Open-label Investigation of the Safety and Efficacy of DIABECELL in Patients With Type 1 Diabetes Mellitus
Condition: Type 1 Diabetes
MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 1
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to establish the efficacy and safety of xenotransplantation of
      DIABECELL [immunoprotected (alginate-encapsulated) porcine islets] in patients with
      established type 1 diabetes mellitus
    

Detailed description: 
      Intraperitoneal islet transplantation has the potential to ameliorate type 1 diabetes
      mellitus and avert the long-term consequences of chronic diabetes which cannot be achieved by
      conventional insulin treatment.

      As donor human islets are not available in sufficient numbers, porcine islets are the best
      alternative source as they are recognised as the most physiologically compatible xenogeneic
      insulin-producing cells. Although the use of pig-derived cells raises the risk of xenotic
      infections, this can be minimised by obtaining cells from designated pathogen-free (DPF)
      animals bred in isolation and monitored to be free of specified pathogens. The worldwide
      experience to date in more than 200 patients who have received transplants of pig tissue has
      not demonstrated evidence of transmitted xenotic infections.

      As animal-derived tissues have to be protected from immune rejection when transplanted into
      humans, transplants are usually accompanied by immunosuppressive therapy. However, porcine
      islets are preferably transplanted without the use of immunosuppressive drugs which cause
      significant morbidity. To protect them from immune rejection, the islets can be encapsulated
      in alginate microcapsules which permit the inward passage of nutrients and glucose and the
      outward passage of insulin. Alginate-encapsulated porcine islets transplanted without
      immunosuppressive drugs have survived rejection for many months in animal studies.

      DIABECELL comprises neonatal porcine islets encapsulated in alginate microcapsules. DIABECELL
      has been safely transplanted in healthy and diabetic mice, rats, rabbits, dogs and non-human
      primates. Following DIABECELL transplants, the requirement for daily insulin was
      significantly reduced in diabetic rats and non-human primates.

      The optimal dose and frequency of transplantation of the current DIABECELL preparation for
      the treatment of type 1 diabetes in humans can only be determined in clinical trials. The
      intention of this phase IIb clinical trial is to obtain at least 52 weeks safety and
      preliminary efficacy data in type 1 diabetic patients following transplantation of two
      effective doses of DIABECELL into the peritoneal cavity.
    

Intervention name(s): DIABECELL
Intervention type(s): Device
Intervention description(s): two transplants of 10,000 IEQ/kg DIABECELL (administered at least 12 weeks apart)- total of 20,000 IEQ/kg
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: xenotransplantation; type 1 diabetes; porcine islets