TerminatedNot Applicable

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

Stopped: Lack of enrollment and participant compliance with surveys.

United States, Italy, Netherlands15 participantsStarted 2021-07-02

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Participant must be greater than or equal to (≥)18 years of age; * Participant must be enrolled in Study AG348-C-008. Exclusion Criteria: \- Participant is enrolled in an Agios-sponsored clinical study involving treatment with a pyruvate kinase activator.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Trial details

NCT IDNCT04964323

SponsorAgios Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2023-06-30

View on ClinicalTrials.gov More Pyruvate Kinase Deficiency trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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