Acquired partial lipodystrophy

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ORPHA:79087OMIM:608709E88.1
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6Active trials32Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Acquired partial lipodystrophy (APL) is a rare condition where the body loses fat tissue from certain areas, most commonly the face, arms, and upper body, while fat may build up in the lower body, especially the hips and legs. Unlike some forms of lipodystrophy that people are born with, APL develops during a person's lifetime — usually in childhood or early adulthood. It is also known as Barraquer-Simons syndrome, named after the doctors who first described it. The loss of fat under the skin changes the way a person looks, which can be emotionally difficult. But APL also affects how the body handles fats and sugars in the blood, which can lead to serious health problems over time. Many people with APL develop high levels of fats called triglycerides in the blood, insulin resistance, and sometimes type 2 diabetes. A particularly important concern is kidney disease — a condition called membranoproliferative glomerulonephritis (MPGN) affects roughly a third of people with APL and can be serious if not caught early. There is no cure for APL, but treatments focus on managing complications like high blood fats, diabetes, and kidney problems. Some people also explore cosmetic options to address changes in their appearance. Regular monitoring by a team of specialists is key to staying as healthy as possible.

Also known as:

Key symptoms:

Gradual loss of fat from the face, giving a sunken or hollow appearanceLoss of fat from the arms, shoulders, and upper bodyUnusual buildup of fat in the hips, thighs, and lower bodyHigh levels of fats (triglycerides) in the bloodInsulin resistance or type 2 diabetesKidney problems, including protein in the urineLow levels of a blood protein called complement C3Fatigue and low energyChanges in skin appearance where fat has been lostSwelling in the legs or ankles if kidney disease develops

Clinical phenotype terms (17)— hover any for plain English
Progeroid facial appearanceHP:0005328Decreased circulating complement C3 concentrationHP:0005421Increased total lymphocyte countHP:0100827GlomerulopathyHP:0100820
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

6 events
Feb 2024Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

Amryt Pharma — PHASE4

TrialRECRUITING
Jan 2024Feasibility of Adipose Tissue Triglyceride (TG) Labelling in Familial Partial Lipodystrophy (FPLD)

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

TrialRECRUITING
Jan 2023Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) — PHASE2

TrialRECRUITING
Dec 2021Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL

Amryt Pharma — PHASE3

TrialACTIVE NOT RECRUITING
Jan 2020Low Energy Diet and Familial Partial Lipodystrophy

Cambridge University Hospitals NHS Foundation Trust — NA

TrialRECRUITING
Oct 2014Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) — PHASE2

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Acquired partial lipodystrophy.

6 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

6 recruitingView all trials with filters →
Phase 31 trial
Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL
Phase 3
Active
· Sites: Birmingham, Alabama; Sacramento, California +29 more · Age: 1299 yrs
Phase 41 trial
Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
Phase 4
Actively Recruiting
· Sites: Lille; Paris +10 more · Age: 1299 yrs
Phase 22 trials
Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy
Phase 2
Active
PI: Rebecca J Brown, M.D. (National Institute of Diabetes and Digestive and K) · Sites: Bethesda, Maryland · Age: 098 yrs
Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance
Phase 2
Actively Recruiting
PI: Rebecca J Brown, M.D. (National Institute of Diabetes and Digestive and K) · Sites: Bethesda, Maryland · Age: 1870 yrs
N/A1 trial
Low Energy Diet and Familial Partial Lipodystrophy
N/A
Actively Recruiting
PI: David Savage (Wellcome Trust-MRC Institute of Metabolic Science,) · Sites: Cambridge · Age: 1899 yrs
Other1 trial
Feasibility of Adipose Tissue Triglyceride (TG) Labelling in Familial Partial Lipodystrophy (FPLD)
Actively Recruiting
PI: Rebecca J Brown, M.D. (National Institute of Diabetes and Digestive and K) · Sites: Bethesda, Maryland · Age: 1865 yrs

