Table 2 Novel symptomatic pharmaceuticals developed in clinical trials for PWS

Oxytocin

NCT02205034;

Phase 1, 2

Improve suckling in PWS infants; reduce appetite drive, improve social skills, and decrease disruptive behaviors in PWS children [43, 44]

NCT02013258

Phase 1

NCT02804373

Phase 2, 3

Oxytocin analogue (Carbetocin)

NCT03649477

Phase 3

Improve hyperphagia and behavioral symptoms [45]

K⁺-ATP channel agonists (Diazoxide, DCCR)

NCT03440814

Phase 3

Ameliorate hyperphagia, improve lipids and insulin resistance, reduce aggressive behaviors [46]

NCT02034071

Phase 1, 2

NCT03714373

Phase 3

UAG analogue (AZP-531)

NCT03790865

Phase 2,3

Improve hyperphagia and metabolic parameters [47]

GLP-1 receptor agonists (Liraglutide, Exenatide)

NCT02527200

Phase 3

Improve hyperphagia [48]

NCT01444898

Not applicable

NCT00551343

Not applicable

MetAP2 inhibitor (ZGN-440)

NCT01818921

Phase 2

Reduce body weight and improve hyperphagia-related behaviors [49]

MC4R agonist (Setmelanotide)

NCT02311673

Phase 2

Improve hyperphagia and result in weight loss [50, 51]

GOAT inhibitors (GLWL 01)

NCT03274856

Phase 2

Reduce food intake [52]

CB1R antagonists (JD5037, CBD)

NCT02844933

Phase 2

Suppresses appetite, improve metabolic issues, increase energy expenditure [53, 54]

NCT03458416

Phase 2

NCT05098509

Phase 2,3

Antiepileptics (Topiramate)

NCT02810483

Phase 3

Attenuates self-injurious behaviors, correct eating behaviors, control body weight [55, 56]

NCT00065923

Not applicable