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Volumn 9, Issue 1, 2017, Pages

A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome

Author keywords

Adolescents; Children; Clinical trial; Fragile X syndrome; Ganaxolone

Indexed keywords

GANAXOLONE; PLACEBO;

EID: 85026675364     PISSN: 18661947     EISSN: 18661955     Source Type: Journal    
DOI: 10.1186/s11689-017-9207-8     Document Type: Article
Times cited : (60)

References (43)
  • 1
    • 84871373139 scopus 로고    scopus 로고
    • FMR1 CGG allele size and prevalence ascertained through newborn screening in the United States
    • 1:CAS:528:DC%2BC3sXktV2hsLo%3D 23259642 4064316
    • Tassone F, et al. FMR1 CGG allele size and prevalence ascertained through newborn screening in the United States. Genome Med. 2012;4(12):100.
    • (2012) Genome Med , vol.4 , Issue.12 , pp. 100
    • Tassone, F.1
  • 2
    • 84905215735 scopus 로고    scopus 로고
    • Longitudinal profiles of adaptive behavior in fragile X syndrome
    • 25070318 4187230
    • Klaiman C, et al. Longitudinal profiles of adaptive behavior in fragile X syndrome. Pediatrics. 2014;134(2):315-24.
    • (2014) Pediatrics , vol.134 , Issue.2 , pp. 315-324
    • Klaiman, C.1
  • 3
    • 0027176361 scopus 로고
    • The FMR-1 protein is cytoplasmic, most abundant in neurons and appears normal in carriers of a fragile X premutation
    • 1:CAS:528:DyaK3sXlsFOlsL8%3D 8401578
    • Devys D, et al. The FMR-1 protein is cytoplasmic, most abundant in neurons and appears normal in carriers of a fragile X premutation. Nat Genet. 1993;4(4):335-40.
    • (1993) Nat Genet , vol.4 , Issue.4 , pp. 335-340
    • Devys, D.1
  • 4
    • 84888788450 scopus 로고    scopus 로고
    • The FMRP regulon: From targets to disease convergence
    • 24167470 3807044
    • Fernandez E, Rajan N, Bagni C. The FMRP regulon: from targets to disease convergence. Front Neurosci. 2013;7:191.
    • (2013) Front Neurosci , vol.7 , pp. 191
    • Fernandez, E.1    Rajan, N.2    Bagni, C.3
  • 5
    • 3042647610 scopus 로고    scopus 로고
    • The mGluR theory of fragile X mental retardation
    • 1:CAS:528:DC%2BD2cXlt1ans7w%3D 15219735
    • Bear MF, Huber KM, Warren ST. The mGluR theory of fragile X mental retardation. Trends Neurosci. 2004;27(7):370-7.
    • (2004) Trends Neurosci , vol.27 , Issue.7 , pp. 370-377
    • Bear, M.F.1    Huber, K.M.2    Warren, S.T.3
  • 6
    • 84937630810 scopus 로고    scopus 로고
    • Therapeutic strategies in fragile X syndrome: From bench to bedside and back
    • 1:CAS:528:DC%2BC2MXovVerurs%3D 25986746 4489963
    • Gross C, et al. Therapeutic strategies in fragile X syndrome: from bench to bedside and back. Neurotherapeutics. 2015;12(3):584-608.
    • (2015) Neurotherapeutics , vol.12 , Issue.3 , pp. 584-608
    • Gross, C.1
  • 7
    • 78650937072 scopus 로고    scopus 로고
    • Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056
    • 1:CAS:528:DC%2BC3MXhsFWisbw%3D 21209411
    • Jacquemont S, et al. Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056. Sci Transl Med. 2011;3(64):64ra1.
    • (2011) Sci Transl Med , vol.3 , Issue.64 , pp. 64ra1
    • Jacquemont, S.1
  • 8
    • 84954289367 scopus 로고    scopus 로고
    • Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials
    • 26764156
    • Berry-Kravis E, et al. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016;8(321):321ra5.
