Cyclophosphamide in the Treatment of Associated Acquired Lipodystrophy Syndrome With Type 1 Diabetes

Study Overview
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Brief Summary
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This study evaluates the change of insulin resistance and glucose metabolism of patients with panniculitis associated acquired lipodystrophy syndrome and type 1 diabetes with the treatment of cyclophosphamide.

Detailed Description
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Patients with panniculitis associated acquired lipodystrophy syndrome and type 1 diabetes have difficulty in blood glucose management due to the presence of both severe insulin resistance and complete insulin deficiency. It is often necessary to use insulin doses several times that of other children of the same age with type 1 diabetes. Since autoimmune response is the main cause of panniculitis associated acquired lipodystrophy syndrome, immunosuppressive agents can suppress immune response, prevent and alleviate the progression of panniculitis and acquired lipodystrophy syndrome, and improve insulin resistance caused by subcutaneous fat deficiency. Cyclophosphamide is a classic immunosuppressive agent. This study hopes to improve insulin resistance of patients with panniculitis associated acquired lipodystrophy syndrome and type 1 diabetes by cyclophosphamide treatment, thereby reducing insulin dosage and improving glucose metabolism.

Official Title
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Cyclophosphamide in the Treatment of Panniculitis Associated Acquired Lipodystrophy Syndrome With Type 1 Diabetes

Conditions
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Lipodystrophy Acquired, Type1diabetes

Intervention / Treatment
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* Drug: Cyclophosphamide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Meet the diagnostic criteria for panniculitis: fat biopsy suggests inflammatory infiltration. 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: Mature and effective treatment methods are available. HIV, HBV and HCV were positive. A the active period of infection. At the active stage of malignant tumors. Combination of other fatal diseases. Existence of mental and psychological diseases.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Interventional<br>Drug:Cyclophosphamide Dosage form: intravenous infusion Dosage: 500 mg/m2 of BSA Frequency: every 4 weeks Duration: 24 weeks | Drug: Cyclophosphamide<br>* Admitted to the hospital every 4 weeks. Cyclophosphamide was intravenously instilled (500mg mg/m2 of BSA) after contraindications are excluded. ECG monitoring will be taken during the medication. A total of 6 treatments will be performed.<br>* Other names: CYC;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of the 1st, 2nd and 3rd days' insulin requirements after the last cyclophosphamide treatment, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | week 21 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of the 1st, 2nd and 3rd days' insulin requirements after the first cyclophosphamidethe, blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | week 1 | 
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of the 1st, 2nd and 3rd days' insulin requirements after the 2nd cyclophosphamide, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | week 5 | 
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of the 1st, 2nd and 3rd days' insulin requirements after the 3rd cyclophosphamide, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | week 9 | 
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of the 1st, 2nd and 3rd days' insulin requirements after the 4th cyclophosphamide, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | week 13 | 
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of the 1st, 2nd and 3rd days' insulin requirements after the 5th cyclophosphamide, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | week 17 | 
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of 3 days' insulin requirements 3 months after the last cyclophosphamide treatment, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | from the completion of treatment to 3 months | 
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of 3 days' insulin requirements 6 months after the last cyclophosphamide treatment, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | from the completion of treatment to 6 months | 
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of 3 days' insulin requirements 9 months after the last cyclophosphamide treatment, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | from the completion of treatment to 9 months | 
| Average daily insulin dosage | Average daily insulin dosage of continuous three days (the average of 3 days' insulin requirements 12 months after the last cyclophosphamide treatment, the blood glucose meets the ISPAD guideline as premeal 4.0-7.0 mmol/L, postmeal 5.0-10.0 mmol/L, prebed 4.4-7.8 mmol/L) | from the completion of treatment to 12 months | 
| HbA1c level | HbA1c level | week 1 | 
| HbA1c level | HbA1c level | week 5 | 
| HbA1c level | HbA1c level | week 9 | 
| HbA1c level | HbA1c level | week 13 | 
| HbA1c level | HbA1c level | week 17 | 
| HbA1c level | HbA1c level | week 21 | 
| HbA1c level | HbA1c level | from the completion of treatment to 3 months | 
| HbA1c level | HbA1c level | from the completion of treatment to 6 months | 
| HbA1c level | HbA1c level | from the completion of treatment to 9 months | 
| HbA1c level | HbA1c level | from the completion of treatment to 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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panniculitis associated acquired generalised lipodystrophy, cyclophosphamide