This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ipsen Pharma

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

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

Trial duration

23 Sep 2020 → 22 Jun 2026

Decision date (initial)

2024-07-11

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Ipsen Pharma, France

External identifiers

EU CT number

2024-512086-14-00

EudraCT number

2019-003807-37

ClinicalTrials.gov

NCT04336722

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in children with biliary atresia (BA) post Kasai hepatoportoenterostomy(HPE) based native liver survival (NLS) of up to 104 weeks.

Secondary objectives 4

1. 1. To evaluate the effect of odevixibat compared to placebo on the time to onset of sentinel events
2. 2. To evaluate the effect of odevixibat compared to placebo on total bilirubin after 13, 26, 52, and 104 weeks
3. 3. To evaluate the effect of odevixibat compared to placebo on serum bile acids after 13, 26, 52, and 104 weeks
4. 4. To assess the long-term safety and tolerability of repeated daily doses of odevixibat compared to placebo for 104 weeks in children with BA post Kasai HPE

Conditions and MedDRA coding

Biliary Atresia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. A male or female patient with a clinical diagnosis of BA
2. 2. Age at Kasai HPE ≤90 days
3. 3. Eligible to start treatment within 3 weeks post-Kasai HPE

Exclusion criteria 9

1. 1. Patients with intractable ascites
2. 2. Ileal resection surgery
3. 3. ALT ≥10× upper limit of normal (ULN) at screening
4. 4. Patient on total parenteral nutrition, or not able to take study drug orally, at randomization
5. 5. Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
6. 6. Choledochal cystic disease
7. 7. INR >1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
8. 8. Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements
9. 9. Weight < 3.5kg at randomization

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary efficacy endpoint is the time from randomization to first occurrence of liver transplant, or death, during the 104 week treatment period.

Secondary endpoints 6

1. 1. Proportion of patients who are alive and have not undergone a liver transplant after 104 weeks.
2. 2. Time to onset of first sentinel event during the 104-week treatment period. Sentinel events are defined in the protocol
3. 3. Total bilirubin level after 13, 26, 52, and 104 weeks.
4. 4. Serum bile acid level after 13, 26, 52, and 104 weeks.
5. 5. Time to pediatric end-stage liver disease (PELD) score ≥15.
6. 6. Safety parameters including AEs, SAEs, findings on physical examination, laboratory assessments (including fat-soluble vitamins and lipids) and abdominal ultrasound.

Investigational products

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

Test 2

Placebo 1

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 10

Locations

8 EU/EEA countries · 19 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 61 files

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

Application history

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