    • (2016) Sci Transl Med , vol.8 , Issue.321 , pp. 321ra5
    • Berry-Kravis, E.1
  • 9
    • 85026641212 scopus 로고    scopus 로고
    • Effect of two doses of Basimglurant on behavioral symptoms in adolescent & adult patients with fragile X syndrome: Results from FRAGXIS, a double-blind, placebo controlled study
    • Salt Lake City
    • Quiroz J, et al. Effect of two doses of Basimglurant on behavioral symptoms in adolescent & adult patients with fragile X syndrome: results from FRAGXIS, a double-blind, placebo controlled study, in International Meeting for Autism Research. Salt Lake City;2015
    • (2015) International Meeting for Autism Research
    • Quiroz, J.1
  • 10
    • 85026677281 scopus 로고    scopus 로고
    • Safety and exploratory efficacy of Basimglurant in pediatric patients with fragile X syndrome: A randomized, double-blind, placebo-controlled study
    • Salt Lake City
    • Quiroz J, et al. Safety and exploratory efficacy of Basimglurant in pediatric patients with fragile X syndrome: a randomized, double-blind, placebo-controlled study, in International Meeting for Autism Research. Salt Lake City; 2015
    • (2015) International Meeting for Autism Research
    • Quiroz, J.1
  • 11
    • 69849090240 scopus 로고    scopus 로고
    • Fragile X syndrome: From molecular genetics to therapy
    • 19724010
    • D'Hulst C, Kooy RF. Fragile X syndrome: from molecular genetics to therapy. J Med Genet. 2009;46(9):577-84.
    • (2009) J Med Genet , vol.46 , Issue.9 , pp. 577-584
    • D'Hulst, C.1    Kooy, R.F.2
  • 12
    • 84899579632 scopus 로고    scopus 로고
    • Fragile X syndrome neurobiology translates into rational therapy
    • 24508819
    • Braat S, Kooy RF. Fragile X syndrome neurobiology translates into rational therapy. Drug Discov Today. 2014;19(4):510-9.
    • (2014) Drug Discov Today , vol.19 , Issue.4 , pp. 510-519
    • Braat, S.1    Kooy, R.F.2
  • 13
    • 84930531864 scopus 로고    scopus 로고
    • A receptor as a therapeutic target for neurodevelopmental disorders
    • 1:CAS:528:DC%2BC2MXhtVejurjK 26050032
    • A receptor as a therapeutic target for neurodevelopmental disorders. Neuron. 2015;86(5):1119-30.
    • (2015) Neuron , vol.86 , Issue.5 , pp. 1119-1130
    • Braat, S.1    Kooy, R.F.2
  • 14
    • 84942036668 scopus 로고    scopus 로고
    • A receptors in the brain of fragile X patients
    • 26222316 4519313
    • A receptors in the brain of fragile X patients. PLoS One. 2015;10(7):e0131486.
    • (2015) PLoS One , vol.10 , Issue.7 , pp. e0131486
    • D'Hulst, C.1
  • 15
    • 84942021759 scopus 로고    scopus 로고
    • The GABAA receptor is an FMRP target with therapeutic potential in fragile X syndrome
    • 1:CAS:528:DC%2BC2MXhslGlurfJ 25790165 4827888
    • Braat S, et al. The GABAA receptor is an FMRP target with therapeutic potential in fragile X syndrome. Cell Cycle. 2015;14(18):2985-95.
    • (2015) Cell Cycle , vol.14 , Issue.18 , pp. 2985-2995
    • Braat, S.1
  • 16
    • 84908384229 scopus 로고    scopus 로고
    • Insights into GABAAergic system deficits in fragile X syndrome lead to clinical trials
    • 1:CAS:528:DC%2BC2cXhsV2ntbvM 25016041
    • Braat S, Kooy RF. Insights into GABAAergic system deficits in fragile X syndrome lead to clinical trials. Neuropharmacology. 2015;88:48-54.
