Disease · MONDO_0008300
Prader-Willi syndrome
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. Prader-Willi syndrome. Diseases, record MONDO_0008300. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0008300
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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.
- Other names
- Prader-Labhart-Willi syndrome, Prader-Willi-Labhart syndrome, Willi-Prader syndrome
- Open Targets
- Highest stage of its drugs
- Approval, for 1 of its 22 drugs, from DailyMed and EMA Human Drugs
- Open Targets
Drugs22
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 |
|---|---|---|---|---|---|---|---|---|
| SOMATROPIN | CHEMBL1201621 | Protein | Approval | DailyMed and EMA Human Drugs | 13 | 10 | 10 | Drugs and targets |
| OXYTOCIN | CHEMBL395429 | Protein | Phase 3 | ClinicalTrials.gov | 12 | 12 | 12 | Drugs and targets |
| DIAZOXIDE CHOLINE | CHEMBL4297276 | Small molecule | Phase 3 | ClinicalTrials.gov and TTD | 7 | 5 | 5 | Drugs and targets |
| CANNABIDIOL | CHEMBL190461 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| OCTREOTIDE | CHEMBL1680 | Protein | Phase 3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| PITOLISANT | CHEMBL462605 | Small molecule | Phase 3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| CARBETOCIN | CHEMBL3301668 | Protein | Phase 3 | ClinicalTrials.gov | 3 | 2 | 2 | Drugs and targets |
| BELORANIB | CHEMBL4297504 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| DENATONIUM ACETATE MONOHYDRATE | CHEMBL5314947 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| TOPIRAMATE | CHEMBL220492 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
1 to 10 of 22
See all 22 drugsTargets56
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 |
|---|---|---|---|---|---|---|
| CNR1 | Cannabinoid receptor 1 | 2 | Cannabinoid CB1 receptor antagonist; Cannabinoid CB1 receptor negative allosteric modulator | Phase 3 | https://conductscience.org/library/genes/1268 | Drugs and targets |
| GLP1R | Glucagon-like peptide 1 receptor | 2 | Glucagon-like peptide 1 receptor agonist | Phase 3 | https://conductscience.org/library/genes/2740 | Drugs and targets |
| OXTR | Oxytocin receptor | 2 | Oxytocin receptor agonist | Phase 3 | https://conductscience.org/library/genes/5021 | Drugs and targets |
| ADRB1 | Beta-1 adrenergic receptor | 1 | Beta-1 adrenergic receptor antagonist | Phase 2 | https://conductscience.org/library/genes/153 | Drugs and targets |
| CA2 | Carbonic anhydrase 2 | 1 | Carbonic anhydrase II inhibitor | Phase 3 | https://conductscience.org/library/genes/760 | Drugs and targets |
| CA4 | Carbonic anhydrase 4 | 1 | Carbonic anhydrase IV inhibitor | Phase 3 | https://conductscience.org/library/genes/762 | Drugs and targets |
| CYP19A1 | Aromatase | 1 | Cytochrome P450 19A1 inhibitor | Unknown | https://conductscience.org/library/genes/1588 | Drugs and targets |
| GABRA1 | GABA-A receptor; anion channel | 1 | GABA-A receptor; anion channel positive modulator | Phase 3 | https://conductscience.org/library/genes/2554 | Drugs and targets |
| GABRA2 | GABA-A receptor; anion channel | 1 | GABA-A receptor; anion channel positive modulator | Phase 3 | https://conductscience.org/library/genes/2555 | Drugs and targets |
| GABRA3 | GABA-A receptor; anion channel | 1 | GABA-A receptor; anion channel positive modulator | Phase 3 | https://conductscience.org/library/genes/2556 | Drugs and targets |
1 to 10 of 56
See all 56 targetsTrials56
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 |
|---|---|---|---|---|---|---|---|---|
| NCT03790865 | Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome | Phase 2/3 | 158 | 2021-02-17 | Prader-Willi Syndrome, Hyperphagia | LIVOLETIDE | Its record names the disease | ClinicalTrials.gov |
