The DRIVE PK trial for people with Pyruvate Kinase deficiency
DRIVE PK is a phase 2 clinical trial for an investigational therapy, AG-348, developed to treat the underlying cause of Pyruvate Kinase (PK) deficiency.1,2 AG-348 is an oral medication that is being studied to determine whether it may increase the levels of healthy red blood cells in the body that are critical for people with PK deficiency.1,2 It is not known if AG-348 will help with increasing the levels of healthy red blood cells in the body—that is why this study is being conducted.
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A closer look at PK deficiency
PK deficiency is a rare inherited disease that can result in lifelong problems.1,2 It occurs when the protein (or enzyme) that is needed to make healthy (or mature) red blood cells is deficient—this enzyme is called Pyruvate Kinase.1 Because of this enzyme deficiency, people with PK deficiency have fewer healthy red blood cells (a condition known as anemia),1 and may experience signs and symptoms impacting quality of life, including:
- Fatigue (extreme tiredness)3
- Jaundice (a yellowing of the skin and/or whites of the eyes)4
- Shortness of breath3,5
People with PK deficiency may also require frequent blood transfusions to help manage their PK deficiency.4 Though doctors can help manage signs and symptoms, there is no approved drug that treats the underlying cause of PK deficiency.2
The AG-348 DRIVE PK trial
The DRIVE PK trial will study an investigational therapy, AG-348, which is being developed to treat the underlying cause of this disease—the deficiency of the Pyruvate Kinase enzyme.1,2 AG-348 attaches to the defective enzyme and may help to activate it, which, in turn, may help increase the amount of healthy red blood cells in the body.2 This could help people with PK deficiency avoid the damaging effects of the disease, but this has not been proven.
In the DRIVE PK trial, doctors will study different doses of AG-348 to see if the treatment is safe and well tolerated in adults. Doctors will also look for early signs that AG-348 helps increase the amount of healthy red blood cells. This trial will last up to 24 weeks, and all participants who qualify to participate in the study will receive AG-348. There is no cost to be a part of this trial.
Sign up for more information on the DRIVE PK trial and PK deficiency.