Stopped: Lack of enrollment and participant compliance with surveys.
The primary objective of this study is to understand and characterize the health-related quality of life (HRQoL) and disease burden of adult participants with PK deficiency receiving routine clinical care. This study is an observational (i.e., noninterventional), longitudinal, multicenter, global registry for participants with PK deficiency, a rare nonspherocytic hemolytic anemia. This study will be open for enrollment for 2 years and all enrolled participants will be followed prospectively for up to 96 weeks. Data will be collected from participants who have provided informed consent and authorization pursuant to applicable laws and regulations.
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Change from Baseline in Pyruvate Kinase Deficiency Diary (PKDD) Score
Timeframe: Baseline, up to Day 672
Change from Baseline in Pyruvate Kinase Deficiency Impact Assessment (PKDIA) Score
Timeframe: Baseline, up to Day 672
Change from Baseline in Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI: SHP) 2.0 Score
Timeframe: Baseline, up to Day 672
Change from Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An)
Timeframe: Baseline, up to Day 672