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

NCT04964323 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 15

Last updated 2023-09-07

No results posted yet for this study

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.

Conditions

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

Sponsors & Collaborators

  • Agios Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Affairs · Agios Pharmaceuticals, Inc.

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-02
Primary Completion
2023-06-30
Completion
2023-06-30

Countries

  • United States
  • Italy
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04964323 on ClinicalTrials.gov