New pill shows promise for rare liver disease
NCT ID NCT03333928
First seen Jun 27, 2026 · Last updated Aug 26, 2026 · Updated 2 times
Summary
This study tested a drug called HTD1801 in 59 adults with primary sclerosing cholangitis (PSC), a rare liver disease. Participants took either a low dose, a high dose, or a placebo for 18 weeks. The main goal was to see if the drug could lower a liver enzyme called ALP, which is a marker of liver damage.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- HTD1801 (a drug taken as tablets)
- What this could lead to
- If it works, this could point toward a treatment to control PSC and improve liver health.
- What could go wrong
- This is an early Phase 2 trial with only 59 people, so results may not apply to everyone. The drug may not work better than placebo or could have side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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59 people
The number who actually took part.
- Started
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Feb 2018
- Finished
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Aug 2020
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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: 1. 5-amino salicylic acid drugs, 2. azathioprine, 3. 6-mercaptopurine, or methotrexate 4. 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. Exclusion Criteria: * 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; * 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;
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Arizona Liver Health
Chandler, Arizona, 85224, United States
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Arizona Liver Health
Tuscon, Arizona, 85711, United States
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Aspen Woods Clinic
Calgary, Alberta, T3H 0V5, Canada
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Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
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Cumberland Research Associates
Fayetteville, North Carolina, 28304, United States
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Florida Research Institute
Lakewood Rch, Florida, 34211, United States
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Fresno Clinical Research Center
Fresno, California, 93720, United States
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Gastro One
Germantown, Tennessee, 38138, United States
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Gastrointestinal Associates
Flowood, Mississippi, 39232, United States
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Keck School of Medicine of USC
Los Angeles, California, 90033, United States
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Mercy Medical Center
Baltimore, Maryland, 21202, United States
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Michigan Medicine University of Michigan
Ann Arbor, Michigan, 48109, United States
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Mount Sinai - Icahn School of Medicine
New York, New York, 10029, United States
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Pinnacle Clinical Research
Austin, Texas, 78746, United States
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Pinnacle Clinical Research
San Antonio, Texas, 78229, United States
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South Denver Gastroenterology, PC
Englewood, Colorado, 80113, United States
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Swedish Medical Center
Seattle, Washington, 98104, United States
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Toronto Centre for Liver Disease, Toronto General Hospital
Toronto, Ontario, M5G 2C4, Canada
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University of Colorado, Denver
Aurora, Colorado, 80045, United States
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University of Miami
Miami, Florida, 33136, United States
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University of Washington
Seattle, Washington, 98104, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
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Wake Forest Baptist Health
Winston-Salem, North Carolina, 27157, United States
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Walter Reed National Military Medical Center
Bethesda, Maryland, 20889, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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Yale School of Medicine
New Haven, Connecticut, 06520, United States
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