    • (2015) Neuropharmacology , vol.88 , pp. 48-54
    • Braat, S.1    Kooy, R.F.2
  • 17
    • 34447621203 scopus 로고    scopus 로고
    • A receptor: A novel target for treatment of fragile X?
    • 17590448
    • A receptor: a novel target for treatment of fragile X? Trends Neurosci. 2007;30(8):425-31.
    • (2007) Trends Neurosci , vol.30 , Issue.8 , pp. 425-431
    • D'Hulst, C.1    Kooy, R.F.2
  • 18
    • 58249135425 scopus 로고    scopus 로고
    • Expression of the GABAergic system in animal models for fragile X syndrome and fragile X associated tremor/ataxia syndrome (FXTAS)
    • 19070606
    • D'Hulst C, et al. Expression of the GABAergic system in animal models for fragile X syndrome and fragile X associated tremor/ataxia syndrome (FXTAS). Brain Res. 2009;1253:176-83.
    • (2009) Brain Res , vol.1253 , pp. 176-183
    • D'Hulst, C.1
  • 19
    • 30744433092 scopus 로고    scopus 로고
    • Expression profiling suggests underexpression of the GABA(A) receptor subunit delta in the fragile X knockout mouse model
    • 1:CAS:528:DC%2BD28XmsVGntw%3D%3D 16199166
    • Gantois I, et al. Expression profiling suggests underexpression of the GABA(A) receptor subunit delta in the fragile X knockout mouse model. Neurobiol Dis. 2006;21(2):346-57.
    • (2006) Neurobiol Dis , vol.21 , Issue.2 , pp. 346-357
    • Gantois, I.1
  • 20
    • 33750726094 scopus 로고    scopus 로고
    • Decreased expression of the GABAA receptor in fragile X syndrome
    • 17046729
    • D'Hulst C, et al. Decreased expression of the GABAA receptor in fragile X syndrome. Brain Res. 2006;1121(1):238-45.
    • (2006) Brain Res , vol.1121 , Issue.1 , pp. 238-245
    • D'Hulst, C.1
  • 21
    • 84856578911 scopus 로고    scopus 로고
    • Pharmacological treatment of fragile X syndrome with GABAergic drugs in a knockout mouse model
    • 1:CAS:528:DC%2BC38Xjs1antb0%3D 22285772
    • Heulens I, et al. Pharmacological treatment of fragile X syndrome with GABAergic drugs in a knockout mouse model. Behav Brain Res. 2012;229(1):244-9.
    • (2012) Behav Brain Res , vol.229 , Issue.1 , pp. 244-249
    • Heulens, I.1
  • 22
    • 77954851827 scopus 로고    scopus 로고
    • Defective GABAergic neurotransmission and pharmacological rescue of neuronal hyperexcitability in the amygdala in a mouse model of fragile X syndrome
    • 1:CAS:528:DC%2BC3cXpsl2isrk%3D 20660275 2948869
    • Olmos-Serrano JL, et al. Defective GABAergic neurotransmission and pharmacological rescue of neuronal hyperexcitability in the amygdala in a mouse model of fragile X syndrome. J Neurosci. 2010;30(29):9929-38.
    • (2010) J Neurosci , vol.30 , Issue.29 , pp. 9929-9938
    • Olmos-Serrano, J.L.1
  • 23
    • 83055184199 scopus 로고    scopus 로고
    • The GABA(A) receptor agonist THIP ameliorates specific behavioral deficits in the mouse model of fragile X syndrome
    • 1:CAS:528:DC%2BC3MXhs1GktbrJ 22067669 3254038
    • Olmos-Serrano JL, Corbin JG, Burns MP. The GABA(A) receptor agonist THIP ameliorates specific behavioral deficits in the mouse model of fragile X syndrome. Dev Neurosci. 2011;33(5):395-403.
