Trial Search Results
Pyruvate Kinase Deficiency Natural History Study
The purpose of this study is to describe the range and incidence of symptoms, treatments, and complications related to pyruvate kinase deficiency (PKD). Eligible patients are those of all ages with known PKD or with a hemolytic anemia and a family member with PKD. The study will collect retrospective medical history, routine clinical care data, and quality of life measures at baseline and annually for patients with PKD.
Stanford is currently not accepting patients for this trial.
Boston Children's Hospital
Collaborator: Agios Pharmaceuticals, Inc.
- Patients of all ages with biochemically or genetically diagnosed PKD.
- Patients with a hemolytic anemia AND a family member with genetically diagnosed PKD
- The participant or the guardian of the participant is willing and able to give written
informed consent and/or assent.
- The participant or the guardian of the participant is unwilling or unable to give
written informed consent and/or assent.
Ages Eligible for Study
N/A - N/A
Genders Eligible for Study