Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

NCT02057692 | Completed Phase 2 Results DMC

• 1 other identifier: LUM001-301
• Study Type: interventional
• Target: 37
• Locations: 2 countries
• Sites: 13

Brief Summary

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.

Conditions

Alagille Syndrome

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level

  Baseline, Week 13/Early Termination

• Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels

  Baseline, Week 13/Early Termination

• Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations

  Baseline, Week 13/Early Termination

Other Outcomes (1)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

  From the start of study drug administration up to Week 17

Study Arms (2)

LUM001

EXPERIMENTAL

LUM001 for oral administration

Drug: LUM001

Placebo

PLACEBO COMPARATOR

Placebo administered orally once each day

Drug: Placebo

Interventions

LUM001 DRUG

LUM001 administered orally

LUM001

Placebo DRUG

Placebo administered orally

Placebo

Eligibility Criteria

• Age: 12 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of Alagille Syndrome
• Evidence of cholestasis
• Moderate to severe pruritus
• Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures

You may not qualify if:

• Surgical disruption of the enterohepatic circulation
• Liver transplant
• History or presence of other concomitant liver disease
• Females who are pregnant or lactating
• Known HIV infection

Sponsors & Collaborators

• Mirum Pharmaceuticals, Inc.: lead
• Childhood Liver Disease Research and Education Network: collaborator

Study Sites (13)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

University of California at San Francisco

San Francisco, California, 94143, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Baylor College of Medicine/Texas Children's Hospital

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84113, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Alagille Syndrome

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Results Point of Contact

• Title: Study Physician
• Organization: Mirum

medinfo@mirumpharma.com

1 650-667-4085

Study Officials

• Study Director

  Mirum

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 5, 2014
• First Posted: February 7, 2014
• Study Start: November 24, 2014
• Primary Completion: November 16, 2016
• Study Completion: November 16, 2016
• Last Updated: March 26, 2019
• Results First Posted: January 23, 2018

Record last verified: 2019-03

Locations

US (12); CA (1)