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

NCT03936829 | Active Not Recruiting

• 1 other identifier: C-PAALS&T1D
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Lipodystrophy Acquired; Type1diabetes

Keywords

panniculitis associated acquired generalised lipodystrophy; cyclophosphamide

Outcome Measures

Primary Outcomes (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 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 Outcomes (19)

• Average daily insulin dosage

  week 1

• Average daily insulin dosage

  week 5

• Average daily insulin dosage

  week 9

• Average daily insulin dosage

  week 13

• Average daily insulin dosage

  week 17

Study Arms (1)

Interventional

EXPERIMENTAL

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

Drug: Cyclophosphamide

Interventions

Cyclophosphamide DRUG

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.

Also known as: CYC

Interventional

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

Location

Related Publications (13)

• 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.

  PMID: 26390101 BACKGROUND

• 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.

  PMID: 30076164 BACKGROUND

• 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.

  PMID: 27967300 BACKGROUND

• 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.

  PMID: 24689899 BACKGROUND

• Li S, Yang H, Guo P, Ao X, Wan J, Li Q, Tan L. Efficacy and safety of immunosuppressive medications for steroid-resistant nephrotic syndrome in children: a systematic review and network meta-analysis. Oncotarget. 2017 Aug 21;8(42):73050-73062. doi: 10.18632/oncotarget.20377. eCollection 2017 Sep 22.

  PMID: 29069848 BACKGROUND

• Brown RJ, Meehan CA, Cochran E, Rother KI, Kleiner DE, Walter M, Gorden P. Effects of Metreleptin in Pediatric Patients With Lipodystrophy. J Clin Endocrinol Metab. 2017 May 1;102(5):1511-1519. doi: 10.1210/jc.2016-3628.

  PMID: 28324110 BACKGROUND

• Chong AY, Lupsa BC, Cochran EK, Gorden P. Efficacy of leptin therapy in the different forms of human lipodystrophy. Diabetologia. 2010 Jan;53(1):27-35. doi: 10.1007/s00125-009-1502-9. Epub 2009 Sep 2.

  PMID: 19727665 BACKGROUND

• Joffe BI, Panz VR, Raal FJ. From lipodystrophy syndromes to diabetes mellitus. Lancet. 2001 May 5;357(9266):1379-81. doi: 10.1016/S0140-6736(00)04616-X. No abstract available.

  PMID: 11356429 BACKGROUND

• Hussain I, Patni N, Garg A. Lipodystrophies, dyslipidaemias and atherosclerotic cardiovascular disease. Pathology. 2019 Feb;51(2):202-212. doi: 10.1016/j.pathol.2018.11.004. Epub 2018 Dec 27.

  PMID: 30595509 BACKGROUND

• Weingartner JS, Zedek DC, Burkhart CN, Morrell DS. Lupus erythematosus panniculitis in children: report of three cases and review of previously reported cases. Pediatr Dermatol. 2012 Mar-Apr;29(2):169-76. doi: 10.1111/j.1525-1470.2011.01544.x. Epub 2011 Nov 8.

  PMID: 22066977 BACKGROUND

• Diker-Cohen T, Cochran E, Gorden P, Brown RJ. Partial and generalized lipodystrophy: comparison of baseline characteristics and response to metreleptin. J Clin Endocrinol Metab. 2015 May;100(5):1802-10. doi: 10.1210/jc.2014-4491. Epub 2015 Mar 3.

  PMID: 25734254 BACKGROUND

• Ale'ed A, Alsonbul A, Al-Mayouf SM. Safety and efficacy of combined cyclophosphamide and rituximab treatment in recalcitrant childhood lupus. Rheumatol Int. 2014 Apr;34(4):529-33. doi: 10.1007/s00296-013-2896-8. Epub 2013 Nov 12.

  PMID: 24218286 BACKGROUND

• Park JY, Chong AY, Cochran EK, Kleiner DE, Haller MJ, Schatz DA, Gorden P. Type 1 diabetes associated with acquired generalized lipodystrophy and insulin resistance: the effect of long-term leptin therapy. J Clin Endocrinol Metab. 2008 Jan;93(1):26-31. doi: 10.1210/jc.2007-1856. Epub 2007 Oct 16.

  PMID: 17940115 BACKGROUND

MeSH Terms

Interventions

Cyclophosphamide

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• Feihong Luo

  Children's Hospital of Fudan University

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 25, 2019
• First Posted: May 3, 2019
• Study Start: April 28, 2019
• Primary Completion (Estimated): May 31, 2028
• Study Completion (Estimated): May 31, 2030
• Last Updated: March 17, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)