Clinical trial NCT02574676: Quality of Life (QOL) Registry for Patients With AL Amyloidosis
Official title: Prospective Observational Study Measuring the Short-Form36 ( SF-36v2) and Other QOL Tools in an AL Amyloidosis Population
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: AL Amyloidosis
Amyloidosis
Lead sponsor: Prothena Biosciences Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Amyloidosis Research Consortium
name: Amyloidosis Support Groups
name: Amyloidosis Foundation
name: Quality Metrics
First posted: 2015-10-14
Enrollment: 343
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: ECOLOGIC_OR_COMMUNITY
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with Al Amyloidosis

Brief summary: This is an online registry to document the psychometric properties of SF-36v2 among patients with AL Amyloidosis, to document patients' burden of disease, to better understand the patient's experience and to follow quality of life issues using a variety of QOL measures.


Eligibility criteria: Inclusion Criteria:

* Patients must have AL Amyloidosis

Exclusion Criteria:

Primary outcomes:
measure: Psychometric evaluation of short-form 36 (SF-36v2) in patients with AL Amyloidosis
timeFrame: Change from Baseline to 12 months
Secondary outcomes:
measure: Calculate burden of illness from participants using SF-36 Mental Component Score (MCS) and Physical Component Score (PCS) compared with sample from US general population and three other chronic conditions: Congestive Heart Failure, Lymphoma and COPD
timeFrame: Baseline- cross sectional
measure: Assess the pathway to diagnosis and treatment through a disease history specific questionnaire collecting organ involvement, initial symptoms, diagnosis duration, treatment received and other disease characteristics
timeFrame: Baseline- cross sectional
measure: Psychometric evaluation of Kansas City Cardiomyopathy Questionnaire (KCCQ-12) in patients with AL Amyloidosis
timeFrame: Change from Baseline to 12 months
measure: Psychometric evaluation of Patient Global Impression-Severity Scale (PGI-S) in patients with AL Amyloidosis
timeFrame: Change from Baseline to 12 months
measure: Psychometric evaluation of Patient Global Assessment of Functioning (GAF) Scale in patients with AL Amyloidosis
timeFrame: Change from Baseline to 12 months
measure: Psychometric evaluation of Hematology Patient Reported Symptom Screen (HPRSS) in patients with AL Amyloidosis
timeFrame: Change from Baseline to 12 months
measure: Psychometric evaluation of Work Productivity and Activity Questionnaire: Specific Health Problem V2.0 (WPAI:SHP) in patients with AL Amyloidosis
timeFrame: Change from Baseline to 12 months
measure: Psychometric evaluation of MOS 6-Item Sleep Scale Standard in patients with AL Amyloidosis
timeFrame: Change from Baseline to 12 months
Other outcomes:
Locations:
city: South San Francisco
country: United States
facility: Online registry
state: California
Study officials:
References:
citation: Bayliss M, McCausland KL, Guthrie SD, White MK. The burden of amyloid light chain amyloidosis on health-related quality of life. Orphanet J Rare Dis. 2017 Jan 19;12(1):15. doi: 10.1186/s13023-016-0564-2.