206. 脆弱Ｘ症候群 Fragile X syndrome Clinical trials / Disease details
臨床試験数 : 108 / 薬物数 : 91 - (DrugBank : 36) / 標的遺伝子数 : 52 - 標的パスウェイ数 : 77
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05301361 (ClinicalTrials.gov) | February 1, 2023 | 8/3/2022 | Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities | Sensitivity of the NIH Toolbox Cognition Battery to Stimulant Treatment in Intellectual Disabilities | Intellectual Disability;Fragile X Syndrome;Down Syndrome;Attention Deficit Hyperactivity Disorder | Drug: Methylphenidate Oral Solution | University of California, Davis | NULL | Recruiting | 6 Years | 17 Years | All | 68 | Phase 1 | United States |
| 2 | NCT05367960 (ClinicalTrials.gov) | November 1, 2022 | 29/4/2022 | An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome | An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome | Fragile X Syndrome | Drug: Zatolmilast/ BPN14770 | Tetra Discovery Partners | NULL | Enrolling by invitation | 12 Years | 45 Years | Male | 300 | Phase 3 | United States |
| 3 | NCT05358886 (ClinicalTrials.gov) | November 1, 2022 | 27/4/2022 | A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome | A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome | Fragile X Syndrome | Drug: BPN14770/ zatolmilast;Drug: Placebo | Tetra Discovery Partners | NULL | Recruiting | 18 Years | 45 Years | Male | 150 | Phase 3 | United States |
| 4 | NCT05418049 (ClinicalTrials.gov) | September 8, 2022 | 31/5/2022 | Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge | Evaluating the Neurophysiologic and Clinical Effects of Single-Dose Baclofen, Roflumilast, Memantine, and Placebo in Fragile X Syndrome | Fragile X Syndrome | Drug: Baclofen;Drug: Memantine;Drug: Roflumilast;Drug: Placebo | Children's Hospital Medical Center, Cincinnati | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Recruiting | 18 Years | 45 Years | All | 45 | Phase 2 | United States |
| 5 | NCT04823052 (ClinicalTrials.gov) | May 25, 2022 | 24/3/2021 | Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40 | A Phase 2, Placebo-controlled, Adaptive Design Study to Explore the Safety and Efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) and Possible Other Treatments in Adolescent and Adult Males With Fragile X Syndrome (FXS) | Fragile X Syndrome | Drug: Sulindac (HLX-0201), dose strength 1;Drug: Sulindac (HLX-0201), dose strength 2;Drug: Placebo;Drug: Gaboxadol (HLX-0206) | Healx Limited | NULL | Withdrawn | 13 Years | 40 Years | Male | 0 | Phase 2 | United States;Australia |
| 6 | NCT05163808 (ClinicalTrials.gov) | March 29, 2022 | 15/12/2021 | A Randomized Study of BPN14770 in Male Adolescents (Aged 12 to < 18 Years) With Fragile X Syndrome | A Randomized, Double-blind, Placebo-controlled, Two-Part Study of High and Low Dose BPN14770 in Male Adolescents (Aged 12 to < 18 Years) With Fragile X Syndrome | Fragile X Syndrome | Drug: zatolmilast;Drug: Placebo | Tetra Discovery Partners | NULL | Recruiting | 12 Years | 18 Years | Male | 150 | Phase 2/Phase 3 | United States |
| 7 | NCT05120505 (ClinicalTrials.gov) | December 29, 2021 | 3/11/2021 | Metformin in Children With Fragile X Syndrome | Efficacy and Safety of Metformin in the Treatment of Fragile X Syndrome | Fragile X Syndrome;Metformin | Drug: Metformin;Drug: Placebo | Children's Hospital of Fudan University | NULL | Recruiting | 2 Years | 16 Years | All | 20 | Phase 4 | China |
| 8 | NCT05030129 (ClinicalTrials.gov) | October 7, 2021 | 5/8/2021 | Single Blind Study of Ergoloid Mesylates, 5-HTP and the Combination in Adult Males With Fragile X Syndrome | An Exploratory Single Blind Study of Ergoloid Mesylates, 5-Hydroxytryptophan, and the Combination in Adult Males With Fragile X Syndrome | Fragile X Syndrome | Dietary Supplement: 5-Hydroxytryptophan / Vitamin B6;Drug: Ergoloid Mesylate;Other: Placebo | Elizabeth Berry-Kravis | NULL | Recruiting | 18 Years | 45 Years | Male | 15 | Phase 2 | United States |
| 9 | NCT04977986 (ClinicalTrials.gov) | September 13, 2021 | 20/7/2021 | Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome | A Randomized, Double-Blind, Placebo-Controlled, Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children, Adolescents and Young Adults With Fragile X Syndrome - RECONNECT | Fragile X Syndrome | Drug: ZYN002 - transdermal gel;Drug: Placebo | Zynerba Pharmaceuticals, Inc. | NULL | Recruiting | 3 Years | 23 Years | All | 204 | Phase 3 | United States;Australia;Ireland;United Kingdom |
| 10 | NCT04314856 (ClinicalTrials.gov) | January 12, 2021 | 11/3/2020 | Novel Clinical Target in Fragile X Syndrome | Sigma-1 Receptors: A Novel Clinical Target in Fragile X Syndrome | Fragile X Syndrome (FXS) | Drug: 18F-FTC-146 | Guido A. Davidzon, MD, SM | NULL | Withdrawn | 18 Years | 65 Years | All | 0 | Phase 1 | United States |
| 11 | NCT04698551 (ClinicalTrials.gov) | September 1, 2020 | 1/9/2020 | NIPD on cffDNA for Triplet Repeat Diseases | Comparison of Two NGS Phasing Techniques of Parental Haplotypes for NIPD of Triplet Expansion Diseases | Myotonic Dystrophy 1;Huntington Disease;Fragile X Syndrome | Genetic: Non invasive prenatal diagnosis | University Hospital, Montpellier | Agence de La Biomédecine | Active, not recruiting | 18 Months | N/A | All | 36 | France | |
| 12 | NCT04141163 (ClinicalTrials.gov) | October 29, 2019 | 24/10/2019 | Metformin in Patients With Fragile X | A Parallel Group Design Randomized Double-Blind Trial of Metformin Treatment in Patients With Fragile X Syndrome on Safety and Effects on Cognition, Anxiety, Attention and Biomarkers | Fragile X Syndrome | Drug: Metformin;Drug: Placebo oral tablet | Rowan University | FRAXA Research Foundation;University of Pennsylvania | Recruiting | 18 Years | 50 Years | Male | 40 | Phase 1/Phase 2 | United States |
| 13 | NCT03862950 (ClinicalTrials.gov) | May 24, 2019 | 26/2/2019 | A Trial of Metformin in Individuals With Fragile X Syndrome (Met) | A Double-Blind, Placebo-Controlled Trial of Metformin in Individuals With Fragile X Syndrome (FXS) | Fragile X Syndrome;Fragile X Mental Retardation Syndrome;Mental Retardation, X-Linked;Genetic Diseases, X-Linked;Trinucleotide Repeat Expansion;Fra(X) Syndrome;Intellectual Disability;FXS;Neurobehavioral Manifestations;Sex Chromosome Disorders | Drug: Placebo Medication;Drug: Metformin | University of Alberta | St. Justine's Hospital | Recruiting | 6 Years | 35 Years | All | 120 | Phase 2 | Canada |