    • (2011) Dev Neurosci , vol.33 , Issue.5 , pp. 395-403
    • Olmos-Serrano, J.L.1    Corbin, J.G.2    Burns, M.P.3
  • 24
    • 14444270606 scopus 로고    scopus 로고
    • Characterization of the anticonvulsant properties of ganaxolone (CCD 1042; 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one), a selective, high-affinity, steroid modulator of the gamma-aminobutyric acid(A) receptor
    • 1:CAS:528:DyaK2sXhvF2mtLY%3D 9067315
    • Carter RB, et al. Characterization of the anticonvulsant properties of ganaxolone (CCD 1042; 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one), a selective, high-affinity, steroid modulator of the gamma-aminobutyric acid(A) receptor. J Pharmacol Exp Ther. 1997;280(3):1284-95.
    • (1997) J Pharmacol Exp Ther , vol.280 , Issue.3 , pp. 1284-1295
    • Carter, R.B.1
  • 25
    • 84891784115 scopus 로고    scopus 로고
    • Neurosteroid interactions with synaptic and extrasynaptic GABA(A) receptors: Regulation of subunit plasticity, phasic and tonic inhibition, and neuronal network excitability
    • 1:CAS:528:DC%2BC3sXhsFaqs73K
    • Carver CM, Reddy DS. Neurosteroid interactions with synaptic and extrasynaptic GABA(A) receptors: regulation of subunit plasticity, phasic and tonic inhibition, and neuronal network excitability. Psychopharmacology (Berl). 2013;230(2):151-88.
    • (2013) Psychopharmacology (Berl) , vol.230 , Issue.2 , pp. 151-188
    • Carver, C.M.1    Reddy, D.S.2
  • 26
    • 33846213568 scopus 로고    scopus 로고
    • Ganaxolone
    • 1:CAS:528:DC%2BD2sXisFaksLY%3D 17199022
    • Nohria V, Giller E. Ganaxolone. Neurotherapeutics. 2007;4(1):102-5.
    • (2007) Neurotherapeutics , vol.4 , Issue.1 , pp. 102-105
    • Nohria, V.1    Giller, E.2
  • 27
    • 0030869099 scopus 로고    scopus 로고
    • Initial human experience with ganaxolone, a neuroactive steroid with antiepileptic activity
    • 1:CAS:528:DyaK2sXmt1KksbY%3D 9579942
    • Monaghan EP, et al. Initial human experience with ganaxolone, a neuroactive steroid with antiepileptic activity. Epilepsia. 1997;38(9):1026-31.
    • (1997) Epilepsia , vol.38 , Issue.9 , pp. 1026-1031
    • Monaghan, E.P.1
  • 28
    • 0031888527 scopus 로고    scopus 로고
    • Reversal of behavioral effects of pentylenetetrazol by the neuroactive steroid ganaxolone
    • 1:CAS:528:DyaK1cXhs1yhurk%3D 9495844
    • Beekman M, et al. Reversal of behavioral effects of pentylenetetrazol by the neuroactive steroid ganaxolone. J Pharmacol Exp Ther. 1998;284(3):868-77.
    • (1998) J Pharmacol Exp Ther , vol.284 , Issue.3 , pp. 868-877
    • Beekman, M.1
  • 29
    • 35148899318 scopus 로고    scopus 로고
    • Clinical evaluation of ganaxolone in pediatric and adolescent patients with refractory epilepsy
    • 1:CAS:528:DC%2BD2sXht12rs7zN 17634060
    • Pieribone VA, et al. Clinical evaluation of ganaxolone in pediatric and adolescent patients with refractory epilepsy. Epilepsia. 2007;48(10):1870-4.
    • (2007) Epilepsia , vol.48 , Issue.10 , pp. 1870-1874
    • Pieribone, V.A.1
  • 30
    • 0033759542 scopus 로고    scopus 로고
    • Ganaxolone for treating intractable infantile spasms: A multicenter, open-label, add-on trial
    • 1:CAS:528:DC%2BD3cXoslGmtLs%3D 11074186
    • Kerrigan JF, et al. Ganaxolone for treating intractable infantile spasms: a multicenter, open-label, add-on trial. Epilepsy Res. 2000;42(2-3):133-9.
