Alagille Syndrome Clinical Trial
— ITCHOfficial title:
The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease
Verified date | March 2019 |
Source | Mirum Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.
Status | Completed |
Enrollment | 37 |
Est. completion date | November 16, 2016 |
Est. primary completion date | November 16, 2016 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 12 Months to 18 Years |
Eligibility |
Inclusion Criteria: 1. Diagnosis of Alagille Syndrome 2. Evidence of cholestasis 3. Moderate to severe pruritus 4. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures Exclusion Criteria: 1. Surgical disruption of the enterohepatic circulation 2. Liver transplant 3. History or presence of other concomitant liver disease 4. Females who are pregnant or lactating 5. Known HIV infection |
Country | Name | City | State |
---|---|---|---|
Canada | The Hospital for Sick Children | Toronto | Ontario |
United States | Children's Healthcare of Atlanta | Atlanta | Georgia |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Baylor College of Medicine/Texas Children's Hospital | Houston | Texas |
United States | Riley Hospital for Children | Indianapolis | Indiana |
United States | Children's Hospital Los Angeles | Los Angeles | California |
United States | The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania |
United States | University of Utah | Salt Lake City | Utah |
United States | University of California at San Francisco | San Francisco | California |
United States | Seattle Children's Hospital | Seattle | Washington |
Lead Sponsor | Collaborator |
---|---|
Mirum Pharmaceuticals, Inc. | Childhood Liver Disease Research and Education Network |
United States, Canada,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An AE was 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 related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment. | From the start of study drug administration up to Week 17 | |
Primary | Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus | Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period. | Baseline, Week 13/Early Termination | |
Secondary | Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level | Fasting sBA level was measured by using a liquid chromatography mass spectrometry method. | Baseline, Week 13/Early Termination | |
Secondary | Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels | Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here. | Baseline, Week 13/Early Termination | |
Secondary | Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations | Liver enzyme levels of total bilirubin and direct bilirubin were reported here. | Baseline, Week 13/Early Termination |
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