. 2019 Mar 14;13:344–358. doi: 10.1016/j.omtm.2019.03.001

Table 3.

Compounds Tested in Humans with PWS but Not in Preclinical Models of PWS

Type of Drug	Compound/Procedure	Clinical Trials in PWS	Company/Trial Center	Outcomes	Clinical Trial Registration
Growth hormone	growth hormone FDA approved	prescribed as needed	Karolinska University Hospital; Novo Nordisk	body composition; linear growth; bone mineral density; cognitive and adaptive function	NCT00372125 NCT00705172
Stimulant	modafinil FDA approved	case series, open label ¹⁶¹	Hôpital Purpan, Toulouse, France	sleepiness	N/A
Synthetic somatostatin	octreotide FDA approved	phase II ¹⁶²	British Columbia’s Children’s Hospital	BMI; appetite; behavior; ghrelin concentration	NCT00175305
Anticonvulsant	topiramate FDA approved	phase II phase III	University of Florida-Brain Institute Hopitaux de Paris	self-injurious behavior eating disorders; self-mutilation; irritability and impulsivity; metabolic status	NCT00065923 NCT02810483
Aromatase Inhibitor	anastrozole FDA approved	phase II	Hôpital Armand Trousseau, Paris	bone maturation related to pathological adrenarche	NCT01520467
Serotonin-noradrenaline-dopamine reuptake inhibitor/beta-blocker	tesomet (tesofensine and metoprolol) Investigational/FDA approved	phase II	Saniona	body weight	NCT03149445 EudraCT:2016-003694-18
Ghrelin pathway	GLWL-01 investigational	phase II	GLWL Research	hyperphagia, HQ-CT score	NCT03274856
Methionine aminopeptidase 2 inhibitor	beloranib not approved	phase II phase III ¹⁵¹	Zafgen	HQ-CT; weight; fat and lean; QOL	NCT01818921 NCT02179151 EudraCT:2015-000660-33
Probiotic	Bifidobacterium lactis B94 (probiotic) supplement	phase II	University of Florida, Gainesville	stool frequency	NCT03277157
Brain stimulation	vagal nerve stimulation FDA approved	exploratory	University of Cambridge ¹⁶³	behavior	N/A
Brain stimulation	deep brain stimulation FDA approved	phase I	Federal University of São Paulo	weight	NCT02297022
Brain stimulation	transcranial brain stimulation FDA approved	exploratory ¹⁶⁴ phase II	University Kansas Medical Center Federal University of São Paulo Laval, Canada	hyperphagia; depression food cravings questionnaire; brain activity	NCT01863017 NCT03324906 NCT02758262

Examples of therapeutics in development not yet studied in PWS: Pitolisant (Wakix), ZGN-1061. Examples of therapeutics in PWS clinical studies but not controlled trials: ketogenic diet, tofogliflozin (SGLT2 inhibitor), metformin, naltrexone/bupropion (Contrave), N-acetylcysteine, risperidone. N/A, not applicable; HQ-CT, Hyperphagia Questionnaire for Clinical Trials; QOL, quality of life.