Past and Present Fragile X Syndrome Clinical Trials

End Yr: Start Yr: Drug: Phase: Primary Outcome Measure: Gender: Age: Sponsor: Trial Size: NCT Link: Notes:
2018 2013 Acamprosate 1 Social avoidance – ABC-C All 5-23 Children's Hospital Medical Center, Cincinnati 46 NCT01911455 Icon: Open in a new window

n/a
2020 2016 Acamprosate, Lovastatin, Minocycline and Baclofen 1 Brain activity, clinical impression – EEG and CGI-I All 15-55 Children's Hospital Medical Center, Cincinnati 29 NCT02998151 Icon: Open in a new window

Results: Neurophysiological and Acute Pharmacological Studies in FXS Patients — Study Results
2005 2002 Ampakine CX516 (Ampalex®) 2 Memory – various measures All 18-50 RespireRx 49 NCT00054730 Icon: Open in a new window

Results have been submitted, but are not yet available. Also see: Effect of CX516, an AMPA-modulating compound, on cognition and behavior in fragile X syndrome: a controlled trial
2013 2011 Arbaclofen 3 Social avoidance – ABC-C All 12-50 Seaside Therapeutics 125 NCT01282268 Icon: Open in a new window

Results: Effects of STX209 (arbaclofen) on neurobehavioral function in children and adults with fragile X syndrome: a randomized, controlled, phase 2 trial
2013 2011 Arbaclofen 3 Lethargy, social withdrawal – ABC-C All 5-11 Seaside Therapeutics 172 NCT01325220 Icon: Open in a new window

Results: Effects of STX209 (arbaclofen) on neurobehavioral function in children and adults with fragile X syndrome: a randomized, controlled, phase 2 trial
2010 2008 Arbaclofen 2 Irritability – ABC-C All 6-40 Seaside Therapeutics 45 NCT01013480 Icon: Open in a new window

Results: Effects of STX209 (arbaclofen) on neurobehavioral function in children and adults with fragile X syndrome: a randomized, controlled, phase 2 trial
2015 2010 Ascorbic Acid and Alpha-Tocopherol 2 Hyperactivity – Conners Males 6-18 The Mediterranean Institute for the Advance of Biotechnology and Health Research 30 NCT01329770 Icon: Open in a new window

Ascorbic acid is Vitamin C and alpha-tocopherol is Vitamin E.
Results: A combination of ascorbic acid and α-tocopherol to test the effectiveness and safety in the fragile X syndrome: study protocol for a phase II, randomized, placebo-controlled trial
2020 2018 AZD7325 1 Safety – APP All 18-50 CCHMC 15 NCT03140813 Icon: Open in a new window

n/a
2014 2012 Basimglurant (RO4917523) 2 Anxiety, depression – ADAMS All 14-50 Roche 185 NCT01517698 Icon: Open in a new window

Results: Effect of Two Doses of Basimglurant on Behavioral Symptoms in Adolescent and Adult Patients with Fragile X Syndrome; Results from Fragxis, a Double-Blind, Placebo Controlled Study
2014 2012 Basimglurant (RO4917523) 2 Safety All 5-13 Hoffmann-La Roche 47 NCT01750957 Icon: Open in a new window

Results: From a presentation of the International Society of Autism Research (ISAR): Safety and Exploratory Efficacy of Basimglurant in Pediatric Patients with Fragile X Syndrome: A Randomized, Double-Blind, Placebo-Controlled Study
2021 2018 BPN14770 2 Safety, tolerability – Treatment emergent AE (TEAE) Males 18-45 Tetra Discovery Partners 30 NCT03569631 Icon: Open in a new window

Results: Inhibition of phosphodiesterase-4D in adults with fragile X syndrome: a randomized, placebo-controlled, phase 2 clinical trial
2014 2011 Mavoglurant (AFQ056) 2 Fragile X behaviors – ABC-Cfx All 12-17 Novartis 139 NCT01357239 Icon: Open in a new window

Results: Mavoglurant in adolescents with fragile X syndrome: analysis of Clinical Global Impression-Improvement source data from a double-blind therapeutic study followed by an open-label, long-term extension study
2020 2018 Gaboxadol (OV101) 2 Safety – Incidence of AEs (secondary was ABC-C and CGI-I) Males 13-22 Ovid Therapeutics 23 NCT03697161 Icon: Open in a new window