| NCT03649477 | Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome | Phase 3 | 130 | 2021-11-17 | Prader-Willi Syndrome | CARBETOCIN | Its record names the disease | ClinicalTrials.gov |
| NCT03440814 | A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome | Phase 3 | 127 | 2023-09-21 | Prader-Willi Syndrome | DIAZOXIDE CHOLINE | Its record names the disease | ClinicalTrials.gov |
| NCT04257929 | A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension | Phase 2 | 65 | 2025-10-22 | Prader-Willi Syndrome | PITOLISANT | Its record names the disease | ClinicalTrials.gov |
| NCT02527200 | Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome | Phase 3 | 56 | 2022-02-03 | Metabolism and Nutrition Disorder, Obesity | LIRAGLUTIDE | Its record names the disease | ClinicalTrials.gov |
| NCT02311673 | Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome | Phase 2 | 40 | 2023-07-27 | Prader-Willi Syndrome | SETMELANOTIDE | Its record names the disease | ClinicalTrials.gov |
| NCT01968187 | Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome | Phase 2 | 38 | 2025-03-27 | Hyperphagia in Prader-Willi Syndrome | CARBETOCIN | Its record names the disease | ClinicalTrials.gov |
| NCT04697381 | Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS | Phase 3 | 33 | 2024-06-05 | Prader-Willi Syndrome | SOMATROPIN | Its record names the disease | ClinicalTrials.gov |
| NCT03197662 | Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome | Phase 2 | 32 | 2025-02-07 | Prader-Willi Syndrome, Hyperphagia | OXYTOCIN | Its record names the disease | ClinicalTrials.gov |
| NCT02629991 | Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome | Phase 2 | 23 | 2021-10-07 | Prader-Willi Syndrome, Hyperphagia | OXYTOCIN | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 56
See all 56 trialsComparisons in its trials25
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 |
|---|---|---|---|---|---|
| DCCR vs Placebo for DCCR: deaths from any cause | 0 of 84 | 0 of 42 | 126 | NCT03440814 | Trial comparisons |
| DCCR vs Placebo for DCCR: serious adverse events | 6 of 84 | 0 of 42 | 126 | NCT03440814 | Trial comparisons |
Setmelanotide 1.5 mg (Open-Label Extension) vs Setmelanotide 2.5 mg (Open-Label Extension): Number of Participants With Clinically Significant Percent Change From Baseline in Body Mass Measured Using DEXA - Period 4 | 0 of 19 | 0 of 20 | 39 | NCT02311673 | Trial comparisons |
Setmelanotide 1.5 mg (Open-Label Extension) vs Setmelanotide 2.5 mg (Open-Label Extension): Number of Participants With Clinically Significant Percent Change From Baseline in Body Fat Measured Using DEXA - Period 4 | 0 of 19 | 0 of 20 | 39 | NCT02311673 | Trial comparisons |
| Carbetocin (FE 992097) vs Placebo: deaths from any cause | 0 of 17 | 0 of 20 | 37 | NCT01968187 | Trial comparisons |
| Carbetocin (FE 992097) vs Placebo: serious adverse events | 0 of 17 | 0 of 20 | 37 | NCT01968187 | Trial comparisons |
| Experimental: Intranasal Oxytocin (IN-OXT) vs Placebo Comparator: Matched Placebo: deaths from any cause | 0 of 15 | 0 of 17 | 32 | NCT03197662 | Trial comparisons |
| Experimental: Intranasal Oxytocin (IN-OXT) vs Placebo Comparator: Matched Placebo: serious adverse events | 0 of 15 | 0 of 17 | 32 | NCT03197662 | Trial comparisons |
| Intranasal Oxytocin (IN-OXT) vs Matched Placebo: deaths from any cause | 0 of 11 | 0 of 12 | 23 | NCT02629991 | Trial comparisons |
| Intranasal Oxytocin (IN-OXT) vs Matched Placebo: serious adverse events | 0 of 11 | 0 of 12 | 23 | NCT02629991 | Trial comparisons |
1 to 10 of 25
See all 25 comparisons in its trialsPapers from its trials29
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
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_0008300
GET https://conductscience.org/api/diseases/v1/MONDO_0008300/drugs?page=2