CompletedNot Applicable

Oral Iron for Erythropoietic Protoporphyrias

United States16 participantsStarted 2016-12

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Enrollment in the Longitudinal Study of the Porphyrias * Male or female age ≥18 years * History of nonblistering cutaneous photosensitivity * Biochemical findings - A marked increase in erythrocyte protoporphyrin (total erythrocyte protoporphyrin \>400 ug/dL) with a predominance of metal-free protoporphyrin * Serum ferritin ≤30 ng/mL at baseline * Able to tolerate oral iron Exclusion Criteria: * History of liver or bone marrow transplant or clinically significant liver dysfunction as determined by the investigator * Known or suspected allergy to oral iron based on patient report * Clinical evidence of active and ongoing GI bleeding * Use of any other clinical or experimental therapy in the past 3 months * Individuals with elevations of porphyrins in plasma or erythrocytes due to other diseases (i.e. secondary porphyrinemia) such as liver and bone marrow diseases * Patients with any clinically significant comorbid conditions, which in the opinion of the investigator, precludes participation

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in Erythrocyte Protoporphyrin Levels

Timeframe: Baseline and at 12 months

Trial details

NCT IDNCT02979249

SponsorIcahn School of Medicine at Mount Sinai

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2019-07-19

Results submitted2020-08-24

View on ClinicalTrials.gov More Erythropoietic Protoporphyria trials