Erythropoietic Protoporphyria Clinical Trial
— EPPOfficial title:
Effect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic Protoporphyrias
Verified date | August 2020 |
Source | Icahn School of Medicine at Mount Sinai |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 16 |
Est. completion date | July 19, 2019 |
Est. primary completion date | July 19, 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
United States | University of Alabama, Birmingham | Birmingham | Alabama |
United States | University of Texas Medical Branch | Galveston | Texas |
United States | Icahn School of Medicine at Mount Sinai | New York | New York |
United States | University of Utah | Salt Lake City | Utah |
United States | University of California | San Francisco | California |
United States | Wake Forest University Health Sciences | Winston-Salem | North Carolina |
Lead Sponsor | Collaborator |
---|---|
Icahn School of Medicine at Mount Sinai | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in Erythrocyte Protoporphyrin Levels | Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment | Baseline and at 12 months | |
Secondary | Mean Change in EPP-specific Quality of Life Questionnaire | 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. Mean change at 12 months as compared to baseline. | Baseline and 12 months |
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