Clinical Trial of ALNAS1 in Patients with Acute Intermittent Porphyria
Research type
Research Study
Full title
A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AS1 in Patients with Acute Intermittent Porphyria (AIP)
IRAS ID
172825
Contact name
David Rees
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc
Eudract number
2014-003618-10
Duration of Study in the UK
0 years, 11 months, 6 days
Research summary
Acute Intermittent Porphyria (AIP) is a rare disease caused by a defect in an enzyme porphobilinogen deaminase (PBGD) that makes a substance called haem in the liver. Haem is important for our body to use oxygen and for the liver to work properly. Some people with AIP do not have any symptoms. Others experience frequent attacks of severe stomach pain, nausea, vomiting, diarrhoea, muscle weakness, confusion, seizures, and in some cases paralysis.
People with AIP have higher amounts of PBG and ALA, substances that make haem and this causes injury to nerve cells. ALN-AS1 (Study Drug) is thought to work by lowering the amount of PBG and ALA. ALN-AS1 is a ribonucleic acid (RNA) that looks just like a part of the message that tells the liver to make PBG and ALA. It is believed the study drug will temporarily stop the message to make PBG and ALA. This means the liver might make less PBG and ALA, and this could help people with AIP by reducing injury to nerve cells and to which may improve the symptoms they experience.
The purpose of this research study is to see if the study drug is safe and tolerable and see if study drug lowers the amount of porphobilinogen (PBG) and/or 5-aminolevulinic acid (ALA) in the blood or urine of people with AIP or has an effect on other substances (called ‘biomarkers’) in the blood or urine of people with AIP.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
15/LO/0433
Date of REC Opinion
27 Apr 2015
REC opinion
Further Information Favourable Opinion