Alagille Syndrome Clinical Trial
— RISEOfficial 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
Verified date | March 2024 |
Source | Mirum Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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].
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Mirum Pharmaceuticals, Inc. |
United States, Belgium, Brazil, France, Mexico, Poland, United Kingdom,
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 |
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