Clinical trials: Acute Porphyria
Acute Intermittent Porphyria (AIP)
Identification of Acute Intermittent Porphyria Modifying Genes
This study proposes to identify the predisposing/protective modifying genes that underlie the acute attacks in symptomatic patients with Acute Intermittent Porphyria (AIP), an autosomal dominant inborn error of heme biosynthesis.
- Trial name
- NCT05502133
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Acute Hepatic Porphyria
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
- Trial name
- NCT02240784
- Where
- 30 sites in United States, Australia and 18 more
- Volunteers
- 136 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Porphyrias, Cutaneous Porphyrias
Longitudinal Study of the Porphyrias
The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes, and mortality in people with these disorders.
- Trial name
- NCT01561157
- Where
- 16 sites in United States
- Volunteers
- 1,500 people
- Sponsor
- The American Porphyrias Expert Collaborative
Acute Hepatic Porphyria, Hepatic Porphyrias
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic…
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP)
- Trial name
- NCT03547297
- Where
- 12 sites in United States
- Volunteers
- 212 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Hepatic Porphyria
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and…
- Trial name
- ELEVATE (NCT04883905)
- Where
- 30 sites in United States, Belgium and 8 more
- Volunteers
- 150 people
- Sponsor
- Alnylam Pharmaceuticals
Porphyrias
Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria
This is an observational study collecting patient/caregiver reports on suspected medication/drug-induced acute porphyria attacks, as well as safe use of drugs previously labeled "unsafe" or with unknown risk.
- Trial name
- NCT03906214
- Testing
- Web-based Survey
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of South Florida
Acute Hepatic Porphyria
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
- Trial name
- NCT04056481
- Testing
- Givosiran
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Coproporphyria (HCP), Acute Intermittent Porphyria (AIP)
Clinical Diagnosis of Acute Porphyria
The purpose of this study is to test whether a focused questionnaire and laboratory tests can better define risk factors associated with possible genetic porphyria.
- Conditions
- Hereditary Coproporphyria (HCP), Acute Intermittent Porphyria (AIP), Variegate Porphyria (VP)
- Trial name
- NCT01568554
- Where
- 6 sites in United States
- Volunteers
- 148 people
- Sponsor
- University of California, San Francisco
Rare Diseases, Porphyrias
Colombia National Porphyria Registry
porphyria is classified as a rare disease and is produced by defects in the enzymatic activity in the biosynthesis of the heme group that leads to the over-accumulation and excretion of porphyrin precursors in hepatocytes or…
- Trial name
- NCT05496933
- Where
- Colombia
- Volunteers
- 100 people
- Sponsor
- Fundación Grupo de Investigación en Cuidados Intensivos y Obstetricia
Acute Hepatic Porphyria, Acute Intermittent Porphyria
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
The purpose of this study is to evaluate the effect of subcutaneous givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP).
- Conditions
- Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent, Acute Porphyria
- Trial name
- NCT03338816
- Testing
- Givosiran
- Where
- 36 sites in United States, Australia and 16 more
- Volunteers
- 94 people
- Sponsor
- Alnylam Pharmaceuticals