NCT04964323

Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

Pyruvate Kinase Deficiency Global Longitudinal Registry: Patient-Reported Outcomes

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 15 (actual)
• Sponsor: Agios Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

Participants aged ≥18 years and previously enrolled in Study AG348-C-008 (NCT03481738) will be eligible to participate in this study. Data will be submitted via electronic patient-reported outcomes (ePRO) application. All data collection efforts will abide by this protocol and be prospectively disclosed in the study informed consent. This study, with the appropriate participant (and or parent/guardian) consent/assent, may incorporate retrospective data from other properly consented studies done for the purpose of examining the longitudinal natural history of PK deficiency.

Conditions

• Pyruvate Kinase Deficiency
• Pyruvate Kinase Deficiency Anemia
• Anemia
• Anemia, Hemolytic

Timeline

• Start date: 2021-07-02
• Primary completion: 2023-06-30
• Completion: 2023-06-30
• First posted: 2021-07-16
• Last updated: 2023-09-07

Locations

5 sites across 5 countries: United States, Italy, Netherlands, Spain, United Kingdom