Fragile X targeted pharmacotherapy: lessons learned and future directions

Table 1 Clinical trials to date in fragile X syndrome by drug and study type

Drug	Mechanism	Open Label/Chart Review	Phase II	Phase III
Acamprosate	GABA-R agonist	C[98]; C[99]	O
Arbaclofen	GABA_B-R agonist		C[102]	P
Basimglurant	mGlur5 antagonist		P
CX516	AMPAR (+) modulator		C[85]
Donepezil	Anticholinergic	C[132]	C[134]
Fenobam	mGlur5 antagonist	C[73]
Ganaxolone	GABA_AR agonist		O
Lithium	GSK3 inhibitor	C[114]
Lovastatin	ERK inhibitor	C[125]
Mavoglurant^a	mGlur5 antagonist		C[75], C[76, 106]	D
Memantine	NMDAR antagonist	C[84]
Metadoxine ER	GABA agonist		P
Minocycline	MMP9 inhibitor	C[117]	C[117]
Oxytocin	OXTR agonist		C[143]
Riluzole	Glutamate agonist	C[93]
R04917523	mGlurR5 antagonist		D
Trofinetide^b	IGF-1 mimic		P

Key: C completed and published, P completed and pending publication, O ongoing clinical trial, www.clinicaltrials.gov, D discontinued and unpublished
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ISSN: 1866-1955