ClinicalTrials.gg
RecruitingNCT06679270Updated Jul 16, 2026

Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

A Phase 3 interventional study of Metreleptin in Familial Partial Lipodystrophy, sponsored by Chiesi Farmaceutici S.p.A.. Recruiting at 7 sites in United States. Open to participants aged 13 Years and older. Per ClinicalTrials.gov, last updated 2026-07-16.

Sponsored by Chiesi Farmaceutici S.p.A. · Phase 3, Interventional, and Treatment

From the registry’s dates

  • Started Oct 2024; still recruiting 2 years later.
Phase
Phase 3
Study type
Interventional
Enrollment
24
Allocation
Not applicable
Ages
13 Years and older
Sex
All
01

Study summary

This Phase 3 study is an Open Label Extension of the APG-20 Study To Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects with Partial Lipodystrophy

02

Conditions studied

  • Familial Partial Lipodystrophy

Keywords

  • Metreleptin
  • familial partial lipodystrophy
03

In context

Lipodystrophy, Familial Partial

20 studies on the registry are indexed under Lipodystrophy, Familial Partial; 5 are open to participants now.

This study's planned enrollment of 24 is above the median of 16 across 14 interventional studies indexed under Lipodystrophy, Familial Partial.

Browse Lipodystrophy, Familial Partial studies →

Lead sponsor

Chiesi Farmaceutici S.p.A. is the lead sponsor of 185 studies on the registry; 22 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 11 (61%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
13 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Age ≥13 years of age, inclusive, at the time of signing the form (ICF).
  2. Subjects must have completed the Parent study APG-20 and, in the opinion of the Investigator and Sponsor, have been compliant with study procedures through Parent study Month 12 visit.
  3. Negative pregnancy test (urine or serum) for female subjects of
  4. Female subjects must be (defined as cessation of menses for at least 1 year), surgically sterile (hysterectomy, bilateral oophorectomy, or tubal ligation), or willing to use a highly effective method of (such methods include combined [estrogen and progestogen containing] hormonal contraception associated with inhibition of ovulation: oral/intravaginal; transdermal/progestogen-only hormonal contraception associated with inhibition of ovulation: oral/injectable; implantable/intrauterine device [IUD]/intrauterine hormone-releasing system [IUS]/bilateral tubal occlusion/vasectomized partner/sexual abstinence) for the duration of the study (from the time they sign an ICF, until 4 weeks after the last dose of study treatment). Hormonal contraception alone (including oral, injectable, transdermal, and implantable) is not acceptable; an additional barrier method must be used. Intravaginal hormonal contraception or IUS alone are allowed per Investigator's discretion. Subjects on oral contraceptives will not be required to discontinue medication. Subjects will not be permitted to commence oral contraceptives while taking study treatment during the study.
  5. Male subjects must be surgically sterile or willing to use an acceptable method of contraception for the duration of the study (from the time they sign an ICF), until 4 weeks after the last dose of study treatment. An acceptable method of contraception would be a barrier method, such as condoms, restraining from having sex, or a partner using the approved methods of contraception for female subjects as per Inclusion Criteria #4.
  6. Subjects who are blood/egg/sperm donors should be willing to halt donations during the study and for 4 weeks following their last dose of study treatment.
  7. Subjects who are willing to provide informed consent/assent prior to any study-specific procedures. If a minor, the subject has a parent or legal guardian able to read, understand, and sign the ICF and/or a Child Assent Form (if applicable), communicate with the Investigator, and understand and comply with the protocol requirements. Adolescent subjects must also read and understand the Child Assent Form.
  8. Subjects who are willing to follow the dietary restrictions recommended by the Investigator.

