156. Rett syndrome
36 clinical trials,   49 drugs   (DrugBank: 19 drugs),   77 drug target genes,   112 drug target pathways

Searched query = "Rett syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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1NCT04304482
(ClinicalTrials.gov)
July 1, 20208/3/2020ANAVEX2-73 Study in Pediatric Patients With Rett SyndromeA Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett SyndromeRett SyndromeDrug: ANAVEX2-73 oral liquid;Drug: Placebo oral liquidAnavex Life Sciences Corp.Anavex Australia Pty Ltd.;Anavex Germany GmbHRecruiting5 Years18 YearsFemale69Phase 2;Phase 3Australia
2EUCTR2019-004345-32-GB
(EUCTR)
26/05/202007/02/2020The use of ANAVEX2-73 as a new therapeutic treatment for Patients with Rett SyndromeA Double-Blind, Randomised, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients with Rett Syndrome - The Safety and Efficacy of ANAVEX2-73 in Patients with Rett Syndrome Rett Syndrome
MedDRA version: 20.0;Level: PT;Classification code 10077709;Term: Rett syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: ANAVEX2-73
INN or Proposed INN: ANAVEX2-73 (blarcamesine)
Other descriptive name: ANA001XHCl (Syntagon) or VEXA-04 (Patheon)
Anavex Germany GmbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: no
30Phase 2Australia;United Kingdom
3EUCTR2019-001605-24-ES
(EUCTR)
04/03/202021/02/2020An open label extension study for long-term safety of GWP42003-P in patients with Rett SyndromeAn open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in patients with Rett Syndrome Rett syndrome (RTT) [typical or atypical]
MedDRA version: 20.0;Level: PT;Classification code 10077709;Term: Rett syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
GW Research LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: no
252Phase 3United States;Canada;Spain;Australia;Italy;United Kingdom
4NCT04252586
(ClinicalTrials.gov)
February 28, 202030/1/2020An Open-label Extension Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett SyndromeAn Open-label Extension Trial to Investigate the Long-term Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett SyndromeRett Syndrome;RTTDrug: GWP42003-PGW Research LtdNULLEnrolling by invitation2 Years18 YearsAll252Phase 3United States;Australia;Canada;Italy;Spain;United Kingdom
5NCT04279314
(ClinicalTrials.gov)
January 29, 202019/2/2020Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett SyndromeA 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett SyndromeRett SyndromeDrug: TrofinetideACADIA Pharmaceuticals Inc.NULLEnrolling by invitation5 Years21 YearsFemale180Phase 3United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
6EUCTR2019-001605-24-GB
(EUCTR)
15/01/202023/10/2019An open label extension study for long-term safety of GWP42003-P in patients with Rett SyndromeAn open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in patients with Rett Syndrome Rett syndrome (RTT) [typical or atypical]
MedDRA version: 20.0;Level: PT;Classification code 10077709;Term: Rett syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: CBD Oral solution, is known as Epidyolex and is the approved name in the EU
Product Name: Cannabidiol (CBD)
Product Code: GWP42003-P
INN or Proposed INN: Cannabidiol
Other descriptive name: CANNABIDIOL
GW Research LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
252Phase 3United States;France;Canada;Spain;Australia;Italy;United Kingdom
7NCT04181723
(ClinicalTrials.gov)
November 6, 201926/11/2019Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett SyndromeRett SyndromeDrug: Trofinetide;Other: PlaceboACADIA Pharmaceuticals Inc.NULLRecruiting5 Years20 YearsFemale184Phase 3United States
8NCT04041713
(ClinicalTrials.gov)
September 1, 201927/6/2019A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett SyndromeA Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett SyndromeRett SyndromeDrug: Rett-T;Other: PlaceboAnagnostou, Evdokia, M.D.Ontario Brain Institute;St. Michael's Hospital, Toronto;Holland Bloorview Kids Rehabilitation HospitalNot yet recruiting2 Years21 YearsFemale35Phase 2Canada
9NCT03848832
(ClinicalTrials.gov)
July 29, 201919/2/2019Efficacy 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 LtdNULLActive, not recruiting2 Years18 YearsFemale252Phase 3United States;Italy;Spain;United Kingdom
10NCT03941444
(ClinicalTrials.gov)
May 6, 20196/5/2019ANAVEX2-73 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.NULLRecruiting18 Years45 YearsFemale30Phase 2Australia;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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11EUCTR2018-003370-27-GB
(EUCTR)
09/04/201924/12/2018A randomized, double-blind, placebo-controlled trial to investigate the efficacy and safety of cannabidiol oral solution (GWP42003-P; CBD-OS) in patients with Rett syndrome.A randomized, double-blind, placebo-controlled trial to investigate the efficacy and safety of cannabidiol oral solution (GWP42003-P; CBD-OS) in patients with Rett syndrome. Rett syndrome (RTT) [typical or atypical]
MedDRA version: 20.0;Level: PT;Classification code 10077709;Term: Rett syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: CBD - Oral Solution, is known as Epidyolex, and is the approved name
Product Name: Cannabidiol (CBD)
Product Code: GWP42003-P
INN or Proposed INN: Cannabidiol
Other descriptive name: CANNABIDIOL
GW Research LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
252Phase 2;Phase 3United States;France;Canada;Spain;Australia;Italy;United Kingdom
12NCT03633058
(ClinicalTrials.gov)
March 12, 20198/8/2018A 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
