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Treatment for Anemia (PKD Trial)

N/A
Waitlist Available
Research Sponsored by Agios Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, up to day 672
Awards & highlights
No Placebo-Only Group

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.

Eligible Conditions
  • Anemia
  • Hemolytic Anemia
  • Pyruvate Kinase Deficiency

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, up to day 672
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, up to day 672 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An)
Change from Baseline in Pyruvate Kinase Deficiency Diary (PKDD) Score
Change from Baseline in Pyruvate Kinase Deficiency Impact Assessment (PKDIA) Score
+1 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
53 Previous Clinical Trials
4,166 Total Patients Enrolled
4 Trials studying Anemia
544 Patients Enrolled for Anemia
Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
40 Previous Clinical Trials
8,586 Total Patients Enrolled
~3 spots leftby Nov 2025
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