To Assess Safety, Tolerability and Efficacy of LJN452 in Patients With Primary Bile Acid Diarrhea.

Novartis

Status and phase: Completed; Phase 2

Conditions: Primary Bile Acid Diarrhea

Treatments: Drug: LJN452; Drug: Placebo to LJN452

Study type: Interventional

Funder types: Industry

Identifiers

NCT02713243

CLJN452X2202

2015-003192-30 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to determine whether LJN452 improves the symptoms of bile acid diarrhea and to assess its safety and tolerability profile in patients with primary bile acid diarrhea (pBAD) to guide decision-making regarding further clinical development in this indication.

Enrollment: 20 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• A history of diarrheal symptoms for at least 3 months prior to dosing - Average stool frequency of at least 2 per day when off therapy AND Average stool form of >5 on Bristol Stool Chart.
• Previous laboratory or radiological confirmation of bile acid malabsorption with either fecal bile acid loss OR 7 day 75Selenium homocholic acid taurine (75SeHCAT) retention.
• Age ≥ 18 years.

Key Exclusion Criteria:

• Patients with other diagnoses leading to diarrhea, including colorectal neoplasia, ulcerative colitis, Crohn's disease, celiac disease, chronic pancreatitis, drug-induced diarrhea or active infection AND Patients who have not been investigated by standard clinical assessments to exclude these disorders.
• Treatment with bile acid sequestrants (colestyramine, colestipol, colesevelam) for 2 weeks before the first dose of LJN452. A washout of 14 days for these agents will be allowed before first dosing.
• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant.
• A positive Hepatitis B surface antigen or Hepatitis C test result.
• History of immunodeficiency diseases, including a positive HIV (ELISA and Western blot) test result.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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20 participants in 2 patient groups

LJN452 followed by placebo

Experimental group

Description:

Randomized patients in this arm will receive single oral dose of LJN452 daily for 14 days. There will be a washout period between 7 to 28 days followed by single oral dose of placebo daily for 14 days.

Treatment:

Drug: LJN452

Drug: Placebo to LJN452

Placebo followed by LJN452

Experimental group

Description:

Randomized patients in this arm will receive single oral dose of placebo daily for 14 days. There will be a washout period between 7 to 28 days followed by single oral dose of LJN452 daily for 14 days.

Treatment:

Drug: LJN452

Drug: Placebo to LJN452