| 14 | NCT03697161 (ClinicalTrials.gov) | September 17, 2018 | 1/10/2018 | A Study of OV101 in Individuals With Fragile X Syndrome | A Phase 2, Randomized, Double-Blind, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of OV101 in Fragile X Syndrome | Fragile X Syndrome (FXS) | Drug: OV101 (gaboxadol) | Ovid Therapeutics Inc. | NULL | Completed | 13 Years | 22 Years | Male | 23 | Phase 2 | United States |
| 15 | NCT03722290 (ClinicalTrials.gov) | September 1, 2018 | 24/10/2018 | Metformin in Children and Adults With Fragile X Syndrome | Evaluate the Efficacy and Safety of Metformin in Children and Adults With Fragile X Syndrome: an Open-label Study | Fragile X Syndrome | Drug: Metformin | Université de Sherbrooke | FRAXA Research Foundation | Completed | 10 Years | 45 Years | All | 15 | Phase 2 | Canada |
| 16 | NCT03569631 (ClinicalTrials.gov) | July 9, 2018 | 25/5/2018 | A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome | A Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study of BPN14770 in Adult Males With Fragile X Syndrome | Fragile X Syndrome;FXS;Fra(X) Syndrome | Drug: BPN14770;Drug: Placebo | Tetra Discovery Partners | NULL | Completed | 18 Years | 45 Years | Male | 30 | Phase 2 | United States |
| 17 | NCT03614663 (ClinicalTrials.gov) | June 12, 2018 | 10/7/2018 | Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) | A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With Fragile X Syndrome | Fragile X Syndrome | Drug: ZYN002 - CBD Transdermal Gel;Other: Placebo Transdermal Gel | Zynerba Pharmaceuticals, Inc. | NULL | Completed | 3 Years | 17 Years | All | 212 | Phase 2/Phase 3 | United States;Australia;New Zealand |
| 18 | NCT03479476 (ClinicalTrials.gov) | April 30, 2018 | 20/3/2018 | A Trial of Metformin in Individuals With Fragile X Syndrome | A Double-Blind, Placebo-Controlled Trial of Metformin in Individuals With Fragile X Syndrome | Fragile X Syndrome;Fragile X Mental Retardation Syndrome;Mental Retardation, X Linked;Genetic Diseases, X-Linked;Trinucleotide Repeat Expansion;Fra(X) Syndrome;Intellectual Disability;FXS;Neurobehavioral Manifestations;Sex Chromosome Disorders | Drug: Placebo Medication;Drug: Metformin | University of California, Davis | NULL | Completed | 6 Years | 25 Years | All | 55 | Phase 2/Phase 3 | United States |
| 19 | NCT03624556 (ClinicalTrials.gov) | January 29, 2018 | 6/6/2018 | Pediatric Exploratory Research Study of EGCG Use and Safety (PERSEUS) | Pediatric Exploratory Research Study of EGCG Use and Safety (PERSEUS) | Down Syndrome;Fragile X Syndrome | Dietary Supplement: EGCG FontUp;Other: Placebo FontUp | Parc de Salut Mar | Hospital Infantil Universitario Niño Jesús, Madrid, Spain;Instituto Hispalense de Pediatría, Sevilla, Spain;Hospital Universitario Marqués de Valdecilla;Institut Jerome Lejeune | Completed | 6 Years | 12 Years | All | 76 | N/A | Spain |
| 20 | NCT03140813 (ClinicalTrials.gov) | January 16, 2018 | 17/4/2017 | An Initial Study of AZD7325 in Adults With Fragile X Syndrome | An Initial Double-Blind, Placebo-Controlled Two-Dose Crossover Study of AZD7325 in Adults With Fragile X Syndrome | Fragile X Syndrome | Drug: AZD7325 (High-Dose);Drug: AZD7325 (Low-Dose);Drug: Placebo oral capsule | Children's Hospital Medical Center, Cincinnati | NULL | Completed | 18 Years | 50 Years | All | 15 | Phase 1 | United States |
| 21 | NCT02920892 (ClinicalTrials.gov) | August 17, 2017 | 28/9/2016 | AFQ056 for Language Learning in Children With FXS | Effects of AFQ056 on Language Learning in Young Children With Fragile X Syndrome (FXS) | Fragile X Syndrome | Drug: AFQ056;Other: Placebo;Other: Language Intervention | Elizabeth Berry-Kravis | National Institute of Neurological Disorders and Stroke (NINDS) | Completed | 32 Months | 6 Years | All | 99 | Phase 2 | United States |
| 22 | NCT03109756 (ClinicalTrials.gov) | April 3, 2017 | 6/4/2017 | Single Dose Pharmacokinetic (PK) Study | A Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman Syndrome | Angelman Syndrome;Fragile X Syndrome | Drug: OV101 | Ovid Therapeutics Inc. | NULL | Completed | 13 Years | 17 Years | All | 12 | Phase 1 | United States |
| 23 | NCT04308954 (ClinicalTrials.gov) | November 1, 2016 | 11/3/2020 | Neuroimaging GABA Physiology in Fragile X Syndrome | Cross-Species Multi-Modal Neuroimaging to Investigate GABA Physiology in Fragile X Syndrome | Fragile X Syndrome (FXS);Idiopathic Intellectual Developmental Disorder (IDD) | Drug: [18F]flumazenil | Stanford University | NULL | Terminated | 18 Years | 30 Years | Male | 17 | Phase 1 | United States |
| 24 | EUCTR2015-005460-42-ES (EUCTR) | 07/07/2016 | 20/04/2016 | Clinical trial to evaluate ascorbic acid (vitamin C) and tocopherol (vitamin E) in combination versus placebo for the treatment of cognitive and behavioral disorders in children with fragile x syndrome | Phase III clinical trial, double-blind, cross-way, to evaluate the safety and efficacy ascorbic acid (vitamin C) and tocopherol (vitamin E) combination versus placebo for the treatment of cognitive and behavioral disorders in children with fragile x syndrome | Fragile x syndrome MedDRA version: 19.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Vitamin C INN or Proposed INN: Vitamin C Other descriptive name: ACIDUM ASCORBICUM D6 Product Name: Vitamin E INN or Proposed INN: Vitamin E Other descriptive name: TOCOPHERYL ACETATE | Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud | NULL | Not Recruiting | Female: no Male: yes | Phase 3 | Spain | |||
| 25 | NCT02719951 (ClinicalTrials.gov) | April 2016 | 9/3/2016 | Glutaminergic Transmission in Autism : Molecular Imaging Exploration | Glutaminergic Transmission in Autism : Molecular Imaging Exploration | Autism;Fragile-X Syndrome (FXS);Healthy Volunteers | Drug: [18F]FPEB PET imaging;Other: Biological samples;Other: MRI | University Hospital, Tours | NULL | Completed | 18 Years | 45 Years | Male | 27 | Phase 1 | France |
| 26 | NCT02680379 (ClinicalTrials.gov) | March 2016 | 25/1/2016 | Combined Treatment of Minocycline and Lovastatin to Treat Individuals With Fragile X Syndrome | A Pilot Study Exploring the Safety and Synergistic Effect of a Minocycline/Lovastatin Combined Treatment on the Behavior of Individuals With Fragile X Syndrome; Validation of New Biochemical and Neurophysiological Markers (LovaMiX) | Fragile X Syndrome | Drug: Minocycline, then Minocycline/Lovastatin;Drug: Lovastatin, then Minocycline/Lovastatin | Université de Sherbrooke | FRAXA Research Foundation | Completed | 8 Years | 45 Years | All | 22 | Phase 2 | Canada |
