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Table 1 Double-blind, placebo-controlled treatment studies of individuals with fragile X

From: Updated report on tools to measure outcomes of clinical trials in fragile X syndrome

FDA register number

Compound

Mechanism

Cohort (years)

Sample size

Outcomes measured (OM)

Phase/sponsor

Results/publications

     

Primary1

Secondary2

  
  

14/22, 64%

      

NCT00788073

Arbaclofen (STX209)

GABA-B agonist

6–50

63

ABC-C_I

ABC-CFX, CGI-S, CGI-I, Vineland-II, SRS, RBANS, SB5, PPVT

II/Seaside

OM:1 not met, 2↓ABC-CFX-SA [112]

NCT01282268

Arbaclofen

GABA-B agonist

12–50

125

ABC-CFX_SA

CGI-S, CGI-I, Vineland-II, ADHD-RS, SB5, PSI, CSHQ

III/Seaside

OM:1 not met [46]

NCT01325220

Arbaclofen

GABA-B agonist

5–11

172

ABC-CFX_SA

CGI-S, CGI-I, Vineland-II, ADHD-RS, SB5, PSI, CSHQ

III/Seaside

OM:1 not met, 2↓ABC-CFX-I, ↓PSI [46, 113]

NCT01725152

Ganaxolone

GABA-A agonist

6–17

60

CGI-I

ABC-CFX, ADAMS, PPI, KiTAP, ERP, ET, SNAP-IV, PARS

II/Marinus

Pending

NCT02126995

Metadoxine

GABA transporter

14–50

62

ADHD-RS

ABC-CFX, CGI-S, CGI-I, Vineland-II, SRS, KiTAP, ERP, ET, PARS

II/Alcobra

OM:1 not met, 2↑Vineland II DL [44, 64]

NCT00718341

Mavoglurant (AFQ056)

mGluR5 antagonist

18–35

30

ABC-C_T

ABC-CFX, CGI-S, CGI-I, SRS, KiTAP, ET, VAS, RBANS..

II/Novartis

OM:1 not met,2 ↓ABC-C_T (n=7 FM) [261]

NCT01253629

Mavoglurant

mGluR5 antagonist

18–45

175

ABC-C_T

ABC-C, CGI-S, CGI-I, SRS, KiTAP

IIb/Novartis

OM:1 & 2 not met, tolerated well [9]

NCT01357239

Mavoglurant

mGluR5 antagonist

12–17

139

ABC-C_T

ABC-C, CGI-S, CGI-I, SRS, KiTAP

IIb/Novartis

OM:1 & 2 not met, tolerated well [9]

NCT01015430

Basimglurant (RO4917523)

mGluR5 antagonist

18–50

40

Safety, PK

ABC-C, ADAMS, CGI-S, CGI-I, KiTAP, ET, VAS, RBANS

IIa/Roche

Not in public domain

NCT01517698

Basimglurant

mGluR5 antagonist

14–40

185

ADAMS_T

ABC-C, ADAMS, CGI-S, CGI-I, WISC-IV, CBI-M

IIb/Roche

Not in public domain

NCT01750957

Basimglurant

mGluR5 antagonist

5–13

180

Safety

PK, ABC-C, ADAMS, CGI-S, CGI-I, WISC-IV, CBI-M

IIb/Roche

Not in public domain

NCT01911455

Acamprosate

GABA/Glutamate

5–23

48

ABC-C_SW

CGI-S, CGI-I, VAS, ABC, PARS, ADAMS, CSQ, ELS, SRS

II/III Cinn Child

Pending

NCT01894958

Trofinetide

IGF-1 analog

14–40

70

Safety

CGI-S, CGI-I, ABC-CFX, SRS, FXS scale, VAS-P/C, KiTAP

II/Neuren

OM: 1Tolerated well, 2-several improved [63]

NCT00054730

CX516

Ampakine

18+

49

Memory

CGI-I, multiple Z scores, SNAP-IV

II/Cortex

OM: 1 Tolerated well,2↑CGI-I in a subset [40]

NCT01053156

Minocycline

MMP-9

3–16

66

CGI-I,VAS

VAS-mb, ABC-C, Vineland-II, EVT-2

II/UC Davis

OM:1 ↑CGI-I, 2↑VAS, ↓MMP-9; [87]

