Partial LipodystrophyPhase 4NCT06484868
Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
A Phase 4 study testing Metreleptin in people with Partial Lipodystrophy, run by Amryt Pharma at 12 sites in 4 countries.
Trial summary
This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- 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
- 12 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 8 Feb 2024to Feb 2028
- Phase
- Phase 4
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed diagnosis of familial or acquired partial lipodystrophy
Who cannot join
- Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, before the screening visit.
- Other protocol defined inclusion/exclusion criteria apply
Trial locations and contacts
12 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 4 countries
Italy5 sites, 5 recruiting
- Recruiting
Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi
Bologna
Alessandra Gambineri
- Not yet recruiting
Universitá degli Studi "Magna Graecia" di Catanzaro
Catanzaro
Antonio Aversa
- Recruiting
Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
Novara
Flavia Prodam
- Recruiting
Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello
Pisa
Giovanni Ceccarini
- Recruiting
A.S.U Integrata di Udine - Presidio Ospedaliero Santa Maria della Misericordia
Udine
Annalisa Sechi
France4 sites, 4 recruiting
- Recruiting
Centre Hospitalier Régional Universitaire de Lille (CHRU) - Hôpital Claude Huriez
Lille
Marie-Christine Vantyghem
- Recruiting
Hopital Pitie-Salpetriere
Paris
Fabrizio Andreelli
- Recruiting
Hôpital Saint-Antoine
Paris
Corinne Vigouroux
- Recruiting
Centre Hospitalier Lyon-Sud
Pierre-Bénite
Emmanuel Disse
Germany2 sites, 2 recruiting
- Recruiting
Charite-Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)
Berlin
Thomas Bobbert
- Not yet recruiting
Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
Ulm
Martin Wabitsch
United Kingdom1 site, 1 recruiting
- Not yet recruiting
Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
Cambridge
Anna Stears
About this study
Official title: A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy
This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy
- Conditions
- Partial Lipodystrophy
- Being tested
- Metreleptin
- Keywords
- Metreleptin
- Medical subject headings
- Lipodystrophy, Partial, Acquired
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.
Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.
To evaluate the efficacy (HbA1c) of metreleptin treatment in patients with PL
Measured: 12 months
Number of patients with decrease of at least 30% in triglycerides (TG) at Month 12 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L)
To evaluate the efficacy (TG) of metreleptin treatment in patients with PL
Measured: 12 months
Secondary outcomes (4)
Number of patients with decrease of at least 0.5% in HbA1c at Month 24 compared to Baseline or HbA1c <6.5 % at Month 24, in patients with baseline HbA1c ≥6.5%.
To evaluate the long-term efficacy of metreleptin treatment in patients with PL
Measured: 24 months
Number of patients with decrease of at least 30% in TG levels at Month 24 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L).
To evaluate the long-term efficacy of metreleptin treatment in patients with PL
Measured: 24 months
Change from baseline in liver volume at Month 12 and Month 24
To assess changes in liver volume.
Measured: 12 months and 24 months
Incidence of, Treatment emergent adverse events (TEAEs), Deaths and other serious adverse events (SAEs), Treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation
To evaluate the safety of metreleptin treatment in patients with PL
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2024
- Study start8 Feb 2024
- Primary completionFeb 2028 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Partial LipodystrophyStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Amryt Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06484868
- Sponsor's protocol ID
- APL-22
- Other IDs
- 2022-502950-14-00
- Status last verified
- Jul 2025
- First submitted
- 26 Jun 2024
- Last updated
- 3 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy
- ClinicalTrials.gov ID
- NCT06484868
- Acronym / sponsor ID
- APL-22
- Conditions
- Partial Lipodystrophy
- Keywords
- Metreleptin
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Amryt PharmaIndustry (pharma, biotech, devices)
- Study start
- 8 Feb 2024 (actual)
- Primary completion
- Feb 2028 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 3 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Metreleptin | Experimental | Drug: MetreleptinMetreleptin [Recombinant-methionyl human Leptin; r-metHuLeptin] for daily injection is a sterile, white, solid lyophilised cake |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Confirmed diagnosis of familial or acquired partial lipodystrophy
Key exclusion criteria (2 listed)
- Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, before the screening visit.
- Other protocol defined inclusion/exclusion criteria apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MetreleptinMetreleptin is a recombinant human leptin analog that is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency | Drug | Metreleptin |
Primary outcome
Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.
Time frame: 12 months
To evaluate the efficacy (HbA1c) of metreleptin treatment in patients with PL
Number of patients with decrease of at least 30% in triglycerides (TG) at Month 12 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L)
Time frame: 12 months
To evaluate the efficacy (TG) of metreleptin treatment in patients with PL
4 secondary outcomes
Number of patients with decrease of at least 0.5% in HbA1c at Month 24 compared to Baseline or HbA1c <6.5 % at Month 24, in patients with baseline HbA1c ≥6.5%.
Time frame: 24 months
Number of patients with decrease of at least 30% in TG levels at Month 24 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L).
Time frame: 24 months
Change from baseline in liver volume at Month 12 and Month 24
Time frame: 12 months and 24 months
Incidence of, Treatment emergent adverse events (TEAEs), Deaths and other serious adverse events (SAEs), Treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation
Time frame: 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
12 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Metreleptin in people with Partial Lipodystrophy, run by Amryt Pharma at 12 sites in 4 countries.
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 1 inclusion and 2 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: looking for participants now, as of the sponsor's last update on 3 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Italy, France, Germany, United Kingdom. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Janet Boylan (+3905212791, clinicaltrials_info@chiesi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%. (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Jul 2025.