156. Rett syndrome Clinical trials / Disease details
Clinical trials : 44 / Drugs : 61 - (DrugBank : 23) / Drug target genes : 57 - Drug target pathways : 83
| 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 | NCT05625568 (ClinicalTrials.gov) | March 2023 | 7/11/2022 | Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients | Phase 2, Double-Blind, Randomized, Placebo-Controlled Clinical Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Female Patients | Rett Syndrome | Drug: VYNT-0126;Drug: Placebo | Vyant Bio | NULL | Not yet recruiting | 18 Years | 45 Years | Female | 48 | Phase 2 | NULL |
| 2 | NCT05606614 (ClinicalTrials.gov) | November 8, 2022 | 28/10/2022 | Safety and Efficacy of TSHA-102 in Adult Females With Rett Syndrome (REVEAL Adult Study) | Open-label, Randomized, Dose-escalation and Dose-expansion Study of the Safety and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, in the Treatment of Adult Females With Rett Syndrome | Rett Syndrome | Genetic: TSHA-102 | Taysha Gene Therapies, Inc. | NULL | Recruiting | 18 Years | N/A | Female | 12 | Phase 1/Phase 2 | Canada |
| 3 | NCT05420805 (ClinicalTrials.gov) | April 1, 2022 | 13/6/2022 | Protective Role of Pre-/ Post-biotics on Gut Inflammation, Dysbiosis, and Life Quality in Rett Syndrome (Biotics_RTT) | Protective Effects of Pre- and Post-biotics on Gut Inflammation, Microbiota Diversity, Epileptogenesis, and Quality of Life in Rett Syndrome | Rett Syndrome;Dysbiosis;Epilepsy;Quality of Life | Dietary Supplement: ALAC, inulin, FOS, and sodium butyrate;Dietary Supplement: Sodium butyrate and zinc oxide | Azienda Ospedaliera Universitaria Senese | Kolfarma s.r.l. - Italy;European Institute of Oncology | Recruiting | 3 Years | N/A | Female | 30 | N/A | Italy |
| 4 | NCT04988867 (ClinicalTrials.gov) | September 22, 2021 | 23/7/2021 | An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome | An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome | Rett Syndrome | Drug: Trofinetide | ACADIA Pharmaceuticals Inc. | NULL | Active, not recruiting | 2 Years | 5 Years | Female | 15 | Phase 2/Phase 3 | United States |
| 5 | NCT04776746 (ClinicalTrials.gov) | November 8, 2020 | 29/11/2020 | Open-Label Extension Study of Trofinetide for Rett Syndrome | An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome | Rett Syndrome | Drug: trofinetide | ACADIA Pharmaceuticals Inc. | NULL | Enrolling by invitation | 5 Years | 22 Years | Female | 153 | Phase 3 | United States |
| 6 | EUCTR2019-001605-24-IT (EUCTR) | 02/11/2020 | 21/01/2021 | An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome | An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in patients with Rett Syndrome - NA | 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 soluzione orale nota come Epidyolex e con tale nome è stato approvato in UE Product Name: Cannabidiolo Product Code: [GWP42003-P] INN or Proposed INN: cannabidiolo Other descriptive name: cannabidiol | GW RESEARCH LTD. | NULL | NA | Female: yes Male: no | 252 | Phase 3 | United States;Canada;Spain;Australia;United Kingdom;Italy | ||
| 7 | NCT04304482 (ClinicalTrials.gov) | July 1, 2020 | 8/3/2020 | ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome | ANAVEX2-73-RS-003 is a Phase 2/3, Double-blind, Randomized, Placebo-controlled Safety and Efficacy Study in Pediatric Patients With RTT | Rett Syndrome | Drug: ANAVEX2-73 oral liquid;Drug: Placebo oral liquid | Anavex Life Sciences Corp. | Anavex Australia Pty Ltd.;Anavex Germany GmbH | Active, not recruiting | 5 Years | 17 Years | Female | 92 | Phase 2/Phase 3 | Australia;Canada;United Kingdom |
| 8 | EUCTR2019-004345-32-GB (EUCTR) | 26/05/2020 | 07/02/2020 | The use of ANAVEX2-73 as a new therapeutic treatment for Patients with Rett Syndrome | A 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 GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: no | 30 | Phase 2 | Australia;United Kingdom | ||
| 9 | EUCTR2019-001605-24-ES (EUCTR) | 04/03/2020 | 21/02/2020 | An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome | An 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: 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 Ltd | NULL | Not Recruiting | Female: yes Male: no | 252 | Phase 3 | United States;Canada;Spain;Australia;United Kingdom;Italy | ||
| 10 | NCT04252586 (ClinicalTrials.gov) | February 28, 2020 | 30/1/2020 | An Open-label Extension Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome | An 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 | Drug: GWP42003-P | GW Research Ltd | NULL | Terminated | 2 Years | 18 Years | All | 21 | Phase 3 | United States;Australia;Canada;Italy;Spain;United Kingdom |
| 11 | NCT04279314 (ClinicalTrials.gov) | January 29, 2020 | 19/2/2020 | Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome | A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome | Rett Syndrome | Drug: Trofinetide | ACADIA Pharmaceuticals Inc. | NULL | Completed | 5 Years | 21 Years | Female | 154 | Phase 3 | United States |
| 12 | EUCTR2019-001605-24-GB (EUCTR) | 15/01/2020 | 23/10/2019 | An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome | An 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 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 252 | Phase 3 | United States;France;Canada;Spain;Australia;Italy;United Kingdom | ||
| 13 | NCT04181723 (ClinicalTrials.gov) | November 6, 2019 | 26/11/2019 | Study 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 Syndrome | Rett Syndrome | Drug: Trofinetide;Other: Placebo | ACADIA Pharmaceuticals Inc. | NULL | Completed | 5 Years | 20 Years | Female | 187 | Phase 3 | United States |
| 14 | EUCTR2018-003370-27-IT (EUCTR) | 18/09/2019 | 24/05/2021 | 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. | 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. - Not applicable | 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 - Solution Orale, conosciuto come Epidiolex, questo è il nome approvato negli Stati Uniti Product Name: Cannabidiolo (CBD) Product Code: [GWP42003-P] INN or Proposed INN: Cannabidiolo Other descriptive name: Cannabidiol | GW RESEARCH LTD. | NULL | Not Recruiting | Female: yes Male: no | 252 | Phase 3 | United States;Canada;Spain;Germany;United Kingdom;Italy | ||
| 15 | NCT04041713 (ClinicalTrials.gov) | September 1, 2019 | 27/6/2019 | A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome | A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome | Rett Syndrome | Drug: Rett-T;Other: Placebo | Anagnostou, Evdokia, M.D. | Ontario Brain Institute;St. Michael's Hospital, Toronto;Holland Bloorview Kids Rehabilitation Hospital | Not yet recruiting | 2 Years | 21 Years | Female | 35 | Phase 2 | Canada |
| 16 | NCT03848832 (ClinicalTrials.gov) | July 29, 2019 | 19/2/2019 | 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 | Jazz Pharmaceuticals | NULL | Terminated | 2 Years | 18 Years | All | 29 | Phase 3 | United States;Italy;Spain;United Kingdom |
| 17 | NCT03941444 (ClinicalTrials.gov) | May 6, 2019 | 6/5/2019 | 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: Placebo | Anavex Life Sciences Corp. | NULL | Completed | 18 Years | 45 Years | Female | 33 | Phase 3 | Australia;United Kingdom |
| 18 | EUCTR2018-003370-27-GB (EUCTR) | 09/04/2019 | 24/12/2018 | 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. | 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 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 252 | Phase 2;Phase 3 | United States;France;Canada;Spain;Australia;Italy;United Kingdom | ||
| 19 | NCT03633058 (ClinicalTrials.gov) | March 12, 2019 | 8/8/2018 | 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 | Completed | 6 Years | 12 Years | Female | 24 | Phase 2 | United States |
| 20 | NCT03758924 (ClinicalTrials.gov) | February 28, 2019 | 25/11/2018 | 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 | Completed | 18 Years | 45 Years | Female | 31 | Phase 2 | United States |
| 21 | NCT03259971 (ClinicalTrials.gov) | August 25, 2017 | 16/8/2017 | 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 | NULL | Recruiting | 1 Year | 50 Years | All | 500 | N/A | Taiwan |
| 22 | NCT03059160 (ClinicalTrials.gov) | April 1, 2017 | 2/2/2017 | 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 yet recruiting | 5 Years | 18 Years | Female | 10 | Phase 2 | NULL |
| 23 | EUCTR2015-004448-20-GB (EUCTR) | 28/11/2016 | 23/09/2016 | 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 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. | NULL | Not Recruiting | Female: yes Male: yes | 129 | Phase 2 | United States;Australia;Italy;United Kingdom;India | ||
| 24 | NCT02790034 (ClinicalTrials.gov) | August 2016 | 24/5/2016 | 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 | NULL | Terminated | 4 Years | N/A | All | 129 | Phase 2/Phase 3 | United States;Australia;India;Italy;United Kingdom |
| 25 | NCT02696044 (ClinicalTrials.gov) | June 2016 | 25/2/2016 | 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 |
| 26 | NCT02715115 (ClinicalTrials.gov) | March 2016 | 21/2/2016 | 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 | Completed | 5 Years | 15 Years | Female | 82 | Phase 2 | United States |
| 27 | NCT02563860 (ClinicalTrials.gov) | July 2015 | 20/7/2015 | 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 | Completed | 3 Years | N/A | Female | 20 | Phase 2 | United States |
| 28 | NCT02023424 (ClinicalTrials.gov) | January 2014 | 23/12/2013 | 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 | NULL | Recruiting | 6 Years | 15 Years | Female | 10 | Phase 1 | Israel |
| 29 | NCT02153723 (ClinicalTrials.gov) | August 2013 | 26/5/2014 | 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 | Completed | 10 Years | N/A | Female | 10 | Phase 2 | United States |
| 30 | NCT02061137 (ClinicalTrials.gov) | August 2013 | 27/8/2013 | 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 | Completed | 6 Years | N/A | All | 6 | Phase 1/Phase 2 | Switzerland |
| 31 | NCT01703533 (ClinicalTrials.gov) | March 2013 | 4/10/2012 | 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;International Rett Syndrome Foundation | Completed | 16 Years | 45 Years | Female | 67 | Phase 2 | United States |
| 32 | EUCTR2012-005021-76-IT (EUCTR) | 31/01/2013 | 11/12/2012 | 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 Other descriptive name: Alpha-tocotrienol quinone | EDISON PHARMACEUTICALS INC. | NULL | Not Recruiting | Female: yes Male: no | Phase 2 | Italy | |||
| 33 | NCT01777542 (ClinicalTrials.gov) | January 2013 | 23/1/2013 | 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 | Completed | 2 Years | 10 Years | Female | 30 | Phase 2 | United States |
| 34 | NCT01822249 (ClinicalTrials.gov) | January 2013 | 25/3/2013 | 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 | NULL | Completed | N/A | 18 Years | Female | 24 | Phase 2 | Italy |
| 35 | NCT01520363 (ClinicalTrials.gov) | March 2012 | 25/1/2012 | 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) | Completed | 1 Year | 10 Years | All | 57 | Phase 2 | United States |
| 36 | NCT01253317 (ClinicalTrials.gov) | December 2010 | 2/12/2010 | 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 | Completed | 2 Years | 12 Years | Female | 12 | Phase 1 | United States |
| 37 | NCT00990691 (ClinicalTrials.gov) | February 17, 2009 | 6/10/2009 | 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 | NULL | Completed | 4 Years | 18 Years | Female | 36 | Phase 2 | France |
| 38 | NCT05352373 (ClinicalTrials.gov) | March 1, 2008 | 15/4/2022 | The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome | The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome | Rett Syndrome | Dietary Supplement: calcium;Dietary Supplement: placebo | Baylor College of Medicine | NULL | Completed | 5 Years | 30 Years | Female | 32 | N/A | NULL |
| 39 | NCT01147575 (ClinicalTrials.gov) | January 2005 | 17/6/2010 | 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 | NULL | Completed | 3 Years | 24 Years | Female | 21 | N/A | Austria |
| 40 | NCT00069550 (ClinicalTrials.gov) | September 2004 | 29/9/2003 | 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) | NULL | Recruiting | 1 Year | 15 Years | Both | 90 | Phase 3 | United States |
| 41 | NCT00593957 (ClinicalTrials.gov) | August 2004 | 4/1/2008 | 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. | NULL | Terminated | 2 Years | 15 Years | All | 38 | Phase 2 | United States |
| 42 | NCT00261508 (ClinicalTrials.gov) | August 1999 | 2/12/2005 | 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 | NULL | Completed | 5 Years | 12 Years | Both | 80 | Phase 3 | NULL |
| 43 | NCT00004807 (ClinicalTrials.gov) | January 1995 | 24/2/2000 | 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 | Completed | N/A | 45 Years | Both | 120 | N/A | NULL | |
| 44 | EUCTR2008-005571-10-NO (EUCTR) | 09/01/2009 | 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 | Kennedy Krieger Inst. Johns Hopkins Medicine | NULL | NA | Female: yes Male: no | 90 | Phase 2 | Norway |