnosis of PSC as evident by chronic cholestasis of more than six months duration with either a consistent magnetic resonance cholangiopancreatography (MRCP)/endoscopic retrograde cholangiopancreatography (ERCP) showing sclerosing cholangitis;
- If subjects have Inflammatory Bowel Disease (IBD) they will be eligible to participate. If a subject has IBD, documented evidence of IBD must have been evident by prior endoscopy or in previous medical records for ≥6 months. In addition, subjects may only enter the study with a Partial Mayo Score of 0-4, inclusively. Subjects who are on treatment are allowed, provided they are stable for 3 months if taking:
- 5-amino salicylic acid drugs,
- azathioprine,
- 6-mercaptopurine, or methotrexate
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- biologics;
- Have a serum ALP ≥1.5 × upper limit of normal (ULN);
- Be able to understand and sign a written informed consent form (ICF);
- Subjects receiving allowed concomitant medications need to be on stable therapy for 28 days prior to the Baseline visit, with the exception of ursodeoxycholic acid (UDCA), which should be stable for at least 6 weeks prior to the Baseline visit.
Who cannot join
- Presence of documented secondary sclerosing cholangitis (such as ischemic cholangitis, recurrent pancreatitis, intraductal stone disease, severe bacterial cholangitis, surgical or blunt abdominal trauma, recurrent pyogenic cholangitis, choledocholithiasis, toxic sclerosing cholangitis due to chemical agents, or any other cause of secondary sclerosing cholangitis) on prior clinical investigations;
- Small duct PSC;
- Presence of percutaneous drain or bile duct stent;
- History of cholangiocarcinoma or clinical suspicion of new dominant stricture within 1 year by MRCP/ERCP. Presence of dominant stricture without ERCP evidence of cholangiocarcinoma is acceptable if stable for ≥ 1 year;
- Ascending cholangitis within 60 days prior to Screening;
- History of alcohol or substance abuse or dependence;
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- Prior or planned liver transplantation;
- Presence of alternative causes of chronic liver disease, including alcoholic liver disease, nonalcoholic steatohepatitis, primary biliary cirrhosis, autoimmune hepatitis;
- Platelet count below 125,000/mm3, albumin below 3.0 g/dL, International Normalized Ratio (INR) > 1.2, or a history of ascites, or encephalopathy, or history of esophageal variceal bleeding;
- Severe active IBD or flare in colitis activity within the last 90 days requiring intensification of therapy beyond baseline treatment;
Trial locations and contacts
26 sites in 2 countries.
26 sites in 2 countries
United States24 sites
Michigan Medicine University of Michigan
Ann Arbor, Michigan, 48109
University of Colorado, Denver
Aurora, Colorado, 80045
Pinnacle Clinical Research
Austin, Texas, 78746
Mercy Medical Center
Baltimore, Maryland, 21202
Walter Reed National Military Medical Center
Bethesda, Maryland, 20889
Arizona Liver Health
Chandler, Arizona, 85224
South Denver Gastroenterology, PC
Englewood, Colorado, 80113
Cumberland Research Associates
Fayetteville, North Carolina, 28304
Gastrointestinal Associates
Flowood, Mississippi, 39232
Fresno Clinical Research Center
Fresno, California, 93720
Gastro One
Germantown, Tennessee, 38138
Florida Research Institute
Lakewood Rch, Florida, 34211
Keck School of Medicine of USC
Los Angeles, California, 90033
Cedars-Sinai Medical Center
Los Angeles, California, 90048
University of Miami
Miami, Florida, 33136
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Yale School of Medicine
New Haven, Connecticut, 06520
Mount Sinai - Icahn School of Medicine
New York, New York, 10029
Pinnacle Clinical Research
San Antonio, Texas, 78229
Swedish Medical Center
Seattle, Washington, 98104
University of Washington
Seattle, Washington, 98104
Washington University School of Medicine
St Louis, Missouri, 63110
Arizona Liver Health
Tuscon, Arizona, 85711
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27157
Canada2 sites
Aspen Woods Clinic
Calgary, Alberta, T3H 0V5
Toronto Centre for Liver Disease, Toronto General Hospital
Toronto, Ontario, M5G 2C4
About this study
Official title: A Proof-of-Concept and Dose-Ranging Study Investigating the Efficacy and Safety of HTD1801 in Adult Subjects With Primary Sclerosing Cholangitis (PSC)
The study was a dose-ranging, 18-week study comparing two doses of HTD1801 (500 mg BID and 1000 mg BID) to placebo in adult subjects with PSC.
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Being tested
- HTD1801, 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.