13NCT03758924
(ClinicalTrials.gov)
February 28, 201925/11/2018Study 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.orgActive, not recruiting18 Years45 YearsFemale31Phase 2United States
14NCT03259971
(ClinicalTrials.gov)
August 25, 201716/8/2017The 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 HospitalNULLRecruiting1 Year50 YearsAll500N/ATaiwan
15NCT03059160
(ClinicalTrials.gov)
April 1, 20172/2/2017Open 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 yet recruiting5 Years18 YearsFemale10Phase 2NULL
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
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agemin
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PhaseCountries
16EUCTR2015-004448-20-GB
(EUCTR)
28/11/201623/09/2016Sarizotan: 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 Symptoms Rett 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
INN or Proposed INN: sarizotan hydrochloride
Other descriptive name: SARIZOTAN HYDROCHLORIDE
Product Name: sarizotan hydrochloride
Product Code: EMD 128130
INN or Proposed INN: sarizotan hydrochloride
Other descriptive name: SARIZOTAN HYDROCHLORIDE
Product Name: sarizotan hydrochloride
Product Code: EMD 128130
INN or Proposed INN: sarizotan hydrochloride
Other descriptive name: SARIZOTAN HYDROCHLORIDE
Newron Pharmaceuticals S.p.A.NULLNot RecruitingFemale: yes
Male: yes
129Phase 2United States;Australia;Italy;United Kingdom;India
17NCT02790034
(ClinicalTrials.gov)
August 201624/5/2016Evaluation 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 SPANULLTerminated4 YearsN/AAll129Phase 2;Phase 3United States;Australia;India;Italy;United Kingdom
18NCT02696044
(ClinicalTrials.gov)
June 201625/2/2016Treatment 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
19NCT02715115
(ClinicalTrials.gov)
March 201621/2/2016A 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.orgCompleted5 Years15 YearsFemale82Phase 2United States
20NCT02563860
(ClinicalTrials.gov)
July 201520/7/2015Pharmacological 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 TrustCompleted3 YearsN/AFemale20Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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21NCT02023424
(ClinicalTrials.gov)
January 201423/12/2013An 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 CenterNULLRecruiting6 Years15 YearsFemale10Phase 1Israel
22NCT02153723
(ClinicalTrials.gov)
August 201326/5/2014Pharmacological 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 TrustCompleted10 YearsN/AFemale10Phase 2United States
23NCT02061137
(ClinicalTrials.gov)
August 201327/8/2013Study 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, SwitzerlandNovartisCompleted6 YearsN/AAll6Phase 1;Phase 2Switzerland
24NCT01703533
(ClinicalTrials.gov)
March 20134/10/2012A 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;International Rett Syndrome FoundationCompleted16 Years45 YearsFemale67Phase 2United States
25EUCTR2012-005021-76-IT
(EUCTR)
31/01/201311/12/2012Phase 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 Syndrome Rett 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
Other descriptive name: Alpha-tocotrienol quinone
EDISON PHARMACEUTICALS INC.NULLNot RecruitingFemale: yes
Male: no
Phase 2AItaly
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
26NCT01822249
(ClinicalTrials.gov)
January 201325/3/2013Phase 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 IncNULLCompletedN/A18 YearsFemale24Phase 2Italy
27NCT01777542
(ClinicalTrials.gov)
January 201323/1/2013Treatment 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 FoundationCompleted2 Years10 YearsFemale30Phase 2United States
28NCT01520363
(ClinicalTrials.gov)
March 201225/1/2012Placebo 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)Completed1 Year10 YearsAll57Phase 2United States
29NCT01253317
(ClinicalTrials.gov)
December 20102/12/2010Treatment 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 SpeaksCompleted2 Years12 YearsFemale12Phase 1United States
30NCT00990691
(ClinicalTrials.gov)
February 17, 20096/10/2009Pilot 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 MarseilleNULLCompleted4 Years18 YearsFemale36Phase 2France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
31NCT01147575
(ClinicalTrials.gov)
January 200517/6/2010Effects 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 ViennaNULLCompleted3 Years24 YearsFemale21N/AAustria
32NCT00069550
(ClinicalTrials.gov)
September 200429/9/2003Independent 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)NULLRecruiting1 Year15 YearsBoth90Phase 3United States
33NCT00593957
(ClinicalTrials.gov)
August 20044/1/2008Trial of Dextromethorphan in Rett SyndromeTrial of Dextromethorphan in Rett SyndromeRett SyndromeDrug: DextromethorphanHugo W. Moser Research Institute at Kennedy Krieger, Inc.NULLTerminated2 Years15 YearsAll38Phase 2United States
34NCT00261508
(ClinicalTrials.gov)
August 19992/12/2005A 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., CanadaNULLCompleted5 Years12 YearsBoth80Phase 3NULL
35NCT00004807
(ClinicalTrials.gov)
January 199524/2/2000Study 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 UniversityCompletedN/A45 YearsBoth120N/ANULL
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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36EUCTR2008-005571-10-NO
(EUCTR)
09/01/2009DEXTROMETORPHAN IN RETT SYNDROMEDEXTROMETORPHAN IN RETT SYNDROME Rett 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: DextromethorphanKennedy Krieger Inst. Johns Hopkins MedicineNULLNAFemale: yes
Male: no
90Phase 2Norway