Open-label ALN-AS1 in Patients with Acute Intermittent Porphyria
Research type
Research Study
Full title
A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients with Acute Intermittent Porphyria who have Completed a Previous Clinical Study with ALN-AS1
IRAS ID
210748
Contact name
David Rees
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc
Eudract number
2016-002638-54
Clinicaltrials.gov Identifier
126094, IND Number
Duration of Study in the UK
2 years, 11 months, 23 days
Research summary
Acute Intermittent Porphyria (AIP) is a rare, inherited metabolic disorder caused by a defect in an enzyme responsible for making haem (component of haemoglobin) in the liver, resulting in a build-up of haem. Some patients with AIP never develop symptoms, however certain factors such as drugs, hormonal changes and diet can trigger symptoms, causing an attack which usually lasts a few days. Symptoms include severe stomach pain, vomiting, muscle weakness and psychiatric problems (eg. anxiety, confusion). The prevalence of AIP is estimated as 5-10 per 100,000.
Management of attacks usually require hospitalisation and treatment with carbohydrate loading and painkillers. Those who fail to respond require haem administered via a vein however side effects include vein inflammation and blood clots and may have decreased efficacy with repeat administration. Liver transplant is currently the only cure for AIP however transplantations have limitations of organ availability and risk of rejection or infection. There remains a significant unmet need for an efficacious, simple, fast-acting and better tolerated treatment for attacks and prevention of recurrent attacks in patients with AIP.
Alnylam Pharmaceuticals, Inc. have developed a new drug, ALN-AS1, that has been designed to help deliver small interfering ribonucleic acids (siRNA) to the liver. The siRNA inhibits production of ALAS-1 (protein in the body) which is believed to then reduce accumulation of haem.
The study is being done to evaluate the long-term safety and tolerability of ALN-AS1 in patients with AIP, when administered via subcutaneous (under the skin) injection either monthly or every 3 months for 3 years.
This is a multicentre, open-label extension study which will take place across Europe and United States. It is anticipated that up to 24 patients who have completed study ALN-AS1-001 will be enrolled.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
16/WM/0356
Date of REC Opinion
24 Aug 2016
REC opinion
Favourable Opinion