A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age6+
SponsorIpsen

About this trial

This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching.

The main aim of this study is to observe the long-term, everyday safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosed with ALGS.

On (or starting) active odevixibat treatment.

Aged 6 months or older at the time of consent.

Disqualifiers

Currently participating in a clinical trial with odevixibat.

Currently participating in any interventional clinical trial for ALGS.

Have any contraindication to odevixibat as per the locally approved label.

Trial population

Participants with Alagille syndrome (ALGS) who have been prescribed odevixibat by their treating physician will be enrolled into the study. Participants who started odevixibat treatment before study implementation may also be enrolled.

Trial design

Design model

Other

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed

Trial outcomes

Primary outcomes

1

Percentage of participants experiencing adverse events (AEs)

An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.

Time frame
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
2

Percentage of participants experiencing serious adverse events (SAEs)

An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.

Time frame
From first ICF signature and up to end of data collection (approximately 5 years of data collection)

Secondary outcomes

1

Percentage of participants with severe diarrhoea events

Time frame
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
2

Percentage of participants with bloody diarrhoea events

Time frame
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
3

Percentage of participants experiencing diarrhoea events with concurrent dehydration

Time frame
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
4

Percentage of participants experiencing diarrhoea events treated with oral or intravenous rehydration

Time frame
From first ICF signature and up to end of data collection (approximately 5 years of data collection)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.