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

Sponsor
Children's Hospital of Fudan University (Other)
Overall Status
Recruiting
CT.gov ID
NCT03936829
Collaborator
(none)
10
Enrollment
1
Location
1
Arm
85.1
Anticipated Duration (Months)
0.1
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or DiseaseIntervention/TreatmentPhase
N/A

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.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
10 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Cyclophosphamide in the Treatment of Panniculitis Associated Acquired Lipodystrophy Syndrome With Type 1 Diabetes
Actual Study Start Date :
Apr 28, 2019
Anticipated Primary Completion Date :
May 31, 2025
Anticipated Study Completion Date :
May 31, 2026

Arms and Interventions

ArmIntervention/Treatment
Experimental: Interventional

Drug:Cyclophosphamide Dosage form: intravenous infusion Dosage: 500 mg/m2 of BSA Frequency: every 4 weeks Duration: 24 weeks

Drug: Cyclophosphamide
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.
Other Names:
  • CYC
  • Outcome Measures

    Primary Outcome Measures

    1. Average daily insulin dosage [week 21]

      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)

    Secondary Outcome Measures

    1. Average daily insulin dosage [week 1]

      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)

    2. Average daily insulin dosage [week 5]

      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)

    3. Average daily insulin dosage [week 9]

      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)

    4. Average daily insulin dosage [week 13]

      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)

    5. Average daily insulin dosage [week 17]

      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)

    6. Average daily insulin dosage [from the completion of treatment to 3 months]

      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)

    7. Average daily insulin dosage [from the completion of treatment to 6 months]

      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)

    8. Average daily insulin dosage [from the completion of treatment to 9 months]

      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)

    9. Average daily insulin dosage [from the completion of treatment to 12 months]

      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)

    10. HbA1c level [week 1]

      HbA1c level

    11. HbA1c level [week 5]

      HbA1c level

    12. HbA1c level [week 9]

      HbA1c level

    13. HbA1c level [week 13]

      HbA1c level

    14. HbA1c level [week 17]

      HbA1c level

    15. HbA1c level [week 21]

      HbA1c level

    16. HbA1c level [from the completion of treatment to 3 months]

      HbA1c level

    17. HbA1c level [from the completion of treatment to 6 months]

      HbA1c level

    18. HbA1c level [from the completion of treatment to 9 months]

      HbA1c level

    19. HbA1c level [from the completion of treatment to 12 months]

      HbA1c level

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    SiteCityStateCountryPostal Code
    1Children's Hospital of Fudan UniversityShanghaiShanghaiChina

    Sponsors and Collaborators

    • Children's Hospital of Fudan University

    Investigators

    • Study Chair: Feihong Luo, Children's Hospital of Fudan University

    Study Documents (Full-Text)

    More Information

    Publications

    Responsible Party:
    Children's Hospital of Fudan University
    ClinicalTrials.gov Identifier:
    NCT03936829
    Other Study ID Numbers:
    • C-PAALS&T1D
    First Posted:
    May 3, 2019
    Last Update Posted:
    Oct 25, 2021
    Last Verified:
    Mar 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Children's Hospital of Fudan University
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Oct 25, 2021