for people ages 1-18 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:
Philip Rosenthal



This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.

Official Title

A Multicenter Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome


Alagille Syndrome Syndrome Liver Diseases Cholestasis LUM001


You can join if…

Open to people ages 1-18

  1. Male or female, 12 months to 18 years of age.
  2. Completed participation in the LUM001-301 protocol.
  3. Females of childbearing potential must have a negative urine pregnancy test at the Baseline Visit.
  4. Sexually active females must be prepared to use an effective method of contraception during the trial.
  5. Caregivers (and age appropriate subjects) must have access to phone for scheduled calls from study site.
  6. Caregivers (and age appropriate subjects) must be willing and able to complete a daily electronic diary.

You CAN'T join if...

  1. Experienced an adverse event or serious adverse event (SAE) related to the study drug during the LUM001-301 protocol that led to the discontinuation of the subject from the core study.
  2. Any conditions or abnormalities which may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
  3. History or presence of gallstones or kidney stones.
  4. History of non-adherence during the subject's participation in the LUM001-301 protocol. Non-adherence is defined by dosing compliance1 of less than 80% in the LUM001-301 protocol.


  • University of California at San Francisco Children's Hospital
    San Francisco California 94143 United States
  • Children's Hospital Los Angeles
    Los Angeles California 90027 United States


in progress, not accepting new patients
Start Date
Completion Date
Phase 2
Lead Scientist
Philip Rosenthal
Study Type
Last Updated
April 2017