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Lipodystrophy AcquiredNCT03936829

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

A study testing Cyclophosphamide in people with Lipodystrophy Acquired and Type1diabetes, run by Children's Hospital of Fudan University at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Any age
Conditions the study is for
Lipodystrophy Acquired, Type1diabetes
What is being tested?
Cyclophosphamide
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteChina
Study dates
28 Apr 2019to 31 May 2030
Phase
Interventional
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality

Other trials in China

About this study

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

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.

Read the 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.

Being tested
Cyclophosphamide
Keywords
panniculitis associated acquired generalised lipodystrophy, cyclophosphamide

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CyclophosphamideDrug

    Also called CYC

    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.

Study groups in NCT03936829
Study groupRoleWhat they receive
InterventionalExperimentalDrug:Cyclophosphamide Dosage form: intravenous infusion Dosage: 500 mg/m2 of BSA Frequency: every 4 weeks Duration: 24 weeks
Receives: Drug: Cyclophosphamide

What is being measured

The primary outcome is the main question the study is designed to answer.

  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)

    Measured: week 21

Secondary outcomes (19)

  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 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)

    Measured: week 1

  2. 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)

    Measured: week 5

  3. 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)

    Measured: week 9

  4. 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)

    Measured: week 13

  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 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)

    Measured: week 17

  6. 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)

    Measured: from the completion of treatment to 3 months

  7. 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)

    Measured: from the completion of treatment to 6 months

  8. 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)

    Measured: from the completion of treatment to 9 months

  9. 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)

    Measured: from the completion of treatment to 12 months

  10. HbA1c level

    HbA1c level

    Measured: week 1

  11. HbA1c level

    HbA1c level

    Measured: week 5

  12. HbA1c level

    HbA1c level

    Measured: week 9

  13. HbA1c level

    HbA1c level

    Measured: week 13

  14. HbA1c level

    HbA1c level

    Measured: week 17

  15. HbA1c level

    HbA1c level

    Measured: week 21

  16. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 3 months

  17. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 6 months

  18. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 9 months

  19. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 May 2019
  2. Study start28 Apr 2019
  3. Primary completion31 May 2028 (estimated)
  4. Study completion31 May 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03936829
Sponsor's protocol ID
C-PAALS&T1D
Status last verified
Mar 2026
First submitted
25 Apr 2019
Last updated
17 Mar 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 26390101

  • 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.

    PubMed 30076164

  • 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.

    PubMed 27967300

  • 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.

    PubMed 24689899

  • 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.

    PubMed 29069848

  • 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.

    PubMed 28324110

  • 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.

    PubMed 19727665

  • 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.

    PubMed 11356429

  • 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.

    PubMed 30595509

  • 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.

    PubMed 22066977

  • 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.

    PubMed 25734254

  • 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.

    PubMed 24218286

  • 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.

    PubMed 17940115

Questions about this trial

What is this clinical trial studying?

A study testing Cyclophosphamide in people with Lipodystrophy Acquired and Type1diabetes, run by Children's Hospital of Fudan University at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Average daily insulin dosage (time frame: week 21). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03936829, which also lists 13 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2026.

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