| 27 | NCT02642653 (ClinicalTrials.gov) | January 2016 | 21/12/2015 | Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome | Combining Lovastatin and a Parent-Implemented Language Intervention in a Multimodal Treatment for Fragile X Syndrome | Fragile X Syndrome;Genetic Diseases | Drug: Lovastatin;Other: Placebo | University of California, Davis | NULL | Completed | 10 Years | 17 Years | All | 30 | Phase 4 | United States |
| 28 | NCT02998151 (ClinicalTrials.gov) | January 2016 | 1/12/2016 | Neurophysiological and Acute Pharmacological Studies in FXS Patients | Neurophysiological and Acute Pharmacological Studies in FXS Patients | Fragile X Syndrome | Drug: Acamprosate;Drug: Lovastatin;Drug: Minocycline;Drug: Placebo;Drug: Baclofen | Children's Hospital Medical Center, Cincinnati | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | 15 Years | 55 Years | All | 29 | Early Phase 1 | United States |
| 29 | EUCTR2014-000251-89-BE (EUCTR) | 22/10/2014 | 18/08/2014 | A clinical study with an investigational drug called ganaxolone in children with fragile X syndrome | A controlled double-blind crossover trial of ganaxolone in children with fragile X syndrome - GNX study | behavioral problems in children with fragile X syndrome MedDRA version: 17.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Ganaxolone Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT316 INN or Proposed INN: GANAXOLONE | Antwerp University Hospital | NULL | Not Recruiting | Female: yes Male: yes | 60 | United States;Belgium | |||
| 30 | NCT02126995 (ClinicalTrials.gov) | June 2014 | 29/4/2014 | A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome | A 6-week, Randomized, Multicenter, Double-blind, Parallel, Flexed and Fixed-dose Study of MG01CI (Metadoxine Extended-release) Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome | Fragile X Syndrome | Drug: MG01CI extended-release tablet | Alcobra Ltd. | NULL | Completed | 15 Years | 55 Years | Both | 62 | Phase 2 | United States;Israel |
| 31 | NCT01894958 (ClinicalTrials.gov) | January 2014 | 3/7/2013 | A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome | A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Fixed-Dose Study of NNZ-2566 in Fragile X Syndrome | Fragile X Syndrome | Drug: NNZ-2566;Drug: Placebo | Neuren Pharmaceuticals Limited | NULL | Completed | 12 Years | 45 Years | Male | 72 | Phase 2 | United States |
| 32 | EUCTR2011-002379-40-BE (EUCTR) | 02/12/2013 | 24/10/2013 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: Mavoglurant Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: Mavoglurant | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 2 | United States;Spain;Indonesia;Turkey;Israel;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Brazil;Australia;Denmark;Germany;Netherlands;Sweden | ||
| 33 | NCT01911455 (ClinicalTrials.gov) | August 27, 2013 | 26/7/2013 | Study of Acamprosate in Fragile x Syndrome | Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome | Fragile X Syndrome | Drug: acamprosate;Drug: Placebo | Children's Hospital Medical Center, Cincinnati | Rush University Medical Center | Completed | 5 Years | 23 Years | All | 46 | Phase 1 | United States |
| 34 | EUCTR2011-002379-40-NL (EUCTR) | 08/07/2013 | 08/05/2013 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 16.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: Mavoglurant Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: Mavoglurant | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 180 | United States;Spain;Indonesia;Turkey;Israel;Italy;Switzerland;United Kingdom;France;Canada;Brazil;Australia;Denmark;Netherlands;Germany;Sweden | |||
| 35 | EUCTR2010-022638-96-NL (EUCTR) | 08/07/2013 | 08/05/2013 | Safety and efficacy of AFQ056 in adolescent patients with Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 16.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: mavoglurant Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: mavoglurant | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 120 | United States;Spain;Indonesia;Turkey;Israel;Italy;Switzerland;United Kingdom;France;Canada;Brazil;Australia;Denmark;Netherlands;Germany;Sweden | |||
| 36 | NCT01855971 (ClinicalTrials.gov) | June 11, 2013 | 9/5/2013 | Using Epigallocatechin Gallate (EGCG) and Cognitive Training to Modulate Cognitive Performance in Patients With Fragile X Syndrome (TESFX) | Estrogen Receptors Beta (ER-B) as Therapeutic Targets for the Improvement of Cognitive Performance in Fragile-X (TESXF) | Fragile X Syndrome | Dietary Supplement: EGCG;Dietary Supplement: Placebo;Other: Cognitive training | Parc de Salut Mar | NULL | Completed | 18 Years | 60 Years | All | 44 | Phase 2 | Spain |
| 37 | EUCTR2010-022638-96-BE (EUCTR) | 27/05/2013 | 18/04/2013 | Safety and efficacy of AFQ056 in adolescent patients with Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety ofAFQ056 in adolescent patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: Mavoglurant Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: Mavoglurant | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 120 | Phase 2 | United States;Spain;Indonesia;Turkey;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Brazil;Australia;Denmark;Netherlands;Germany;Sweden | ||
| 38 | NCT01750957 (ClinicalTrials.gov) | February 2013 | 11/12/2012 | A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome | A Randomized, Parallel Group, Double-Blind, Placebo-Controlled, Safety and Exploratory Efficacy and Pharmacokinetic, Study of RO4917523 in Pediatric Patients With Fragile X Syndrome | Fragile X Syndrome | Drug: Placebo;Drug: RO4917523 | Hoffmann-La Roche | NULL | Completed | 5 Years | 13 Years | Both | 47 | Phase 2 | United States |
| 39 | NCT01725152 (ClinicalTrials.gov) | November 2012 | 7/11/2012 | Ganaxolone Treatment in Children With Fragile X Syndrome | A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome | Fragile x Syndrome | Drug: Ganaxolone;Drug: Placebo | Marinus Pharmaceuticals | University of California, Davis;U.S. Army Medical Research and Development Command | Completed | 6 Years | 17 Years | All | 59 | Phase 2 | United States;Belgium |
| 40 | EUCTR2011-004349-42-ES (EUCTR) | 16/08/2012 | 21/05/2012 | A study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome | A randomized, double-blind, 12- week, parallel group, placebo-controlled study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome. | Fragile X Syndrome (FXS) MedDRA version: 14.1;Level: LLT;Classification code 10025463;Term: Major depressive disorder, single episode;System Organ Class: 10037175 - Psychiatric disorders;Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03] | Product Name: mGlu5 antagonist Product Code: Ro 491-7523/F18 Other descriptive name: mGlu5 antagonist Product Name: mGlu5 antagonist Product Code: Ro 491-7523/F19 Other descriptive name: mGlu5 antagonist | F. Hoffmann-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 180 | France;United States;Mexico;Canada;Argentina;Spain;Peru;Chile;United Kingdom;Sweden | |||
