CR-BD-001

• Research type

  Research Study

• Full title

  An exploratory, open-label study to evaluate the safety and feasibility of ATIR201, a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells (using photodynamic treatment), as adjuvant treatment to a Tcell depleted haploidentical hematopoietic stem cell transplantation in patients with beta-thalassemia major

• IRAS ID

  214528

• Contact name

  Rob Wynn

• Sponsor organisation

  Kiadis Pharma Netherlands B.V.

• Eudract number

  2016-002959-17

• Duration of Study in the UK

  2 years, 0 months, 1 days

• Research summary

  This exploratory study of ATIR201 in patients is a first step in the clinical development of a new therapeutic strategy in beta-thalassemia major. Beta-thalassemia major is an inborn disease leading to impaired production of hemoglobin and patients typically present with lifethreatening anemia within the first year of life.
  Allogeneic hematopoietic stem cell transplantation (HSCT) is the only treatment option available to date that has shown to lead to cure in these patients, but its use is limited by donor availability, opportunistic infections and the risk of graft-versus-host disease (GVHD). ATIR201 has the potential to address these current risks and limitations related to HSCT. ATIR201 is given as an adjuvant treatment after a haploidentical HSCT. ATIR201 is made of lymphocytes from the same donor as used for the initial HSCT. Prior to HSCT, cells are collected and processed outside the body to eliminate alloreactive T-cells, thereby eliminating potential GVHD-inducing T-cells while sparing the cells which can fight infections.
  After obtaining written informed consent from the patient or legal guardian(s) and informed assent from the patient according to his/her age, patients will receive an HSCT from a haploidentical donor,followed by ATIR201 infusion of T-cells from the same donor at a dose of 2×10E6 viable Tcells/kg between 28 and 32 days after the HSCT.
  All patients treated with ATIR201 will be followed up until 12 months after the HSCT.
  Assessments will be performed at weekly visits from the day of the HSCT (day 0) until 11 weeks after the HSCT (Week 11), at monthly visits from 3 until 6 months after the HSCT, and every 2 months from 6 until 12 months after the HSCT.

• REC name

  North East - York Research Ethics Committee

• REC reference

  17/NE/0364

• Date of REC Opinion

  30 Nov 2016

• REC opinion

  Further Information Favourable Opinion