Mesenchymal Stromal Cells to Treat Type 1 Diabetes in Children and Adolescents

Inclusion criteria

• 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)

Exclusion criteria

• Subjects with body weight >100 kg
• Subjects with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
• Subjects with uncontrolled hypertension (≥160/105 mmHg).
• Subjects with active on-going infections.
• Subjects with latent or previous as well as on-going therapy against tuberculosis, or exposed to tuberculosis or has traveled in areas with a high risk of tuberculosis or mycosis within the last 3 months.
• Subjects with serological evidence of infection with HIV, Treponema pallidum, hepatitis B antigen (subjects with serology consistent with previous vaccination and a history of vaccination are acceptable), or hepatitis C.
• Subjects with any systemic immune suppressive treatment
• Subjects with a known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease.
• Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
• Subjects with known, or previous, malignancy.
• Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin.
• Subjects with GFR <60 ml/min/1.73 m2 body surface.
• Subject with any condition or any circumstance that, in the opinion of the investigator, would make it unsafe to undergo treatment with MSC.
• Known hypersensitivity against any excipients, i.e., dimethyl sulfoxide (DMSO).