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A POC and Dose-Ranging Study of HTD1801 in PSC Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03333928
Recruitment Status : Completed
First Posted : November 7, 2017
Last Update Posted : October 20, 2020
Information provided by (Responsible Party):
HighTide Biopharma Pty Ltd

Brief Summary:
This study evaluates HTD1801 in the treatment of Primary Sclerosing Cholangitis (PSC) in adults. The subjects will be randomised to 3 treatment groups, to receive 500mg, 1000mg and placebo, bid.

Condition or disease Intervention/treatment Phase
Primary Sclerosing Cholangitis (PSC) Drug: HTD1801 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 59 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: A primary parallel group comparison for a 6-week treatment, followed by a 6-week dose-controlled extension, and followed by a 6-week random withdrawal.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Actual Study Start Date : February 9, 2018
Actual Primary Completion Date : April 30, 2020
Actual Study Completion Date : August 14, 2020

Arm Intervention/treatment
Active Comparator: 500mg HTD1801, bid Drug: HTD1801
HTD1801 tablets, 250mg

Active Comparator: 1000mg HTD1801, bid Drug: HTD1801
HTD1801 tablets, 250mg

Placebo Comparator: placebo, bid Drug: Placebo
tablets manufactured to mimic HTD1801 tablets

Primary Outcome Measures :
  1. change of alkaline phosphatase (ALP) [ Time Frame: 6 weeks ]

Secondary Outcome Measures :
  1. change of alkaline phosphatase (ALP) [ Time Frame: up to 18 weeks ]
  2. frequency and severity of adverse events [ Time Frame: 6 weeks ]
  3. frequency and severity of adverse events [ Time Frame: up to 18 weeks ]

Other Outcome Measures:
  1. PK/PD (substudy) [ Time Frame: 6 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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;

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03333928

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Sponsors and Collaborators
HighTide Biopharma Pty Ltd
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Study Director: Adrian Di Bisceglie, MD,FACP,FAASLD HighTide Therapeutics USA, LLC
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Responsible Party: HighTide Biopharma Pty Ltd Identifier: NCT03333928    
Other Study ID Numbers: HTD1801.PCT003
First Posted: November 7, 2017    Key Record Dates
Last Update Posted: October 20, 2020
Last Verified: October 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cholangitis, Sclerosing
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases