DDrare: Database of Drug Development for Rare Diseases

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.

Drug = Acetate; Administration of a high dose of desipramine; Administration of a low dose of desipramine; Alpha-tocotrienol quinone; Anavex2-73; Creatine; Creatine monohydrate; Desipramine; Dextromethorphan; Donepezil; Donepezil hydrochloride; Emd 128130; Epi-743; Fingolimod; Fingolimod (fty720); Glatiramer; Glatiramer acetate; Glatiramer acetate (copaxone®); Gwp42003-p; Human insulin; Ketamine; Lactobacillus plantarum; Lovastatin; Nnz-2566; Probiotic-lactobacillus plantarum ps128; Recombinant human insulin growth factor 1 (rhigf-1); Rhigf-1; Risperidone; Sarizotan; Sarizotan hydrochloride; Tocotrienol; Topiramate; Tridecanoic acid; Triheptanoin

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
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

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