DTX101 in adults with moderate/severe Haemophillia B

• Research type

  Research Study

• Full title

  A Phase I/II Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B

• IRAS ID

  199055

• Contact name

  Allen Poma

• Contact email

  allen.poma@dimensiontx.com

• Sponsor organisation

  Dimension Therapeutics, Inc.

• Eudract number

  2015-001486-67

• Clinicaltrials.gov Identifier

  NCT02618915

• Clinicaltrials.gov Identifier

  16453, IND

• Duration of Study in the UK

  1 years, 7 months, 28 days

• Research summary

  Hemophilia B is an X-linked recessive bleeding disorder that affects approximately 1 in
  20,000 to 25,000 male births. Mutations in the factor IX (FIX) gene,prevent normal blood clotting occurring . The disease is characterized by frequent, spontaneous internal bleeding that can lead to chronic joint damage, intracranial hemorrhage, and even death. In patients with moderate/severe to severe hemophilia B, 90% of all bleeding episodes occur in the joints and, if not treated properly,can lead to debilitating damage and a decreased quality of life.

  The current standard of care for hemophilia B is intravenous (IV) injections of either recombinant or plasma-derived FIX concentrates at the time of a bleed or prophylactically to prevent bleeding. These treatment options do not completely prevent the chronic and debilitating damage to joints and soft tissues that follows a bleeding episode.

  DTX101 is a gene therapy that is intended to produce FIX and promote the normal blood clotting process.A transporter or ´vector´ is used to deliver the FIX to the target cells.The vector in DTX101 is a virus called adeno-associated virus rh10 (AAVrh10).
  Adeno-associated virus is a common virus found in human respiratory systems. In its normal state, it can reproduce and cause respiratory infections like a common cold.The AAVrh10 vector used in DTX101 to deliver FIX, has been changed in the laboratory so that it cannot reproduce or cause an infection

  Subjects will receive a single dose of DTX01 and be monitored for 52 weeks. Clinic visits will occur every 4 days for 12 weeks , followed by 4 weekly visits until week 52. Visits will include a Physical examination, ECG, collection of blood,stool and saliva samples . Subjects will also complete Quality of Life Questionnaires and diaries.

  DIMENSION are conducting this open label single arm multi centre dose finding Study. recruitment will be world wide with 12-18 patients recruited.

• REC name

  North East - York Research Ethics Committee

• REC reference

  16/NE/0078

• Date of REC Opinion

  27 May 2016

• REC opinion

  Further Information Favourable Opinion