| 41 | EUCTR2011-004349-42-GB (EUCTR) | 16/07/2012 | 25/04/2012 | A study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome | A randomized, double-blind, 12- week, parallel group, placebo-controlled study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome. | Fragile X Syndrome (FXS) MedDRA version: 14.1;Level: LLT;Classification code 10025463;Term: Major depressive disorder, single episode;System Organ Class: 100000004873;Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03] | Product Name: mGlu5 antagonist Product Code: Ro 491-7523/F18 Other descriptive name: mGlu5 antagonist Product Name: mGlu5 antagonist Product Code: Ro 491-7523/F19 Other descriptive name: mGlu5 antagonist | F. Hoffmann-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 2 | United States;France;Mexico;Canada;Argentina;Spain;Peru;Chile;United Kingdom;Sweden | ||
| 42 | EUCTR2011-004349-42-SE (EUCTR) | 29/06/2012 | 02/05/2012 | A study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome | A randomized, double-blind, 12- week, parallel group, placebo-controlled study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome. | Fragile X Syndrome (FXS) MedDRA version: 14.1;Level: LLT;Classification code 10025463;Term: Major depressive disorder, single episode;System Organ Class: 100000004873;Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03] | Product Name: mGlu5 antagonist Product Code: Ro 491-7523/F18 Other descriptive name: mGlu5 antagonist Product Name: mGlu5 antagonist Product Code: Ro 491-7523/F19 Other descriptive name: mGlu5 antagonist | F. Hoffmann-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 180 | France;United States;Mexico;Canada;Argentina;Spain;Peru;Chile;United Kingdom;Sweden | |||
| 43 | EUCTR2011-002379-40-FR (EUCTR) | 27/06/2012 | 24/08/2012 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 15.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 2 | United States;Spain;Israel;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Australia;Denmark;Germany;Netherlands;Sweden | ||
| 44 | EUCTR2011-002379-40-ES (EUCTR) | 25/05/2012 | 22/02/2012 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open?label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Farmacéutica S.A | NULL | Not Recruiting | Female: yes Male: yes | 180 | United States;Spain;Israel;Switzerland;United Kingdom;Italy;France;Canada;Australia;Denmark;Germany;Netherlands;Sweden | |||
| 45 | EUCTR2011-004867-65-ES (EUCTR) | 25/05/2012 | 29/03/2012 | Clinical study to assess the pharmacokinetics, safety & tolerability of single and multiple oral doses of AFQ056 in children with Fragile X Syndrome | Sequential, two-period study to assess the pharmacokinetics, safety & tolerability of single and multiple oral doses of AFQ056 in patients with FXS (Fragile X syndrome) aged 5-11 years (Cohort 1) and 3-4 years (Cohort 2) | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 | Novartis Farmacéutica, S.A. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 1 | United States;Spain | ||
| 46 | NCT01517698 (ClinicalTrials.gov) | May 2012 | 20/1/2012 | A Study of RO4917523 in Patients With Fragile X Syndrome | A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome. | Fragile X Syndrome | Drug: Placebo;Drug: RO4917523 0.5 mg;Drug: RO4917523 1.5 mg | Hoffmann-La Roche | NULL | Completed | 14 Years | 50 Years | Both | 185 | Phase 2 | United States;Argentina;Canada;Chile;France;Mexico;Peru;Spain;Sweden;United Kingdom |
| 47 | EUCTR2011-002379-40-GB (EUCTR) | 24/04/2012 | 12/01/2012 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 180 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Belgium;Spain;Denmark;Australia;Netherlands;Germany;Italy;Switzerland;United Kingdom;Sweden | ||
| 48 | EUCTR2011-002379-40-DK (EUCTR) | 28/03/2012 | 28/02/2012 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 16.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 2 | United States;Spain;Indonesia;Turkey;Israel;Italy;Switzerland;United Kingdom;France;Canada;Brazil;Belgium;Denmark;Australia;Netherlands;Germany;Sweden | ||
| 49 | EUCTR2011-001952-12-FR (EUCTR) | 21/03/2012 | 01/09/2011 | Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome | An open-label study to evaluate the long-term safety, tolerability and efficacy of AFQ056 in adult patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 2 | United States;France;Canada;Spain;Denmark;Australia;Germany;Italy;United Kingdom;Switzerland | ||
| 50 | EUCTR2011-002379-40-IT (EUCTR) | 19/03/2012 | 02/03/2012 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: NA Other descriptive name: NA Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: NA Other descriptive name: NA | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 180 | United States;Belgium;Spain;Australia;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | |||
| 51 | EUCTR2011-001952-12-ES (EUCTR) | 08/03/2012 | 01/02/2012 | Seguridad, tolerabilidad y eficacia a largo plazo de AFQ056 en pacientes adultos con síndrome X frágil | Estudio abierto para evaluar la seguridad, tolerabilidad y eficacia a largo plazo de AFQ056 en pacientes adultos con síndrome X frágil | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Farmacéutica S.A | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 2 | United States;France;Canada;Spain;Denmark;Australia;Germany;United Kingdom;Switzerland;Italy | ||
| 52 | NCT01482143 (ClinicalTrials.gov) | March 2012 | 21/11/2011 | Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS) | Sequential, Two-period Study to Assess the Pharmacokinetics, Safety & Tolerability of Single and Multiple Oral Doses of AFQ056 in Patients With FXS (Fragile X Syndrome) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2) | Fragile X Syndrome | Drug: AFQ056 | Novartis Pharmaceuticals | NULL | Completed | 3 Years | 11 Years | All | 21 | Phase 1 | United States;Spain;Switzerland |
| 53 | EUCTR2011-002379-40-DE (EUCTR) | 09/02/2012 | 13/12/2011 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 180 | France;United States;Belgium;Spain;Denmark;Australia;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden | |||
