ALN-TTRSC-001 SAD/MAD

• Research type

  Research Study

• Full title

  A Phase 1, Randomized, Double-blind, Placebo-controlled, Single and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC in Healthy Volunteers

• IRAS ID

  121646

• Contact name

  Joseph Chiesa

• Sponsor organisation

  Alnylam Pharmaceuticals Ltd

• Eudract number

  2012-004203-12

• ISRCTN Number

  N/A

• Research summary

  In this study each group to be dosed will comprise of 4 subjects randomly receiving study drug or placebo as a single dose (single ascending dose -SAD) or as multiple doses (multiple ascending dose -MAD) of ALN-TTRSC or placebo given as injection(s) under the skin. The study will have 4 such groups receiving single doses and 3 groups receiving multiple doses with a possibility of 3 optional groups if required. Subjects will be screened from 45 to 2 days prior to dosing. In the SAD groups subjects will be admitted to the unit on the day before dosing. In each group, the second subject will dosed only after approximately 48 hrs. after the first subject has been dosed. Similarly the third and fourth subjects will be dosed at least 48 hours after the second subject was dosed. Subjects will be discharged approximately 24 hours after dosing. They will return for outpatient visits up to 56 days post-dose. In the MAD group subjects will receive a total of 10 doses: 1 dose each day for 5 days followed by 5 weekly doses on Days 7, 14, 21, 28 and 35. In all groups, apart from the optional groups, the second subject will be dosed with ALN-TTRSC or placebo only after a safety assessment carried out approximately 48 hours after the fifth dose of the first subject. Similarly the 3rd and 4th subjects will be dosed only after a safety assessment is carried out after the fifth dose of the second subject. Subjects will be discharged on Day 5 and will return on an outpatient basis for rest of the dosing. Subjects will be re-admitted to be dosed on Day 35, and discharged next day. Subjects will return on an outpatient basis, for safety assessments and blood sampling for up to 90 days after the first dose.

• REC name

  North East - York Research Ethics Committee

• REC reference

  13/NE/0009

• Date of REC Opinion

  22 Jan 2013

• REC opinion

  Favourable Opinion