NextCell Pharma AB has announced the successful completion of patient treatment in its ProTrans-Young clinical trial, representing a significant milestone in the development of cell therapy for pediatric type 1 diabetes. The Phase II study, which is the company's largest clinical trial to date, has now completed dosing for all 66 enrolled patients across multiple age groups.
Trial Design and Patient Population
ProTrans-Young is an investigator-initiated Phase II clinical trial led by Professor Per-Ola Carlsson at Uppsala University, conducted in collaboration with Linköping and Lund Universities. The study follows a multi-phase design targeting pediatric and adolescent patients with recently diagnosed type 1 diabetes.
The trial began with an open-label safety phase involving six children who received ProTrans treatment. This initial cohort included three patients aged 12-18 years and three aged 7-11 years, followed by a three-month safety follow-up period.
Subsequently, the study progressed to a randomized, placebo-controlled, double-blinded phase including 30 patients aged 12-21 years. These participants were allocated in a 1:1 ratio to receive either ProTrans or placebo treatment. The final cohort consisted of 30 patients aged 7-11 years, also randomized 1:1 to receive either ProTrans or placebo.
Preliminary Efficacy Results
One-year follow-up results for the 12-21 years age group were reported in April 2025, demonstrating a positive trend in preserved insulin production. However, the study encountered an unexpected finding: many patients in both treatment arms remained in remission at the one-year mark, longer than initially anticipated. This extended remission period made it challenging to observe the full treatment effect at this early stage.
Based on previous adult trials with ProTrans, researchers expect the differences between treatment groups to become more pronounced over time as the natural remission period ends. This observation aligns with the typical disease progression pattern in type 1 diabetes, where the honeymoon phase can vary significantly among patients.
ProTrans Technology Platform
The ProTrans platform is based on allogeneic mesenchymal stromal cells (MSCs) that are selected using NextCell's proprietary algorithm. This selection process identifies cells with optimal immunomodulatory function, designed to enhance therapeutic efficacy.
ProTrans is being developed as an off-the-shelf, single-dose infusion therapy. The treatment aims to modulate the immune system and preserve residual beta cell function in patients with autoimmune diabetes, potentially offering a disease-modifying approach rather than merely symptomatic treatment.
Clinical Significance and Future Timeline
"We are pleased to have completed treatment of all patients in the ProTrans-Young study," said Mathias Svahn, CEO of NextCell Pharma. "This is an important milestone in our mission to develop a disease-modifying therapy for children with type 1 diabetes. We are very impressed by the work of the study teams and deeply grateful to the participating patients and their families for their trust."
The completion of patient dosing represents a crucial step toward potentially providing a new therapeutic option for pediatric type 1 diabetes, a condition that typically requires lifelong insulin therapy and carries significant long-term complications.
One-year follow-up data from the complete study population, including all age cohorts, are expected after the summer of 2026. These comprehensive results will provide a more complete picture of ProTrans's safety and efficacy profile across the pediatric age spectrum and may inform future regulatory discussions and potential commercialization strategies.