Exclusion criteria

Exclusion Criteria:

  1. Severe reactions to the study treatment of the Parent study APG-20.
  2. Known to have tested positive for (HIV) or known to be diagnosed with HIV-related LD. Positive HIV test in countries requiring HIV testing.
  3. Are or receiving immunomodulatory drugs.
  4. Estimated (eGFR) \<30 mL/min/1.73m2 calculated by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for subjects ≥18 years of age and by Bedside Schwartz for subjects \<18 years of age.
  5. Diagnosis of hematological abnormalities (including but not limited to clinically significant leukopenia, neutropenia, bone marrow abnormalities, leukemia or lymphoma, or clinically significant pathological lymphadenopathy).
  6. that is ongoing/not in remission or that currently requires or has required active treatment within the past year (with the exception of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ [e.g., breast carcinoma, cervical cancer in situ] that have undergone potentially curative therapy).
  7. For females only: currently pregnant (confirmed with a positive pregnancy test) or breastfeeding.
  8. Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the subject.
Terms in these criteria, in plain language(10)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Postmenopausal

Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
24 participants (estimated)

Study arms

  • Experimental
    Metreleptin

    Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake

    Drug: Metreleptin

Interventions

  • Metreleptin is a recombinant human leptin analog that is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency

06

What researchers measure

Primary outcomes

  1. Evaluate the Incidence and Frequency of Treatment-Emergent Adverse Events (Safety and Tolerability)

    Incidence and frequency of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs), treatment-related TEAEs, adverse events of special interest (AESI), and adverse events leading to study treatment discontinuation/withdrawal from the study

    Time frame: From enrollment to the end of treatment (until the last participant completes 24 months)

Secondary outcomes

  1. To evaluate the long-term efficacy (HbA1c) of daily SC metreleptin treatment in participants with familial partial lipodystrophy (FPLD)

    Glycated hemoglobin (HbA1c) in participants with HbA1c ≥7% at the Parent study Baseline, over time during the OLE

    Time frame: From enrollment to the end of treatment (until the last participant completes 24 months)

  2. To evaluate the long-term efficacy (TGs) of daily SC metreleptin treatment in participants with FPLD

    \- Fasting triglycerides (TGs) in participants with TG ≥500 mg/dL at the Parent study Baseline, over time during the OLE

    Time frame: From enrollment to the end of treatment (until the last participant completes 24 months)

  3. To evaluate the long-term efficacy (FBG) of daily SC metreleptin treatment in participants with FPLD

    * Fasting blood glucose (FBG) in participants with HbA1c ≥7% at the Parent study Baseline, over time during the OLE * FBG in participants with FBG above upper limit of normal (ULN) at the Parent study Baseline, over time during the OLE

    Time frame: From enrollment to the end of treatment (until the last participant completes 24 months)

07

Study locations

7 of 7 sites recruiting
  • University of Alabama
    Birmingham, Alabama 35233, United States
    Recruiting
    • Fernando Ovalle · Contact
    • Fernando Ovalle · Principal investigator
  • Flourish Research
    Boca Raton, Florida 33434, United States
    Recruiting
    • Rasha Youssef · Contact
    • Rasha Youssef · Principal investigator
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
    Recruiting
    • Lindsay Fourman, MD · Contact
    • Lindsay Fourman, MD · Principal investigator
  • University of Michigan
    Ann Arbour, Michigan 48109, United States
    Recruiting
    • Elif Oral, MD · Contact
    • Elif Oral, MD · Principal investigator
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
    Recruiting
    • Vaneeta Bamba, MD · Contact
    • Vaneeta Bamba, MD · Principal investigator
  • UT Southwestern Medical Center
    Dallas, Texas 75390, United States
    Recruiting
    • Abhimanyu Garg, MD · Contact
    • Abhimanyu Garg, MD · Principal investigator
  • The Medical College of Wisconsin
    Milwaukee, Wisconsin 53226, United States
    Recruiting
    • Bradley Javorsky, MD · Contact
    • Bradley Javorsky, MD · Principal investigator
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 16, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06679270
Responsible party
Sponsor
First posted
Nov 7, 2024
Start date
Oct 14, 2024
Primary completion
Mar 2028 (estimated)
Completion
Dec 2028 (estimated)
Last update
Jul 16, 2026

Study contacts

Lori Hartnett, PhD
Contact
clinicaltrials_info@chiesi.com
+3905212791

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Metreleptin

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