156. Rett syndrome
[
27 clinical trials,
34 drugs(DrugBank:
16 drugs),
69 target genes / 105 target pathways ]
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.
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | NCT03848832 | July 29, 2019 | 2 September 2019 | 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 | Drug: GWP42003-P;Drug: Placebo | GW Research Ltd | Recruiting | 2 Years | 18 Years | Female | 252 | Phase 3 | United States;United Kingdom | |
2 | NCT03941444 | May 6, 2019 | 20 May 2019 | ANAVEX2-73 Study in Patients With Rett Syndrome | A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients With Rett Syndrome | Rett Syndrome | Drug: ANAVEX2-73;Drug: Placebos | Anavex Life Sciences Corp. | Recruiting | 18 Years | 45 Years | Female | 30 | Phase 2 | Australia | |
3 | NCT03633058 | March 12, 2019 | 30 September 2019 | A Study to Evaluate Ketamine for the Treatment of Rett Syndrome | A Randomized, Double-blind, Placebo-controlled, Cross-over Study to Assess the Safety, Tolerability and Efficacy of Oral Ketamine for Patients With Rett Syndrome | Rett Syndrome | Drug: Ketamine | Rett Syndrome Research Trust | Vanderbilt University Medical Center | Recruiting | 6 Years | 12 Years | Female | 48 | Phase 2 | United States |
4 | NCT03758924 | February 28, 2019 | 11 November 2019 | Study of ANAVEX2-73 in Patients With Rett Syndrome | A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome | Rett Syndrome | Drug: ANAVEX2-73;Drug: Placebo | Anavex Life Sciences Corp. | International Rett Syndrome Foundation Rettsyndrome.org | Recruiting | 18 Years | 45 Years | Female | 21 | Phase 2 | United States |
5 | NCT03259971 | August 25, 2017 | 25 March 2019 | The Role of Probiotics PS128 in Movement Disorders | The Role of Probiotics PS128 in Movement Disorders | Rett Syndrome;Tourette Syndrome;Tic Disorders | Dietary Supplement: Probiotic-Lactobacillus plantarum PS128;Dietary Supplement: Placebo | National Taiwan University Hospital | Recruiting | 1 Year | 50 Years | All | 500 | N/A | Taiwan | |
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
6 | NCT03059160 | April 1, 2017 | 16 December 2017 | Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. | Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. | Rett Syndrome | Drug: Tridecanoic Acid | Sheba Medical Center | Ultragenyx Pharmaceutical Inc | Not recruiting | 5 Years | 18 Years | Female | 10 | Phase 2 | |
7 | EUCTR2015-004448-20-GB | 28/11/2016 | 7 January 2019 | Sarizotan: 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 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. | Authorised | Female: yes Male: yes | 129 | Phase 2 | United States;Australia;Italy;United Kingdom;India | |||
8 | NCT02790034 | August 2016 | 25 February 2019 | Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms | A Randomised, Double-Blind, Placebo-Controlled 6-month Study to Evaluate the Efficacy, Safety, and Tolerability of Sarizotan in Patients With Rett Syndrome With Respiratory Symptoms | Rett Syndrome | Drug: Sarizotan;Drug: Placebo | Newron Pharmaceuticals SPA | Not recruiting | 4 Years | N/A | All | 129 | Phase 2/Phase 3 | United States;Australia;India;Italy;United Kingdom | |
9 | NCT02696044 | June 2016 | 19 February 2018 | Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin | Treatment 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 Epilepsy | Rett Syndrome | Drug: triheptanoin | Center for Rare Neurological Diseases, Norcross, GA | Ultragenyx Pharmaceutical Inc;Rett Syndrome Research Trust | Recruiting | 2 Years | N/A | Female | 12 | Phase 2 | United States |
10 | NCT02715115 | March 2016 | 5 February 2018 | A Safety Study of NNZ-2566 in Pediatric Rett Syndrome | A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome | Rett Syndrome | Drug: NNZ-2566;Drug: Placebo | Neuren Pharmaceuticals Limited | rettsyndrome.org | Not recruiting | 5 Years | 15 Years | Female | 82 | Phase 2 | United States |
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
11 | NCT02563860 | July 2015 | 22 July 2019 | Pharmacological Treatment of Rett Syndrome With Statins | Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor) | Rett Syndrome | Drug: Lovastatin | Montefiore Medical Center | Rett Syndrome Research Trust | Not recruiting | 3 Years | N/A | Female | 20 | Phase 2 | United States |
12 | NCT02023424 | January 2014 | 19 February 2015 | An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome | An Open Label, Exploratory Study to Investigate the Treatment Effect og Glatiramer Acetate (Copaxone ®) on Girls Woth Rett Syndrome | Rett Syndrome | Drug: Glatiramer Acetate (Copaxone®) | Sheba Medical Center | Recruiting | 6 Years | 15 Years | Female | 10 | Phase 1 | Israel | |
13 | NCT02061137 | August 2013 | 25 June 2018 | Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome | A Phase 1 Clinical Study to Assess Safety and Efficacy of Oral Fingolimod (FTY720) in Children With Rett Syndrome. | Rett's Syndrome | Drug: fingolimod (FTY720) | University Hospital, Basel, Switzerland | Novartis | Not recruiting | 6 Years | N/A | All | 6 | Phase 1/Phase 2 | Switzerland |