| 54 | EUCTR2011-002379-40-SE (EUCTR) | 09/01/2012 | 14/11/2011 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 16.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 180 | United States;France;Belgium;Spain;Denmark;Australia;Netherlands;Germany;United Kingdom;Italy;Switzerland;Sweden | |||
| 55 | NCT01474746 (ClinicalTrials.gov) | January 2012 | 10/11/2011 | Trial of Sertraline to Treat Children With Fragile X Syndrome | A Controlled Trial of Sertraline in Young Children With Fragile X Syndrome | Fragile X Syndrome | Drug: Sertraline;Drug: Placebo | Randi J. Hagerman, MD | NULL | Completed | 24 Months | 68 Months | All | 57 | Phase 2 | United States |
| 56 | EUCTR2010-022638-96-ES (EUCTR) | 01/12/2011 | 18/10/2011 | Seguridad y eficacia de AFQ056 en pacientes adolescentes con Síndrome X Frágil. | Estudio aleatorizado, doble ciego, controlado con placebo, con grupos paralelos para evaluar la eficacia y seguridad de AFQ056 en pacientes adolescentes con síndrome X frágil | Síndrome X Frágil MedDRA version: 14.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Farmacéutica S.A | NULL | Not Recruiting | Female: yes Male: yes | 160 | Phase 2 | United States;France;Belgium;Spain;Denmark;Australia;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 57 | EUCTR2011-001952-12-IT (EUCTR) | 14/11/2011 | 06/03/2012 | Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome | An open-label study to evaluate the long-term safety, tolerability and efficacy of AFQ056 in adult patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: NA Product Code: AFQ056 INN or Proposed INN: NA Other descriptive name: NA Product Name: NA Product Code: AFQ056 INN or Proposed INN: NA Other descriptive name: NA | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 200 | United States;Canada;Spain;Denmark;Australia;Germany;United Kingdom;Switzerland;Italy | |||
| 58 | EUCTR2011-001952-12-DK (EUCTR) | 08/11/2011 | 14/10/2011 | Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome | An open-label study to evaluate the long-term safety, tolerability and efficacy of AFQ056 in adult patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 200 | United States;France;Canada;Spain;Australia;Denmark;Germany;United Kingdom;Switzerland;Italy | |||
| 59 | EUCTR2011-001952-12-GB (EUCTR) | 07/11/2011 | 21/09/2011 | Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome | An open-label study to evaluate the long-term safety, tolerability and efficacy of AFQ056 in adult patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 16.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 200 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Canada;Spain;Denmark;Australia;Germany;Italy;Switzerland;United Kingdom | ||
| 60 | EUCTR2011-001952-12-DE (EUCTR) | 03/11/2011 | 08/08/2011 | Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome | An open-label study to evaluate the long-term safety, tolerability and efficacy of AFQ056 in adult patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 200 | United States;France;Canada;Spain;Denmark;Australia;Germany;United Kingdom;Switzerland;Italy | |||
| 61 | NCT01555333 (ClinicalTrials.gov) | November 2011 | 1/3/2012 | An Open Label Extension Study in Subjects With Fragile X Syndrome | An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacokinetics in Subject With Fragile X Syndrome | Fragile X Syndrome | Drug: arbaclofen | Seaside Therapeutics, Inc. | NULL | Terminated | 5 Years | 50 Years | Both | 357 | Phase 3 | United States |
| 62 | NCT01433354 (ClinicalTrials.gov) | November 2011 | 31/8/2011 | Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label) | An Open-label Study to Evaluate the Long-term Safety and Tolerability of AFQ056 in Adolescent Patients With Fragile X Syndrome | Fragile X Syndrome | Drug: AFQ056 | Novartis Pharmaceuticals | NULL | Terminated | 12 Years | 18 Years | All | 119 | Phase 2/Phase 3 | United States;Australia;Belgium;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom;Brazil;Canada;Turkey |
| 63 | EUCTR2009-013667-19-ES (EUCTR) | 06/10/2011 | 11/08/2011 | Seguridad y eficacia de AFQ056 en pacientes adultos con Síndrome X Frágil. | Estudio aleatorizado, doble ciego, controlado con placebo, con grupos paralelos para evaluar AFQ056 en pacientes adultos con síndrome X frágil | Síndrome X Frágil MedDRA version: 14.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Farmacéutica S.A | NULL | Not Recruiting | Female: yes Male: yes | 160 | France;United States;Canada;Spain;Denmark;Australia;Germany;Italy;United Kingdom;Switzerland | |||
| 64 | NCT01348087 (ClinicalTrials.gov) | August 2011 | 3/5/2011 | Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome | An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome | Fragile X Syndrome | Drug: AFQ056 | Novartis Pharmaceuticals | NULL | Terminated | 18 Years | N/A | All | 148 | Phase 2 | United States;Australia;Canada;Denmark;France;Germany;Italy;Spain;Switzerland;United Kingdom;Brazil;Netherlands |
| 65 | EUCTR2010-022638-96-IT (EUCTR) | 11/07/2011 | 20/03/2012 | A study to evaluate the efficacy of AFQ056 in treating abnormal behaviors showed by adolescents (12-17 year-old) with Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03] | Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: AFQ056 Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: AFQ056 | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 180 | United States;Spain;Denmark;Australia;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | |||
| 66 | EUCTR2010-022638-96-GB (EUCTR) | 15/06/2011 | 21/02/2011 | Safety and efficacy of AFQ056in adolescent patients with Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 160 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Spain;Israel;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Australia;Denmark;Netherlands;Germany;Sweden | ||
| 67 | EUCTR2010-022638-96-SE (EUCTR) | 13/06/2011 | 04/04/2011 | Safety and efficacy of AFQ056 in adolescent patients with Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety ofAFQ056 in adolescent patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 160 | France;United States;Spain;Denmark;Australia;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden | |||
| 68 | NCT01325220 (ClinicalTrials.gov) | June 2011 | 28/3/2011 | Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome | A Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Children With Fragile X Syndrome | Fragile X Syndrome | Drug: arbaclofen;Drug: Placebo | Seaside Therapeutics, Inc. | NULL | Completed | 5 Years | 11 Years | Both | 172 | Phase 3 | United States |
