Using cyclophosphamide to treat lipodystrophy in children with type 1 diabetes
Cyclophosphamide in the Treatment of Panniculitis Associated Acquired Lipodystrophy Syndrome With Type 1 Diabetes
This study is testing if cyclophosphamide can help children with type 1 diabetes and lipodystrophy improve their insulin sensitivity and lower their insulin needs.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Sex | All |
| Sponsor | Children's Hospital of Fudan University Academic / other |
| Drugs / interventions | cyclophosphamide |
| Locations | 1 site (Shanghai, Shanghai) |
| Trial ID | NCT03936829 on ClinicalTrials.gov |
What this trial studies
This study evaluates the effects of cyclophosphamide on insulin resistance and glucose metabolism in children suffering from panniculitis associated acquired lipodystrophy syndrome and type 1 diabetes. The condition leads to severe insulin resistance and requires higher insulin doses for blood glucose management. By using cyclophosphamide, an immunosuppressive agent, the study aims to suppress the autoimmune response, alleviate the progression of the syndrome, and improve insulin sensitivity. The ultimate goal is to reduce insulin dosage and enhance glucose metabolism in affected patients.
Who should consider this trial
Good fit: Ideal candidates are children diagnosed with type 1 diabetes and panniculitis associated acquired lipodystrophy syndrome.
Not a fit: Patients with mature and effective treatment methods available or those with active infections or malignancies may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve blood glucose management and reduce insulin requirements for patients with this rare condition.
How similar studies have performed: While the use of immunosuppressive agents in similar contexts has shown promise, this specific application of cyclophosphamide for this condition is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 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. Meet the diagnostic criteria for panniculitis: fat biopsy suggests inflammatory infiltration. 3. Meet the diagnostic criteria for acquired lipodystrophy syndrome: childhood onset, clinically no nutritional deficiency or catabolism, systemic or partial subcutaneous fat reduction, genetic testing to exclude congenital lipodystrophy syndrome; low leptin level and autoantibodies can aid in diagnosis. 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
- Children's Hospital of Fudan University — Shanghai, Shanghai, China (Recruiting)
Study contacts
- Study coordinator: Feihong Luo
- 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.