NCT01474746

Sertraline

SSRI

2–6

30

CGI-I,VAS

Vineland-II, PSI-4, PLS-5, MSEL

II/UC Davis

OM:1↑CGI-I (46%),2 ↑VAS [262]

NCT01120626

Donepezil

Cholinergic drug

12–29

45

ABC-C_T/CNT

MRS, WM tests, CARS

II/Stanford

Pending

NCT01254945

Oxytocin

Brain peptide

13–28

8

Eye gaze

HR, RSA, HRV, cortisol

Ib/Stanford

OM:1↑Eye gaze, 2↓cortisol [77, 263]

NCT01329770

Vitamins C & E

Antioxidants

6–18

30

DBC, WISC

Tolerability

II/Imabis

Pending [264]

N/A

Melatonin

Hormone

2–15

15

Sleep times

Actiwatch

II/UC Davis

OM:1↑sleep; [265]

N/A

L-AcCarnitine

Brain energy

6–13

63

ConnersGI-P

Vineland-Survey, WISC-R

II/TELETHON

OM:1↓HI/I, 2↑Vineland [266]

N/A

MPH/DAMP

Stimulants

3–11

15

ConnersAbb

ACTeRS, IQ measures

II/Denver Child

OM:1MPH:↓I, 2↓ACTeRS; [267]

  1. Modified from Table 1, Budimirovic and Phan [268] with permission
  2. Abbreviations: FDA Federal Drug Administration, GABA gamma-aminobutyric acid, ABC-C_I Aberrant Behavior Checklist-Community Edition (ABC-C), Irritability subscale, CGI-S Clinician Global Impression-Severity, CGI-I CGI improvement, VABS-II Vineland Adaptive Behavior Scale, Second Edition, SRS Social Responsiveness Scale, RBANS Repeatable Battery for the Assessment of Neurological Status, SB5 Stanford-Binet Version 5, ABC-C FX _SA ABC-CFX, Social Avoidance subscale, PSI Parenting Stress Index, CSHQ Children’s Sleep Habits Questionnaire, PPVT Peabody Picture Vocabulary Test, ADHD-RS Attention-Deficit Hyperactivity Disorder Rating Scale, ABC-C FX ABC-C, FXS-specific, ADAMS Anxiety Depression and Mood Scale, PPI prepulse inhibition, KiTAP Tests of Attentional Performance for Children, ERP evoked response potentials, ET eye tracking, SNAP-IV Swanson, Nolan and Pelhram Rating Scale, PARS Pediatric Anxiety Rating Scale, ABC-C_T ABC-C total score, ADAMS_T ADAMS total score, WISC-IV Wechsler Intelligence Scale for Children, 4th ed, CBI-M Caregiver Burden Inventory-Modified, MMP9 matrix metallopeptidase 9, VAS visual analog scale (VAS-C, clinician rated; VAS-mb, multiple behaviors; VAS-P, parent rated), mGluR5antagonist metabotropic glutamate receptor 1/5 antagonist, GABA/Glutamate GABA/glutamate normalizer, ABC-C_SW ABC-C, Lethargy/Social Withdrawal subscale, CSQ Caregiver Strain Questionnaire, ELS Expressive Language Sampling, Trofinetide Neu-2566-FXS-001 study, IGF-1 insulin-like growth factor, PLS-5 Preschool Language Scale-Version 5, MSEL Mullen Scales of Early Learning, CNT Contingency Naming Task, MRS magnetic resonance spectroscopy, WM working memory tests, CARS Childhood Autism Rating Scale, HR heart rate, RSA respiratory sinus arrhythmia, HRV heart rate variability, SSRI selective serotonin reuptake inhibitor, DBC Developmental Behavior Checklist, EVT-2 Expressive Vocabulary Test, 2nd ed, PK pharmacokinetics, ConnersGI-P Conners General Index-Parent Version, WISC-R WISC-Revised, MPH methylphenidate, DAMP dextroamphetamine, Conners Abb Conners Abbreviated Version, ACTeRS ADD-H Comprehensive Teacher’s Rating Scales, IQ intellectual quotient
  3. 1reflects a primary outcome measure (OM)
  4. 2reflects a secondary outcome measure (OM)
  5. Italized under Mechanism are clinical trials targeting excitatory-inhibitory imbalance in FXS
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Journal of Neurodevelopmental Disorders

ISSN: 1866-1955

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