Clinical Trial: Oral Iron for Erythropoietic Protoporphyrias

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Effect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic Protoporphyrias

Brief Summary: In the medical literature there are conflicting reports on whether iron improves symptoms in patients with EPP and XLP. Giving iron to people who are iron deficient is thought to improve EPP symptoms. However, this has never been systematically tested. Therefore, the purpose of this study is to determine the effect of oral iron for EPP and XLP patients.

Detailed Summary:

This is a clinical trial, which means its purpose is to study an intervention or treatment. In this study all patients with EPP or XLP will be given a standard dose of iron pills and monitored for one year. There is currently no effective Food and Drug Administration (FDA) approved treatment for EPP or XLP in the US. Giving iron to patients with low ferritin (a measure of body iron stores) levels may help improve their EPP symptoms by decreasing erythrocyte protoporphyrin levels.

Patients will be asked come to the study site once every three months over the course of a year for a total of five visits. At these visits the study doctors will check in with participants and some blood and urine samples will be taken. Participants will not be charged for any of the lab tests that are being done for part of this study alone. In between these study visits there will be phone call to check in and see how participants are doing. All patients in this study will receive iron pills at no cost to them.


Sponsor: Icahn School of Medicine at Mount Sinai

Current Primary Outcome: Change in erythrocyte protoporphyrin levels [ Time Frame: Baseline and at 12 months ]

Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment


Original Primary Outcome: Same as current

Current Secondary Outcome: EPP-specific Quality of Life Questionnaire [ Time Frame: At 12 months ]

Quality of life tool designed to capture the specific issues that patients with EPP experience. Score range is 0-100, higher scores represent higher levels or satisfaction, lower scores represent more impacted quality of life.


Original Secondary Outcome: Same as current

Information By: Icahn School of Medicine at Mount Sinai

Dates:
Date Received: November 29, 2016
Date Started: December 2016
Date Completion: August 2019
Last Updated: February 24, 2017
Last Verified: February 2017