ALN-TTR01 in Patients with Amyloidosis of Transthyretin (TTR)

• Research type

  Research Study

• Full title

  A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients with TTR Amyloidosis

• IRAS ID

  48299

• Contact name

  Tim Mant

• Eudract number

  2009-017383-16

• Research summary

  ALN-TTR01 is an investigational new drug that is being developed to treat TTR amyloidosis, a genetic illness that causes protein to accumulate in human tissue (heart, brain and liver.The purpose of this study is to see if a single dose of ALN-TTR01 is safe and can be tolerated without side effects. This includes a review of the side effects of ALN-TTR01 in order to see how much of the drug can be safely given to patients with TTR amyloidosis. In addition, the study will look at the amount of drug that gets into the blood and urine after study drug dosing, and whether the study drug decreases TTR levels in the blood.This study plans to enroll up to 28 patients (males and females) with TTR amyloidosis, ages 18 and older in up to five centres worldwide. Eligible patients will be made aware of the study by individual investigators in clinics of the participating institutions.Each patient may remain in the study for as long as 12 weeks. The study will involve up to a total of 13 visits to the research facility. If a patient??s TTR levels have not returned to at least 80% of the level before they received the study drug by the day 28 visit, the patient may be asked to return to the study doctor every 2 weeks until the final day 70 visit.The proposed research has been designed and will be funded by Alnylam Pharmaceuticals, Inc.

• REC name

  London - London Bridge Research Ethics Committee

• REC reference

  10/H0804/45

• Date of REC Opinion

  19 Jul 2010

• REC opinion

  Further Information Favourable Opinion