Patients

Do you have PK deficiency?

If so, and if you are 18 years of age or older, you may be eligible for a clinical study.

The ACTIVATE and ACTIVATE-T clinical studies are testing the safety and effectiveness of an investigational medication, AG-348.

Talk with your doctor to see if you are eligible.

See if you prequalify
to participate

Understanding PK Deficiency

PK deficiency is a rare, inherited blood disorder caused by decreased activity of an enzyme called pyruvate kinase (PK). Pyruvate kinase is important for red blood cells to have enough energy to develop normally, to function properly, and to live a normal lifespan.

Without sufficient PK enzyme activity, red blood cells lose their shape and break apart, resulting in a condition called hemolytic anemia.

The symptoms of one person with PK deficiency may be different from those of another – in terms of both the types and severity of symptoms. Currently, treatment for PK deficiency is limited to managing the symptoms and complications.

See if you prequalify
to participate

Select a Study and Learn More

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ACTIVATE Clinical Study

About the ACTIVATE Study

The ACTIVATE clinical study is testing the safety and effectiveness of an investigational medication called AG-348 in people with PK deficiency, a rare blood disorder, who are not receiving regular blood transfusions for PK deficiency.

AG-348 is an investigational oral treatment that is designed to help activate the deficient PK enzyme. This, in turn, may help increase the amount of hemoglobin in the body.

The safety and efficacy of AG-348 have not been established. There is no guarantee that AG-348 will receive health authority approval or become commercially available in any country for the uses being investigated.


Are You Eligible for the Study?

You must be age 18 or older and must not be receiving regular blood transfusions for PK deficiency. Talk to your doctor to determine your eligibility to participate in the ACTIVATE clinical study.


Study Sites

The ACTIVATE clinical study is being conducted globally, including several sites in the United States. New site locations will continue to be added. Visit ClinicalTrials.gov to locate a clinical trial site near you.

Locate a clinical trial site

Frequently Asked Questions

Q: What is the ACTIVATE study?

A: The ACTIVATE clinical study is testing the safety and effectiveness of an investigational medication called AG-348 in adults with PK deficiency.

Q: Hasn’t the medication been tested already in an earlier study?

A: Yes, Agios completed enrollment of DRIVE PK, a Phase 2 study evaluating different doses of AG-348, in adults with PK deficiency who were not receiving regular transfusions.

Q: Am I eligible for the ACTIVATE study?

A: To be eligible for the ACTIVATE study, you must meet certain criteria, including being at least 18 years old and not receiving regular blood transfusions for PK deficiency. Talk to your doctor about your eligibility for this study.

Q: What if I’m not able to take part in the study?

A: If you are not eligible to take part in this study, there may be other studies that you could be involved in. Talk with your doctor about other options that may be available to you. Also, Agios is sponsoring a global PK deficiency disease registry, which is open to all people with a diagnosis of PK deficiency that has been confirmed by a genetic test. The disease registry is an observational study, meaning that no specific treatment or tests are being studied. For more information about the registry, ask your doctor or visit www.peakregistry.com.

Agios Activate-T Clinical Trial Logo

ACTIVATE-T Clinical Study

About the ACTIVATE-T Study

The ACTIVATE-T clinical study is testing the safety and effectiveness of an investigational medication called AG-348 in people with PK deficiency, a rare blood disorder, who are receiving regular blood transfusions for PK deficiency.

AG-348 is an investigational oral treatment that is designed to help activate the deficient PK enzyme. This, in turn, may help reduce the number of transfusions these people receive.

The safety and efficacy of AG-348 have not been established. There is no guarantee that AG-348 will receive health authority approval or become commercially available in any country for the uses being investigated.


Are You Eligible for the Study?

You must be age 18 or older and receiving regular blood transfusions for PK deficiency. Talk to your doctor to determine your eligibility to participate in the ACTIVATE-T clinical study.


Study Sites

The ACTIVATE-T clinical study is being conducted globally, including several sites in the United States. New site locations will continue to be added. Visit ClinicalTrials.gov to locate a clinical trial site near you.

Locate a clinical trial site

Frequently Asked Questions

Q: What is the ACTIVATE-T study?

A: The ACTIVATE-T clinical study is testing the safety and effectiveness of an investigational medication called AG-348 in adults with PK deficiency.

Q: Hasn’t the medication been tested already in an earlier study?

A: Yes, Agios completed enrollment of DRIVE PK, a Phase 2 study evaluating different doses of AG-348, in adults with PK deficiency. The goal of the study was to test different doses for safety and effectiveness in people who were not receiving regular blood transfusions.

Q: Am I eligible for the ACTIVATE-T study?

A: To be eligible for the ACTIVATE-T study, you must meet certain criteria, including being at least 18 years old and receiving regular blood transfusions for PK deficiency. Talk to your doctor about your eligibility for this study.

Q: What if I’m not able to take part in the study?

A: If you are not eligible to take part in this study, there may be other studies that you could be involved in. Talk with your doctor about other options that may be available to you. Also, Agios is sponsoring a global PK deficiency disease registry, which is open to all people with a diagnosis of PK deficiency that has been confirmed by a genetic test. The disease registry is an observational study, meaning that no specific treatment or tests are being studied. For more information about the registry, ask your doctor or visit www.peakregistry.com.

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About the Study Sponsor

Agios is a pharmaceutical company dedicated to creating disease-modifying medicines to treat rare genetic metabolic disorders, such as PK deficiency, that are unaddressed by existing therapeutic approaches. Agios is committed to better understanding PK deficiency and the burden experienced by those living with the disease.

o Learn more about Agios at www.agios.com.