Clinical trial NCT06252753: Observational Study Protocol: LIVER-R
Official title: An Observational Multi Center Study to Evaluate Real World Treatment Outcomes of Durvalumab Based Regimens in Hepatobiliary Cancers
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hepatobiliary Cancers
Keywords: Hepatobiliary cancer
Unresectable hepatocellular carcinoma (uHCC)
Advanced biliary tract cancer (aBTC)
Durvalumab
Observatory
Real-world
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
Collaborators: name: ICON plc
First posted: 2024-02-12
Enrollment: 4490
Sex: ALL
Minimum age (years): 18
Maximum age (years): 130
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population includes patients treated with a durvalumab based regimen for hepatobiliary cancer as part of routine clinical practice. The decision to treat patients with a durvalumab based regimen should be independent from the decision to enroll patients in the study. Depending on the country, it is anticipated that 15 to 400 patients may be enrolled in each participating country.

Brief summary: Given the number of anticipated durvalumab-based treatment launches in the hepatobiliary cancer space over the next 3 years, there is a need to capture contemporary real-world data across these indications. LIVER-R is a multicountry, multicenter, observational study of patients with a confirmed diagnosis of hepatobiliary cancer treated with a durvalumab-based regimen as part of routine clinical practice or early access program (EAP). The study design will include primary and secondary data collection. The primary objective of this study is to evaluate the effectiveness of durvalumab-based regimens in real-world settings as measured by real-world overall survival. Other endpoints include demographics, clinical characteristics, clinically significant events of interest, treatment patterns, concomitant medications, and other real-world clinical endpoints (such as duration of treatment, progression-free survival, time to treatment progression, time to next treatment, recurrence-free survival, and time to treatment recurrence).

Detailed description: LIVER-R is a real-world, multi-country, multi-centre study aiming to enroll approximately 2500 pts with unresectable hepatocellular carcinoma (uHCC; n=1135) or advanced biliary tract cancers (aBTCs; n=1355) across 22 countries from North and South America, Europe, the Middle East and the Asia-Pacific region. The study design will include primary and secondary data collection. Primary data will be collected in real-time during the patient's routine visit. Secondary data will be collected from the patient's medical record at enrollment and at prespecified study time points (6-month intervals). The study population includes adult patients whose physician has previously made the decision to treat them with a durvalumab-based regimen for hepatobiliary cancer as part of routine clinical practice or patients receiving treatment through EAP. The study will include a baseline period of up to 5 years before the index date (initiation of a Durvalumab-based regimen) and a follow-up period to the earliest of death, loss to follow-up, withdrawal, or end of study for a maximum follow-up of 2 years for patients with aBTCs or 3 years for patients with uHCC. This is a descriptive, noncomparative study. No formal hypotheses are to be tested. All descriptive analyses will be conducted separately for each primary hepatobiliary cancer indication. Kaplan Meier estimates will be produced for time-to-event outcomes.

Eligibility criteria: Inclusion Criteria:

1. Age ≥18 years and a lawful adult in the country at the index date
2. Confirmed presence of malignancy of primary hepatobiliary cancer (i.e., uHCC or aBTC) by the treating physician
3. Type of hepatobiliary cancer indication is approved to be treated (i.e., positive phase 3 clinical trial read out for HIMALAYA or TOPAZ 1) with a durvalumab based regimen in the respective country or was administered as part of an EAP
4. Informed consent was obtained as per country level regulations on or after the index date

Exclusion Criteria:

1. Currently/was participating or plans to participate in any clinical trial for investigational treatment for hepatobiliary cancers on or after the diagnosis date until the index date
2. Received other systemic therapies for hepatobiliary cancer indication on or after diagnosis date through the index date (e.g., uHCC or aBTC patient who received a systemic treatment for unresectable HCC or advanced BTC, respectively, prior to initiating durvalumab based regimen)
3. Received a liver transplant during the baseline period

