Alagille SyndromePhase 3ASSERTNCT04674761
Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
A Phase 3 study testing Odevixibat in people with Alagille Syndrome, run by Albireo at 32 sites in 13 countries.
Trial summary
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Alagille Syndrome
- What is being tested?
- Odevixibat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 52 participants
- Where
- 32 sitesin 13 countries
- Study dates
- 19 Mar 2021to 9 Sep 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Genetically confirmed diagnosis of Alagille syndrome
- History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument
- Elevated serum bile acid level
Who cannot join
- History or ongoing presence of other types of liver disease (eg. biliary atresia, progressive familial intrahepatic cholestasis, hepatocellular carcinoma)
- History of liver transplant, or a liver transplant is planned within 6 months of randomization
- ALT >10× upper limit of normal (ULN) at screening
- Total bilirubin >15 × ULN at screening
- Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome
Trial locations and contacts
32 sites in 13 countries.
32 sites in 13 countries
United States9 sites
Johns Hopkins Hospital
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
Children's Mercy Hospital and Clinics
Kansas City, Missouri, 64108
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Columbia University Medical Center
New York, New York, 10032
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health Science University School of Medicine
Portland, Oregon, 97239
University of California - San Francisco
San Francisco, California, 94158
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
The Bronx, New York, 10467
France4 sites
Hôpital Femme Mère Enfant de Lyon
Bron, 69677
Antenne pédiatrique du CIC - Hopital Jeanne De Flandre
Lille, 59037
APHM
Marseille, 13385
Hopital Necker Enfants Malades
Paris, 75015
Italy4 sites
ASST Papa Giovanni XXIII
Bergamo, 24127
AOU Meyer
Florence, 50139
Azienda Ospedale Università Padova
Padova, 35128
Bambino Gesù Children's Hospital
Rome, 00165
Germany3 sites
Charité-Universitätsmedizin Berlin
Berlin, 13353
Medizinische Hochschule Hannover
Hanover, 30625
Universitätsklinik für Kinder-und Jugendmedizin Tübingen
Tübingen, 72076
Israel2 sites
Sharie Zedek
Jerusalem, 9103102
Schneider Children's Medical Center of Israel
Petah Tikva, 4920235
Netherlands2 sites
Universitair Medisch Centrum Groningen (UMCG)
Groningen, 9713 GZ
University Medical Centre Utrecht, WKZ
Utrecht, 3584EA
Turkey (Türkiye)2 sites
Hacettepe Üniversitesi İhsan Doğramacı Çocuk Hastanesi
Ankara, 06230
Istanbul University Medical Faculty Hospital
Fatih, 34093
About this study
Official title: A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.
Read the detailed description
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
- Conditions
- Alagille Syndrome
- Being tested
- Odevixibat, Placebo
- Keywords
- ALGS, Alagille syndrome
- Medical subject headings
- Alagille Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OdevixibatDrug
Also called A4250
Odevixibat is a small molecule and selective inhibitor of IBAT.
PlaceboDrug
Placebo identical in appearance to experimental drug (odevixibat).
| Study group | Role | What they receive |
|---|---|---|
| Odevixibat (A4250) | Experimental | Capsules for oral administration once daily for 24 weeks. Receives: Drug: Odevixibat |
| Placebo | Placebo comparator | Capsules for oral administration (to match active) once daily for 24 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Scratching Score
Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.
Measured: Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.
Secondary outcomes (1)
Serum Bile Acid Levels
Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24
Measured: Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2020
- Study start19 Mar 2021
- Primary completion9 Sep 2022
- Study completion9 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Alagille SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Albireo
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04674761
- Sponsor's protocol ID
- A4250-012
- Acronym
- ASSERT
- Status last verified
- Oct 2023
- First submitted
- 10 Dec 2020
- Last updated
- 2 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Ovchinsky N, Aumar M, Baker A, Baumann U, Bufler P, Cananzi M, Czubkowski P, Durmaz O, Fischer R, Indolfi G, Karnsakul WW, Lacaille F, Lee WS, Maggiore G, Rosenthal P, Ruiz M, Sokal E, Sturm E, van der Woerd W, Verkade HJ, Wehrman A, Clemson C, Yu Q, Ni Q, Ruvido J, Manganaro S, Mattsson JP. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2024 Jul;9(7):632-645. doi: 10.1016/S2468-1253(24)00074-8. Epub 2024 Apr 23.
PubMed 38670135
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)
- ClinicalTrials.gov ID
- NCT04674761
- Acronym / sponsor ID
- ASSERT / A4250-012
- Conditions
- Alagille Syndrome
- Keywords
- ALGS, Alagille syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- AlbireoIndustry (pharma, biotech, devices)
- Study start
- 19 Mar 2021 (actual)
- Primary completion
- 9 Sep 2022 (actual)
- Study completion
- 9 Sep 2022 (actual)
- First posted
- 19 Dec 2020 (actual)
- Results
- Posted 2 Nov 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.
Detailed description
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Odevixibat (A4250) | Experimental | Drug: OdevixibatCapsules for oral administration once daily for 24 weeks. |
| Placebo | Placebo comparator | Drug: PlaceboCapsules for oral administration (to match active) once daily for 24 weeks. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Genetically confirmed diagnosis of Alagille syndrome
- History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument
- Elevated serum bile acid level
Key exclusion criteria (5 listed)
- History or ongoing presence of other types of liver disease (eg. biliary atresia, progressive familial intrahepatic cholestasis, hepatocellular carcinoma)
- History of liver transplant, or a liver transplant is planned within 6 months of randomization
- ALT >10× upper limit of normal (ULN) at screening
- Total bilirubin >15 × ULN at screening
- Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OdevixibatAlso: A4250Odevixibat is a small molecule and selective inhibitor of IBAT. | Drug | Odevixibat (A4250) |
| PlaceboPlacebo identical in appearance to experimental drug (odevixibat). | Drug | Placebo |
Primary outcome
Change From Baseline in Scratching Score
Time frame: Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.
Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.
1 secondary outcome
Serum Bile Acid Levels
Time frame: Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38670135Ovchinsky N, Aumar M, Baker A, Baumann U, Bufler P, Cananzi M, Czubkowski P, Durmaz O, Fischer R, Indolfi G, Karnsakul WW, Lacaille F, Lee WS, Maggiore G, Rosenthal P, Ruiz M, Sokal E, Sturm E, van der Woerd W, Verkade HJ, Wehrman A, Clemson C, Yu Q, Ni Q, Ruvido J, Manganaro S, Mattsson JP. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol. 2024 Jul;9(7):632-645. doi: 10.1016/S2468-1253(24)00074-8. Epub 2024 Apr 23.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
32 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Odevixibat in people with Alagille Syndrome, run by Albireo at 32 sites in 13 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, France, Italy, Germany, Israel and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Nov 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Scratching Score (time frame: Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04674761, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Nov 2023.