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The purpose of this multi-center study, which is also known as SPIMM-203, is to understand the long-term effects of the study drug elamipretide on people with primary mitochondrial disease (PMD). Mitochondrial diseases are rare and occur when one or more genetic mutations result in mitochondria failing to create enough energy to support the body, causing damage to the brain, heart, kidneys, muscles, or other organ systems.
The study accepts male and female patients, ages 16 and older, who have a genetically confirmed PMD and who have completed the end-of-study visit in the SPIMM-202 trial.Males and Females: Ages 16 and older
Participants will continue to receive treatment with an injection of a specific dose of elamipretide – either self-administered or administered by a caregiver – for up to 260 weeks, subject to study terms. Other tests, including electrocardiogram, blood and urine sampling, and various physical tests will be done at the study onset and at prescribed intervals. Participants will be seen by researchers at 3 months, 6 months, 12 months, and thereafter every 6 months until their participation in the study ends. Researchers will also phone participants monthly for status checkups.
Duration: Up to 5 years
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Michele Graham, RN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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