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NCT04729751 Clinical Trial

A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).

Alagille Syndrome Clinical Trial

— RISE  

Official title:
Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT04729751
Other study ID # MRX-801
Secondary ID 2020-004628-40
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date September 9, 2021
Est. completion date December 2024

Study information
Verified date March 2024
Source Mirum Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

Description:

This is an open label study where all participants will receive maralixibat treatment.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 12
Est. completion date December 2024
Est. primary completion date August 22, 2023
Accepts healthy volunteers No
Gender All
Age group 0 Days to 364 Days
Eligibility Inclusion Criteria: 1. Body weight of =2.5 kg 2. <12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US). 3. Gestational age =36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of =36 weeks is required. 4. Diagnosis of PFIC or ALGS Exclusion criteria: 1. Predicted complete absence of bile salt excretion pump (BSEP) function 2. History of surgical disruption of the enterohepatic circulation 3. History of liver transplant or imminent need for liver transplant 4. Decompensated cirrhosis 5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion 6. Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• Maralixibat
Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL) 400 µg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base 600 µg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base

Locations
Country Name City State
Belgium Cliniques Universitaires Saint-Luc Brussels
Brazil Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês São Paulo
France Hôpital Kremlin Bicêtre Le Kremlin-Bicêtre
France Hopital Necker Paris
Mexico Consultorio de Joshue David Covarrubias Esquer Zapopan
Poland Instytut Pomnik-Centrum Zdrowia Dziecka Warsaw
United Kingdom King's College Hospital London
United States Texas Children's Hospital Houston Texas
United States Children Hospital LA Los Angeles California
United States Ochsner Hospital for Children New Orleans Louisiana
United States Children's Hospital of Pittsburgh Pittsburgh Pennsylvania
United States University of California - San Francisco San Francisco California
United States Seattle Children's Hospital Seattle Washington
United States Medstar Georgetown University Hospital Washington District of Columbia

Sponsors (1)
Lead Sponsor Collaborator
Mirum Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Belgium,  Brazil,  France,  Mexico,  Poland,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary Frequency of treatment-emergent adverse events [TEAEs] From Baseline through to Week 13
Secondary Change in fasting serum bile acid (sBA) levels From Baseline through to Week 13
Secondary To evaluate the effect on liver enzymes (ALT, AST) and bilirubin From Baseline through to Week 13
Secondary To evaluate the effect on LSVs From Baseline through to Week 13
Secondary To assess the plasma level of maralixibat in infant participants At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit
See also
  Status Clinical Trial Phase
Active, not recruiting NCT05035030 - Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome Phase 3
Recruiting NCT06193928 - Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)
Completed NCT02160782 - Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS) Phase 2
Completed NCT00007033 - Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease N/A
Suspended NCT00571272 - Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Completed NCT02057692 - Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome Phase 2
Active, not recruiting NCT02922751 - FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)
Completed NCT00001642 - Positional Cloning of the Gene(s) Responsible for Alagille Syndrome N/A
Recruiting NCT05488067 - Atorvastatin Therapy on Xanthoma in Alagille Syndrome Phase 4
Completed NCT04674761 - Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome Phase 3
Completed NCT02117713 - An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome Phase 2
Completed NCT02131623 - Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
Completed NCT02963077 - A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384 Phase 1
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Completed NCT03082937 - An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects Phase 1
Completed NCT02047318 - An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS) Phase 2
Completed NCT01903460 - Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome Phase 2
Completed NCT01515631 - Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record Review
Enrolling by invitation NCT05846854 - Decreasing Hemorrhage Risk in Children With Alagille Syndrome N/A
Approved for marketing NCT04530994 - A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

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