INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP

Alnylam Pharmaceuticals

Status: Terminated

Conditions: ALA Dehydratase Deficient Porphyria (ADP); Variegate Porphyria (VP); Hereditary Coproporphyria (HCP); Acute Intermittent Porphyria (AIP); Hepatic Porphyrias; Porphyria Acute; Acute Hepatic Porphyria

Study type: Observational

Funder types: Industry

Identifiers

NCT03547297

ALN-AS1-NT-002

Details and patient eligibility

About

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 process for patients in the future.

Enrollment: 212 patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• ≥ 16 years of age
• Recurrent and severe episodes of abdominal pain that last for at least 24 hours
• At least 1 episode of severe abdominal pain that required an urgent healthcare visit in the past 12 months
• Other symptoms related to AHP that your doctor will discuss with you
• Willing and able to comply with protocol required assessments and provide written informed consent

Exclusion criteria

• Known diagnosis of AHP
• Alternative explanation for abdominal pain with clinical response to treatment specific for alternative diagnosis