| 69 | NCT01357239 (ClinicalTrials.gov) | May 2011 | 18/5/2011 | Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome | A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of AFQ056 in Adolescent Patients With Fragile X Syndrome | Fragile X Syndrome | Drug: AFQ056;Drug: Placebo | Novartis Pharmaceuticals | NULL | Completed | 12 Years | 17 Years | All | 139 | Phase 2 | United States;Australia;Belgium;Canada;Denmark;France;Germany;Indonesia;Israel;Italy;Netherlands;Spain;Sweden;Switzerland;Turkey;United Kingdom;Brazil |
| 70 | NCT01282268 (ClinicalTrials.gov) | May 2011 | 20/1/2011 | Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome | A Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Adolescents and Adults With Fragile X Syndrome | Fragile X Syndrome | Drug: arbaclofen;Drug: placebo | Seaside Therapeutics, Inc. | NULL | Completed | 12 Years | 50 Years | Both | 125 | Phase 3 | United States |
| 71 | NCT01325740 (ClinicalTrials.gov) | May 2011 | 28/3/2011 | A Study to Assess the Tolerability of a Single Dose of STX107 in Adults With Fragile X Syndrome | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of a Single Dose of STX107 in Adults With Fragile X Syndrome | Fragile X Syndrome | Drug: STX107 | Seaside Therapeutics, Inc. | NULL | Suspended | 18 Years | 50 Years | Male | 16 | Phase 2 | United States |
| 72 | EUCTR2010-022638-96-DK (EUCTR) | 27/04/2011 | 18/04/2011 | Safety and efficacy of AFQ056 in adolescent patients with Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety ofAFQ056 in adolescent patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 120 | United States;Spain;Israel;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Denmark;Australia;Netherlands;Germany;Sweden | |||
| 73 | EUCTR2009-013667-19-DE (EUCTR) | 29/12/2010 | 02/11/2010 | Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patientswith Fragile X Syndrome | Fragile X Syndrome MedDRA version: 15.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 160 | France;United States;Canada;Spain;Denmark;Australia;Germany;Italy;United Kingdom;Switzerland | |||
| 74 | EUCTR2009-013667-19-IT (EUCTR) | 22/12/2010 | 24/12/2010 | A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patients with Fragile X Syndrome - ND | A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patients with Fragile X Syndrome - ND | Fragile X Syndrome MedDRA version: 9.1;Level: PT;Classification code 10017324 | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 160 | Spain;Denmark;Germany;United Kingdom;Italy | |||
| 75 | EUCTR2009-013667-19-GB (EUCTR) | 15/12/2010 | 20/10/2010 | Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 160 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Canada;Spain;Denmark;Australia;Germany;Italy;Switzerland;United Kingdom | ||
| 76 | NCT01329770 (ClinicalTrials.gov) | December 2010 | 29/3/2011 | Safety and Efficacy Study of Antioxidants for the Treatment of the Fragile X Syndrome | Phase II Double-blind Randomized Placebo-controlled 1-way Crossover Trial to Investigate Safety and Efficacy of the Ascorbic Acid and Tocopherol for the Treatment of the Fragile X Syndrome | Fragile X Syndrome | Dietary Supplement: Ascorbic Acid (Vitamin C) and Alpha-tocopherol (Vitamin E);Dietary Supplement: Placebo | Yolanda de Diego Otero | NULL | Completed | 6 Years | 18 Years | Male | 30 | Phase 2 | Spain |
| 77 | EUCTR2009-013667-19-DK (EUCTR) | 16/11/2010 | 22/09/2010 | Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patientswith Fragile X Syndrome | Fragile X Syndrome MedDRA version: 15.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 160 | France;United States;Canada;Spain;Australia;Denmark;Germany;Italy;United Kingdom;Switzerland | |||
| 78 | NCT01253629 (ClinicalTrials.gov) | November 2010 | 2/12/2010 | Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome | A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome | Fragile X Syndrome | Drug: AFQ056;Drug: Placebo | Novartis Pharmaceuticals | NULL | Completed | 18 Years | 45 Years | All | 175 | Phase 2 | United States;Australia;Canada;Denmark;France;Germany;Italy;Spain;Switzerland;United Kingdom |
| 79 | NCT01300923 (ClinicalTrials.gov) | August 2010 | 25/8/2010 | Acamprosate in Youth With Fragile X Syndrome | Pilot Study of Acamprosate in Youth With Fragile X Syndrome | Fragile X Syndrome;Autism Spectrum Disorders | Drug: Acamprosate | Indiana University | NULL | Completed | 5 Years | 17 Years | All | 14 | Phase 2 | United States |
| 80 | NCT01053156 (ClinicalTrials.gov) | January 2010 | 19/1/2010 | Trial of Minocycline to Treat Children With Fragile X Syndrome | Randomized Double-Blind Controlled Cross Over Trial of Minocycline in Children With Fragile X Syndrome | Fragile X Syndrome | Drug: minocycline hydrochloride;Drug: Placebo | University of California, Davis | The National Fragile X Foundation | Completed | 42 Months | 16 Years | All | 66 | N/A | United States |
| 81 | NCT01013480 (ClinicalTrials.gov) | November 2009 | 12/11/2009 | An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome | An Open Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects With Fragile X Syndrome | Fragile X Syndrome | Drug: Arbaclofen | Seaside Therapeutics, Inc. | NULL | Terminated | 6 Years | 40 Years | Both | 45 | Phase 2 | United States |
| 82 | NCT01015430 (ClinicalTrials.gov) | November 2009 | 17/11/2009 | A Study With RO4917523 in Patients With Fragile X Syndrome | A Randomized, Double-blind, Placebo-controlled, Pharmacokinetic, Safety and Tolerability, and Exploratory Efficacy and Pharmacodynamic Effects Study of RO4917523 in Adult Patients With Fragile X Syndrome. | Fragile X Syndrome | Drug: Placebo (for RO4917523 ascending doses);Drug: Placebo (for RO4917523 fixed dose);Drug: RO4917523 | Hoffmann-La Roche | NULL | Completed | 18 Years | 50 Years | Both | 40 | Phase 2 | United States |
| 83 | NCT01120626 (ClinicalTrials.gov) | September 2009 | 7/5/2010 | Randomized Controlled Study of Donepezil in Fragile X Syndrome | Augmentation of the Cholinergic System in Fragile X Syndrome: A Double-Blind Placebo-Controlled Randomized Study of Donepezil | Fragile X Syndrome | Drug: donepezil;Drug: sugar pill | Stanford University | National Institute of Mental Health (NIMH);Autism Speaks | Completed | 12 Years | 29 Years | All | 45 | Phase 2 | United States |
