Anaemia & Thalassemia

Stem cells have been used to treat diseases of the blood for many years. Listed below are some trials investigating the use of stem cells from bone marrow and placental tissue to assist patient recovery. Click on the link provided to find out more about each trial. It remains too early to know which approach will be most suitable. We need evidence collected in clinical trials to determine if a proposed stem cell treatment is both safe and effective.

tagged as Anaemia & Thalassemia

  • Human Placental-Derived Stem Cell Transplantation Location: US
    Overview: This trial aims to compare the safety of transplanting combinations of stem cells from placental with cord blood for a number of different malignant and benign haematological disorders. The trial is being conducted by the New York Medical College and will investigate whether giving placental stem cells at the same time as umbilical cord blood effects the survival of the transplanted stem. The trial expects to enrol 30 patients between March 2013 and December 2019 with any one of 16 diseases including several kinds of Leukaemia, Aplastic Anaemia as well as several benign but severely debilitating, incurable enzyme-linked metabolic disorders such as Gaucher's & Nieman-Pick disease. Patients will be monitored for complications and therapeutic benefit for nine months after the injection.
    Link to Clinical Registry
    Trial Design: Safety and Efficacy Study
    Status: Open - Recruiting
    Stem Cell: Placental Stem Cells
  • A Study Evaluating the Safety and Efficacy of the LentiGlobin┬« BB305 Drug Product in Beta-Thalassemia Major Subjects Location: US
    Overview: This trial aims to investigate the safety and efficacy of using gene therapy to treat subjects with β-Thalassemia Major. This trial being sponsored by Bluebird Bio and proposes to recruit 15 patients between July 2013 and July 2017. Participants in the study will receive their own blood stem cells (or CD34+ haematopoietic stem sells) that have been modified to include the proprietary product - LentiGlobin® BB305. The study seeks to explore if the product can result in the production of a protein that is absent in patients with β-Thalassemia Major, thus reversing this common condition. Participants will be monitored for complications following the transfusion and changes in clinical condition, including production of desired haemoglobin [β-A(T87Q)-globin] 18 to 24 months post treatment.
    Link to Clinical Registry
    Trial Design: Safety and Efficacy Study
    Status: Not yet started
    Stem Cell: Adult Blood Stem Cells