Specialists

Showing 25 of 32View all specialists →
DS
David Savage
Specialist
PI on 1 active trial
GA
Geetha Anand
LAKELAND, FL
Specialist
1 Acquired partial lipodystrophy publication
GG
Girish L Gupte
Specialist
1 Acquired partial lipodystrophy publication
RW
Rachel Williams
Specialist
1 Acquired partial lipodystrophy publication
AR
Astor Rodrigues
Specialist
1 Acquired partial lipodystrophy publication
RB
Rachel M Brown
Specialist
1 Acquired partial lipodystrophy publication
KD
Kavinda Dayasiri
Specialist
1 Acquired partial lipodystrophy publication
CB
Christopher Bakewell
Specialist
1 Acquired partial lipodystrophy publication
CV
Chandna Vasandani
Specialist
1 Acquired partial lipodystrophy publication
MH
Michael Hwang
Specialist
1 Acquired partial lipodystrophy publication
CP
Caterina Pelosini
Specialist
2 Acquired partial lipodystrophy publications
CK
Chatchon Kaewkrasaesin
Specialist
1 Acquired partial lipodystrophy publication
RM
Rebecca J Brown, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 7 active trials
VM
Vanessa Rangel Miller, M.S., M.B.A
Specialist
PI on 1 active trial
LB
Leonardo Bighetti
Specialist
1 Acquired partial lipodystrophy publication
EM
Edoardo Luigi Maria Mollero
Specialist
1 Acquired partial lipodystrophy publication
MR
Martina Romanisio
Specialist
1 Acquired partial lipodystrophy publication

Treatment Centers

8 centers
⚗️ Trial Site

National Institutes of Health Clinical Center

📍 Bethesda, Maryland

👤 Payal P Khincha, M.D.

👤 Christopher Grunseich, M.D.

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

Travel Grants

No travel grants are currently matched to Acquired partial lipodystrophy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Acquired partial lipodystrophy

Disease timeline:

New recruiting trial: Feasibility of Adipose Tissue Triglyceride (TG) Labelling in Familial Partial Lipodystrophy (FPLD)

A new clinical trial is recruiting patients for Acquired partial lipodystrophy

New recruiting trial: Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

A new clinical trial is recruiting patients for Acquired partial lipodystrophy

New recruiting trial: Low Energy Diet and Familial Partial Lipodystrophy

A new clinical trial is recruiting patients for Acquired partial lipodystrophy

New recruiting trial: Tirzepatide for Partial Lipodystrophy Treatment: A New Horizon in 2024

A new clinical trial is recruiting patients for Acquired partial lipodystrophy

New recruiting trial: Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

A new clinical trial is recruiting patients for Acquired partial lipodystrophy

New recruiting trial: Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance

A new clinical trial is recruiting patients for Acquired partial lipodystrophy

New trial: Study of Growth Hormone Inhibition Using Pegvisomant in Severe Insulin Resistance

Phase PHASE2 trial recruiting. Pegvisomant

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What tests do I need to check my kidneys, blood fats, and blood sugar, and how often should I have them?,Should I have genetic testing to confirm my diagnosis or rule out other types of lipodystrophy?,What dietary changes will help manage my triglyceride levels and reduce my risk of complications?,Are there any clinical trials or newer treatments I should know about?,What are the warning signs that my kidney function is getting worse, and when should I go to the emergency room?,Can you refer me to a lipodystrophy specialist or center with experience in this condition?,What support is available for the emotional and psychological effects of changes in my appearance?

Common questions about Acquired partial lipodystrophy

What is Acquired partial lipodystrophy?

Acquired partial lipodystrophy (APL) is a rare condition where the body loses fat tissue from certain areas, most commonly the face, arms, and upper body, while fat may build up in the lower body, especially the hips and legs. Unlike some forms of lipodystrophy that people are born with, APL develops during a person's lifetime — usually in childhood or early adulthood. It is also known as Barraquer-Simons syndrome, named after the doctors who first described it. The loss of fat under the skin changes the way a person looks, which can be emotionally difficult. But APL also affects how the body

How is Acquired partial lipodystrophy inherited?

Acquired partial lipodystrophy follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Acquired partial lipodystrophy typically begin?

Typical onset of Acquired partial lipodystrophy is childhood. Age of onset can vary across affected individuals.

Are there clinical trials for Acquired partial lipodystrophy?

Yes — 6 recruiting clinical trials are currently listed for Acquired partial lipodystrophy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Acquired partial lipodystrophy?

25 specialists and care centers treating Acquired partial lipodystrophy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.