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Trial record 2 of 4 for:    CAMEO

Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis (CAMEO)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT02061540
First received: February 11, 2014
Last updated: February 27, 2017
Last verified: February 2017
  Purpose
The study is an open-label study in adults with primary sclerosing cholangitis to evaluate the safety, tolerability, and effect of 14-weeks of daily dosing of LUM001.

Condition Intervention Phase
Primary Sclerosing Cholangitis (PSC) Drug: LUM001 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: A Pilot, Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Primary Sclerosing Cholangitis

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: From start of study drug administration until Week 18 ]
    An Adverse Event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered to be related to the investigational drug product. TEAEs were AEs with a start date on or after the first dose of investigational product and started prior to the last dose of investigational product plus 14 days.

  • Change From Baseline in Fasting Serum Bile Acid Level at Week 14 [ Time Frame: Baseline, Week 14 ]
    Serum bile acid levels were evaluated using blood samples collected.


Secondary Outcome Measures:
  • Change From Baseline in Liver Enzyme Levels in Serum [ Time Frame: Baseline, Week 14 ]
    Levels of liver enzymes such as Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (ALP) in serum were evaluated.

  • Change From Baseline in Bilirubin Levels at Week 14 [ Time Frame: Baseline, Week 14 ]
    Total Bilirubin and Direct (Conjugated) Bilirubin levels were evaluated.

  • Change From Baseline in Pruritus as Measured by Adult Itch Reported Outcome (ItchRO) Weekly Sum Score [ Time Frame: Baseline, Week 14 ]
    The Adult ItchRO instrument was completed twice daily using an electronic diary (eDiary). Each morning and evening score had a range from 0-10, with the higher score indicating increasing itch severity. The following was used for assessing the Adult ItchRO daily score: The score which represented the most severe itching for the day (morning or evening) was taken for each day as the daily score (maximum daily score of 10); If only 1 of the 2 scores was available for the day, the score that was available was used as the daily score; If both the morning and the evening scores were missing, the score was considered missing for the day.


Other Outcome Measures:
  • Change From Baseline for Other Biochemical Markers of Cholestasis: Total Cholesterol, Low Density Lipoprotein Cholesterol [ Time Frame: Baseline, Week 14 ]
    Total cholesterol (TC) level and low density lipoprotein cholesterol (LDLC) level were considered as biochemical markers of cholestasis.


Enrollment: 27
Study Start Date: March 2014
Study Completion Date: February 2016
Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM001
LUM001 administered orally once each day
Drug: LUM001
LUM001 oral dose

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female subjects between the ages of 18-80 years, inclusive.
  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:

  1. Small duct PSC (clinical biochemical and histological features compatible with PSC, but having a normal cholangiogram).
  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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02061540

Locations
United States, California
Scripps Clinic
: La Jolla, California, United States, 92037
University of California at Davis
Sacramento, California, United States, 95817
United States, Colorado
University of Colorado
Aurora, Colorado, United States, 80045
United States, Florida
University of Miami
Miami, Florida, United States, 33136
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27710
Canada, Alberta
University of Calgary Liver Unit
Calgary, Alberta, Canada, T2N 4Z6
Canada, Ontario
University Health Network, Toronto Western Hospital
Toronto, Ontario, Canada, M5T 2S8
United Kingdom
University of Birmingham
Birmingham, England, United Kingdom, B15 2TT
Royal Free Hospital
London, United Kingdom, NW3 2QG
Sponsors and Collaborators
Shire
Investigators
Study Director: Shire Study Physican Shire
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02061540     History of Changes
Other Study ID Numbers: LUM001-401
2014-005558-21 ( EudraCT Number )
Study First Received: February 11, 2014
Results First Received: December 21, 2016
Last Updated: February 27, 2017

Additional relevant MeSH terms:
Cholangitis
Cholangitis, Sclerosing
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases

ClinicalTrials.gov processed this record on July 25, 2017