Disease · MONDO_0002525

inherited lipid metabolism disorder

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. inherited lipid metabolism disorder. Diseases, record MONDO_0002525. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0002525

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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.

Name: inherited lipid metabolism disorder; Open Targets
Open Targets ID: MONDO_0002525; Open Targets
Other names: Disorder of lipid metabolism; Open Targets
Disease Ontology: DOID:3146; Disease Ontology
NCI Thesaurus: C97092; NCI Thesaurus
Orphanet: ORPHA:309005; Orphanet
Highest stage of its drugs: Approval, for 15 of its 112 drugs, from EMA Human Drugs, PMDA and TTD; Open Targets

Drugs112

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
FENOFIBRATE	CHEMBL672	Small molecule	Approval	EMA Human Drugs	21	19	19	Drugs and targets
EZETIMIBE	CHEMBL1138	Small molecule	Approval	EMA Human Drugs	18	17	17	Drugs and targets
NIACIN	CHEMBL573	Small molecule	Approval	TTD	19	14	14	Drugs and targets
SIMVASTATIN	CHEMBL1064	Small molecule	Approval	EMA Human Drugs	11	10	10	Drugs and targets
LAROPIPRANT	CHEMBL426559	Small molecule	Approval	EMA Human Drugs, at withdrawal	9	5	5	Drugs and targets
PRAVASTATIN	CHEMBL1144	Small molecule	Approval	EMA Human Drugs	4	3	3	Drugs and targets
BEMPEDOIC ACID	CHEMBL3545313	Small molecule	Approval	EMA Human Drugs	3	1	1	Drugs and targets
ALIROCUMAB	CHEMBL2109540	Antibody	Approval	EMA Human Drugs	2	1	1	Drugs and targets
CHOLINE FENOFIBRATE	CHEMBL1201745	Small molecule	Approval	TTD	2	1	1	Drugs and targets
EVOLOCUMAB	CHEMBL2364655	Antibody	Approval	EMA Human Drugs	2	1	1	Drugs and targets

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Targets139

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
PPARA	Peroxisome proliferator-activated receptor	10	Peroxisome proliferator-activated receptor agonist; Peroxisome proliferator-activated receptor alpha agonist	Approval	https://conductscience.org/library/genes/5465	Drugs and targets
HMGCR	3-hydroxy-3-methylglutaryl-coenzyme A reductase	7	HMG-CoA reductase inhibitor	Approval	https://conductscience.org/library/genes/3156	Drugs and targets
AGTR1	Type-1 angiotensin II receptor	6	Type-1 angiotensin II receptor antagonist	Phase 3	https://conductscience.org/library/genes/185	Drugs and targets
PPARG	Peroxisome proliferator-activated receptor	6	Peroxisome proliferator-activated receptor agonist; Peroxisome proliferator-activated receptor gamma agonist	Approval	https://conductscience.org/library/genes/5468	Drugs and targets
ADRB1	Adrenergic receptor beta	4	Adrenergic receptor beta antagonist; Beta-1 adrenergic receptor antagonist	Unknown	https://conductscience.org/library/genes/153	Drugs and targets
CETP	Cholesteryl ester transfer protein	4	Cholesteryl ester transfer protein inhibitor	Phase 3	https://conductscience.org/library/genes/1071	Drugs and targets
PCSK9	PCSK9 mRNA	4	PCSK9 mRNA RNAi inhibitor; Subtilisin/kexin type 9 inhibitor	Approval	https://conductscience.org/library/genes/255738	Drugs and targets
DPP4	Dipeptidyl peptidase 4	3	Dipeptidyl peptidase IV inhibitor	Phase 3	https://conductscience.org/library/genes/1803	Drugs and targets
GLP1R	Glucagon-like peptide 1 receptor	3	Glucagon-like peptide 1 receptor agonist; Glucagon-like peptide 1 receptor antagonist	Unknown	https://conductscience.org/library/genes/2740	Drugs and targets
ADRB2	Adrenergic receptor beta	2	Adrenergic receptor beta antagonist; Beta-2 adrenergic receptor antagonist	Unknown	https://conductscience.org/library/genes/154	Drugs and targets