Results: Gaboxadol in Fragile X Syndrome: A 12-Week Randomized, Double-Blind, Parallel-Group, Phase 2a Study
2016 2012 Ganaxolone 2 Clinical impression – CGI All 6-17 Marinus Pharmaceuticals 59 NCT01725152 Icon: Open in a new window

Results: A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome
2020 2016 Lovastatin and PILI 4 Fragile X behaviors, language – ELS All 10-17 UC Davis 30 NCT02642653 Icon: Open in a new window

Results: Controlled trial of lovastatin combined with an open-label treatment of a parent-implemented language intervention in youth with fragile X syndrome
2013 2010 Mavoglurant (AFQ056) 2 Fragile X behaviors – ABC-Cfx All 18-45 Novartis 175 NCT01253629 Icon: Open in a new window

Results: Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056
2023 2017 AFQ056 (Mavoglurant) and Language Intervention 2 Language – Weighted child intentional communication score All 2.5-6 Elizabeth Berry-Kravis, Rush University Medical Center 99 NCT02920892 Icon: Open in a new window

This trial is complete, the results will be published soon.
2009 2008 Mavoglurant (AFQ056) 2 Fragile X behaviors – ABC-Cfx Males 18-35 Novartis 30 NCT00718341 Icon: Open in a new window

Results: Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials
2015 2014 Metadoxine (MG01CI) 2 Inattention – ADHD RS All 15-55 Alcobra 62 NCT02126995 Icon: Open in a new window

Results: Alcobra Announces Results From Phase 2 Clinical Trial of MDX for Fragile X Syndrome
2020 2018 Metformin (Canada) 2 Safety (secondary was behavior and cognition) – AE and ABC-C All 10-45 Université de Sherbrooke 15 NCT03722290 Icon: Open in a new window

Results: Metformin treatment in young children with fragile X syndrome
2021 2018 Metformin (U.S.) 2/3 Fragile X symptoms (language, behavior, weight) – ELS mean NDW score All 6-25 UC Davis 55 NCT03479476 Icon: Open in a new window

Results: Metformin treatment in young children with fragile X syndrome
2010 2007 Oxytocin 2 Fragile X behaviors – Eye contact challenge task Males 13-29 Stanford University 10 NCT01254045 Icon: Open in a new window

Results: Effects of intranasal oxytocin on social anxiety in males with fragile X syndrome
2011 2010 Minocycline n/a Clinical impression – CGI and VAS behavior All 3.5-16 UC Davis 66 NCT01053156 Icon: Open in a new window

Results: A randomized double-blind, placebo-controlled trial of minocycline in children and adolescents with fragile x syndrome
2017 2016 Minocycline and Lovastatin 2 Fragile X behaviors All 13-40 Université de Sherbrooke 23 NCT02680379 Icon: Open in a new window

Results: Drug duo delivers brain, behavioral benefits for fragile X syndrome
2015 2012 Sertraline 2 Cognition, language – Mullen and CGI All 2.5-5 UC Davis 57 NCT01474746 Icon: Open in a new window

Results: A Randomized, Double-Blind, Placebo-Controlled Trial of Low-Dose Sertraline in Young Children With Fragile X Syndrome
2015 2014 Trofinetide 2 Safety (secondary included physiological, behavior, and global/ functional assessments) Males 12-45 Neuren Pharmaceuticals 72 NCT01894958 Icon: Open in a new window

Results: A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Fragile X Syndrome
2020 2018 Zygel (ZYN002) 2/3 Fragile X behaviors – ABC-Cfx All 3-17 Zynerba Pharmaceuticals 212 NCT03614663 Icon: Open in a new window

Full drug name: Zygel (ZYN002) – CBD Transdermal Gel
Phases: Phase 2: ZYN002 – CBD Transdermal Gel; Phase 3: Placebo Transdermal Gel
Results: A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome
2013 2009 Donepezil 2 Executive functioning All 12-29 Stanford University 45 NCT01120626 Icon: Open in a new window

Results: Effectiveness and Safety of Donepezil in Boys With Fragile X Syndrome: A Double-Blind, Randomized, Controlled Pilot Study