N/A
Completed N=16
Oral Iron for Erythropoietic Protoporphyrias
Erythropoietic Protoporphyria · EPP · X-linked Protoporphyria · XLP
Source: ClinicalTrials.gov NCT02979249 ↗
Enrolled (actual)
16
Serious AEs
30.0%
Results posted
Sep 2020
Primary outcomePrimary: Change in Erythrocyte Protoporphyrin Levels — 2314.6; 2240.4 µg/dL
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Erythrocyte Protoporphyrin Levels |
2314.6; 2240.4 | — |
| SECONDARY Mean Change in EPP-specific Quality of Life Questionnaire |
3.3 | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT02979249). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.