Primary outcomes:
measure: Real-world overall survival (rwOS)
timeFrame: rwOS will be assessed as OS rates at 6, 12, 24 months (both aBTC and uHCC), and 36 months (uHCC), median OS for a maximum follow-up of 2 and 3 years for aBTC and uHCC, respectively.
Secondary outcomes:
measure: Real-world duration of treatment (rwDOT)
timeFrame: rwDOT will be assessed as median DOT for a maximum follow-up of 2 years (aBTC) or 3 years (uHCC).
measure: Real-world progression free survival (rwPFS)
timeFrame: rwPFS will be assessed as PFS rates at 6, 12, 24 months (both aBTC and uHCC), and 36 months (uHCC), median PFS for a maximum follow-up of 2 and 3 years for aBTC and uHCC, respectively.
measure: Real-world time to progression (rwTTP)
timeFrame: rwTTP will be assessed as median TTP for a maximum follow-up of 2 years (aBTC) or 3 years (uHCC).
measure: Real-world time to next treatment (rwTTNT)
timeFrame: rwTTNT will be assessed as median TTNT for a maximum follow-up of 2 years (aBTC) or 3 years (uHCC).
measure: Real-world recurrence free survival (rwRFS)
timeFrame: rwRFS will be assessed as RFS rates at 6, 12, 24 months (both aBTC and uHCC), and 36 months (uHCC), median RFS for a maximum follow-up of 2 and 3 years for aBTC and uHCC, respectively.
measure: Real-world time to recurrence (rwTTR)
timeFrame: rwTTR will be assessed as median TTR for a maximum follow-up of 2 years (aBTC) or 3 years (uHCC).
measure: Patient demographic and clinical characteristics
timeFrame: Patient demographic and clinical characteristics will be captured up to 5 years prior to the index date.
measure: Percentage of patients receiving each treatment regimen
timeFrame: Treatment patterns will be captured from the index date until the earliest of death, loss to follow-up, withdrawal, or end of study for a maximum follow-up of 2 years for patients with aBTCs or 3 years for patients with uHCC.
measure: Clinically significant events (CSEs) of interest leading to a medical intervention
timeFrame: CSEs will be captured from the index date through 90 days after the last administration of durvalumab-based regimen or death, whichever occurs first.
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Research Site
state: Alabama
city: Mobile
country: United States
facility: Research Site
state: Alabama
city: Phoenix
country: United States
facility: Research Site
state: Arizona
city: Coronado
country: United States
facility: Research Site
state: California
city: Los Angeles
country: United States
facility: Research Site
state: California
city: Walnut Creek
country: United States
facility: Research Site
state: California
city: Clermont
country: United States
facility: Research Site
state: Florida
city: Gainesville
country: United States
facility: Research Site
state: Florida
city: Tampa
country: United States
facility: Research Site
state: Florida
city: Augusta
country: United States
facility: Research Site
state: Georgia
city: Evergreen Park
country: United States
facility: Research Site
state: Illinois
city: Hinsdale
country: United States
facility: Research Site
state: Illinois
city: Baton Rouge
country: United States
facility: Research Site
state: Louisiana
city: White Plains
country: United States
facility: Research Site
state: New York
city: Canton
country: United States
facility: Research Site
state: Ohio
city: Bethlehem
country: United States
facility: Research Site
state: Pennsylvania
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Dallas
country: United States
facility: Research Site
state: Texas
city: Temple
country: United States
facility: Research Site
state: Texas
city: Spokane
country: United States
facility: Research Site
state: Washington
city: Garran
country: Australia
facility: Research Site
state: Australian Capital Territory
city: Heidelberg
country: Australia
facility: Research Site
state: Victoria
city: Melbourne
country: Australia
facility: Research Site
state: Victoria
city: Murdoch
country: Australia
facility: Research Site
state: Western Australia
city: Linz
country: Austria
facility: Research Site
state: Upper Austria
city: Bonheiden
country: Belgium
facility: Research Site
state: Antwerp
city: Leuven
country: Belgium
facility: Research Site
state: Brabant
city: Liege
country: Belgium
facility: Research Site
state: Wallonia
city: Stene
country: Belgium
facility: Research Site
state: West Flanders
city: Curitiba
country: Brazil
facility: Research Site
state: Parana
city: Porto Alegre
country: Brazil
facility: Research Site
state: Rio Grande Do Sul
city: Recife - PE
country: Brazil
facility: Research Site
city: Sao Paulo
country: Brazil
facility: Research Site
city: Sao Paulo
country: Brazil
facility: Research Site
city: Halifax
country: Canada
facility: Research Site
state: Nova Scotia
city: Strasbourg
country: France
facility: Research Site
state: Alsace
city: Grenoble
country: France
facility: Research Site
state: Auvergne-Rh ne-Alpes
city: St Malo
country: France
facility: Research Site
state: Bretagne
city: Mulhouse
country: France
facility: Research Site
state: Grand Est
city: Boulogne-Sur-Mer
country: France
facility: Research Site
state: Hauts-de-France
city: Paris
