Disease · MONDO_0005439
Trials71
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. 1 more is not shown, since the checks do not confirm it.
1 to 20 of 71
Download this page as JSON| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT03814187 | Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C | Phase 3 | 3,275 | 2024-03-06 | ASCVD, Elevated Cholesterol, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia | INCLISIRAN | Its record names the disease | ClinicalTrials.gov |
| NCT03694197 | Long Term Safety Study of PRALUENT | Phase 4 | 1,389 | 2021-06-15 | Heterozygous Familial Hypercholesterolemia, Non-familial Hypercholesterolemia | ALIROCUMAB | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT02597127 | Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C) | Phase 2 | 501 | 2019-05-17 | Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia, Diabetes | INCLISIRAN | Its record names the disease | ClinicalTrials.gov |
| NCT03397121 | Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH) | Phase 3 | 482 | 2020-10-28 | Heterozygous Familial Hypercholesterolemia, Elevated Cholesterol | INCLISIRAN SODIUM | Its record names the disease | ClinicalTrials.gov |
| NCT01968980 | A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia | Phase 3 | 370 | 2017-05-19 | Heterozygous Familial Hypercholesterolemia | BOCOCIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT01730040 | Study of the Efficacy and Safety of Alirocumab (REGN727/SAR236553) in Combination With Other Lipid-modifying Treatment (LMT) (ODYSSEY OPTIONS I) | Phase 3 | 355 | 2015-08-31 | Hypercholesterolemia | ALIROCUMAB, ATORVASTATIN, EZETIMIBE, ROSUVASTATIN | Its record names the disease | ClinicalTrials.gov |
| NCT05425745 | Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies. | Phase 3 | 354 | 2025-06-11 | Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia, Lipid Metabolism Disorder, Metabolic Disease, Lipid Metabolism, Inborn Errors, Genetic Disease, Inborn, Hyperlipoproteinemias | OBICETRAPIB | Its record names the disease | ClinicalTrials.gov |
| NCT01078675 | An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia | Phase 3 | 315 | 2015-02-02 | Familial Hypercholesterolaemia | ROSUVASTATIN | Its record names the disease | ClinicalTrials.gov |
| NCT01475825 | A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol | Phase 3 | 309 | 2019-03-14 | Hypercholesterolemia, Heterozygous Familial | MIPOMERSEN SODIUM | Its record names the disease | ClinicalTrials.gov |
| NCT01524289 | Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020) | Phase 3 | 306 | 2019-08-28 | Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial | ANACETRAPIB | Its record names the disease | ClinicalTrials.gov |
| NCT05952869 | A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH) | Phase 3 | 303 | 2026-02-24 | Hypercholesterolemia, Familial Hypercholesterolemia | ENLICITIDE, ENLICITIDE DECANOATE | Its record names the disease | ClinicalTrials.gov |
| NCT01624142 | Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders | Phase 2/3 | 300 | 2019-01-28 | Severe Familial Hypercholesterolemia | EVOLOCUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT01709500 | Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy) | Phase 3 | 249 | 2015-10-28 | Heterozygous Familial Hypercholesterolemia | ALIROCUMAB | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT00355615 | PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin | Phase 3 | 173 | 2009-07-20 | Familial Hypercholesterolemia | ROSUVASTATIN | Its record names the disease | ClinicalTrials.gov |
| NCT02624869 | Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) | Phase 3 | 163 | 2022-01-14 | Familial Hypercholesterolemia | EVOLOCUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT02392559 | Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders | Phase 3 | 158 | 2020-07-15 | Heterozygous Familial Hypercholesterolemia | EVOLOCUMAB | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT00474240 | AEGR-733 and Atorvastatin 20 mg vs. Monotherapy in Moderate Hypercholesterolemia | Phase 2 | 157 | 2013-02-21 | Hypercholesterolemia | ATORVASTATIN, LOMITAPIDE, LOMITAPIDE MESYLATE | Its record names the disease | ClinicalTrials.gov |
| NCT00694109 | An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia | Phase 3 | 144 | 2015-12-21 | Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders | MIPOMERSEN | Its record names the disease | ClinicalTrials.gov |
| NCT04652726 | Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia | Phase 3 | 141 | 2025-06-10 | Familial Hypercholesterolemia - Heterozygous | INCLISIRAN | Its record names the disease | ClinicalTrials.gov |
| NCT00706849 | Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease | Phase 3 | 124 | 2013-03-21 | Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease | MIPOMERSEN | Its record names the disease | ClinicalTrials.gov |
diseases-2026-09-18-0.1.0
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
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GET https://conductscience.org/api/diseases/v1/MONDO_0005439/trials