Using stem cells with standard therapy to treat new type 1 diabetes in children

Investigation of Dose Escalation and Cohort Expansion Study on the Safety and Efficacy of Allogeneic Umbilical Cord Mesenchymal Stem Cells(UCMSCs) Combined With Standard Therapy for Newly Diagnosed Type 1 Diabetes

This study is testing if giving children with newly diagnosed type 1 diabetes stem cells from umbilical cords along with their regular treatment can help them keep making insulin and manage their diabetes better.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 8 Years to 18 Years
• Sex: All
• Sponsor: Shanghai Changzheng Hospital (Academic / other)
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT06407297 on ClinicalTrials.gov

What this trial studies

This study investigates the safety and tolerability of intravenous infusion of allogeneic umbilical cord mesenchymal stem cells (UCMSCs) in pediatric patients aged 8-18 who have been newly diagnosed with type 1 diabetes. It consists of two parts: an open, dose-escalating study with 9 patients, followed by a randomized, double-blinded, placebo-controlled study involving 24 patients. The study aims to assess not only the safety of UCMSCs but also their effectiveness in preserving insulin production and improving metabolic control over a one-year follow-up period.

Who should consider this trial

Good fit: Ideal candidates are children aged 8 to 18 years who have been diagnosed with type 1 diabetes within the last 6 months.

Not a fit: Patients with uncontrolled diabetic ketoacidosis or those with severe allergic reactions may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could enhance insulin production and improve diabetes management in children with newly diagnosed type 1 diabetes.

How similar studies have performed: While the use of stem cells in diabetes treatment is an emerging field, this specific approach has not been widely tested, making it a novel investigation.

Eligibility criteria

Inclusion Criteria:

1. Meets the diagnostic criteria for diabetes according to the World Health Organization (WHO) guidelines from 1999.
2. Presence of at least one pancreatic autoantibody, or idiopathic type 1 diabetes mellitus (T1DM) with negative autoantibody testing.
3. Ages 8 to 18 years, regardless of gender.
4. Newly diagnosed T1DM within the past 6 months.
5. Fasting C-peptide ≥0.1 nmol/L and postprandial 2-hour C-peptide \>0.2 nmol/L.
6. Voluntary acceptance of stem cell transplantation therapy by the individual, their family members, or legal guardians, and signing of an informed consent form.

Exclusion Criteria:

1. Diabetic ketoacidosis is not under control.
2. Severe allergic constitution.
3. BMI \<14 or \>35.
4. History of other autoimmune diseases, hematologic disorders.
5. HIV positive, carrier of viral hepatitis, active phase of viral hepatitis, or other uncontrolled infectious diseases.
6. History of acute pancreatitis, pulmonary embolism, or other thrombotic diseases, as well as severe diseases of the heart, liver, kidneys, respiratory system, nervous system, etc.
7. Suffering from gestational diabetes, monogenic diabetes, diabetes caused by pancreatic injury, or other secondary diabetes (such as Cushing's syndrome, thyroid dysfunction, or acromegaly-induced diabetes);
8. Pregnancy or planning pregnancy within 3 months before or after treatment, as well as breastfeeding women.
9. Mental illness, alcohol or drug abuse, inability to comply with treatment.
10. Known or suspected tumors.
11. According to the investigator's judgment, there are other clinical conditions that may endanger the safety of the subjects.

Where this trial is running

Shanghai, Shanghai Municipality

• Shanghai Changzheng Hospital — Shanghai, Shanghai Municipality, China (Recruiting)

Study contacts

• Principal investigator: Hao Yin — Shanghai Changzheng Hospital
• Study coordinator: Hao Yin

Conditions Newly Diagnosed Type 1 Diabetes Mellitus