Current Research Studies Part 2 of 2Below you will find a list of current studies with short summaries. Please consider being a part of a study. The research is a key to your cure!7204: Clinical Diagnosis of Acute PorphyriaWe will enroll individuals who are first-degree relatives (child, sibling, parent, or grandparent) of a patient with a diagnosis of one of the acute porphyrias (index case). We are interested in all three types of acute porphyria in which the index case has been confirmed by genetic testing: Acute Intermittent Porphyria (AIP), Hereditary Corproporphyria (HCP) or Variegate Porphyria (VP). Participants (the first-degree relatives) must not have had any genetic testing as yet. They will have an initial visit during which they will complete a history questionnaire and have routine laboratory tests, including genetic testing for porphyria. The researchers will use this data to develop a Clinical Profile of the risk factors associated with being a genetic carrier of acute porphyria.7205: Measuring the Effects of Isoniazid Treatment on Erythrocyte and Plasma Protoporphyrin IX Concentration in Patients with Erythropoietic ProtoporphyriaThis is an interventional study (study in which investigators give research subjects a particular medicine (in this case isoniazid) to measure how the subjects' health changes while using the medicine). To participate, all participants must also be enrolled in the Longitudinal Study of the Porphyrias and be willing to share their medical records with researchers. In the initial visit, participants will have a general physical exam and blood drawn for lab work. Participants will also be prescribed a dose of 300 mg of Isoniazid that they will need to take daily. Participants will need to have a follow up visit every two weeks for twelve weeks. Each follow up visit will include a blood test to see how the Isoniazid is working.7206: Hydroxychloroquine vs. phlebotomy for porphyria cutanea tardaThis study compares two treatments for PCT. The initial treatment phase may take up to 6 months. During this phase subjects are followed every 2 weeks. After the treatment phase subjects will be followed at least 3 years.Study patients will be characterized in terms of known risk factors for PCT, including drinking alcohol, smoking, hepatitis C, HIV infection, estrogen use, and genetic characteristics.Blood plasma and urine porphyrin concentrations will be measured at 2-week intervals for up to 6 months. After that participants will be followed every 2 months for the first year and every 6 months for the second year. There will be one final follow-up visit in the third year.7207: Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact)This is a longitudinal study (study taking place over a long period of time) of about 150 individuals with EPP. Participants enrolled in this study should also be enrolled in the "Longitudinal Study of the Porphyrias" Those participating in this study will be evaluated yearly. Follow up visits can be conducted with the participant's local physicians.Please contact the APF for more information or to get enrolled in the studies 713.266.9617.
"Remember....Research is the key to your cure!"
University of Texas Medical Branch in Galveston, TX is recruiting patients with acute porphyria attacks.
During the trial, both glucose and hemin given by an infusion through the veins and are considered standard treatments for attacks of porphyria. Hemin has been on the market since 1983, and based on experience over the past 30+ years is believed to be the most effective treatment. But a good study to prove that it is effective has not been done, and this has impaired its acceptance by doctors and its availability to patients. This study is designed to provide the needed evidence that has not been available before.
The research questions are:
- To evaluate the effectiveness of glucose and Panhematin, compared to glucose alone treatment for acute attacks of porphyria
- To evaluate the safety of glucose and Panhematin, compared to glucose alone for acute attacks of porphyria.
This is a clinical trial, which means its purpose is to study an intervention or treatment. It is also a randomized, controlled clinical trial, which means there is a treatment group given active drug and a control group given an inactive placebo. In this study, both groups are also treated with glucose, which is what the Food and Drug Administration (FDA) currently recommends before treatment with Panhematin. The study will occur in the hospital, with planned follow up visits in the outpatient clinic or by telephone.
To be eligible to participate in this study, you must:
Be an individual with one on the following, documented by lab reports:
- Acute intermittent porphyria (AIP)
- Hereditary coproporphyria (HCP)
- Variegate porphyria (VP)
- Be in an acute attack of porphyria.
Contact the Amy Chapman~ Amy.APF@gmail.com orAPF for enrollment and more details 713.266.9617.
"Remember....Research is the key to your cure!"