Disease · MONDO_0020088
familial partial lipodystrophy
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. familial partial lipodystrophy. Diseases, record MONDO_0020088. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0020088
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
- FPLD, congenital partial lipodystrophy, genetic partial lipodystrophy, lipodystrophy, familial partial
- Open Targets
Drugs6
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| METRELEPTIN | CHEMBL2107857 | Protein | Approval | EMA Human Drugs | 1 | 0 | 0 | Drugs and targets |
| VOLANESORSEN | CHEMBL3544994 | Oligonucleotide | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| MIBAVADEMAB | CHEMBL4650509 | Antibody | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| OBETICHOLIC ACID | CHEMBL566315 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| VUPANORSEN | CHEMBL4298197 | Oligonucleotide | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| GEMCABENE | CHEMBL2110686 | Small molecule | Phase 1/2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets5
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| LEPR | Leptin receptor | 2 | Leptin receptor agonist | Approval | https://conductscience.org/library/genes/3953 | Drugs and targets |
| ANGPTL3 | Angiopoietin like 3 (ANGPTL3) mRNA | 1 | Angiopoietin like 3 (ANGPTL3) mRNA antisense inhibitor | Phase 2 | https://conductscience.org/library/genes/27329 | Drugs and targets |
| APOC3 | Apolipoprotein C-III mRNA 3'UTR | 1 | Apolipoprotein C-III mRNA 3'UTR antisense inhibitor | Phase 2/3 | https://conductscience.org/library/genes/345 | Drugs and targets |
| ASGR1 | Asialoglycoprotein receptor 1 | 1 | Asialoglycoprotein receptor 1 binding agent | Phase 2 | https://conductscience.org/library/genes/432 | Drugs and targets |
| NR1H4 | Bile acid receptor | 1 | Bile acid receptor FXR agonist | Phase 2 | https://conductscience.org/library/genes/9971 | Drugs and targets |
Trials5
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT02527343 | The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy | Phase 2/3 | 40 | 2021-10-18 | Familial Partial Lipodystrophy | VOLANESORSEN | Its record names the disease | ClinicalTrials.gov |
| NCT05088460 | A Study to Examine the Effects of the Leptin Receptor (LEPR) Agonist Antibody REGN4461 in Adult Patients With Familial Partial Lipodystrophy (FPLD) | Phase 2 | 20 | 2025-07-02 | Familial Partial Lipodystrophy, Metabolic Abnormalities | MIBAVADEMAB | Its record names the disease | ClinicalTrials.gov |
| NCT02430077 | Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients | Phase 2 | 10 | 2023-12-13 | Familial Partial Lipodystrophy | OBETICHOLIC ACID | Its record names the disease | ClinicalTrials.gov |
| NCT03508687 | Study of Gemcabene in Adults With FPLD | Phase 1/2 | 5 | 2020-08-17 | Familial Partial Lipodystrophy, Hypertriglyceridemia, Fatty Liver, NASH - Nonalcoholic Steatohepatitis | GEMCABENE | Its record names the disease | ClinicalTrials.gov |
| NCT03514420 | Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL) | Phase 2 | 4 | 2021-02-16 | Familial Partial Lipodystrophy | VUPANORSEN | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials5
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Active Capsule of Obeticholic Acid vs Placebo for Obeticholic Acid: deaths from any cause | 0 of 10 | 0 of 10 | 20 | NCT02430077 | Trial comparisons |
| Active Capsule of Obeticholic Acid vs Placebo for Obeticholic Acid: serious adverse events | 0 of 10 | 0 of 10 | 20 | NCT02430077 | Trial comparisons |
| Arm 2 Combined Cohort A + B: REGN4461 to REGN4461 vs Arm 1 Combined Cohort A + B: Placebo to REGN4461: Number of Participants With Treatment-emergent Anti-drug Antibody (ADA) Response | 0 of 10 | 0 of 10 | 20 | NCT05088460 | Trial comparisons |
| Group 1: 300 mg Gemcabene Daily Week 1-24 vs Group 2: 600mg Gemcabene Daily Week 12-24: deaths from any cause | 0 of 2 | 0 of 3 | 5 | NCT03508687 | Trial comparisons |
| Group 1: 300 mg Gemcabene Daily Week 1-24 vs Group 2: 600mg Gemcabene Daily Week 12-24: serious adverse events | 0 of 2 | 1 of 3 | 5 | NCT03508687 | Trial comparisons |
Papers from its trials1
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
| PMID | Listed as | Trials listing it |
|---|---|---|
| 37164837 | DERIVED | 1 |
Source PubMed
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0020088
GET https://conductscience.org/api/diseases/v1/MONDO_0020088/drugs