    • (2000) Epilepsy Res , vol.42 , Issue.2-3 , pp. 133-139
    • Kerrigan, J.F.1
  • 31
    • 39749156744 scopus 로고
    • The Snijders-Oomen nonverbal intelligence tests. General intelligence tests or tests for learning potential?
    • K. JS Hamers A.J.J.M. Ruijssenaar (eds) Swets & Zeitlinger B.V Amsterdam/Lisse
    • Tellegen PJ, Laros JA. The Snijders-Oomen nonverbal intelligence tests. General intelligence tests or tests for learning potential? In: JS Hamers K, Ruijssenaar AJJM, editors. Learning potential assessment: theoretical, methodological and practical issues. Amsterdam/Lisse: Swets & Zeitlinger B.V; 1993. p. 267-83.
    • (1993) Learning Potential Assessment: Theoretical, Methodological and Practical Issues , pp. 267-283
    • Tellegen, P.J.1    Laros, J.A.2
  • 32
    • 0036718715 scopus 로고    scopus 로고
    • The Pediatric Anxiety Rating Scale (PARS): Development and psychometric properties
    • (RUPP), the Research Units on Pediatric Psychopharmacology Anxiety Study Group
    • (RUPP), the Research Units on Pediatric Psychopharmacology Anxiety Study Group. The Pediatric Anxiety Rating Scale (PARS): development and psychometric properties. J Am Acad Child Adolesc Psychiatry. 2002;41(9):1061-9.
    • (2002) J Am Acad Child Adolesc Psychiatry , vol.41 , Issue.9 , pp. 1061-1069
  • 33
    • 0346787627 scopus 로고    scopus 로고
    • Reliability and validity of an assessment instrument for anxiety, depression, and mood among individuals with mental retardation
    • 14714931
    • Esbensen AJ, et al. Reliability and validity of an assessment instrument for anxiety, depression, and mood among individuals with mental retardation. J Autism Dev Disord. 2003;33(6):617-29.
    • (2003) J Autism Dev Disord , vol.33 , Issue.6 , pp. 617-629
    • Esbensen, A.J.1
  • 34
    • 84863875429 scopus 로고    scopus 로고
    • Psychometric study of the aberrant behavior checklist in fragile X syndrome and implications for targeted treatment
    • 21972117 3290710
    • Sansone SM, et al. Psychometric study of the aberrant behavior checklist in fragile X syndrome and implications for targeted treatment. J Autism Dev Disord. 2012;42(7):1377-92.
    • (2012) J Autism Dev Disord , vol.42 , Issue.7 , pp. 1377-1392
    • Sansone, S.M.1
  • 35
    • 0035141470 scopus 로고    scopus 로고
    • Clinical relevance of the primary findings of the MTA: Success rates based on severity of ADHD and ODD symptoms at the end of treatment
    • 1:STN:280:DC%2BD3M7ktFCntg%3D%3D 11211365
    • Swanson JM, et al. Clinical relevance of the primary findings of the MTA: success rates based on severity of ADHD and ODD symptoms at the end of treatment. J Am Acad Child Adolesc Psychiatry. 2001;40(2):168-79.
    • (2001) J Am Acad Child Adolesc Psychiatry , vol.40 , Issue.2 , pp. 168-179
    • Swanson, J.M.1
  • 36
    • 84932103949 scopus 로고    scopus 로고
    • Developmental changes in cognitive and behavioural functioning of adolescents with fragile-X syndrome
    • 1:STN:280:DC%2BC2M%2FltlGhug%3D%3D 25160119
    • Frolli A, Piscopo S, Conson M. Developmental changes in cognitive and behavioural functioning of adolescents with fragile-X syndrome. J Intellect Disabil Res. 2015;59(7):613-21.
