Clinical trial NCT02848521: A Study Measuring Quality of Life, Treatment Preference and Satisfaction of ADPKD Patients in Europe
Official title: A Prospective, Non-interventional Study Measuring Quality of Life, Treatment Preference and Treatment Satisfaction of Autosomal Dominant Polycystic Kidney Disease Patients in Europe
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Autosomal Dominant Polycystic Kidney Disease
Keywords: ADPKD
CKD
Rapid progressor
quality of life
DCE
Lead sponsor: Otsuka Pharmaceutical Europe Ltd
Sponsor class: INDUSTRY
First posted: 2016-07-28
Enrollment: 407
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The study population will be adult patients with ADPKD in CKD Stages 1 -3, with evidence of rapidly progressing disease according to the investigator. Investigator must be ADPKD experts qualified by experience and ability to perform the study and be working at an ADPKD reference centre.

Brief summary: This is a prospective, non-interventional study (NIS) measuring health-related quality of life (HRQoL), treatment satisfaction, and other patient-reported outcomes (PROs) of ADPKD patients in Europe.

Detailed description: This is a prospective, non-interventional study (NIS) measuring HRQoL, treatment satisfaction, and other PROs of ADPKD patients in Europe. The study aims to enrol at least 486 patients. Data will be prospectively collected at clinics, from medical notes and via PRO measures for each patient at Baseline, Month 1, Month 3 and subsequently at 3 month intervals up to and including the final assessment (18 months maximum follow-up time). No visits with the patients' treating physician will be imposed by the protocol. Any clinical visits will occur as per normal clinical practice

Eligibility criteria: Inclusion Criteria:

* Male and female aged ≥ 18 years.
* Patient has a diagnosis of ADPKD between CKD Stages 1-3, and is deemed by their treating physician to likely have rapidly progressing disease.
* Patient has a life expectancy greater than 18 months at time of enrolment.
* Patient is able and willing to give informed consent, if required according to local regulations.
* Patient is fluent in local language.

Exclusion Criteria:

* Patient is currently participating in, or has in the last 12 months participated in an interventional clinical trial.
* Presence of any condition/circumstance which in the opinion of the investigator could significantly limit the complete follow up of the patient.
* Inability of the patient to complete PROs remotely.

Primary outcomes:
measure: Mean rate of change (%) in Physical Health Composite Scale (PCS) scores of the 12-Item Short Form Survey (SF-12)
timeFrame: From Baseline to end of study (maximum of 18 months)
Secondary outcomes:
measure: Mean rate of change (%) in Mental Health Composite Scale (MCS) scores of the SF-12
timeFrame: From Baseline to end of study (maximum of 18 months)
measure: Mean ADPKD-IS score changes
timeFrame: From Baseline to end of study (maximum of 18 months)
measure: Mean Treatment Satisfaction Questionnaire for Medication (TSQM-9) score changes
timeFrame: From Baseline to end of study (maximum of 18 months)
measure: Mean ADPKD-Urinary Impact Scale (UIS) score changes
timeFrame: From Baseline to end of study (maximum of 18 months)
measure: Description of real-world ADPKD treatment patterns (number of subjects taking different treatments)
timeFrame: From Baseline to end of study (maximum of 18 months)
measure: Description of real-world ADPKD treatment patterns (percentage of subjects taking different treatments)
timeFrame: From Baseline to end of study (maximum of 18 months)
Other outcomes:
measure: Mean ADPKD-Pain and Discomfort Scale(PDS) score change from baseline to end of study (exploratory)
timeFrame: From Baseline to end of study (maximum of 18 months)
measure: Overall odds ratio of discrete-choice experiment (DCE) for patient preference to the addition of a disease modifying treatment versus no change to local Standard of Care (SoC)
timeFrame: At baseline and at the end of the study (18 months)
Locations:
city: Vienna
country: Austria
facility: AKH Wien
city: Villach
country: Austria
facility: LKH Villach
city: Brussels
country: Belgium
facility: UCL Brussels
city: Brussels
country: Belgium
facility: UZ Brussels
city: Leuven
country: Belgium
facility: UZ Leuven/Gasthuisberg KUL
city: Liege
country: Belgium
facility: CHU Liège - Domaine Universitaire du Sart Tilman
city: Lille
country: France
facility: Clinique La Louviere
city: Nimes
country: France
facility: CHU Nimes - Hôpital CAREMEAU
city: Paris
country: France
facility: Hopital Tenon
city: Paris
country: France
facility: Necker Hospital
city: Tours
country: France
facility: CHU TOURS - Hôpital Bretonneau
city: Berlin
country: Germany
facility: Charite Berlin
city: Gottingen
country: Germany
facility: Universitätsmedizin Göttingen
city: Lubeck
country: Germany
facility: University Medical Center Schleswig-Holstein
city: Barcelona
country: Spain
facility: Fundación Puigvert
city: Barcelona
country: Spain
facility: Hospital Clinic
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Getafe
country: Spain
facility: Hospital Universitario de Getafe
city: Genève
country: Switzerland
facility: University Hospital of Geneve
city: Zürich
country: Switzerland
facility: University Hospital of Zurich
city: Edinburgh
country: United Kingdom
facility: Edinburgh Royal Infirmary
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
city: Liverpool
country: United Kingdom
facility: Aintree University Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital
city: London
country: United Kingdom
facility: Royal London Hospital
city: Newcastle
country: United Kingdom
facility: Freeman Hospital
Study officials:
affiliation: Otsuka Europe
name: Medical Department
role: STUDY_DIRECTOR
References:
citation: Joly D, Quinn J, Mokiou S, O'Reilly K, Sanchez-Covisa J, Wang-Silvanto J, Doll H. Rationale and study protocol of ACQUIRE, a prospective, observational study measuring quality of life, treatment preference and treatment satisfaction of autosomal dominant polycystic kidney disease (ADPKD) patients in Europe. BMC Nephrol. 2020 Jul 24;21(1):298. doi: 10.1186/s12882-020-01927-1.