A Double-blinded, Randomized, Parallel, Placebo-controlled Trial of Wharton's Jelly-derived Allogeneic Mesenchymal Stromal Cells to Treat Type 1 Diabetes in Children and Adolescents
This is a combined phase 1 and 2 study in 66 subjects, male or female, between 7-21 years of age that have recently (\< 6 months) been diagnosed with type 1 diabetes. The first phase 1 part of the study includes six subjects openly receiving allogeneic Wharton's jelly derived mesenchymal stromal cells as the Advanced Therapy Medicinal Product (ATMP) Protrans, three each in the age ranges 7-11 and 12-18.The second part is a randomized, double-blinded placebo-controlled phase 2 study in parallel design comparing allogeneic Wharton's jelly derived mesenchymal stromal cells treatment (as Protrans) to placebo in children and adolescent subjects (7-21 years of age) diagnosed with type 1 diabetes, The primary objectives of this study will be to investigate the safety, tolerance and efficacy after an allogieneic infusion of Wharton's jelly derived mesenchymal stromal cells.
• Written informed consent for participation of the study (for subjects below 18 years of age also from both caregivers), given before undergoing any study-specific procedures
• Clinical history compatible with type 1 diabetes diagnosed less than 6 months before enrolment
• In the first part of the study, six subjects, three between 7-11 and three between 12-18 years of age (both groups inclusive at both ends), will be included. The sixty subjects in the second part of the study are stratified by age (12-21 and 7-11 years, respectively) and randomized to one of two treatment arms (active or placebo), with a 6-month safety delay for the younger stratum.
• Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol.
• Fasting plasma C-peptide concentration \>0.12 nmol/L.
• Subjects of child-bearing potential must agree to using adequate contraception until one year after the administration of WJMSC/Placebo. Adequate contraception is as follows:
‣ oral (except low-dose gestagen (lynestrenol and noretisteron), injectable or implanted hormonal contraceptives.
⁃ intrauterine device
⁃ intrauterine system (for example progestin-releasing coil)
⁃ vasectomized male (with appropriate postvasectomy documentation of the absence of sperm in the ejaculate)