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

Primary Purpose

Lipodystrophy Acquired, Type1diabetes

Status
Recruiting
Phase
Not Applicable
Locations
China
Study Type
Interventional
Intervention
Cyclophosphamide
Sponsored by
Children's Hospital of Fudan University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Lipodystrophy Acquired focused on measuring panniculitis associated acquired generalised lipodystrophy, cyclophosphamide

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital of Fudan UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Interventional

Arm Description

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

Outcomes

Primary Outcome Measures

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)

Secondary Outcome Measures

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)
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)
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)
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)
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)
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)
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)
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)
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)
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level

Full Information

First Posted
April 25, 2019
Last Updated
July 30, 2023
Sponsor
Children's Hospital of Fudan University
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1. Study Identification

Unique Protocol Identification Number
NCT03936829
Brief Title
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

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 28, 2019 (Actual)
Primary Completion Date
May 31, 2025 (Anticipated)
Study Completion Date
May 31, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital of Fudan University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lipodystrophy Acquired, Type1diabetes
Keywords
panniculitis associated acquired generalised lipodystrophy, cyclophosphamide

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Interventional
Arm Type
Experimental
Arm Description
Drug:Cyclophosphamide Dosage form: intravenous infusion Dosage: 500 mg/m2 of BSA Frequency: every 4 weeks Duration: 24 weeks
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide
Other Intervention Name(s)
CYC
Intervention Description
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.
Primary Outcome Measure Information:
Title
Average daily insulin dosage
Description
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)
Time Frame
week 21
Secondary Outcome Measure Information:
Title
Average daily insulin dosage
Description
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)
Time Frame
week 1
Title
Average daily insulin dosage
Description
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)
Time Frame
week 5
Title
Average daily insulin dosage
Description
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)
Time Frame
week 9
Title
Average daily insulin dosage
Description
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)
Time Frame
week 13
Title
Average daily insulin dosage
Description
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)
Time Frame
week 17
Title
Average daily insulin dosage
Description
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)
Time Frame
from the completion of treatment to 3 months
Title
Average daily insulin dosage
Description
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)
Time Frame
from the completion of treatment to 6 months
Title
Average daily insulin dosage
Description
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)
Time Frame
from the completion of treatment to 9 months
Title
Average daily insulin dosage
Description
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)
Time Frame
from the completion of treatment to 12 months
Title
HbA1c level
Description
HbA1c level
Time Frame
week 1
Title
HbA1c level
Description
HbA1c level
Time Frame
week 5
Title
HbA1c level
Description
HbA1c level
Time Frame
week 9
Title
HbA1c level
Description
HbA1c level
Time Frame
week 13
Title
HbA1c level
Description
HbA1c level
Time Frame
week 17
Title
HbA1c level
Description
HbA1c level
Time Frame
week 21
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 3 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 6 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 9 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 12 months

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Feihong Luo
Phone
+862164931226
Ext
+862164931226
Email
luofh@fudan.edu.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Zhenran H Xu
Phone
+862164931226
Ext
+862164931226
Email
xu_zhenran@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Feihong Luo
Organizational Affiliation
Children's Hospital of Fudan University
Official's Role
Study Chair
Facility Information:
Facility Name
Children's Hospital of Fudan University
City
Shanghai
State/Province
Shanghai
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Feihong Luo
Phone
+862164931226
Ext
+862164931226
Email
luofh@fudan.edu.cn
First Name & Middle Initial & Last Name & Degree
Zhenran Xu
Phone
+862164931226
Ext
+862164931226
Email
xu_zhenran@163.com

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
26390101
Citation
Lebastchi J, Ajluni N, Neidert A, Oral EA. A Report of Three Cases With Acquired Generalized Lipodystrophy With Distinct Autoimmune Conditions Treated With Metreleptin. J Clin Endocrinol Metab. 2015 Nov;100(11):3967-70. doi: 10.1210/jc.2015-2589. Epub 2015 Sep 21.
Results Reference
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PubMed Identifier
30076164
Citation
Kumar R, Pilania RK, Bhatia A, Dayal D. Acquired generalised lipodystrophy and type 1 diabetes mellitus in a child: a rare and implacable association. BMJ Case Rep. 2018 Aug 3;2018:bcr2018225553. doi: 10.1136/bcr-2018-225553.
Results Reference
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PubMed Identifier
27967300
Citation
Gupta N, Asi N, Farah W, Almasri J, Barrionuevo P, Alsawas M, Wang Z, Haymond MW, Brown RJ, Murad MH. Clinical Features and Management of Non-HIV-Related Lipodystrophy in Children: A Systematic Review. J Clin Endocrinol Metab. 2017 Feb 1;102(2):363-374. doi: 10.1210/jc.2016-2271.
Results Reference
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PubMed Identifier
24689899
Citation
Carroll M, Mellick N, Wagner G. Dermatomyositis panniculitis: a case report. Australas J Dermatol. 2015 Aug;56(3):224-6. doi: 10.1111/ajd.12172. Epub 2014 Apr 1.
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PubMed Identifier
29069848
Citation
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Results Reference
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PubMed Identifier
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Citation
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Citation
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Learn more about this trial

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

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