Primary Biliary CholangitisPhase 2PRONTO-PBCNCT04604652
Open-Label Study of HTD1801 in Adult Subjects With Primary Biliary Cholangitis
A Phase 2 study testing HTD1801 (BUDCA) in people with Primary Biliary Cholangitis and Primary Biliary Cirrhosis, run by HighTide Therapeutics (Hong Kong) Limited at 12 sites in United States.
Trial summary
The purpose of this open-label study is to evaluate the safety and tolerability of HDT1801 (BUDCA) over 12 weeks in adult subjects with PBC who have an inadequate response to standard therapy. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Biliary Cholangitis, Primary Biliary Cirrhosis, Cholangitis
- What is being tested?
- HTD1801 (BUDCA)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 12 sitesUnited States
- Study dates
- 27 May 2021to 31 May 2022
- 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
- Have a clinical diagnosis of PBC as confirmed by patient history consistent with the American Association for the Study of Liver Diseases (AASLD) Practice Guideline confirmed by two of the following three criteria:
- Biochemical evidence of cholestasis with elevation of ALP activity
- Presence of antimitochondrial antibody (AMA)
- Histopathologic evidence of non-suppurative cholangitis and destruction of small or medium-sized bile ducts if biopsy performed Note: historical AMA and liver biopsy data may be used but must be recorded in source documentation.
- Has been taking a stable, adequate dose of at least (13-15 mg/kg/day) of UDCA for at least 6 months with a serum ALP of at least ≥1.5 × ULN at any time after being on UDCA for >6 months (historical value) and at Screening. If the historical ALP was obtained less than 6 months prior to study start as part of standard of care, the subject may be screened and a second ALP value should be obtained as part of screening, There must be at least a 4-week interval between the ALP values and the ALP values must be ≥1.5 × ULN
- If the subject is taking cholestyramine or other bile acid sequestrant for pruritus, must be on a stable dose no more than once a day for at least 8 weeks prior to Baseline visit. Must be willing and able to take cholestyramine at least 2 hours before or after study medication
- Females of child-bearing potential and males participating in the study must either agree to use at least two approved barrier methods of contraception or be completely abstinent from sexual intercourse, if this is their usual and preferred lifestyle, throughout the duration of the study and for three months after stopping study drug. Females who are postmenopausal must have appropriate documentation
- Able to provide consent
Who cannot join
- Uncontrolled concomitant autoimmune hepatitis (AIH). Subject should be on no more than 5 mg per day of prednisone (or equivalent dose for other corticosteroids) or no more than 150 mg per day of azathioprine at stable doses and serum ALT should be ≤ 5 × ULN. Enrollment of subjects with controlled AIH will be limited to a total of 5 subjects.
- History of alcohol or substance abuse
- Prior liver transplantation or currently listed for liver transplantation
- History of chronic viral hepatitis, types B or C
- Platelet count ≤150,000/mm3, albumin <3.0 g/dL, International Normalized Ratio (INR) >1.2, or a history of ascites, or encephalopathy, or history of variceal bleeding
- Total bilirubin >1.3 × ULN unless subject has Gilbert Syndrome. If subject has increased total bilirubin due to Gilbert's Syndrome, then direct bilirubin should be <0.3 mg/dL.
Show all 22Show fewer
- Hemoglobin <10 g/dL for males or females
- Serum TSH level <0.1 or >10 u/mL (subject may be re-screened if hyper- or hypothyroidism has been corrected)
- Renal impairment with eGFR <60 ml/min (CKD stages 3, 4 or 5)
- Human immunodeficiency virus (HIV)-1 or HIV-2 infection by history
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency
- History of malignancy within the past 2 years or ongoing malignancy other than basal cell carcinoma, or resected noninvasive cutaneous squamous cell carcinoma
- Active, serious infections that require parenteral antibiotic or antifungal therapy within 30 days prior to Screening
- Major surgical procedure within 30 days of Screening or prior solid organ transplantation
- Females who are pregnant or breastfeeding
- Current or anticipated treatment with radiation therapy, cytotoxic chemotherapeutic agents, and immune-modulating agents (such as interleukins, interferons)
- Diseases that may result in increased serum ALP activities from sources other than the biliary system (e.g., Paget's disease of bone, osteomalacia)
- Allergy to the clinical trial material or its components
- Having received any experimental medications within 28 days prior to Screening
- Use of bezafibrate or fenofibrate within 28 days prior to first day of IP dosing
- Use of obeticholic acid (OCA) within 28 days prior to first day of IP dosing
- Any other clinically significant disorders or prior therapy that, in the opinion of the investigator, would make the subject unsuitable for the study or unable to comply with the dosing and protocol requirements
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Piedmont Healthcare
Atlanta, Georgia, 30309
Massachusetts General Hospital
Boston, Massachusetts, 02114
Baylor Research Institute
Dallas, Texas, 75246
Henry Ford Health Services
Detroit, Michigan, 48202
Northshore University Hospital
Manhasset, New York, 11030
University of Miami Schiff Center for Liver Disease
Miami, Florida, 33136
Liver Institute of Virginia
Newport News, Virginia, 23602
University GI
Providence, Rhode Island, 02905
Bon Secours Liver Institute of Richmond
Richmond, Virginia, 23226
The Texas Liver Institute
San Antonio, Texas, 78215
Liver Institute Northwest
Seattle, Washington, 98105
St. Louis University
St Louis, Missouri, 63104
About this study
Official title: A Phase 2 Open Label, Proof of Concept Study of HTD1801 (BUDCA) in Adult Subjects With Primary Biliary Cholangitis (PBC) and an Inadequate Response to Standard Therapy - PRONTO-PBC
The purpose of this open-label study is to evaluate the safety and tolerability of HDT1801 (BUDCA) over 12 weeks in adult subjects with PBC who have an inadequate response to standard therapy. Inadequate response is defined as persistently elevated serum alkaline phosphatase at greater than or equal to1.5 times the upper limits of normal for the testing lab in spite of having been on adequate doses of standard therapy with UDCA (ursodeoxycholic acid) at 13-15 mg/kg for at least 6 months.
