156. Rett syndrome Clinical trials / Disease details
Clinical trials : 44 / Drugs : 61 - (DrugBank : 23) / Drug target genes : 57 - Drug target pathways : 83
Showing 1 to 10 of 44 diseases
| 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 Phase 2, Double-Blind, Randomized, Placebo-Controlled Clinical Study of VYNT-0126 in the Treatment o ... | 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 Open-label, Randomized, Dose-escalation and Dose-expansion Study of the Safety and Preliminary Effic ... | 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 Role of Pre-/ Post-biotics on Gut Inflammation, Dysbiosis, and Life Quality in Rett Syndr ... | Protective Effects of Pre- and Post-biotics on Gut Inflammation, Microbiota Diversity, Epileptogenesis, and Quality of Life in Rett Syndrome Protective Effects of Pre- and Post-biotics on Gut Inflammation, Microbiota Diversity, Epileptogenes ... | Rett Syndrome;Dysbiosis;Epilepsy;Quality of Life | Dietary Supplement: ALAC, inulin, FOS, and sodium butyrate;Dietary Supplement: Sodium butyrate and zinc oxide Dietary Supplement: ALAC, inulin, FOS, and sodium butyrate;Dietary Supplement: Sodium butyrate and z ... | 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 ... | 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 ... | 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 An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP4 ... | 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] Rett syndrome(RTT) [typical or atypical] MedDRA version: 20.0;Level: PT;Classification code 10077709 ... | 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 Trade Name: CBDsoluzione orale nota come Epidyolex e con tale nome è stato approvato in UE Product Na ... | 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 ANAVEX2-73-RS-003 is a Phase 2/3, Double-blind, Randomized, Placebo-controlled Safety and Efficacy S ... | 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 A Double-Blind, Randomised, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients ... | 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] Rett Syndrome MedDRA version: 20.0;Level: PT;Classification code 10077709;Term: Rett syndrome;System ... | Product Name: ANAVEX2-73 INN or Proposed INN: ANAVEX2-73 (blarcamesine) Other descriptive name: ANA001XHCl (Syntagon) or VEXA-04 (Patheon) Product Name: ANAVEX2-73 INN or Proposed INN: ANAVEX2-73(blarcamesine) Other descriptive name: ANA001X ... | 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 An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP4 ... | 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] Rett syndrome(RTT) [typical or atypical] MedDRA version: 20.0;Level: PT;Classification code 10077709 ... | 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 Trade Name: Epidyolexand is the approved name in the EU Product Name: Cannabidiol(CBD) Product Code: G ... | 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 Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Ret ... | An Open-label Extension Trial to Investigate the Long-term Safety 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 (GWP4 ... | 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 |