14 | NCT02153723 | August 2013 | 28 April 2015 | Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) | Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) | Rett Syndrome | Drug: Glatiramer Acetate | Montefiore Medical Center | Rett Syndrome Research Trust | Not recruiting | 10 Years | N/A | Female | 20 | Phase 2 | United States |
15 | NCT01703533 | March 2013 | 12 February 2018 | A Safety Study of NNZ-2566 in Patients With Rett Syndrome | A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Escalation Study of NNZ-2566 in Rett Syndrome | Rett Syndrome | Drug: NNZ-2566;Drug: Placebo | Neuren Pharmaceuticals Limited | Baylor College of Medicine;Texas Children's Hospital;International Rett Syndrome Foundation | Not recruiting | 16 Years | 45 Years | Female | 67 | Phase 2 | United States |
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
16 | EUCTR2012-005021-76-IT | 31/01/2013 | 16 December 2013 | Phase 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 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 Recruiting | Female: yes Male: no | Phase 2A | Italy | ||||
17 | NCT01777542 | January 2013 | 11 June 2018 | Treatment of Rett Syndrome With Recombinant Human IGF-1 | Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With Recombinant Human IGF-1(Mecasermin [rDNA] Injection) | Rett Syndrome | Drug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1);Drug: Placebo | Boston Children’s Hospital | International Rett Syndrome Foundation | Not recruiting | 2 Years | 10 Years | Female | 30 | Phase 2 | United States |
18 | NCT01822249 | January 2013 | 20 August 2018 | Phase 2 Study of EPI-743 for Treatment of Rett Syndrome | A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome | Rett Syndrome | Drug: EPI-743;Drug: Placebo | Edison Pharmaceuticals Inc | Not recruiting | N/A | 18 Years | Female | 24 | Phase 2 | Italy | |
19 | NCT01520363 | March 2012 | 5 February 2018 | Placebo Controlled Trial of Dextromethorphan in Rett Syndrome | Placebo Controlled Trial of Dextromethorphan in Rett Syndrome | Rett Syndrome | Drug: dextromethorphan;Drug: placebo | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | The Johns Hopkins Institute for Clinical and Translational Research (ICTR);FDA Office of Orphan Products Development | Not recruiting | 1 Year | 10 Years | All | 57 | Phase 2 | United States |
20 | NCT01253317 | December 2010 | 16 December 2017 | Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection) | Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With IGF-1 | Rett Syndrome | Drug: rhIGF-1 | Boston Children’s Hospital | International Rett Syndrome Foundation;Autism Speaks | Not recruiting | 2 Years | 12 Years | Female | 12 | Phase 1 | United States |
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
21 | NCT00990691 | February 17, 2009 | 20 August 2018 | Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome | Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome | Rett Syndrome | Drug: Administration of a high dose of desipramine;Drug: Administration of a low dose of desipramine;Drug: Administration of a placebo | Assistance Publique Hopitaux De Marseille | Not recruiting | 4 Years | 18 Years | Female | 36 | Phase 2 | France | |
22 | EUCTR2008-005571-10-NO | 2 October 2017 | DEXTROMETORPHAN IN RETT SYNDROME | DEXTROMETORPHAN 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 care | Product Name: Dextromethorphan Pharmaceutical Form: Capsule, soft | Kennedy Krieger Inst. Johns Hopkins Medicine | Not Available | Female: yes Male: no | 90 | Phase 2 | Norway | ||||
23 | NCT01147575 | January 2005 | 19 February 2015 | Effects of Creatine Supplementation in Rett Syndrome | Effects of Creatine Supplementation in Rett Syndrome: A Randomized, Placebo-controlled Trial | Rett Syndrome | Dietary Supplement: Creatine monohydrate;Dietary Supplement: Placebo | Medical University of Vienna | Not recruiting | 3 Years | 24 Years | Female | 21 | N/A | Austria | |
24 | NCT00069550 | September 2004 | 19 February 2015 | Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome | Pathogenesis of Rett Syndrome: Natural History and Treatment | Rett Syndrome | Drug: dextromethorphan;Drug: donepezil hydrochloride | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Recruiting | 1 Year | 15 Years | Both | 90 | Phase 3 | United States | |
25 | NCT00593957 | August 2004 | 19 October 2017 | Trial of Dextromethorphan in Rett Syndrome | Trial of Dextromethorphan in Rett Syndrome | Rett Syndrome | Drug: Dextromethorphan | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Not recruiting | 2 Years | 15 Years | All | 38 | Phase 2 | United States | |
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
26 | NCT00261508 | August 1999 | 19 February 2015 | A 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 Study | DCild Development Disorders, Pervasive;Autistic Disorder;Developmental Disabilities;Asperger Syndrome;Rett Syndrome | Drug: risperidone | Janssen-Ortho Inc., Canada | Not recruiting | 5 Years | 12 Years | Both | 80 | Phase 3 | ||
27 | NCT00004807 | January 1995 | 19 February 2015 | Study of the Pathogenesis of Rett Syndrome | Rett Syndrome | Drug: dextromethorphan;Drug: topiramate;Drug: Donepezil | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Johns Hopkins University | Not recruiting | N/A | 45 Years | Both | 120 | N/A |