- Conditions
- Primary Biliary Cholangitis, Primary Biliary Cirrhosis, Cholangitis, Cholestasis, Biliary Tract Diseases, Bile Duct Stricture
- Being tested
- HTD1801 (BUDCA)
- Keywords
- Open-label
- Medical subject headings
- Liver Cirrhosis, Biliary, Cholangitis, Cholestasis, Biliary Tract Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HTD1801 (BUDCA)Drug
HTD1801 (BUDCA) 250 mg tablets. Dose 1000 mg twice daily with food for 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Open-label | Experimental | HTD1801 (BUDCA) 250 mg tablets. Dosed at 1000 mg BID with food. Receives: Drug: HTD1801 (BUDCA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in Alkaline Phosphatase (ALP) at Week 12 Compared to Baseline
To evaluate the effects of HTD1801 on serum ALP in adult subjects with PBC who have experienced an inadequate response to standard therapy.
Inadequate response is defined as ALP ≥1.5 × upper limit of normal (ULN) despite having been on adequate doses of ursodeoxycholic acid (UDCA) for at least 6 months. A reduction in ALP represents an improvement.
Measured: Baseline to Week 12
Secondary outcomes (6)
Change in Total Bilirubin From Baseline to Week 12
To evaluate the effects of HTD1801 on serum markers of cholestasis. A reduction in total bilirubin represents an improvement in a marker of cholestasis.
Measured: Baseline to Week 12
Change in Serum Gamma-glutamyl Transferase (GGT) From Baseline to Week 12
To evaluate the effects of HTD1801 on serum markers of cholestasis. A decrease in GGT represents an improvement in a marker of cholestasis.
Measured: Baseline to Week 12
Change in Serum Total Cholesterol From Baseline to Week 12
To evaluate the effects of HTD1801 on serum lipids. A reduction in total cholesterol represents an improvement in serum lipids.
Measured: Baseline to Week 12
Change in Immunoglobulin M (IgM) From Baseline to Week 12
To evaluate the effects of HTD1801 on serum markers of inflammation. A reduction in IgM represents an improvement in a marker of inflammation.
Measured: Baseline to Week 12
Change in GLOBE Score Between Baseline and Week 12
The GLOBE score is a validated risk assessment tool providing an estimate of transplant-free survival for patients with PBC. It was developed by the GLOBAL PBC Study Group using Cox regression model on over 4,000 patients with PBC. Lower GLOBE score predicts lower risk. It is calculated using age and levels of ALP, total bilirubin, platelets, and albumin.
Measured: Baseline to Week 12
Change in Pruritus as Measured by Pruritus Visual Analog Score (VAS) From Baseline to Week 12
Pruritus Visual Analog Scale (VAS) is a self-reported instrument for measurement of itch intensity using 24-hour recall period. Subjects were asked to rate the average intensity of their itch on a 10 cm horizontal line ranging from 0 cm (no itch) to 10 cm (worst imaginable itch). A decrease in the Pruritus VAS represents an improvement in itch intensity.
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.
- First posted27 Oct 2020
- Study start27 May 2021
- Primary completion31 May 2022
- Study completion31 May 2022
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.
