The paediatric diabetes study gets the green light to treat the age group 7 – 11 years
NextCell Pharma AB (publ) ("NextCell") announces that the Data Safety Monitoring Board for the ProTrans-Young study has recommended the principal investigator, Professor Per-Ola Carlsson, to continue with treatment of children in the younger age group 7 – 11 years. This decision is based on a review of all available safety data from the 36 children and adolescents who have participated in the study so far. The recruitment of the 30 children aged 7-11 will begin in August 2024.
The first part of the ProTrans-Young study is a safety part (phase Ib) where all 6 patients aged 7-18 years are treated with ProTrans. The second part of the study is randomized and placebo-controlled (phase II) and a total of 60 children and adolescents with type-1 diabetes will be treated, of which 30 with ProTrans and 30 with placebo. The first 30 patients aged 12-21 have undergone treatment and been on follow-up visits for at least 3 months. The Data Safety Monitoring Board has now reviewed the safety and recommended that the study continue with treatment of the remaining 30 patients aged 7-11.
Information in Swedish about the clinical trial and participation can be found at: https://www.nextcellpharma.com/studie
Treatment with ProTrans aims to stop the autoimmune destruction of insulin-producing cells. The children and adolescents in ProTrans-Young are treated with ProTrans within 6 months of being diagnosed with type-1 diabetes. The purpose is to be able to maintain as much of their insulin production as long as possible, even if they will continue to need extra insulin. The body's own insulin production buffers blood sugar and reduces the risk of fluctuations that can lead to serious complications.
A subgroup analysis of the treatment effect of ProTrans in 30 adolescents in the age group 12-21 years, is expected to be presented in April 2025. The measure of effectiveness is the difference in endogenous insulin production between ProTrans and placebo-treated patients one year after treatment. Topline results from the entire study are expected by the end of 2026. Previous studies with ProTrans have been limited to adults, therefore only children under 18 years of age are included in the first part of the study. The other part is divided into two age groups that are commonly used in clinical trials in type 1 diabetes, 7-11 and 12-21 years respectively.
The principal investigators are Professor Per-Ola Carlsson, Uppsala University and Uppsala University Hospital, with the co-investigators Professor Helena Elding Larsson, Skåne University Hospital and Professor Johnny Ludvigsson, Linköping University Hospital. The Data Safety Monitoring Board consists of Chairman Professor Ulf Smith, Sahlgrenska and Professor Mikael Rydén, Karolinska and Professor Anders Fasth, Queen Silivia Children's Hospital.
The complete title of the clinical trial is: “A Double-blinded, Randomized, Parallel, Placebo-controlled trial of Wharton´s Jelly-derived Allogeneic Mesenchymal Stromal Cells to treat Type I Diabetes in Children and Adolescents” (EudraCT 2020-004520-42).
Uppsala University Hospital is the sponsor of the study, which is funded by research grants. NextCell contributes with ProTrans and placebo in the study. In addition, the company will support the trial with logistics, documentation and expertise.