201. Angelman syndrome Clinical trials / Disease details
Clinical trials : 25 / Drugs : 40 - (DrugBank : 11) / Drug target genes : 22 - Drug target pathways : 20
| 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 | NCT04863794 (ClinicalTrials.gov) | April 29, 2021 | 26/4/2021 | A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants | A Non-Randomized, Open-Label, Adaptive, Single Center, Positron Emission Tomography (Pet) Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants | Angelman Syndrome | Drug: RO7248824 | Hoffmann-La Roche | NULL | Completed | 25 Years | 55 Years | Male | 21 | Phase 1 | Netherlands |
| 2 | EUCTR2019-003787-48-NL (EUCTR) | 22/09/2020 | 08/10/2020 | Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. | AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME | Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 Product Code: RO7248824/F03-01 (Diluent for RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 | F. Hoffmann-La Roche | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 1 | United States;Canada;Spain;Netherlands;United Kingdom;Italy | ||
| 3 | NCT04428281 (ClinicalTrials.gov) | August 19, 2020 | 9/6/2020 | A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome | An Open-Label, Multicenter Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome | Angelman Syndrome | Drug: RO7248824 | Hoffmann-La Roche | NULL | Recruiting | 1 Year | 12 Years | All | 74 | Phase 1 | United States;Italy;Netherlands;Spain;Canada;United Kingdom |
| 4 | EUCTR2019-003787-48-IT (EUCTR) | 03/07/2020 | 15/06/2021 | Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. | AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME - TANGELO | Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: - Product Code: [RO7248824/F02-01] INN or Proposed INN: Non disponibile Other descriptive name: RO7248824 | F. HOFFMANN - LA ROCHE LTD. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 1 | United States;Canada;Spain;Netherlands;United Kingdom;Italy | ||
| 5 | EUCTR2019-003787-48-GB (EUCTR) | 11/06/2020 | 16/03/2020 | Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. | AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME | Angelman Syndrome MedDRA version: 20.0;Level: PT;Classification code 10049004;Term: Angelman's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 Product Code: RO7248824/F03-01 (Diluent for RO7248824/F02-01 INN or Proposed INN: Not available Other descriptive name: RO7248824 | F. Hoffmann-La Roche | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 1 | United States;Canada;Spain;Netherlands;Italy;United Kingdom |