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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03333928
Recruitment Status : Recruiting
First Posted : November 7, 2017
Last Update Posted : July 26, 2019
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)
Estimated Enrollment : 90 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
Estimated Primary Completion Date : October 12, 2019
Estimated Study Completion Date : February 3, 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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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Contact: S Gamble, Ph. D. 301-801-8815

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United States, Arizona
The Institute for Liver Health Recruiting
Chandler, Arizona, United States, 85224
Contact: Anita Kohli, MD    480-686-8874   
Contact: Dana M Robledo, MD,MBA    480-686-8874   
The Institute for Liver Health Recruiting
Tucson, Arizona, United States, 86711
Contact: Anita Kohli, MD    480-686-8874   
Contact: Mario A Gutierrez Casale, MD, MPH    520-274-1224   
United States, California
Fresno Clinical Research Center Recruiting
Fresno, California, United States, 93720
Contact: Muhammad Y Sheikh, MD    559-970-3441   
Contact: Hina Fatima, SC    559-826-2200   
Cedars-Sinai Medical Center Recruiting
Los Angeles, California, United States, 90048
Contact: Laura Sarmiento, CCRP    310-423-4295   
Contact: Jenny Park, CCRP    310-423-8762   
United States, Colorado
University of Colorado, Denver Recruiting
Aurora, Colorado, United States, 80045
Contact: Joselin Gonzalez-Diaz       JOSELIN.GONZALEZ-DIAZ@UCDENVER.EDU   
Contact: Lisa Forman, MD    720-848-2292   
Principal Investigator: Lisa Forman, MD         
United States, Connecticut
Yale School of Medicine Recruiting
New Haven, Connecticut, United States, 06520
Contact: Marina Silveira, MD    203-737-6060   
Contact: Laura Cusack    203-737-6839   
Principal Investigator: Marina Silveira, MD         
United States, Florida
Florida Research Institute Recruiting
Lakewood Ranch, Florida, United States, 34211
Contact: Tracy L Balentine, RN, MS, CCRC    941-727-7772   
Contact: Johanna Valentin, MBA    941-727-7772   
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Cynthia Levy, MD    305-243-4615   
Contact: Tiffannia T Grant    305-243-4649   
Principal Investigator: Cynthia Levy, MD         
United States, Kentucky
University of Louisville Recruiting
Louisville, Kentucky, United States, 40202
Contact: Karen Beatty, RN    502-852-3383   
Contact: Matthew Cave, MD    502-852-6189   
Principal Investigator: Matthew C. Cave, MD         
United States, Maryland
Mercy Medical Center Recruiting
Baltimore, Maryland, United States, 21202
Contact: Polly Robarts    410-843-2075   
Contact: Sung Cho    410-576-5389   
Principal Investigator: Paul Thuluvath, MD         
Walter Reed National Military Medical Center Recruiting
Bethesda, Maryland, United States, 20889
Contact: Dawn M Torres, MD    301-295-4451   
Contact: Jeffrey Bloch, MS    301-319-8713   
United States, Mississippi
Gastrointestinal Associates Recruiting
Flowood, Mississippi, United States, 39232
Contact: Nolan Runnels, RN    601-863-0395   
Contact: Lynda Richards    601-863-0395   
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Michael Klebert, PhD    314-454-0058   
Contact: Kevin Korenblat, MD    314-454-8141   
Principal Investigator: Kevin Korenblat, MD         
United States, New York
Mount Sinai - Icahn School of Medicine Recruiting
New York, New York, United States, 10029
Contact: Jawad Ahmad, MD    212-241-0255   
Contact: Brandy Haydel    212-241-0255   
United States, North Carolina
Cumberland Research Associates Recruiting
Fayetteville, North Carolina, United States, 28304
Contact: Christina M Steele    910-484-8163   
Contact: Loretta Joyce    910-484-8163   
Wake Forest Baptist Health Recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Dee W. Faust    336-713-1442   
Contact: Sean R. Rudnick, MD    336-713-7381   
Principal Investigator: Sean R. Rudnick, MD         
United States, Tennessee
Gastro One Recruiting
Germantown, Tennessee, United States, 38138
Contact: Alice Bunnell    901-682-2450   
Contact: Amanda Berry, NP    901-309-6035   
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37212
Contact: Mary C. Vozar, RN, BSN, MSN    615-936-1745   
Contact: Tina C. Higginbotham, MPA    615-322-4643   
Principal Investigator: Michael Porayko, MD         
United States, Texas
Pinnacle Clinical Research Recruiting
Austin, Texas, United States, 78746
Contact: Nadege Gunn, MD    512-384-4560   
Pinnacle Clinical Research Recruiting
San Antonio, Texas, United States, 78229
Contact: Leslie M DeHart, MSN, FNP-C    210-982-0320 ext 1451   
Contact: Shannon Patteson, FNPC   
United States, Washington
Swedish Medical Center Recruiting
Seattle, Washington, United States, 98104
Contact: Brook A Quist, CRC    206-386-2427   
Contact: Jodie Davila, RN    206-215-4384   
Principal Investigator: Kris V. Kowdley, MD         
University of Washington Recruiting
Seattle, Washington, United States, 98104
Contact: Lynn Gemmell, MBA, CRC    206-744-7063   
Contact: Peter Nguyen    206-744-3402   
Principal Investigator: Charles Landis, MD, PhD         
Sponsors and Collaborators
HighTide Biopharma Pty Ltd

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Responsible Party: HighTide Biopharma Pty Ltd Identifier: NCT03333928     History of Changes
Other Study ID Numbers: HTD1801.PCT003
First Posted: November 7, 2017    Key Record Dates
Last Update Posted: July 26, 2019
Last Verified: July 2019

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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