Table 2.
Categories of Compounds Tested in Both Preclinical (Mouse) Models of PWS and in Humans with PWS
| Type of Drug | Compound | Mouse Model of PWS: Reference | Clinical Trials in PWS | Company/Trial Center | Outcomes | NCT or EudraCT |
|---|---|---|---|---|---|---|
| Neuropeptide hormones | oxytocin (examples only) FDA approved |
Magel2: 5 Magel2: 29 |
phase II phase III, 133,135,136,149 |
many sites | infant suckling; food intake; hyperphagia; behavior; social behavior |
NCT03197662 NCT02205034 NCT02013258 EudraCT: 2010-022370-14 EudraCT: 2017-003423-30 |
| carbetocin/FE 992097 EU approved | not trialed | phase II phase III, 150 |
Ferring Levo Therapeutics |
hyperphagia; behavior | NCT01968187 | |
| K+-ATP channel agonist | diazoxide FDA approved |
Magel2: 38 | not trialed | |||
| DCCR/extended release diazoxide choline investigational |
not trialed | phase II phase III |
Essentialis Soleno Therapeutics |
hyperphagia; REE |
NCT02034071 NCT02893618 NCT03440814 |
|
| Melanocortin 4 receptor agonists | MT-II/Melanotan 2 not approved |
Magel2: 6 | not trialed | |||
| RM-493 or setmelanotide investigational |
Magel2: 34 | phase II | Rhythm | weight loss; hyperphagia-related behavior | NCT02311673 | |
| GLP-1 receptor agonists | exenatide (Byetta) FDA approved |
Snord116: 113 | exploratory, open label phase II 153,166 |
Children’s Hospital Los Angeles Aintree University Hospital |
weight; metabolism ghrelin levels |
NCT01444898 EudraCT: 2010-023179-25 |
| exenatide extended release (Bydureon) | not trialed | phase III | Garvan Foundation, Australia | gastric emptying | ACTRN12616000710426 | |
| liraglutide (Saxenda) | not trialed | phase III | Novo Nordisk | BMI; hyperphagia; metabolism |
NCT02527200 EudraCT: 2014-004415-37 |
|
| Cannabinoids | oleoyl α-methyl serine (HU-671) investigational |
Magel2: 37 | ||||
| JD5037 or SLV-319 (ibipinabant) research only |
Magel2: 36 | |||||
| OEA (oleoylethanolamide) supplement |
Magel2: 39 | |||||
| cannabidiol (CBD) oral solution Under review by FDA |
phase II | Insys Therapeutics | hyperphagi, weight |
NCT02844933 NCT03458416 |
||
| rimonabant (Acomplia) withdrawn |
phase II 167 | Cornell Medical College Karolinska University Hospital | obesity |
NCT00603109 EudraCT: 2007-006305-25 |
||
| Ghrelin analog | ghrelin receptor agonist HM01 | Snord116: 114 | ||||
| livoletide (AZP-531) GLWL 01 |
phase IIa 168 phase II phase II |
Alize Millendo Therapeutics GLWL Research |
blood glucose levels; weight hyperphagia hyperphagia, behavior |
EudraCT: 2014-001670-34 NCT03790865 NCT03274856 |
||
| Natural supplement | cactus extract from Caralluma fimbriata | Snord116: 165 | ||||
| cactus extract from Caralluma fimbriata | phase I 169 | Victoria University, Australia | hyperphagia; behavior | ACTRN12611000334909 | ||