    • (2015) J Intellect Disabil Res , vol.59 , Issue.7 , pp. 613-621
    • Frolli, A.1    Piscopo, S.2    Conson, M.3
  • 37
    • 79952316385 scopus 로고    scopus 로고
    • Reduced GABAA receptors and benzodiazepine binding sites in the posterior cingulate cortex and fusiform gyrus in autism
    • 1:CAS:528:DC%2BC3MXisFGnu74%3D 20858465
    • Oblak AL, Gibbs TT, Blatt GJ. Reduced GABAA receptors and benzodiazepine binding sites in the posterior cingulate cortex and fusiform gyrus in autism. Brain Res. 2011;1380:218-28.
    • (2011) Brain Res , vol.1380 , pp. 218-228
    • Oblak, A.L.1    Gibbs, T.T.2    Blatt, G.J.3
  • 38
    • 58549113548 scopus 로고    scopus 로고
    • GABA(A) receptor downregulation in brains of subjects with autism
    • 18821008
    • Fatemi SH, et al. GABA(A) receptor downregulation in brains of subjects with autism. J Autism Dev Disord. 2009;39(2):223-30.
    • (2009) J Autism Dev Disord , vol.39 , Issue.2 , pp. 223-230
    • Fatemi, S.H.1
  • 39
    • 84952895502 scopus 로고    scopus 로고
    • Evaluation of the neuroactive steroid ganaxolone on social and repetitive behaviors in the BTBR mouse model of autism
    • 1:CAS:528:DC%2BC2MXhslOnsLjI
    • Kazdoba TM, et al. Evaluation of the neuroactive steroid ganaxolone on social and repetitive behaviors in the BTBR mouse model of autism. Psychopharmacology (Berl). 2016;233(2):309-23.
    • (2016) Psychopharmacology (Berl) , vol.233 , Issue.2 , pp. 309-323
    • Kazdoba, T.M.1
  • 40
    • 84925463449 scopus 로고    scopus 로고
    • Use of emotional cues for lexical learning: A comparison of autism spectrum disorder and fragile X syndrome
    • 25318904 4369166
    • Thurman AJ, et al. Use of emotional cues for lexical learning: a comparison of autism spectrum disorder and fragile X syndrome. J Autism Dev Disord. 2015;45(4):1042-61.
    • (2015) J Autism Dev Disord , vol.45 , Issue.4 , pp. 1042-1061
    • Thurman, A.J.1
  • 41
    • 84931560678 scopus 로고    scopus 로고
    • Symptoms of autism in males with fragile X syndrome: A comparison to nonsyndromic ASD using current ADI-R scores
    • 24414079 4096070
    • McDuffie A, et al. Symptoms of autism in males with fragile X syndrome: a comparison to nonsyndromic ASD using current ADI-R scores. J Autism Dev Disord. 2015;45(7):1925-37.
    • (2015) J Autism Dev Disord , vol.45 , Issue.7 , pp. 1925-1937
    • McDuffie, A.1
  • 42
    • 84927609960 scopus 로고    scopus 로고
    • Effect of speaker gaze on word learning in fragile X syndrome: A comparison with nonsyndromic autism spectrum disorder
    • 25629603 4675125
    • Benjamin DP, et al. Effect of speaker gaze on word learning in fragile X syndrome: a comparison with nonsyndromic autism spectrum disorder. J Speech Lang Hear Res. 2015;58(2):383-95.
    • (2015) J Speech Lang Hear Res , vol.58 , Issue.2 , pp. 383-395
    • Benjamin, D.P.1
  • 43
    • 84897106498 scopus 로고    scopus 로고
    • Psychiatric symptoms in boys with fragile X syndrome: A comparison with nonsyndromic autism spectrum disorder
    • 24629733 4009990
    • Thurman AJ, et al. Psychiatric symptoms in boys with fragile X syndrome: a comparison with nonsyndromic autism spectrum disorder. Res Dev Disabil. 2014;35(5):1072-86.
    • (2014) Res Dev Disabil , vol.35 , Issue.5 , pp. 1072-1086
    • Thurman, A.J.1


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