HTD1801Drug
HTD1801 tablets, 250 mg
PlaceboDrug
tablets manufactured to mimic HTD1801 tablets
| Study group | Role | What they receive |
|---|---|---|
| HTD1801 500 mg BID | Active comparator | Not described Receives: Drug: HTD1801 |
| HTD1801 1000 mg BID | Active comparator | Not described Receives: Drug: HTD1801 |
| Placebo BID | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change in Serum Alkaline Phosphatase (ALP) From Baseline to Week 6 in Period 1
Measured: Baseline to Week 6
Secondary outcomes (14)
Percentage of Subjects Who Achieve ALP of <1.5 x ULN at the End of Week 6 (Period 1)
Measured: Baseline to Week 6
Percentage of Subjects Who Achieve a 50% Decrease in ALP at the End of Week 6 (Period 1)
Measured: Baseline to Week 6
Percentage of Subjects Who Normalize ALP at the End of Week 6 (Period 1)
Measured: Baseline to Week 6
Absolute Change in Serum Total Bilirubin at the End of Week 6 (Period 1)
Measured: Baseline to Week 6
Absolute Change in Serum ALP From Week 6 to Week 12 (Period 2)
Measured: Week 6 to Week 12
Percentage of Subjects Who Achieve ALP of <1.5 x ULN at the End of Week 12 (Period 2)
Measured: Week 6 to Week 12
Percentage of Patients Who Achieve a 50% Decrease in ALP at the End of Week 12 (Period 2)
Measured: Week 6 to Week 12
Percentage of Patients Who Normalize ALP at the End of Week 12 (Period 2)
Measured: Week 6 to Week 12
Absolute Change in Serum Total Bilirubin at the End of Week 12 (Period 2)
Measured: Week 6 to Week 12
Absolute Change in Serum ALP From Week 12 to Week 18 (Period 3)
Change in serum ALP between a new baseline at Week 12 and the final value at Week 18 for all subjects following the randomized withdrawal
Measured: Week 12 to Week 18
Percentage of Patients Who Achieve ALP of <1.5 x ULN at the End of Week 18 (Period 3)
The percentage of patients who achieve ALP of <1.5 x ULN at the end of week 18 (Period 3)
Measured: Week 12 to Week 18
Percentage of Patients Who Achieve a 50% Decrease in ALP at the End of Week 18 (Period 3)
Measured: Week 12 to Week 18
Percentage of Subjects Who Normalize ALP at the End of Week 18 (Period 3)
Measured: Week 12 to Week 18
Absolute Change in Serum Total Bilirubin at the End of Week 18 (Period 3)
Measured: Week 12 to Week 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2017
- Study start9 Feb 2018
- Primary completion30 Apr 2020
- Study completion14 Aug 2020
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 Cholangitis (PSC)Studies 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
- Model description
- A primary parallel group comparison for a 6-week treatment period (Period 1), followed by a 6-week dose-controlled extension (Period 2), and followed by a 6-week placebo-controlled randomized withdrawal (Period 3).
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- HighTide Therapeutics (Hong Kong) Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Adrian Di Bisceglie, MD,FACP,FAASLD, Study director, HighTide Therapeutics USA, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03333928
- Sponsor's protocol ID
- HTD1801.PCT003
- Status last verified
- Aug 2026
- First submitted
- 27 Oct 2017
- Last updated
- 25 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Kowdley KV, Forman L, Eksteen B, Gunn N, Sundaram V, Landis C, Harrison SA, Levy C, Liberman A, Di Bisceglie AM, Hirschfield GM. A Randomized, Dose-Finding, Proof-of-Concept Study of Berberine Ursodeoxycholate in Patients With Primary Sclerosing Cholangitis. Am J Gastroenterol. 2022 Nov 1;117(11):1805-1815. doi: 10.14309/ajg.0000000000001956. Epub 2022 Aug 22.
PubMed 36327436
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Proof-of-Concept and Dose-Ranging Study Investigating the Efficacy and Safety of HTD1801 in Adult Subjects With Primary Sclerosing Cholangitis (PSC)
- ClinicalTrials.gov ID
- NCT03333928
- Acronym / sponsor ID
- HTD1801.PCT003
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 59 participants (actual)
- Lead sponsor
- HighTide Therapeutics (Hong Kong) LimitedIndustry (pharma, biotech, devices)
- Study start
- 9 Feb 2018 (actual)
- Primary completion
- 30 Apr 2020 (actual)
- Study completion
- 14 Aug 2020 (actual)
- First posted
- 7 Nov 2017 (actual)
- Results
- Posted 6 Apr 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study was a dose-ranging, 18-week study comparing two doses of HTD1801 (500 mg BID and 1000 mg BID) to placebo in adult subjects with PSC.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- A primary parallel group comparison for a 6-week treatment period (Period 1), followed by a 6-week dose-controlled extension (Period 2), and followed by a 6-week placebo-controlled randomized withdrawal (Period 3).
