Clinical trial NCT03936829: Cyclophosphamide in the Treatment of Associated Acquired Lipodystrophy Syndrome With Type 1 Diabetes
Official title: Cyclophosphamide in the Treatment of Panniculitis Associated Acquired Lipodystrophy Syndrome With Type 1 Diabetes
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Lipodystrophy Acquired
Type1diabetes
Keywords: panniculitis associated acquired generalised lipodystrophy
cyclophosphamide
Lead sponsor: Children's Hospital of Fudan University
Sponsor class: OTHER
First posted: 2019-05-03
Enrollment: 10
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility 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.

Primary outcomes:
measure: Average daily insulin dosage
timeFrame: week 21
Secondary outcomes:
measure: Average daily insulin dosage
timeFrame: week 1
measure: Average daily insulin dosage
timeFrame: week 5
measure: Average daily insulin dosage
timeFrame: week 9
measure: Average daily insulin dosage
timeFrame: week 13
measure: Average daily insulin dosage
timeFrame: week 17
measure: Average daily insulin dosage
timeFrame: from the completion of treatment to 3 months
measure: Average daily insulin dosage
timeFrame: from the completion of treatment to 6 months
measure: Average daily insulin dosage
timeFrame: from the completion of treatment to 9 months
measure: Average daily insulin dosage
timeFrame: from the completion of treatment to 12 months
measure: HbA1c level
timeFrame: week 1
measure: HbA1c level
timeFrame: week 5
measure: HbA1c level
timeFrame: week 9
measure: HbA1c level
timeFrame: week 13
measure: HbA1c level
timeFrame: week 17
measure: HbA1c level
timeFrame: week 21
measure: HbA1c level
timeFrame: from the completion of treatment to 3 months
measure: HbA1c level
timeFrame: from the completion of treatment to 6 months
measure: HbA1c level
timeFrame: from the completion of treatment to 9 months
measure: HbA1c level
timeFrame: from the completion of treatment to 12 months
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: Feihong Luo
role: STUDY_CHAIR
References:
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