Trial record 2 of 2 for:
PEDFIC 1
Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
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| ClinicalTrials.gov Identifier: NCT03659916 |
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Recruitment Status :
Recruiting
First Posted : September 6, 2018
Last Update Posted : December 24, 2020
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Sponsor:
Albireo
Information provided by (Responsible Party):
Albireo
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Brief Summary:
Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in children with PFIC.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Progressive Familial Intrahepatic Cholestasis | Drug: A4250 (odevixibat) | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 120 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) |
| Actual Study Start Date : | September 28, 2018 |
| Estimated Primary Completion Date : | December 2021 |
| Estimated Study Completion Date : | December 2021 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Progressive familial intrahepatic cholestasis
| Arm | Intervention/treatment |
|---|---|
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Experimental: A4250
Capsules for oral administration (120 ug/kg) once daily for 72 weeks
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Drug: A4250 (odevixibat)
A4250 is a small molecule and selective inhibitor of IBAT |
Primary Outcome Measures :
- Change in Pruritus [ Time Frame: From baseline over 72 weeks ]Change in pruritus as indexed by caregiver-reported (Albireo ObsRO instrument) observed scratching
- Change in serum bile acids [ Time Frame: From baseline up to week 72 ]
Secondary Outcome Measures :
- All-cause mortality [ Time Frame: From baseline to weeks 24, 48, and 72 ]
- Number of patients undergoing biliary diversion surgery [ Time Frame: From baseline to weeks 24, 48, and 72 ]
- Number of patients undergoing liver transplantation [ Time Frame: From baseline to weeks 24, 48, and 72 ]
- Change in growth [ Time Frame: From baseline to weeks 24, 48, and 72 ]The linear growth deficit compared to the standard growth curve
- Change in AST to platelet ratio idex (APRI) score [ Time Frame: From baseline to week 72 ]
- Change in Fib-4 score [ Time Frame: From baseline to week 72 ]
- Change in pediatric end-stage liver disease (PELD)/model for end-stage liver disease (MELD) score [ Time Frame: From baseline to week 72 ]
- Change in use of antipruritic medication [ Time Frame: From baseline to weeks 24, 48, and 72 ]
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| Ages Eligible for Study: | up to 100 Years (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria Cohort 1:
- Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of intolerable symptoms after completing at least 12 weeks of treatment
- Signed informed consent and assent as appropriate
- Patients expected to have a consistent caregiver for the duration of the study
- Caregivers (and age appropriate patients) must be willing and able to use an eDiary device as required by the study
Inclusion Criteria Cohort 2:
- A male or female patient with a clinical diagnosis of PFIC and with a body weight ≥5 kg
- Patient must have clinical genetic confirmation of PFIC
- Patient must have elevated serum bile acid levels
- Patient must have history of significant pruritus
- Age appropriate patients are expected to have a consistent caregiver for the duration of the study
- Caregivers and age-appropriate patients (≥8 years of age) must be willing and able to use an eDiary device as required by the study
Exclusion Criteria Cohort 1:
- Decompensated liver disease: coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
- Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter
- Patients not compliant with treatment in study A4250-005
- Any other conditions or abnormalities which, in the opinion of the investigator or Medical Monitor, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
Exclusion Criteria Cohort 2:
- Known pathologic variations of the ABCB11 gene that have been demonstrated to result in complete absence of the BSEP protein
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Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
- Biliary atresia of any kind
- Benign recurrent intrahepatic cholestasis, indicated by any history of normal serum bile acids
- Suspected or proven liver cancer or metastasis to the liver on imaging studies
- Histopathology on liver biopsy is suggestive of alternate non-PFIC related etiology of cholestasis
- Patient with past medical history or ongoing chronic (i.e., >3 months) diarrhea
- Any patient with suspected or confirmed cancers except for basal cell carcinoma
- Patient has had a liver transplant, or a liver transplant is planned within 6 months of the Screening/Inclusion Visit
- Decompensated liver disease
- Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC
- Patient previously treated with an IBAT inhibitor and whose pruritus did not respond to treatment
- Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03659916
Contacts
| Contact: Albireo Pharma | (857) 378-2035 | medinfo@albireopharma.com |
Locations
Show 41 study locations
Sponsors and Collaborators
Albireo
| Responsible Party: | Albireo |
| ClinicalTrials.gov Identifier: | NCT03659916 |
| Other Study ID Numbers: |
A4250-008 |
| First Posted: | September 6, 2018 Key Record Dates |
| Last Update Posted: | December 24, 2020 |
| Last Verified: | December 2020 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Albireo:
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Pediatric Cholestasis |
Additional relevant MeSH terms:
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Cholestasis Cholestasis, Intrahepatic Bile Duct Diseases |
Biliary Tract Diseases Digestive System Diseases Liver Diseases |