1 to 10 of 139

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Trials164

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. 6 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00961636	A Long-term Study of ERN/LRPT (Extended Release Niacin/Laropiprant [MK0524A]) in Patients With Dyslipidemia (0524A-102)	Phase 3	1,152	2012-02-17	Dyslipidemia	LAROPIPRANT, NIACIN	Open Targets' reading of its record	ClinicalTrials.gov
NCT00463606	A Multicenter Study Comparing the Safety and Efficacy of ABT-335 and Rosuvastatin Calcium Combination Therapy to Monotherapy in Subjects With Dyslipidemia	Phase 3	760	2012-08-01	Hypercholesterolemia, Dyslipidemia	FENOFIBRIC ACID, ROSUVASTATIN	Open Targets' reading of its record	ClinicalTrials.gov
NCT00542386	A Study of MCI-196 in Chronic Kidney Disease Subjects on Dialysis With Hyperphosphatemia and Dyslipidaemia	Phase 3	642	2014-10-06	Chronic Kidney Disease, Dialysis, Hyperphosphatemia, Dyslipidemia	COLESTILAN CHLORIDE	Open Targets' reading of its record	ClinicalTrials.gov
NCT00639158	Safety and Efficacy Study Comparing ABT-335 Coadministered With Atorvastatin and Ezetimibe to Atorvastatin Coadministered With Ezetimibe in Subjects With Multiple Abnormal Lipid (Fat) Levels in the Blood	Phase 3	543	2009-11-05	Dyslipidemias, Coronary Heart Disease, Combined (Atherogenic) Dyslipidemia, Mixed Dyslipidemia	FENOFIBRIC ACID	Open Targets' reading of its record	ClinicalTrials.gov
NCT00770653	Efficacy of Pioglitazone/Metformin Combination Therapy in Subjects With Type 2 Diabetes Mellitus and Dyslipidemia.	Phase 3	305	2010-10-06	Diabetes Mellitus, Dyslipidemias	GLIMEPIRIDE, METFORMIN, PIOGLITAZONE	Open Targets' reading of its record	ClinicalTrials.gov
NCT01218204	A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Administering Multiple Oral Doses of GSK1292263 Alone and With Atorvastatin	Phase 2	287	2017-11-08	Dyslipidaemias, Dyslipidemias	EZETIMIBE, GSK-1292263	Open Targets' reading of its record	ClinicalTrials.gov
NCT04516291	A Dose-Ranging Study With Vupanorsen (TRANSLATE-TIMI 70)	Phase 2	286	2022-10-04	Dyslipidemias, Hyperlipidemias, Hyperlipoproteinemias	VUPANORSEN	Open Targets' reading of its record	ClinicalTrials.gov
NCT00858637	Efficacy and Safety Study of MCI-196 Versus Simvastatin for Dyslipidaemia in Chronic Kidney Disease (CKD) Subjects on Dialysis	Phase 3	260	2014-10-17	Chronic Kidney Disease	COLESTILAN CHLORIDE, SIMVASTATIN	Open Targets' reading of its record	ClinicalTrials.gov
NCT00246376	Diet, Exercise, Niacin, and Fenofibrate to Reduce Heart Disease Risk Factors in Individuals With HIV Lipodystrophy or Dyslipidemia	Not applicable	221	2016-03-21	Cardiovascular Diseases, Heart Diseases, HIV Infections, Hyperlipidemia, Hypertriglyceridemia, Insulin Resistance, Atherosclerosis	FENOFIBRATE, NIACIN	Open Targets' reading of its record	ClinicalTrials.gov
NCT00504829	Study of Atorvastatin/Fenofibrate (LCP-AtorFen) Combination Therapy in Dyslipidemia	Phase 2	220	2020-02-05	Dyslipidemia	ATORVASTATIN, FENOFIBRATE	Open Targets' reading of its record	ClinicalTrials.gov

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Comparisons in its trials18

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
ABT-335 + 40 mg Atorvastatin + 10 mg Ezetimibe vs Placebo + 40 mg Atorvastatin + 10 mg Ezetimibe: serious adverse events	3 of 272	5 of 270	542	NCT00639158	Trial comparisons
Pioglitazone 15 mg and Metformin 850 mg BID vs Glimepiride 2 mg and Metformin 850 mg BID: serious adverse events	17 of 153	8 of 149	302	NCT00770653	Trial comparisons
Pioglitazone 15 mg and Metformin 850 mg BID vs Glimepiride 2 mg and Metformin 850 mg BID: Intake of Study Medication Greater Than 80% and Less Than 120%.	136 of 146	137 of 142	288	NCT00770653	Trial comparisons
MCI-196 (Active) + Simvastatin (Placebo)/ Comparison Phase vs Simvastatin (Active) + MCI-196 (Placebo)/ Comparion Phase: serious adverse events	16 of 128	12 of 131	259	NCT00858637	Trial comparisons
Pitavastatin 4 mg vs Atorvastatin 40 mg: serious adverse events	8 of 143	5 of 71	214	NCT00344370	Trial comparisons
Pitavastatin 4 mg vs Atorvastatin 40 mg: NCEP LDL-C Target Attainment	110 of 141	55 of 71	212	NCT00344370	Trial comparisons
Atorvastatin 10 mg vs Atorvastatin 80 mg: Number of Participants With Abnormal Clinically Significant ECG Findings- Part B (Run-in)	0 of 59	0 of 25	84	NCT01218204	Trial comparisons
Simvastatin vs Placebo: serious adverse events	0 of 30	0 of 30	60	NCT00935259	Trial comparisons
Ezetimibe vs Placebo: serious adverse events	0 of 20	0 of 20	40	NCT01849068	Trial comparisons
Pioglitazone Group vs Comparator Group: serious adverse events	0 of 16	0 of 14	30	NCT01156597	Trial comparisons

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Papers from its trials193

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	Source
39924184	RESULT	1	PubMed
39322570	RESULT	1	PubMed
38616112	RESULT	1	PubMed
37405785	RESULT	1	PubMed
35953719	RESULT	1	PubMed
31860457	RESULT	1	PubMed
28478132	RESULT	1	PubMed
27616593	RESULT	1	PubMed
27532918	RESULT	1	PubMed
27317162	RESULT	1	PubMed

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Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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_0002525

GET https://conductscience.org/api/diseases/v1/MONDO_0002525/drugs?page=2

API reference For AI assistants