Familial Partial LipodystrophyPhase 3NCT06679270
Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
A Phase 3 study testing Metreleptin in people with Familial Partial Lipodystrophy, run by Chiesi Farmaceutici S.p.A. at 7 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Familial Partial Lipodystrophy
- What is being tested?
- Metreleptin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 7 sitesUnited States
- Study dates
- 14 Oct 2024to Dec 2028
- Phase
- Phase 3
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 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥13 years of age, inclusive, at the time of signing the informed consent form (ICF).
- 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.
- Negative pregnancy test (urine or serum) for female subjects of childbearing potential
- Female subjects must be postmenopausal (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 contraception (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.
- 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.
- 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.
- 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.
- Subjects who are willing to follow the dietary restrictions recommended by the Investigator.
Who cannot join
- Severe hypersensitivity reactions to the study treatment of the Parent study APG-20.
- Known to have tested positive for human immunodeficiency virus (HIV) or known to be diagnosed with HIV-related LD. Positive HIV test in countries requiring HIV testing.
- Are immunocompromised or receiving immunomodulatory drugs.
- Estimated glomerular filtration rate (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.
- Diagnosis of clinically significant hematological abnormalities (including but not limited to clinically significant leukopenia, neutropenia, bone marrow abnormalities, leukemia or lymphoma, or clinically significant pathological lymphadenopathy).
- Malignancy 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).
- For females only: currently pregnant (confirmed with a positive pregnancy test) or breastfeeding.
- Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the subject.
Trial locations and contacts
7 sites in 1 country. Contact the study team or a site near you to ask about taking part.
7 sites in 1 country
United States7 sites, 7 recruiting
- Recruiting
University of Michigan
Ann Arbour, Michigan, 48109
Elif Oral, MD
Elif Oral, MD
- Recruiting
University of Alabama
Birmingham, Alabama, 35233
Fernando Ovalle
Fernando Ovalle
- Recruiting
Flourish Research
Boca Raton, Florida, 33434
Rasha Youssef
Rasha Youssef
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
Lindsay Fourman, MD
Lindsay Fourman, MD
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75390
Abhimanyu Garg, MD
Abhimanyu Garg, MD
- Recruiting
The Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Bradley Javorsky, MD
Bradley Javorsky, MD
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Vaneeta Bamba, MD
Vaneeta Bamba, MD
About this study
Official title: An Open-label Extension of APG-20 Study to Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy
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
- Conditions
- Familial Partial Lipodystrophy
- Being tested
- Metreleptin
- Keywords
- Metreleptin, familial partial lipodystrophy
- Medical subject headings
- Lipodystrophy, Familial Partial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetreleptinDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Metreleptin | Experimental | Metreleptin [Recombinant-methionyl human Leptin; r-metHuLeptin] for daily injection is a sterile, white, solid lyophilised cake Receives: Drug: Metreleptin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: From enrollment to the end of treatment (until the last participant completes 24 months)
Secondary outcomes (3)
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
Measured: From enrollment to the end of treatment (until the last participant completes 24 months)
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
Measured: From enrollment to the end of treatment (until the last participant completes 24 months)
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
Measured: From enrollment to the end of treatment (until the last participant completes 24 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2024
- Study start14 Oct 2024
- Primary completionMar 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial Partial LipodystrophyStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06679270
- Sponsor's protocol ID
- APG-20-OLE
- Other IDs
- APG-20 OLE
- Status last verified
- Jul 2026
- First submitted
- 5 Nov 2024
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No