Bitopertin access program for rare blood porphyria diseases
Part of Digestive system, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This program provides access to bitopertin, an experimental medicine being tested for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP)—rare genetic conditions affecting how your body processes heme, a component of blood. The program aims to offer the medication to eligible patients while safety is being studied.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 12 or older at the time of enrollment
- Confirmed diagnosis of EPP or XLP (by genetic testing or blood porphyrin analysis)
- Hemoglobin level of at least 10 g/dL (a measure of red blood cell health)
- Healthy liver function and no history of liver transplant
- Willingness to avoid certain medications and foods that interact with bitopertin
- If female and able to become pregnant: negative pregnancy test and agreement to use contraception
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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