- Other trials for Primary Biliary 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
- Not applicable
- Intervention model
- Single group
- Model description
- Single group open label
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- HighTide Therapeutics (Hong Kong) Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Adrian DiBisceglie, MD, Study director, HighTide Therapeutics Biopharma Pty.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04604652
- Sponsor's protocol ID
- HTD1801.PCT013
- Acronym
- PRONTO-PBC
- Status last verified
- Aug 2026
- First submitted
- 16 Oct 2020
- Last updated
- 25 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
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 Phase 2 Open Label, Proof of Concept Study of HTD1801 (BUDCA) in Adult Subjects With Primary Biliary Cholangitis (PBC) and an Inadequate Response to Standard Therapy - PRONTO-PBC
- ClinicalTrials.gov ID
- NCT04604652
- Acronym / sponsor ID
- PRONTO-PBC / HTD1801.PCT013
- Conditions
- Primary Biliary Cholangitis, Primary Biliary Cirrhosis, Cholangitis, Cholestasis, Biliary Tract Diseases, Bile Duct Stricture
- Keywords
- Open-label
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- HighTide Therapeutics (Hong Kong) LimitedIndustry (pharma, biotech, devices)
- Study start
- 27 May 2021 (actual)
- Primary completion
- 31 May 2022 (actual)
- Study completion
- 31 May 2022 (actual)
- First posted
- 27 Oct 2020 (actual)
- Results
- Posted 24 Apr 2024
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 open-label study is to evaluate the safety and tolerability of HDT1801 (BUDCA) over 12 weeks in adult subjects with PBC who have an inadequate response to standard therapy. Inadequate response is defined as persistently elevated serum alkaline phosphatase at greater than or equal to1.5 times the upper limits of normal for the testing lab in spite of having been on adequate doses of standard therapy with UDCA (ursodeoxycholic acid) at 13-15 mg/kg for at least 6 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single group open label
| Arm | Type | Receives |
|---|---|---|
| Open-label | Experimental | Drug: HTD1801 (BUDCA)HTD1801 (BUDCA) 250 mg tablets. Dosed at 1000 mg BID with food. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Have a clinical diagnosis of PBC as confirmed by patient history consistent with the American Association for the Study of Liver Diseases (AASLD) Practice Guideline confirmed by two of the following three criteria:
- Has been taking a stable, adequate dose of at least (13-15 mg/kg/day) of UDCA for at least 6 months with a serum ALP of at least ≥1.5 × ULN at any time after being on UDCA for >6 months (historical value) and at Screening. If the historical ALP was obtained less than 6 months prior to study start as part of standard of care, the subject may be screened and a second ALP value should be obtained as part of screening, There must be at least a 4-week interval between the ALP values and the ALP values must be ≥1.5 × ULN
- If the subject is taking cholestyramine or other bile acid sequestrant for pruritus, must be on a stable dose no more than once a day for at least 8 weeks prior to Baseline visit. Must be willing and able to take cholestyramine at least 2 hours before or after study medication
- Females of child-bearing potential and males participating in the study must either agree to use at least two approved barrier methods of contraception or be completely abstinent from sexual intercourse, if this is their usual and preferred lifestyle, throughout the duration of the study and for three months after stopping study drug. Females who are postmenopausal must have appropriate documentation
- Able to provide consent
Key exclusion criteria (22 listed)
- Uncontrolled concomitant autoimmune hepatitis (AIH). Subject should be on no more than 5 mg per day of prednisone (or equivalent dose for other corticosteroids) or no more than 150 mg per day of azathioprine at stable doses and serum ALT should be ≤ 5 × ULN. Enrollment of subjects with controlled AIH will be limited to a total of 5 subjects.
- History of alcohol or substance abuse
- Prior liver transplantation or currently listed for liver transplantation
- History of chronic viral hepatitis, types B or C
- Platelet count ≤150,000/mm3, albumin <3.0 g/dL, International Normalized Ratio (INR) >1.2, or a history of ascites, or encephalopathy, or history of variceal bleeding
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 |
|---|---|---|
| HTD1801 (BUDCA)HTD1801 (BUDCA) 250 mg tablets. Dose 1000 mg twice daily with food for 12 weeks. | Drug | Open-label |
Primary outcome
Percent Change in Alkaline Phosphatase (ALP) at Week 12 Compared to Baseline
Time frame: Baseline to Week 12
To evaluate the effects of HTD1801 on serum ALP in adult subjects with PBC who have experienced an inadequate response to standard therapy. Inadequate response is defined as ALP ≥1.5 × upper limit of normal (ULN) despite having been on adequate doses of ursodeoxycholic acid (UDCA) for at least 6 months. A reduction in ALP represents an improvement.
6 secondary outcomes
Change in Total Bilirubin From Baseline to Week 12
Time frame: Baseline to Week 12
Change in Serum Gamma-glutamyl Transferase (GGT) From Baseline to Week 12
Time frame: Baseline to Week 12
Change in Serum Total Cholesterol From Baseline to Week 12
Time frame: Baseline to Week 12
Change in Immunoglobulin M (IgM) From Baseline to Week 12
Time frame: Baseline to Week 12
Change in GLOBE Score Between Baseline and Week 12
Time frame: Baseline to Week 12
Change in Pruritus as Measured by Pruritus Visual Analog Score (VAS) From Baseline to Week 12
Time frame: Baseline to Week 12.
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.
- Individual participant dataSharing planned: No
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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 DiBisceglie, MD study director
HighTide Therapeutics Biopharma Pty.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HTD1801 (BUDCA) in people with Primary Biliary Cholangitis and Primary Biliary Cirrhosis, run by HighTide Therapeutics (Hong Kong) Limited at 12 sites in United States.
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 8 inclusion and 22 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 12 sites in United States. 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 24 Apr 2024). 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?
Percent Change in Alkaline Phosphatase (ALP) at Week 12 Compared to Baseline (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?
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.