Transplanting stem cells and islet cells in children with type 1 diabetes and immune deficiency
Sequential Transplantation of Umbilical Cord Blood Stem Cells and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency Type 1 Diabetes Mellitus
This study is testing if giving children with type 1 diabetes and immune problems a transplant of stem cells followed by insulin-producing cells can help them feel better and possibly cure their conditions.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | Children's Hospital of Fudan University Academic / other |
| Locations | 1 site (Shanghai, Shanghai Municipality) |
| Trial ID | NCT03835312 on ClinicalTrials.gov |
What this trial studies
This study evaluates the effectiveness of sequential transplantation of umbilical cord blood stem cells followed by islet cells in children and adolescents diagnosed with monogenic immunodeficiency type 1 diabetes mellitus. The initial procedure involves transplanting umbilical cord blood stem cells to help restore immune function, after which patients with stable immune recovery will receive islet cell transplantation. This innovative approach aims to address the complications associated with both diabetes and immune deficiency, potentially offering a cure for affected children.
Who should consider this trial
Good fit: Ideal candidates for this study are children and adolescents with type 1 diabetes mellitus and associated genetic immunodeficiency.
Not a fit: Patients who do not have genetic immunodeficiency or those with other forms of diabetes may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a potential cure for children suffering from monogenic immunodeficiency type 1 diabetes, significantly improving their quality of life.
How similar studies have performed: While this approach is innovative, similar studies involving stem cell and islet cell transplantation have shown promise in treating diabetes, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1.Type 1 diabetes mellitus children with genetic immunodeficiency 1. Meet the diagnostic criteria of type 1 diabetes mellitus: clinical manifestations of typical diabetes mellitus include polyphagia, polyuria, weight loss, or diabetic ketoacidosis, confirmed by blood sugar level, islet function and autoimmune antibody. 2. Existence of extrapancreatic organ damage: (1) inflammatory bowel disease, (2) impairment of renal function, (3) repeated infection of mouth, skin, anus or whole body, (4) immune hepatitis, (5) persistent chronic immune iridocyclitis, (6) immune adrenalinitis leading to adrenocortical dysfunction, (7) pituitary inflammation leading to hypophysis, (8) rheumatoid disease, (9) immune vasculitis, (10) systemic lupus erythematosus, (11) other organs besides thyroid function damage. Suffering from one or more of above diseases. Recurrence after receiving regular clinical treatment, including symptomatic treatment of organ protective drugs. 3. Gene mutation was found according to gene diagnosis: gene mutation was found by gene sequencing. Literature searches at home and abroad confirmed that the defect of the gene resulted in autoimmune or immune dysfunction, resulting in multiple organ dysfunction and poor prognosis. Exclusion Criteria: 1. Mature and effective treatment methods are available. 2. HIV, HBV and HCV were positive. 3. A the active period of infection. 4. At the active stage of malignant tumors. 5. Combination of other fatal diseases. 6. Existence of mental and psychological diseases.
Where this trial is running
Shanghai, Shanghai Municipality
- Children's Hospital of Fudan University — Shanghai, Shanghai Municipality, China (Recruiting)
Study contacts
- Study coordinator: Luo Feihong
- Email: luofh@fudan.edu.cn
- Phone: +862164931226
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.