Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis
NCT ID: NCT02061540
Last Updated: 2019-03-29
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
27 participants
INTERVENTIONAL
2014-03-31
2016-02-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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LUM001
LUM001 administered orally once each day
LUM001
LUM001 oral dose
Interventions
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LUM001
LUM001 oral dose
Eligibility Criteria
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Inclusion Criteria
2. Diagnosis of PSC
3. If inflammatory bowel disease (IBD) is present, disease activity ≤ 2 (normal to moderate), using the physician assessment on the Mayo ulcerative colitis (UC) disease activity score.
4. Patients receiving azathioprine for intestinal bowel disease are eligible to participate in the study provided that they have had no IBD exacerbations for at least 6 months.
5. Females of childbearing potential must have a negative serum pregnancy test \[β human chorionic gonadotropin (β-hCG)\] during screening and negative urine pregnancy test at the baseline/Day 0 visit.
6. Sexually active females must be postmenopausal, surgically sterile, or if premenopausal, be prepared to use an effective method (≤ 1% failure rate) of contraception during the trial.
7. Ability to read and understand English in order to use the study-related questionnaires and the text on the eDiary screen.
8. Must be willing and able to use an eDiary daily for a minimum of 20 weeks.
9. Must digitally accept the licensing agreement in the eDiary software at the outset of the study.
10. Must complete at least 10 eDiary Adult ItchRO reports (AM or PM) during each of two consecutive weeks of the screening period prior to allocation to treatment (maximum possible reports = 14 per week).
11. Access to phone for scheduled calls from study site.
12. Must agree to comply with the study protocol procedures and provide written informed consent.
Exclusion Criteria
2. Presence of a dominant stricture unless brushings and/or biopsies of the stricture are negative for dysplasia or malignancy within 6 months of screening.
3. Surgical or endoscopic biliary tree interventions for treatment of clinically significant strictures within 6 months of screening.
4. IBD flare (Mayo UC disease activity score \> 5 including endoscopic evaluation) within 3 months prior to screening.
5. Secondary cause of sclerosing cholangitis (e.g., choledocholithiasis, post-surgical biliary stricture, intra-arterial chemotherapy, recurrent pancreatitis, IgG4 associated cholangiopathy, AIDS cholangiopathy).
6. AST or ALT ≥ 5 x ULN at screening.
7. History or presence of any other concomitant significant liver disease as assessed by the Investigator.
8. Medical conditions that may cause nonhepatic increases in ALP (e.g., Paget's disease).
9. Known history of human immunodeficiency virus (HIV) infection.
10. The anticipated need for a surgical procedure within 20 weeks from randomization.
11. Any female who is pregnant or lactating or who is planning to become pregnant within 20 weeks of randomization.
12. History of cancer, except for basal or squamous cell carcinoma of the skin, or with any laboratory or physical exam or diagnostic procedure finding suggestive of current malignancy.
13. Family history of any documented hereditary cancer syndrome.
14. History of alcohol or other substance abuse within 1 year prior to screening.
15. Receipt of an investigational drug, biologic, or medical device within 30 days prior to Screening, or 5 half-lives of the study agent, whichever is longer.
16. History of noncompliance with medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to noncompliance with the study protocol.
17. Any other conditions or abnormalities which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
18 Years
80 Years
ALL
No
Sponsors
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Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Mirum
Locations
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Scripps Clinic
La Jolla, California, United States
University of California at Davis
Sacramento, California, United States
University of Colorado
Aurora, Colorado, United States
University of Miami
Miami, Florida, United States
Duke University
Durham, North Carolina, United States
University of Calgary Liver Unit
Calgary, Alberta, Canada
University Health Network, Toronto Western Hospital
Toronto, Ontario, Canada
University of Birmingham
Birmingham, England, United Kingdom
Royal Free Hospital
London, , United Kingdom
Countries
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References
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Bowlus CL, Eksteen B, Cheung AC, Thorburn D, Moylan CA, Pockros PJ, Forman LM, Dorenbaum A, Hirschfield GM, Kennedy C, Jaecklin T, McKibben A, Chien E, Baek M, Vig P, Levy C. Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study. Hepatol Commun. 2023 May 15;7(6):e0153. doi: 10.1097/HC9.0000000000000153. eCollection 2023 Jun 1.
Other Identifiers
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2014-005558-21
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
LUM001-401
Identifier Type: -
Identifier Source: org_study_id
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