Ignite Creation Date:
2024-05-06 @ 4:35 PM
Last Modification Date:
2024-10-26 @ 2:13 PM
Study NCT ID:
NCT05035030
Status:
RECRUITING
Last Update Posted:
2024-07-09
First Post:
2021-08-13
Brief Title:
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
Sponsor:
Albireo an Ipsen Company
Organization:
Ipsen
Study Overview
Official Title:
An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat A4250 in Patients With Alagille Syndrome ASSERT-EXT
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ASSERT-EXT
Brief Summary:
The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome ALGS
The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver heart skeleton eyes and kidneys Common symptoms which often develop during the first three months of life include blockage of the flow of bile from the liver cholestasis yellowing of the skin and mucous membranes jaundice poor weight gain and growth and severe itching pruritis
The drug used for the study is odevixibat and was authorized for the treatment of cholestatic pruritus in infants with ALGS over 12 months of age by the United States Food and Drug Administration on 13 June 2023
The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver heart skeleton eyes and kidneys Common symptoms which often develop during the first three months of life include blockage of the flow of bile from the liver cholestasis yellowing of the skin and mucous membranes jaundice poor weight gain and growth and severe itching pruritis
The drug used for the study is odevixibat and was authorized for the treatment of cholestatic pruritus in infants with ALGS over 12 months of age by the United States Food and Drug Administration on 13 June 2023
Detailed Description:
This Phase 3 open-label multi-center extension study will have two groups of participants Cohort 1 participants who participated in Study A4250-012 NCT04674761 ASSERT and meet the entry criteria for this study and Cohort 2 infants under 12 months of age with ALGS
The study will consist of 2 or 3 periods
1 A Treatment period of 72 weeks cohort 1 or 12 weeks cohort 2 Participants will visit the clinic every 4 to 12 weeks and will receive a dose of 120 μgkg odevixibat daily
2 An Optional extension period where participants who wish to continue receiving odevixibat after the treatment period will have the opportunity to remain on treatment with visits every 16 weeks until the drug is commercially available The optional extension is available provided continued use is supported by the risk-benefit profile the participant has not been previously withdrawn or discontinued from the study and the study is not terminated by the Sponsor
3 A Safety follow-up period of 4 weeks cohort 1 or 2 weeks cohort 2 The Safety Follow-up Period will not occur for those who remain on treatment in the optional extension period
Participants will need to complete an e-diary and questionnaires throughout the study cohort 1 only Participants will undergo blood samplings urine collections cohort 1 only physical examinations and clinical evaluations They may continue some other medications but the details need to be recorded
The study will consist of 2 or 3 periods
1 A Treatment period of 72 weeks cohort 1 or 12 weeks cohort 2 Participants will visit the clinic every 4 to 12 weeks and will receive a dose of 120 μgkg odevixibat daily
2 An Optional extension period where participants who wish to continue receiving odevixibat after the treatment period will have the opportunity to remain on treatment with visits every 16 weeks until the drug is commercially available The optional extension is available provided continued use is supported by the risk-benefit profile the participant has not been previously withdrawn or discontinued from the study and the study is not terminated by the Sponsor
3 A Safety follow-up period of 4 weeks cohort 1 or 2 weeks cohort 2 The Safety Follow-up Period will not occur for those who remain on treatment in the optional extension period
Participants will need to complete an e-diary and questionnaires throughout the study cohort 1 only Participants will undergo blood samplings urine collections cohort 1 only physical examinations and clinical evaluations They may continue some other medications but the details need to be recorded
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2023-509028-17-00 | CTIS | None | None |