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RP-L301 for Pyruvate Kinase Deficiency
Study Summary
This trial is testing a new gene therapy for people with Pyruvate Kinase Deficiency, a rare blood disorder. The goal is to find out if the therapy is safe.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
Frequently Asked Questions
What potential risks does RP-L301 pose for patients?
"Due to the Phase 1 trial nature of RP-L301, our team at Power estimates its safety as a 1. Data regarding efficacy and safety is still limited in this case."
How many participants are currently undergoing this research project?
"Affirmative, according to clinicaltrials.gov information this medical trial is presently seeking out potential participants. This experiment was initially posted on July 6th 2020 and recently updated on September 22nd 2021, with a requirement for the recruitment of six patients from one site."
Who is eligible to enroll in this trial?
"6 applicants will be accepted into this clinical trial, provided they meet the criteria of pyruvate metabolism and inborn errors disorders as well as being between 8 to 50 years old."
Are there any open slots available for participation in this investigation?
"According to the clinicaltrials.gov data, this research is recruiting patients presently; it was posted on July 6th 2020 and last updated September 22nd 2021."
Does the age restriction for participants in this study extend to minors?
"This research project is offering enrollment to individuals aged 8 and over, but not exceeding 50 years of age."
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