REGN7257 in Patients with Severe Aplastic Anemia

  • Research type

    Research Study

  • Full title

    A Phase 1/2 Study of REGN7257 (Anti-Interleukin 2 Receptor Subunit Gamma [IL2RG] Monoclonal Antibody) in Patients with Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy.

  • IRAS ID

    285419

  • Contact name

    Austin Gladston Kulasekararaj

  • Contact email

    Austin.kulasekararaj@nhs.net

  • Sponsor organisation

    Regeneron Pharmaceuticals, Inc.

  • Eudract number

    2020-002031-29

  • Duration of Study in the UK

    2 years, 0 months, 1 days

  • Research summary

    Severe Aplastic Anaemia (SAA),is a disease of the bone marrow resulting in an impairment of the production of blood cells.\n\nThe main purpose of this two-part study (Part A and Part B) is to test how safe and tolerable REGN7257 is in participants with Severe Aplastic Anaemia (SAA) when other Immunosuppressive therapy (ISTs) have not worked well.\n\nPart A will employ single ascending dose (SAD) escalation cohorts to assess safety and determine a preferred dose(s) for the expansion cohort(s).\n\nPart B will involve 1 or more expansion cohorts at the preferred dose(s) to assess safety and efficacy.\n\nUp to 48 participants (Part A: up to 18 participants, Part B: up to 30 participants) at approximately 15 research centres will be participating.\n\nThe study is divided into the following:\n•\tA screening period of up to 4 weeks, \n•\tA treatment period of 1 day \n•\tA follow-up period, where the study doctor and study staff will monitor participant’s health over a 12-month duration after participant receive the study drug treatment.\n•\tA second treatment of REGN7257 may be considered if participant’s respond to the first treatment of REGN7257 and then relapse. This second treatment is given at the discretion of participant’s study doctor following consultation with Regeneron. If participant receive a second treatment of REGN7257, an additional follow-up period of 12 months applies, beginning after completion of the second treatment of REGN7257. \n\nDuration of study participation may last up to 108 weeks. If participants do not receive a second treatment of REGN7257, their total participation in the study may last up to 56 weeks. \n\n

  • REC name

    Yorkshire & The Humber - Leeds West Research Ethics Committee

  • REC reference

    20/YH/0229

  • Date of REC Opinion

    5 Nov 2020

  • REC opinion

    Further Information Favourable Opinion