ACTIVATE and ACTIVATE-T Clinical Trials
Pyruvate Kinase Deficiency is a manageable disease; however, there are currently no approved medicines that treat the underlying cause of the disorder. Due to a deficiency in the enzyme, pyruvate kinase, people with pk deficiency are anemic, which means they have a reduced number of healthy red blood cells. At this time Agios Pharmaceuticals is conducting its ACTIVATE and ACTIVATE – T Clinical Trials for the oral medication, AG-348. This therapy has been developed as a potential treatment for PK Deficiency. The study is being conducted to determine the safety and effectiveness of AG-348. To learn more about these studies visit Agios’s Activate Clinical Trial website.
“The decision to participate in a clinical trial is an IMPORTANT one. ASK QUESTIONS! Read about participating in clinical research. Talk to researchers, physicians, family members, and friends to help decide if a trial is right for you.”
PKD Clinical Trial Participant
For updated information on pyruvate kinase deficiency clinical studies, go to http://www.clinicaltrials.gov.
Watch this video to learn more about what a clinical trial is and how it works.
Part of the Roche Drawn to Science video Series
Natural History Study
The purpose of this study is to describe the symptoms, treatments, and complications related to Pyruvate Kinase deficiency (PKD). Patients of all ages are eligible to enroll in the study. Participants will be asked to fill in questionnaires regarding their quality of life. In addition, the study team will collect past medical history and routine clinical care information. Those who are interested in participating or want more information about this study can contact the following researchers:
Dr. Rachael Grace
Boston Children’s Hospital
Phone: (617) 919-2144
You may go to the following link to get more information about this study:
Pyruvate Kinase Deficiency Natural History Study(PKD NHS)
Dr. Eduard J van Beers
University Medical Centre
Utrecht, the Netherlands
T: +31 88 75 584 50
F: +31 88 75 554 38
ForGeTPKD – gene therapy
Dr. José Carlos Segovia
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)
Avda. Complutense 22 28040 Madrid • Spain