281102 - Prophylactic and on-demand treatment of cTTP with BAX 930

• Research type

  Research Study

• Full title

  A phase 3, prospective, randomized, controlled, open-label, multicenter, 2 period crossover study with a single arm continuation evaluating the safety and efficacy of BAX 930 (rADAMTS13) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP, Upshaw-Schulman Syndrome [USS], hereditary thrombotic thrombocytopenic purpura [hTTP])

• IRAS ID

  227050

• Contact name

  Judit Koranyi

• Contact email

  Judit.koranyi@shire.com

• Sponsor organisation

  Baxalta Innovations GmbH

• Eudract number

  2017-000858-18

• Clinicaltrials.gov Identifier

  IND Number, 015219

• Duration of Study in the UK

  3 years, 9 months, 30 days

• Research summary

  Summary of Research

  Thrombotic thrombocytopenic purpura (TTP) is a rare, life-threatening disease. TTP causes problems with the blood system, including low platelet count, blood clots in small blood vessels and low iron levels caused by loss of red blood cells; these can cause damage to multiple organs (mainly kidney, heart, and brain). In patients with TTP, the enzyme ADAMTS13 is faulty.
  The medication being evaluated in this study, BAX930, is an artificially made ADAMTS13, that could improve current treatment of TTP. The effectiveness of BAX930 will be evaluated as well as whether it causes side effects. BAX930 will be compared with standard TTP treatments, e.g. fresh frozen plasma (FFP), solvent-detergent treated plasma from donors, or concentrates of factor VIII.
  The study is comparing BAX930 to standard treatment in participants between 0-70 years old in two different scenarios:
  Prophylaxis cohort. Prophylaxis is when a medicine is given to prevent a condition from happening or worsening. In people with severe TTP, prophylaxis is used to prevent episodes of the condition that can affect the central nervous system and kidneys. The duration of treatment will last approximately 18 months.
  On-demand treatment cohort. Sudden episodes of TTP are urgently treated to stop the loss of blood cells and improve symptoms patients are having. Upon resolution of the acute event, subjects may choose to move to the prophylaxis cohort of the study.
  While the current treatment of TTP via plasma infusions is effective, the therapy can cause allergic reaction and carries a very small but not negligible risk of transmitting blood-borne viruses, BAX 930 does not carry this risk. FFP infusions in patients undergoing long-term preventative treatment, can cause haemorrhage related to the insertion of a catheter, blood clots and infection. An additional risk is the availability of FFP, which can fluctuate, a risk eliminated with BAX 930.

  Summary of Results

  The plain language summary (that was previously approved by the study team) needs to be updated and re-approved. Once re-approved, it will be posted on Takeda‘s disclosure website clinicaltrials.takeda.com.
  Link to study details page of study 281102: https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fclick.pstmrk.it%2F3ts%2Fclinicaltrials.takeda.com%252Fstudy-detail%252F5f6b5fc84db2bf003ab4602d%253F%253Fpage%253D1%2526idFilter%253D281102%2FNBTI%2FxfG7AQ%2FAQ%2F0dab4a42-0bb7-4b93-b338-3752ffc1c881%2F2%2FJbPIeS8mKi&data=05%7C02%7Clondoncentral.rec%40hra.nhs.uk%7C1e1296fc7d0f4307e47008dd6149f9fc%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638773692526697659%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=9rbR1biVv1XbUQPCstWqdQETH%2Bd9v8gOoXfxuWeY36g%3D&reserved=0
  

• REC name

  London - Central Research Ethics Committee

• REC reference

  17/LO/1157

• Date of REC Opinion

  12 Sep 2017

• REC opinion

  Further Information Favourable Opinion