A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ipsen Pharma

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

6 Jan 2022 → ongoing

Decision date (initial)

2024-02-19

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Ipsen Pharma

External identifiers

EU CT number

2023-509028-17-00

EudraCT number

2021-000996-36

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT)

Secondary objectives 3

1. 1. To demonstrate a sustained effect of odevixibat on serum bile acids in patients with ALGS who have completed study A4250-012
2. 2. To evaluate an effect of odevixibat on parameters related to quality of life
3. 3. To evaluate the long-term safety and tolerability of repeated daily doses of odevixibat in patients with ALGS

Conditions and MedDRA coding

Alagille Syndrome

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. 1. Completion of the 24-week Treatment Period of Study A4250-012
2. 2. Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study
3. 3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study
4. 4. Sexually active males and females must agree to use a reliable contraceptive method with ≤ 1% failure rate (such as intra-uterine device or complete abstinence) from signed informed consent through 90 days after last dose of study drug.

Exclusion criteria 4

1. 1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
2. 2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012
3. 3. Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
4. 4. Known hypersensitivity to any components of odevixibat

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.

Secondary endpoints 8

1. • Change in serum bile acid levels from baseline to Week 72;
2. • Change from baseline through Week 72 in patient reported and observer reported itching and scratching severity scores, respectively, for the morning and evening assessment;
3. • Percentage of patients achieving a clinically meaningful decrease in pruritus (pruritus responders) at each visit as measured by the Albireo ObsRO/patient reported outcomes (PRO) instruments;
4. • Change from baseline to Week 72 in sleep parameters as measured with the Albireo ObsRO/PRO instruments (e.g. tiredness and number of awakenings);
5. • Change from baseline to Week 72 in Pediatric Quality of Life Inventory (PedsQL) scores
6. • Assessment of Global Symptom Relief from baseline to Weeks 4, 12, 24, 48, and 72 as measured by patient, caregiver, and clinician Global impression of Symptoms (PGIS, CaGIS, CGIS) items
7. • Assessment of Global Symptom Relief as measured by patient, caregiver, and clinician Global Impression of Change (PGIC, CaGIC, CGIC) items at Weeks 4, 12, 24, 48, and 72
8. • Change in serum bile acid levels from baseline through Week 72

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ipsen Pharma

Sponsor organisation

Ipsen Pharma

Address

70 Rue Balard

City

Paris

Postcode

75015

Country

France

Scientific contact point

Organisation

Ipsen Pharma

Contact name

Clinical Operations Department

Public contact point

Organisation

Ipsen Pharma

Contact name

Clinical Operations Department

Third parties 8

Locations

6 EU/EEA countries · 12 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 63 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

9 submissions — initial application plus substantial / non-substantial modifications