Phase 2
N=20
To Assess Safety, Tolerability and Efficacy of LJN452 in Patients With Primary Bile Acid Diarrhea.
Primary Bile Acid Diarrhea
Bottom Line
View on ClinicalTrials.gov: NCT02713243 ↗Enrolled (actual)
20
Serious AEs
0.0%
Results posted
May 2019
Primary outcome: Primary: Number of Patients Reported With Adverse Events , Serious Adverse Events and Death. — 9; 14; 0; 4 count of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- LJN452 (Drug); Placebo to LJN452 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Jan 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients Reported With Adverse Events , Serious Adverse Events and Death. |
9; 14; 0; 4; 0; 0 | — |
| PRIMARY Stool Frequency at Baseline, Week 1 (Period 1 & Period 2), Week 2 (Period 1 & Period 2), and Week 1 & 2 Combined |
18.0; 15.9; 20.0; 16.2; 18.8; 17.5 | 0.01 sig |
| PRIMARY Stool Form at Baseline, Week 1 (Period 1 & Period 2), Week 2 (Period 1 & Period 2), and Week 1 & 2 Combined |
5.5; 5.1; 5.3; 5.3; 4.9; 5.1 | 0.533 |
| SECONDARY Area Under the Plasma Concentration-time Profile (AUCtau) of LJN452 |
23.6; 22.1 | — |
| SECONDARY (Cmax) of LJN452 |
1.63; 1.82 | — |
| SECONDARY Time to Reach Maximum Concentration After Drug Administration (Tmax) |
5.00; 5.00 | — |
| SECONDARY Total Dose of Rescue Medication Used at Baseline, Week 1 (Period 1 & Period 2), Week 2 (Period 1 & Period 2), and Week 1 & 2 Combined |
0.9; 1.3; 0.6; 0.7; 0.5; 0.7 | — |
Summary
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.
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT02713243). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.