drugset / Trial / NCT01550705
Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria
Terminated
11 enrolled
University of Utah
Icahn School of Medicine at Mount Sinai · collabNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · collabUniversity of Alabama at Birmingham · collabUniversity of California, San Francisco · collabUniversity of Texas · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2012-03
- Primary completion
- 2015-12
- Completion
- 2015-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Isoniazid | Small molecule | 5 mg/kg | Oral |