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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02061540
• Date of registration: 11/02/2014
• Prospective Registration: Yes
• Primary sponsor: Mirum Pharmaceuticals, Inc.
• Public title: Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis CAMEO
• Scientific 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
• Date of first enrolment: March 2014
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02061540
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Canada, United Kingdom, United States

Contacts

• Name: Study Director
• Affiliation: Mirum

Key inclusion & exclusion 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 [ß human
chorionic gonadotropin (ß-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.



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Sclerosing Cholangitis (PSC)

Intervention(s)

Drug: LUM001

Primary Outcome(s)

Change From Baseline in Fasting Serum Bile Acid Level at Week 14 [Time Frame: Baseline, Week 14]

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [Time Frame: From start of study drug administration until Week 18]

Secondary Outcome(s)

Change From Baseline in Pruritus as Measured by Adult Itch Reported Outcome (ItchRO) Weekly Sum Score [Time Frame: Baseline, Week 14]

Change From Baseline in Liver Enzyme Levels in Serum [Time Frame: Baseline, Week 14]

Change From Baseline in Bilirubin Levels at Week 14 [Time Frame: Baseline, Week 14]

Secondary ID(s)

LUM001-401

2014-005558-21

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 12/04/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02061540