Phase 1 study of ALN-AT3SC in Healthy Males and Haemophilia Patients

• Research type

  Research Study

• Full title

  A Phase 1 Single-ascending and Multiple-ascending Dose, Safety, Tolerability and Pharmacokinetics Study of Subcutaneously Administered ALN-AT3SC in Healthy Adult Male Volunteers and Male Hemophilia A or B Patients (Moderate or Severe Hemophilia)

• IRAS ID

  139954

• Contact name

  David Bevan

• Contact email

  David.H.Bevan@gstt.nhs.uk

• Sponsor organisation

  Alnylam Pharmaceuticals, Inc.

• Eudract number

  2013-003135-29

• Research summary

  Haemophilia is a rare bleeding disorder in which blood does not clot normally because the body is unable to produce or has a defective blood factor protein (for example factor VIII or IX), which is required for blood clotting.
  People with haemophilia may bleed for a longer periods of time after an injury or, they may develop bleeds spontaneously. Haemophilia is usually an inherited disorder, meaning it is passed down from parents to children.
  ALNAT3SC is a new experimental medicine being developed by Alnylam Pharmaceuticals, Inc (the Sponsor) for the possible treatment of haemophilia. ALN AT3SC works by reducing the amount of a protein called antithrombin (AT) produced in the liver which has a role in preventing blood from clotting. ALN-AT3SC works by reducing the liver’s production of AT protein. In patients with haemophilia, researchers want to test whether lowering AT protein may help them clot more easily leading to improved hemostasis. ALN-AT3SC has not previously been tested in humans. In studies performed in animals, ALN-AT3SC led to lower amounts of AT protein in the blood. The purpose of this new study is to see if single doses of ALN-AT3SC are safe in normal healthy volunteers and if multiple doses of ALN-AT3SC are safe in patients with hemophilia. 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 has any impact on AT levels in the blood. ALN-AT3SC will be administered by subcutaneous injection (under the skin of abdomen). Up to 24 adult males are expected to be enrolled into this study from the UK. Up to 18 adult males with moderate to severe hemophilia A and B are planned for enrollment into this study from sites in UK, and a small number of other hemophilia centers in other parts of the world.

• REC name

  London - London Bridge Research Ethics Committee

• REC reference

  13/LO/1620

• Date of REC Opinion

  18 Dec 2013

• REC opinion

  Further Information Favourable Opinion