A Phase 1 Study of BIVV009 in Patients with Chronic ITP (BIVV009-201)

• Research type

  Research Study

• Full title

  A Phase 1 Safety, Tolerability, and Pharmacokinetics & Pharmacodynamics Study of Multiple-dose BIVV009 in Patients with Chronic Immune Thrombocytopenia (ITP)

• IRAS ID

  252958

• Contact name

  Marie Scully

• Contact email

  m.scully@ucl.ac.uk

• Sponsor organisation

  Bioverativ USA Inc.

• Eudract number

  2018-001692-20

• Clinicaltrials.gov Identifier

  NCT03275454

• Duration of Study in the UK

  3 years, 1 months, 0 days

• Research summary

  Chronic Immune Thrombocytopenia (ITP) is an autoimmune blood disorder. People with ITP have a lower than normal level of blood cells called platelets because they are destroyed by the body's own immune system. As platelets are required for normal blood clotting, patients with chronic ITP are at a higher risk of spontaneous bruising and bleeding. Whilst the exact mechanism is unknown, it is thought that the complement proteins of the immune system may be involved in the destruction of platelets and/or inhibition of platelet production.
  
  Existing first-line treatments for ITP try to prevent major bleeding rather than correct abnormal platelet counts. In some patients these therapies and subsequent second-line therapies may be ineffective. Bioverativ USA Inc. is developing a new medication, BIVV009, that is designed to act on the immune system complement protein, C1s, to prevent platelet destruction.

  This is a phase 1, open-label study to evaluate the safety, tolerability, pharmacokinetics (the impact of the body on the drug) and pharmacodynamics(the impact of the drug on the body) of multiple dose BIVV009 in chronic ITP patients.

  The study will be conducted in two parts:
  Part A: Eligible patients will receive a fixed dose infusion of BIVV009 every 2 weeks followed by a 9-week safety follow up period.
  Part B (Long-term treatment): Participants from Part A who are considered responders to BIVV009, will continue biweekly dosing of BIVV009 for an additional 52-week treatment period followed by a 9-week safety follow up period.

  This is a multicentre study which will recruit up to 16 patients with Chronic ITP from research sites in the UK, Germany and USA.

• REC name

  London - Brent Research Ethics Committee

• REC reference

  18/LO/1918

• Date of REC Opinion

  8 Mar 2019

• REC opinion

  Further Information Favourable Opinion