| 84 | NCT00965432 (ClinicalTrials.gov) | September 2009 | 24/8/2009 | A Single-Dose Study in Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107 | A Single-Dose, Randomized, Double-Blind, Placebo-Controlled, Dose-Escalation Study in Healthy, Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107 | Fragile X Syndrome | Drug: STX107 | Seaside Therapeutics, Inc. | National Institutes of Health (NIH) | Completed | 18 Years | 50 Years | Male | 40 | Phase 1 | United States |
| 85 | NCT00892580 (ClinicalTrials.gov) | May 2009 | 1/5/2009 | Biomarker and DNA Collection in Subjects Participating in Protocol 22003 | Biomarker Testing and DNA Collection in Subjects Participating in an Open-Label, Flexible-Dose Evaluation of the Efficacy, Safety, and Tolerability of STX209 in the Treatment of Irritability in Subjects With Autism Spectrum Disorders | Fragile X Syndrome | Drug: STX209 | Seaside Therapeutics, Inc. | NULL | Completed | 6 Years | 17 Years | Both | 32 | N/A | United States |
| 86 | NCT00895752 (ClinicalTrials.gov) | April 2009 | 6/5/2009 | Riluzole in Fragile X Syndrome | Riluzole in Fragile X Syndrome: A Pilot Study Incorporating Biomarker Assay | Fragile X Syndrome | Drug: Riluzole | Indiana University | Indiana Clinical and Translational Sciences Institute | Completed | 18 Years | N/A | All | 6 | Phase 4 | United States |
| 87 | NCT00870974 (ClinicalTrials.gov) | March 2009 | 26/3/2009 | A PET Brain Imaging Study of mGluR5 in Subjects With Neuropsychiatric Conditions | Evaluation of [18F]PEB and Positron Emission Tomography (PET) as a Marker of mGluR5 in Subjects w/ Neuropsychiatric Conditions | Parkinson Disease;Huntington Disease;Autistic Spectrum Disorders;Fragile X Syndrome;Alzheimer Disease;Mild Cognitive Impairment | Drug: [18F]FPEB | Institute for Neurodegenerative Disorders | NULL | Completed | 18 Years | 85 Years | All | 48 | Phase 1 | United States |
| 88 | NCT00823368 (ClinicalTrials.gov) | January 2009 | 13/1/2009 | Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001 | Biomarker Testing and DNA Collection in Subjects Participating in a Double-Blind, Placebo-Controlled, Crossover, Flexible-Dose Evaluation of the Efficacy, Safety, and Tolerability of STX209 in the Treatment of Irritability in Subjects With Fragile X Syndrome | Fragile X Syndrome | Drug: STX209 | Seaside Therapeutics, Inc. | NULL | Completed | 6 Years | 40 Years | Both | N/A | United States | |
| 89 | NCT00788073 (ClinicalTrials.gov) | November 2008 | 7/11/2008 | Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome | A Double-Blind, Placebo-Controlled, Crossover, Flexible-Dose Evaluation of the Efficacy, Safety and Tolerability of STX209 in the Treatment of Irritability in Subjects With Fragile X Syndrome | Fragile X Syndrome | Drug: STX209;Drug: Placebo | Seaside Therapeutics, Inc. | NULL | Completed | 6 Years | 40 Years | All | 63 | Phase 2 | United States |
| 90 | EUCTR2008-003287-18-BE (EUCTR) | 17/09/2008 | 03/09/2008 | Functional consequences of decreased RNA expression of certain GABA(A) receptor subunits in fragile X patients using Positron Emission Tomography and [11C]flumazenil. - PET-FRAX-GABA | Functional consequences of decreased RNA expression of certain GABA(A) receptor subunits in fragile X patients using Positron Emission Tomography and [11C]flumazenil. - PET-FRAX-GABA | Fragile X syndrome is the most common form of inherited mental retardation with a prevalence of 1/4000 males and 1/6000 females. Patients are characterized by a mild to severe form of cognitive impairment and light dysmorphic features. Autism-like behaviour, hyperactivity and epilepsy can also be associated with the disease. On the molecular level the disease is caused by a dynamic mutation of a CGG repeat in the 5' UTR of the FMR1 gene. | Product Name: 11C-flumazenil | University Antwerp | University Hospital Leuven gasthuisberg | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | Belgium | ||||
| 91 | EUCTR2007-005088-82-IT (EUCTR) | 11/06/2008 | 09/04/2008 | A multi-centre, randomized, double-blind, placebo-controlled, two-period, crossover proof-of-concept study in male patients with Fragile X Syndrome to assess the efficacy, safety and tolerability of multiple oral doses of AFQ056 - ND | A multi-centre, randomized, double-blind, placebo-controlled, two-period, crossover proof-of-concept study in male patients with Fragile X Syndrome to assess the efficacy, safety and tolerability of multiple oral doses of AFQ056 - ND | Fragile X Syndrome MedDRA version: 9.1;Level: LLT;Classification code 10017324;Term: Fragile X syndrome | Product Code: AFQ056 Product Code: AFQ056 | NOVARTIS FARMA | NULL | Not Recruiting | Female: no Male: yes | 30 | France;Italy | |||
| 92 | NCT00718341 (ClinicalTrials.gov) | June 2008 | 17/7/2008 | Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients | A Multi-centre, Randomized, Double-blind, Placebo Controlled, Two-period, Crossover Proof-of-concept Study in Male Patients With Fragile X Syndrome to Assess the Efficacy, Safety and Tolerability of Multiple Oral Doses of AFQ056 | Fragile X Syndrome | Drug: AF056;Drug: Placebo | Novartis | NULL | Completed | 18 Years | 35 Years | Male | 30 | Phase 2 | France;Italy;Switzerland |
| 93 | EUCTR2007-005088-82-FR (EUCTR) | 15/05/2008 | 20/11/2007 | A multi-centre, randomized, double-blind, placebo-controlled, two-period, crossover proof-of-concept study in patients with Fragile X Syndrome to assess the efficacy, safety and tolerability of multiple oral doses of AFQ056 | A multi-centre, randomized, double-blind, placebo-controlled, two-period, crossover proof-of-concept study in patients with Fragile X Syndrome to assess the efficacy, safety and tolerability of multiple oral doses of AFQ056 | Fragile X Syndrome MedDRA version: 9.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome | Product Code: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: no Male: yes | 30 | France;Italy | |||
| 94 | NCT00637221 (ClinicalTrials.gov) | March 2008 | 3/3/2008 | Open Label Study Investigating Safety and Efficacy of NPL2009 50 mg - 150 mg on Prepulse Inhibition Tests and Continuous Performance Tasks, Adults With Fragile X Syndrome | An Open Label Exploratory Study to Investigate the Safety and Effects of NPL-2009 ( 50 mg - 150 mg Single Dose) on Prepulse Inhibition Tests and Continuous Performance Tasks, in Adults With Fragile X Syndrome | Fragile X Syndrome | Drug: NPL-2009 | Neuropharm | NULL | Completed | 18 Years | 45 Years | Both | 12 | Phase 1/Phase 2 | United States |
| 95 | NCT00858689 (ClinicalTrials.gov) | October 2007 | 9/3/2009 | Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome | Add-on Pilot Trial of Minocycline in Fragile X Syndrome | Fragile X Syndrome | Drug: Minocycline | FRAXA Research Foundation | Fragile X Research Foundation of Canada | Completed | 13 Years | 35 Years | All | 20 | N/A | Canada |
