Status: Recruiting
Background
The porphyrias are a group of rare metabolic diseases that may present in childhood or adult life and are due to deficiencies of enzymes in the heme biosynthetic pathway. Porphyrias have various symptoms depending on the type, but these can range from neurological symptoms to sun sensitivity. See the descriptions of each type to get more information. The natural history of these disorders is not well described and it is not known why some patients are more severe than others. Therefore, the purpose of this long-term follow-up study is to collect a large group of patients with the different types of porphyria and to provide a better understanding of the natural history of these disorders. The hope is that this information will help in developing new forms of treatment.
The research aims are:
Contact the American Porphyria Foundation to learn more about this study and be placed in contact with the research coordinator. (general@porphyriafoundation.org; 301-347-7166)
Participating Locations: |
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1 - University of Texas Medical Branch (UTMB - Galveston, TX) |
Panhematin Prevention Study
Are you currently using Panhematin® to prevent acute porphyria attacks? Dr. Karl Anderson, esteemed Porphyria Expert at The University of Texas Medical Branch in Galveston, Texas is currently seeking patients for a Panhematin® Prevention study. The purpose of this study is to determine if Panhematin® is effective for prevention of acute attacks of porphyria. We are seeking 20-30 patients who currently receive prophylactic preventative heme treatment. You may be receiving heme weekly, bi-weekly or once a month and have successfully prevented attacks. Participation in this study will involve traveling to the study site for participation. Travel to the study site will be included.
RESEARCH STUDY: |
STUDY DESCRIPTION: |
WHO CAN PARTICIPATE: |
PARTICIPATING SITES: |
Panhematin Prevention Study |
The purpose of this study is to determine if Panhematin® is effective for prevention of acute attacks of porphyria. |
Patients diagnosed with AIP, VP or HCP |
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Contact the American Porphyria Foundation to learn more about this study and be placed in contact with the research coordinator. (general@porphyriafoundation.org; 301-347-7166)
Contact the American Porphyria Foundation to learn more about this study and be placed in contact with the research coordinator. (general@porphyriafoundation.org; 301-347-7166)
No active recruitment for PCT at this time. Contact the American Porphyria Foundation to learn more about this study and be placed in contact with the research coordinator. (general@porphyriafoundation.org; 301-347-7166)