Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria
Status:
Terminated
Trial end date:
2015-12-01
Target enrollment:
Participant gender:
Summary
In erythropoietic protoporphyria there is an accumulation of protoporphyrin IX (PPIX) in the
plasma and liver. The reason it builds up is either the last step to make heme, insertion of
iron into PPIX, is rate limiting or there is an increase in activity in the first step in the
heme pathway.
It may be possible to decrease the amount of PPIX made and see a decrease in symptoms. The
first step to make heme is the key step in the pathway and it uses vitamin B6 as a cofactor.
If the investigators can limit the amount of vitamin B6 the investigators can possibly reduce
the activity of this rate limiting step. With decreased activity of the enzyme it may be
possible for the body to utilize all the PPIX that is made so that none builds up.
Phase:
N/A
Details
Lead Sponsor:
University of Utah
Collaborators:
Icahn School of Medicine at Mount Sinai National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) University of Alabama at Birmingham University of California, San Francisco University of Texas