| 96 | NCT00420459 (ClinicalTrials.gov) | April 2007 | 9/1/2007 | A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome | Aripiprazole in Fragile X Syndrome | Fragile X Syndrome | Drug: Aripiprazole | Indiana University School of Medicine | NULL | Completed | 5 Years | 35 Years | All | 12 | Phase 2 | United States |
| 97 | NCT01254045 (ClinicalTrials.gov) | February 2007 | 2/12/2010 | Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome | Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome | Fragile X Syndrome | Drug: placebo;Drug: oxytocin 24IU;Drug: oxytocin 48IU | Stanford University | NULL | Completed | 13 Years | 29 Years | Male | 10 | Phase 2 | United States |
| 98 | NCT00362843 (ClinicalTrials.gov) | August 22, 2006 | 9/8/2006 | Protein Synthesis in the Brain of Patients With Fragile X Syndrome | PET Measurement of Regional Rates of Cerebral Protein Synthesis in Subjects With Fragile X Syndrome | Fragile X Syndrome;Healthy Volunteers | Drug: [15-O]water | National Institute of Mental Health (NIMH) | NULL | Completed | 18 Years | 24 Years | Male | 147 | United States | |
| 99 | NCT00220584 (ClinicalTrials.gov) | July 2005 | 15/9/2005 | An Open-Label Trial of Donepezil in Fragile X Syndrome | An Open-Label Trial of Donepezil in Fragile X Syndrome | Fragile X Syndrome | Drug: donepezil | Stanford University | NULL | Completed | 14 Years | N/A | Both | 10 | Phase 1 | United States |
| 100 | NCT00054730 (ClinicalTrials.gov) | June 2002 | 7/2/2003 | Effects of CX516 on Functioning in Fragile X Syndrome and Autism | Effects of Ampakine CX516 (Ampalex®) on Functioning in Fragile X Syndrome and Autism | Fragile X Syndrome;Autism | Drug: CX516 (Ampalex®) | Cortex Pharmaceuticals | FRAXA Foundation | Completed | 18 Years | 50 Years | Both | Phase 2 | United States | |
| 101 | NCT00768820 (ClinicalTrials.gov) | May 2001 | 7/10/2008 | The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome | The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome (VCFS), Williams Syndrome (WS)and Fragile X Syndrome Characterization, Treatment and Examining the Connection to Developmental and Molecular Factors | Velocardiofacial Syndrome;Williams Syndrome;Fragile X Syndrome | Drug: methylphenidate, fluoxetin, risperidone | The Chaim Sheba Medical Center | NULL | Recruiting | N/A | N/A | All | 400 | Phase 4 | Israel |
| 102 | EUCTR2021-002542-33-IE (EUCTR) | 10/08/2021 | Effects and Safety of Test drug ZYN002, applied as a gel onto the skin of children and adolescents with a certain hereditary disease called Fragile X Syndrome | A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome - RECONNECT - RECONNECT | Fragile X syndrome (FXS) is a rare genetic disorder caused by the deficiency or absence of Fragile X mental retardation protein (FMRP), an RNA-binding protein and the gene product of the FMR1 gene. FXS is most commonly caused by silencing of the FMR1 gene due to a trinucleotide repeat expansion. In FXS patients, the relative absence of functional FMRP is associated with critical impairments in neurodevelopment and learning, as well as disruption to other neuronal and non-neuronal functions. MedDRA version: 20.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: ZYN002 INN or Proposed INN: n/a Other descriptive name: CANNABIDIOL | Zynerba Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 204 | Phase 3 | United States;Ireland;Australia;United Kingdom | |||
| 103 | EUCTR2010-022638-96-DE (EUCTR) | 26/04/2011 | Safety and efficacy of AFQ056 in adolescent patients with Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety ofAFQ056 in adolescent patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 15.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 160 | United States;Spain;Israel;Switzerland;United Kingdom;Italy;France;Canada;Australia;Denmark;Germany;Netherlands;Sweden | ||||
| 104 | EUCTR2010-022638-96-Outside-EU/EEA (EUCTR) | 31/10/2011 | Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome - | Fragile X Syndrome MedDRA version: 14.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: mavoglurant Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: mavoglurant | Novartis Pharma Services AG | NULL | NA | Female: yes Male: yes | 160 | Australia;United States | ||||
| 105 | EUCTR2011-002379-40-Outside-EU/EEA (EUCTR) | 15/12/2011 | Long-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label) | An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | NA | Female: yes Male: yes | 180 | Australia;Switzerland;United States | ||||
| 106 | EUCTR2010-019353-18-Outside-EU/EEA (EUCTR) | 31/10/2011 | Two-period, single-dose pharmacokinetic study in male adolescent patients with Fragile X Syndrome (FXS). | A sequential, open-label, two-period study to assess the pharmacokinetics, safety and tolerability of two dose levels of AFQ056 in male, adolescent patients with Fragile X Syndrome (12 to 18 years inclusive) | Fragile X Syndrome MedDRA version: 14.0;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: mavoglurant Product Name: AFQ056 Product Code: AFQ056 INN or Proposed INN: mavoglurant | Novartis Pharma Services AG | NULL | NA | Female: no Male: yes | 12 | Switzerland | ||||
| 107 | EUCTR2011-004867-65-Outside-EU/EEA (EUCTR) | 29/02/2012 | Clinical study to assess the pharmacokinetics, safety & tolerability of single and multiple oral doses of AFQ056 in children with Fragile X Syndrome | Sequential, two-period study to assess the pharmacokinetics, safety & tolerability of single and multiple oral doses of AFQ056 in patients with FXS (Fragile X syndrome) aged 5-11 years (Cohort 1) and 3-4 years (Cohort 2) | Fragile X Syndrome MedDRA version: 14.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | NA | Female: yes Male: yes | 24 | Spain;United States | ||||
| 108 | EUCTR2010-022638-96-FR (EUCTR) | 12/04/2011 | A study to evaluate the efficacy of AFQ056 in treating abnormal behaviors showed by adolescents (12-17 year-old) with Fragile X Syndrome | A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome | Fragile X Syndrome MedDRA version: 13.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03] | Product Name: AFQ056 Product Code: AFQ056 Product Name: AFQ056 Product Code: AFQ056 | Novartis Pharma Services AG | NULL | NA | Female: yes Male: yes | 180 | Phase 2 | United States;France;Belgium;Spain;Denmark;Australia;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden |