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 156. レット症候群 [臨床試験数:27,薬物数:34(DrugBank:16),標的遺伝子数:69,標的パスウェイ数:105] 

Searched query = "Rett syndrome"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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size
PhaseCountries
1NCT03848832July 29, 20192 September 2019Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett SyndromeA Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett SyndromeRett Syndrome;RTTDrug: GWP42003-P;Drug: PlaceboGW Research LtdRecruiting2 Years18 YearsFemale252Phase 3United States;United Kingdom
2NCT03941444May 6, 201920 May 2019ANAVEX2-73 Study in Patients With Rett SyndromeA Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett SyndromeRett SyndromeDrug: ANAVEX2-73;Drug: PlacebosAnavex Life Sciences Corp.Recruiting18 Years45 YearsFemale30Phase 2Australia
3NCT03633058March 12, 201930 September 2019A Study to Evaluate Ketamine for the Treatment of Rett SyndromeA Randomized, Double-blind, Placebo-controlled, Cross-over Study to Assess the Safety, Tolerability and Efficacy of Oral Ketamine for Patients With Rett SyndromeRett SyndromeDrug: KetamineRett Syndrome Research TrustVanderbilt University Medical CenterRecruiting6 Years12 YearsFemale48Phase 2United States
4NCT03758924February 28, 201911 November 2019Study of ANAVEX2-73 in Patients With Rett SyndromeA Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett SyndromeRett SyndromeDrug: ANAVEX2-73;Drug: PlaceboAnavex Life Sciences Corp.International Rett Syndrome Foundation Rettsyndrome.orgRecruiting18 Years45 YearsFemale21Phase 2United States
5NCT03259971August 25, 201725 March 2019The Role of Probiotics PS128 in Movement DisordersThe Role of Probiotics PS128 in Movement DisordersRett Syndrome;Tourette Syndrome;Tic DisordersDietary Supplement: Probiotic-Lactobacillus plantarum PS128;Dietary Supplement: PlaceboNational Taiwan University HospitalRecruiting1 Year50 YearsAll500N/ATaiwan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
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PhaseCountries
6NCT03059160April 1, 201716 December 2017Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.Rett SyndromeDrug: Tridecanoic AcidSheba Medical CenterUltragenyx Pharmaceutical IncNot recruiting5 Years18 YearsFemale10Phase 2
7EUCTR2015-004448-20-GB28/11/20167 January 2019Sarizotan: Treatment in patients with Rett Syndrome with Respiratory Symptoms.A Randomized, Double-Blind, Placebo-Controlled, Six-Month Study to Evaluate the Efficacy, Safety and Tolerability of Sarizotan in Patients with Rett Syndrome with Respiratory SymptomsRett syndrome
MedDRA version: 20.0 Level: LLT Classification code 10039000 Term: Rett's disorder System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: sarizotan hydrochloride
Product Code: EMD 128130
Pharmaceutical Form: Capsule
INN or Proposed INN: sarizotan hydrochloride
CAS Number: 195068-07-6
Other descriptive name: SARIZOTAN HYDROCHLORIDE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use
Product Name: sarizotan hydrochloride
Product Code: EMD 128130
Pharmaceutical Form: Capsule
INN or Proposed INN: sarizotan hydrochloride
CAS Number: 195068-07-6
Other descriptive name: SARIZOTAN HYDROCHLORIDE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use
Product Name: sarizotan hydrochloride
Product Code: EMD 128130
Pharmaceutical Form: Capsule
INN or Proposed INN: sarizotan hydrochloride
CAS Number: 195068-07-6
Other descriptive name: SARIZOTAN HYDROCHLORIDE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use
Newron Pharmaceuticals S.p.A.AuthorisedFemale: yes
Male: yes
129Phase 2United States;Australia;Italy;United Kingdom;India
8NCT02790034August 201625 February 2019Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory SymptomsA Randomised, Double-Blind, Placebo-Controlled 6-month Study to Evaluate the Efficacy, Safety, and Tolerability of Sarizotan in Patients With Rett Syndrome With Respiratory SymptomsRett SyndromeDrug: Sarizotan;Drug: PlaceboNewron Pharmaceuticals SPANot recruiting4 YearsN/AAll129Phase 2/Phase 3United States;Australia;India;Italy;United Kingdom
9NCT02696044June 201619 February 2018Treatment of Mitochondrial Dysfunction in Rett Syndrome With TriheptanoinTreatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin: An Open-label, 10-subject Clinical Trial of UX007 (Triheptanoin) in the Treatment of Mitochondrial Dysfunction in Participants With Rett Syndrome, Dyskinesia, and EpilepsyRett SyndromeDrug: triheptanoinCenter for Rare Neurological Diseases, Norcross, GAUltragenyx Pharmaceutical Inc;Rett Syndrome Research TrustRecruiting2 YearsN/AFemale12Phase 2United States
10NCT02715115March 20165 February 2018A Safety Study of NNZ-2566 in Pediatric Rett SyndromeA Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett SyndromeRett SyndromeDrug: NNZ-2566;Drug: PlaceboNeuren Pharmaceuticals Limitedrettsyndrome.orgNot recruiting5 Years15 YearsFemale82Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT02563860July 201522 July 2019Pharmacological Treatment of Rett Syndrome With StatinsPharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)Rett SyndromeDrug: LovastatinMontefiore Medical CenterRett Syndrome Research TrustNot recruiting3 YearsN/AFemale20Phase 2United States
12NCT02023424January 201419 February 2015An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett SyndromeAn Open Label, Exploratory Study to Investigate the Treatment Effect og Glatiramer Acetate (Copaxone ®) on Girls Woth Rett SyndromeRett SyndromeDrug: Glatiramer Acetate (Copaxone®)Sheba Medical CenterRecruiting6 Years15 YearsFemale10Phase 1Israel
13NCT02061137August 201325 June 2018Study to Assess Safety and Efficacy of Fingolimod in Children With Rett SyndromeA Phase 1 Clinical Study to Assess Safety and Efficacy of Oral Fingolimod (FTY720) in Children With Rett Syndrome.Rett's SyndromeDrug: fingolimod (FTY720)University Hospital, Basel, SwitzerlandNovartisNot recruiting6 YearsN/AAll6Phase 1/Phase 2Switzerland
14NCT02153723August 201328 April 2015Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)Rett SyndromeDrug: Glatiramer AcetateMontefiore Medical CenterRett Syndrome Research TrustNot recruiting10 YearsN/AFemale20Phase 2United States
15NCT01703533March 201312 February 2018A Safety Study of NNZ-2566 in Patients With Rett SyndromeA Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Escalation Study of NNZ-2566 in Rett SyndromeRett SyndromeDrug: NNZ-2566;Drug: PlaceboNeuren Pharmaceuticals LimitedBaylor College of Medicine;Texas Children's Hospital;International Rett Syndrome FoundationNot recruiting16 Years45 YearsFemale67Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2012-005021-76-IT31/01/201316 December 2013Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett SyndromeRett Syndrome
MedDRA version: 14.1 Level: HLGT Classification code 10021605 Term: Inborn errors of metabolism System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: EPI-743
Product Code: NA
Pharmaceutical Form: Oral solution
CAS Number: 1213269-98-7
Current Sponsor code: EPI-743
Other descriptive name: Alpha-tocotrienol quinone
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 1.5-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use
EDISON PHARMACEUTICALS INC.Not RecruitingFemale: yes
Male: no
Phase 2AItaly
17NCT01777542January 201311 June 2018Treatment of Rett Syndrome With Recombinant Human IGF-1Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With Recombinant Human IGF-1(Mecasermin [rDNA] Injection)Rett SyndromeDrug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1);Drug: PlaceboBoston Children’s HospitalInternational Rett Syndrome FoundationNot recruiting2 Years10 YearsFemale30Phase 2United States
18NCT01822249January 201320 August 2018Phase 2 Study of EPI-743 for Treatment of Rett SyndromeA Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett SyndromeRett SyndromeDrug: EPI-743;Drug: PlaceboEdison Pharmaceuticals IncNot recruitingN/A18 YearsFemale24Phase 2Italy
19NCT01520363March 20125 February 2018Placebo Controlled Trial of Dextromethorphan in Rett SyndromePlacebo Controlled Trial of Dextromethorphan in Rett SyndromeRett SyndromeDrug: dextromethorphan;Drug: placeboHugo W. Moser Research Institute at Kennedy Krieger, Inc.The Johns Hopkins Institute for Clinical and Translational Research (ICTR);FDA Office of Orphan Products DevelopmentNot recruiting1 Year10 YearsAll57Phase 2United States
20NCT01253317December 201016 December 2017Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With IGF-1Rett SyndromeDrug: rhIGF-1Boston Children’s HospitalInternational Rett Syndrome Foundation;Autism SpeaksNot recruiting2 Years12 YearsFemale12Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT00990691February 17, 200920 August 2018Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett SyndromePilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett SyndromeRett SyndromeDrug: Administration of a high dose of desipramine;Drug: Administration of a low dose of desipramine;Drug: Administration of a placeboAssistance Publique Hopitaux De MarseilleNot recruiting4 Years18 YearsFemale36Phase 2France
22EUCTR2008-005571-10-NO2 October 2017DEXTROMETORPHAN IN RETT SYNDROMEDEXTROMETORPHAN IN RETT SYNDROMERett syndrome (RTT) is a neurological disorder with devastating consequences on the brain. It is characterized by stagnation of development followed by regression, both occurring between age 6 months and 3 years. The clinical picture is dominated by cognitive impairment, loss of communication skills, purposeful hand movements, hand stereotypies, progressive deceleration of head growth, and abnormal locomotion. RTT syndrome presently has no effective therapy other than pallitative careProduct Name: Dextromethorphan
Pharmaceutical Form: Capsule, soft
Kennedy Krieger Inst. Johns Hopkins MedicineNot AvailableFemale: yes
Male: no
90Phase 2Norway
23NCT01147575January 200519 February 2015Effects of Creatine Supplementation in Rett SyndromeEffects of Creatine Supplementation in Rett Syndrome: A Randomized, Placebo-controlled TrialRett SyndromeDietary Supplement: Creatine monohydrate;Dietary Supplement: PlaceboMedical University of ViennaNot recruiting3 Years24 YearsFemale21N/AAustria
24NCT00069550September 200419 February 2015Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett SyndromePathogenesis of Rett Syndrome: Natural History and TreatmentRett SyndromeDrug: dextromethorphan;Drug: donepezil hydrochlorideEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Recruiting1 Year15 YearsBoth90Phase 3United States
25NCT00593957August 200419 October 2017Trial of Dextromethorphan in Rett SyndromeTrial of Dextromethorphan in Rett SyndromeRett SyndromeDrug: DextromethorphanHugo W. Moser Research Institute at Kennedy Krieger, Inc.Not recruiting2 Years15 YearsAll38Phase 2United States
No.TrialIDDate_
enrollement
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on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Status
Inclusion_
agemin
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agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT00261508August 199919 February 2015A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD)Efficacy And Safety Of Risperidone In The Treatment Of Children With Autistic Disorder And Other Pervasive Developmental Disorders: A Canadian, Multicenter, Double-Blind, Placebo-Controlled StudyDCild Development Disorders, Pervasive;Autistic Disorder;Developmental Disabilities;Asperger Syndrome;Rett SyndromeDrug: risperidoneJanssen-Ortho Inc., CanadaNot recruiting5 Years12 YearsBoth80Phase 3
27NCT00004807January 199519 February 2015Study of the Pathogenesis of Rett SyndromeRett SyndromeDrug: dextromethorphan;Drug: topiramate;Drug: DonepezilEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Johns Hopkins UniversityNot recruitingN/A45 YearsBoth120N/A

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