Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria (INHEPP)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01550705|
Recruitment Status : Terminated (Interim analysis demonstrated the treatment was not effective)
First Posted : March 12, 2012
Results First Posted : January 16, 2017
Last Update Posted : January 16, 2017
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.
|Condition or disease||Intervention/treatment||Phase|
|Erythropoietic Protoporphyria (EPP) X Linked Erythropoietic Protoporphyria||Drug: Isoniazid||Not Applicable|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||11 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Quantification of the Effects of Isoniazid Treatment on Erythrocyte and Plasma Protoporphyrin IX Concentration and Plasma Aminolevulinic Acid in Patients With Erythropoietic Protoporphyria|
|Study Start Date :||March 2012|
|Actual Primary Completion Date :||December 2015|
|Actual Study Completion Date :||December 2015|
Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check.
Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.
- Change in Plasma Protoporphyrin IX Level [ Time Frame: Baseline and 3 Months ]Plasma Protoporphyrin IX will be measured at baseline and at 3 months
- Participants With Increased Sun Sensitivity [ Time Frame: Baseline and 3 Months ]Study participants were asked to report after 3 months if they had experienced an increase in subjective measures of sun sensitivity during the trial. Reported outcome is the number of study participants who reported increased sun sensitivity
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01550705
|United States, Utah|
|University of Utah School of Medicine|
|Salt Lake City, Utah, United States, 84132|
|Principal Investigator:||John D Phillips, PhD||University of Utah|