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

Trial ID: NCT05061030

Interventional - Phase 1/2 - Active, not recruiting

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.

Conditions

• Type1diabetes

Interventions

• the ATMP Protrans

Eligibility

Inclusion criteria

• 1. 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
• 2. Clinical history compatible with type 1 diabetes diagnosed less than 6 months before enrolment
• 3. 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.
• 4. Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol.
• 5. Fasting plasma C-peptide concentration \>0.12 nmol/L.
• 6. 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:
• 1. oral (except low-dose gestagen (lynestrenol and noretisteron), injectable or implanted hormonal contraceptives.
• 2. intrauterine device
• 3. intrauterine system (for example progestin-releasing coil)
• 4. vasectomized male (with appropriate postvasectomy documentation of the absence of sperm in the ejaculate)

Exclusion criteria

• 1. Subjects with body weight \>100 kg
• 2. Subjects with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
• 3. Subjects with uncontrolled hypertension (≥160/105 mmHg).
• 4. Subjects with active on-going infections.
• 5. 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.
• 6. 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.
• 7. Subjects with any systemic immune suppressive treatment
• 8. Subjects with a known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease.
• 9. 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.
• 10. Subjects with known, or previous, malignancy.
• 11. Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin.
• 12. Subjects with GFR \<60 ml/min/1.73 m2 body surface.
• 13. Subject with any condition or any circumstance that, in the opinion of the investigator, would make it unsafe to undergo treatment with MSC.
• 14. Known hypersensitivity against any excipients, i.e., dimethyl sulfoxide (DMSO).

Sponsor

Uppsala University Hospital

Source: ClinicalTrials.gov

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