| Arm | Type | Receives |
|---|---|---|
| HTD1801 500 mg BID | Active comparator | Drug: HTD1801 |
| HTD1801 1000 mg BID | Active comparator | Drug: HTD1801 |
| Placebo BID | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male or female between 18 and 75 years of age;
- Have a clinical diagnosis of PSC as evident by chronic cholestasis of more than six months duration with either a consistent magnetic resonance cholangiopancreatography (MRCP)/endoscopic retrograde cholangiopancreatography (ERCP) showing sclerosing cholangitis;
- If subjects have Inflammatory Bowel Disease (IBD) they will be eligible to participate. If a subject has IBD, documented evidence of IBD must have been evident by prior endoscopy or in previous medical records for ≥6 months. In addition, subjects may only enter the study with a Partial Mayo Score of 0-4, inclusively. Subjects who are on treatment are allowed, provided they are stable for 3 months if taking:
- Have a serum ALP ≥1.5 × upper limit of normal (ULN);
- Be able to understand and sign a written informed consent form (ICF);
Key exclusion criteria (10 listed)
- Presence of documented secondary sclerosing cholangitis (such as ischemic cholangitis, recurrent pancreatitis, intraductal stone disease, severe bacterial cholangitis, surgical or blunt abdominal trauma, recurrent pyogenic cholangitis, choledocholithiasis, toxic sclerosing cholangitis due to chemical agents, or any other cause of secondary sclerosing cholangitis) on prior clinical investigations;
- Small duct PSC;
- Presence of percutaneous drain or bile duct stent;
- History of cholangiocarcinoma or clinical suspicion of new dominant stricture within 1 year by MRCP/ERCP. Presence of dominant stricture without ERCP evidence of cholangiocarcinoma is acceptable if stable for ≥ 1 year;
- Ascending cholangitis within 60 days prior to 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 |
|---|---|---|
| HTD1801HTD1801 tablets, 250 mg | Drug | HTD1801 1000 mg BID, HTD1801 500 mg BID |
| Placebotablets manufactured to mimic HTD1801 tablets | Drug | Placebo BID |
Primary outcome
Absolute Change in Serum Alkaline Phosphatase (ALP) From Baseline to Week 6 in Period 1
Time frame: Baseline to Week 6
14 secondary outcomes
Percentage of Subjects Who Achieve ALP of <1.5 x ULN at the End of Week 6 (Period 1)
Time frame: Baseline to Week 6
Percentage of Subjects Who Achieve a 50% Decrease in ALP at the End of Week 6 (Period 1)
Time frame: Baseline to Week 6
Percentage of Subjects Who Normalize ALP at the End of Week 6 (Period 1)
Time frame: Baseline to Week 6
Absolute Change in Serum Total Bilirubin at the End of Week 6 (Period 1)
Time frame: Baseline to Week 6
Absolute Change in Serum ALP From Week 6 to Week 12 (Period 2)
Time frame: Week 6 to Week 12
Percentage of Subjects Who Achieve ALP of <1.5 x ULN at the End of Week 12 (Period 2)
Time frame: Week 6 to Week 12
Percentage of Patients Who Achieve a 50% Decrease in ALP at the End of Week 12 (Period 2)
Time frame: Week 6 to Week 12
Percentage of Patients Who Normalize ALP at the End of Week 12 (Period 2)
Time frame: Week 6 to Week 12
Absolute Change in Serum Total Bilirubin at the End of Week 12 (Period 2)
Time frame: Week 6 to Week 12
Absolute Change in Serum ALP From Week 12 to Week 18 (Period 3)
Time frame: Week 12 to Week 18
Percentage of Patients Who Achieve ALP of <1.5 x ULN at the End of Week 18 (Period 3)
Time frame: Week 12 to Week 18
Percentage of Patients Who Achieve a 50% Decrease in ALP at the End of Week 18 (Period 3)
Time frame: Week 12 to Week 18
Percentage of Subjects Who Normalize ALP at the End of Week 18 (Period 3)
Time frame: Week 12 to Week 18
Absolute Change in Serum Total Bilirubin at the End of Week 18 (Period 3)
Time frame: Week 12 to Week 18
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36327436Kowdley KV, Forman L, Eksteen B, Gunn N, Sundaram V, Landis C, Harrison SA, Levy C, Liberman A, Di Bisceglie AM, Hirschfield GM. A Randomized, Dose-Finding, Proof-of-Concept Study of Berberine Ursodeoxycholate in Patients With Primary Sclerosing Cholangitis. Am J Gastroenterol. 2022 Nov 1;117(11):1805-1815. doi: 10.14309/ajg.0000000000001956. Epub 2022 Aug 22.
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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.
Adrian Di Bisceglie, MD,FACP,FAASLD study director
HighTide Therapeutics USA, LLC
26 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HTD1801 in people with Primary Sclerosing Cholangitis (PSC), run by HighTide Therapeutics (Hong Kong) Limited at 26 sites in 2 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 10 inclusion and 10 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 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Canada. 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. 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 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 6 Apr 2022). 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?
Absolute Change in Serum Alkaline Phosphatase (ALP) From Baseline to Week 6 in Period 1 (time frame: Baseline to Week 6). 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 NCT03333928, 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 25 Aug 2026.