country: France
facility: Research Site
state: Ile-de-France
city: Paris
country: France
facility: Research Site
state: Ile-de-France
city: Poitiers
country: France
facility: Research Site
state: Nouvelle-Aquitaine
city: Avignon
country: France
facility: Research Site
state: Provence-Alpes-Cote d'Azur
city: Marseille
country: France
facility: Research Site
state: Provence-Alpes-Cote d'Azur
city: Heidenheim
country: Germany
facility: Research Site
state: Baden Wurttemberg
city: Konstanz
country: Germany
facility: Research Site
state: Baden-Wurttemberg
city: Mannheim
country: Germany
facility: Research Site
state: Baden-Wurttemberg
city: Augsburg
country: Germany
facility: Research Site
state: Bavaria
city: Muenchen
country: Germany
facility: Research Site
state: Bavaria
city: Bremenhaven
country: Germany
facility: Research Site
state: Bremen
city: Wiesbaden
country: Germany
facility: Research Site
state: Hesse Land
city: Kassel
country: Germany
facility: Research Site
state: Hesse
city: Marburg
country: Germany
facility: Research Site
state: Hesse
city: Goettingen
country: Germany
facility: Research Site
state: Lower Saxony
city: Hannover
country: Germany
facility: Research Site
state: Lower Saxony
city: Hannover
country: Germany
facility: Research Site
state: Lower Saxony
city: Aachen
country: Germany
facility: Research Site
state: North Rhine-Westphalia
city: Bochum
country: Germany
facility: Research Site
state: North Rhine-Westphalia
city: Dortmund
country: Germany
facility: Research Site
state: North Rhine-Westphalia
city: Moers
country: Germany
facility: Research Site
state: North Rhine-Westphalia
city: Stolberg
country: Germany
facility: Research Site
state: North Rhine-Westphalia
city: Koblenz
country: Germany
facility: Research Site
state: Rhineland-Palatinate
city: Halle (Saale)
country: Germany
facility: Research Site
state: Saxony-Anhalt
city: Magdeburg
country: Germany
facility: Research Site
state: Saxony-Anhalt
city: Dresden
country: Germany
facility: Research Site
state: Saxony
city: Leipzig
country: Germany
facility: Research Site
state: Saxony
city: Neumuenster
country: Germany
facility: Research Site
state: Schleswig-Holstein
city: Berlin
country: Germany
facility: Research Site
city: Hamburg
country: Germany
facility: Research Site
city: Hamburg
country: Germany
facility: Research Site
city: Chaidari
country: Greece
facility: Research Site
state: Athens
city: Athens
country: Greece
facility: Research Site
state: Attica
city: Athens
country: Greece
facility: Research Site
state: Attica
city: Thessaloniki
country: Greece
facility: Research Site
state: Central Macedonia
city: Thessaloniki
country: Greece
facility: Research Site
state: Central Macedonia
city: Larissa
country: Greece
facility: Research Site
state: Thessaly
city: Ramat Gan
country: Israel
facility: Research Site
state: Tel-Aviv
city: Jerusalem
country: Israel
facility: Research Site
city: Tel-Aviv
country: Israel
facility: Research Site
city: Naples
country: Italy
facility: Research Site
state: Campania
city: Rome
country: Italy
facility: Research Site
state: Lazio
city: Rome
country: Italy
facility: Research Site
state: Lazio
city: Brescia
country: Italy
facility: Research Site
state: Lombardy
city: Milan
country: Italy
facility: Research Site
state: Lombardy
city: Milan
country: Italy
facility: Research Site
state: Lombardy
city: Rozzano
country: Italy
facility: Research Site
state: Lombardy
city: Turin
country: Italy
facility: Research Site
state: Piemonte
city: Monserrato
country: Italy
facility: Research Site
state: Sardinia
city: Pisa
country: Italy
facility: Research Site
state: Tuscany
city: Nagoya
country: Japan
facility: Research Site
state: Aichi-Ken
city: Kashiwa-shi
country: Japan
facility: Research Site
state: Chiba
city: Kanazawa-shi
country: Japan
facility: Research Site
state: Ishikawa
city: Yokohama-shi
country: Japan
facility: Research Site
state: Kanagawa
city: Kyoto-shi
country: Japan
facility: Research Site
state: Kyoto
city: Sendai-shi
country: Japan
facility: Research Site
state: Miyagi
city: Osaka-Sayama
country: Japan
facility: Research Site
state: Osaka
city: Lisboa
country: Portugal
facility: Research Site
city: San Juan
country: Puerto Rico
facility: Research Site
city: Bucuresti
country: Romania
facility: Research Site
city: Cluj
country: Romania
facility: Research Site
city: Singapore
country: Singapore
facility: Research Site
city: Santiago de Compostela
country: Spain
facility: Research Site
state: A Coruna
city: Santander
country: Spain
facility: Research Site
state: Cantabria
city: Poniente Sur
country: Spain
facility: Research Site
state: Cordoba
city: Ourense
country: Spain
facility: Research Site
state: Galicia
city: El Palmar
country: Spain
facility: Research Site
state: Murcia
city: Barcelona
country: Spain
facility: Research Site
city: Pamplona
country: Spain
facility: Research Site
city: Kaohsiung City
country: Taiwan
facility: Research Site
city: Taipei City
country: Taiwan
facility: Research Site
city: Taipei City
country: Taiwan
facility: Research Site
city: Taoyuan City
country: Taiwan
facility: Research Site
Study officials:
References: