Open-label Investigation of the Safety and Effectiveness of DIABECELL(R) in Patients With Type I Diabetes Mellitus
|ClinicalTrials.gov Identifier: NCT00940173|
Recruitment Status : Completed
First Posted : July 15, 2009
Last Update Posted : October 20, 2017
|Condition or disease||Intervention/treatment||Phase|
|Type 1 Diabetes||Device: DIABECELL(R)||Phase 1 Phase 2|
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, and have been retrieved from a diabetic patient over 9.5 years after intraperitoneal transplantation and shown to contain viable islets that stain positive for insulin.
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 I/IIa clinical trial is to obtain at least 52 weeks safety and preliminary efficacy data in type 1 diabetic patients following transplantation of a single low effective dose of DIABECELL® into the peritoneal cavity.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||16 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I/IIa Open-label Investigation of the Safety and Effectiveness of DIABECELL(R) [Immunoprotected (Alginate-Encapsulated) Porcine Islets for Xenotransplantation] in Patients With Type I Diabetes Mellitus|
|Study Start Date :||July 2009|
|Primary Completion Date :||October 2013|
|Study Completion Date :||October 2013|
Dose Group 1 (Receiving a dose of 10,000 IEQ/kg of DIABECELL(R))
10,000 IEQ/kg injected into the peritoneal cavity via laparoscopy
Dose Group 2 (Receiving a dose of 15,000 IEQ/kg of DIABECELL(R))
15,000 IEQ/kg injected into the peritoneal cavity via laparoscopy
Dose Group 3 (Receiving a dose of 20,000 IEQ/kg of DIABECELL(R))
20,000 IEQ/kg injected into the peritoneal cavity via laparoscopy
Dose Group 4 (Receiving a dose of 5,000 IEQ/kg of DIABECELL(R))
5,000 IEQ/kg injected into the peritoneal cavity via laparoscopy
- To establish the safety of xenotransplantation of DIABECELL(R) [immunoprotected (alginate-encapsulated) porcine islets] [ Time Frame: 52 Weeks ]
- To establish preliminary evidence of the efficacy of DIABECELL(R), as measured by a reduction in serial HbA1C levels [ Time Frame: 52 weeks ]
- To establish whether DIABECELL(R) causes an improvement in glucose lability determined from continuous glucose monitoring [ Time Frame: 52 Weeks ]
- To determine whether DIABECELL(R) causes a reduction in hypoglycaemia and nocturnal hypoglycaemia [ Time Frame: 52 Weeks ]
- To determine whether DIABECELL(R) causes a reduction in insulin dose [ Time Frame: 52 Weeks ]
- To determine whether DIABECELL(R) causes an improvement in endogenous insulin secretion [ Time Frame: 52 Weeks ]
- To determine whether DIABECELL(R) causes an improvement in quality-of-life [ Time Frame: 52 Weeks ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00940173
|Centre for Clinical Research and Effective Practice|
|Auckland, New Zealand|
|Principal Investigator:||John Baker, MB ChB||Centre for Clinical Research and Effective Practice|