GTX-102 ( DrugBank: GTX-102 )


2 diseases
IDDisease name (Link within this page)Number of trials
65Primary immunodeficiency1
201Angelman syndrome1

65. Primary immunodeficiency


Clinical trials : 500 Drugs : 614 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 217
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT05531890
(ClinicalTrials.gov)
September 13, 202231/8/2022Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy SubjectsCrossover Study to Evaluate the Comparative Bioavailability, Pharmacokinetics, and Safety of GTX-102 Administered as an Oral Spray Compared to Intramuscular Injection and an Oral Solution of Betamethasone in Healthy SubjectsAtaxia TelangiectasiaDrug: GTX-102 medium dose fast Period 1 and Period 2;Drug: GTX-102 medium dose slow Period 1 and Period 2;Drug: GTX-102 high dose fast Period 1 and Period 2;Drug: GTX-102 low dose fast Period 1 and Period 2;Drug: Betamethasone solution as intramuscular injection Period 1 and Period 2;Drug: Betamethasone Oral Solution Period 1 and Period 2Acasti Pharma Inc.NULLActive, not recruiting18 Years55 YearsAll48Phase 1Canada

201. Angelman syndrome


Clinical trials : 25 Drugs : 40 - (DrugBank : 11) / Drug target genes : 22 - Drug target pathways : 20
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04259281
(ClinicalTrials.gov)
February 24, 202031/1/2020A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeA Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)Angelman SyndromeDrug: GTX-102Ultragenyx Pharmaceutical IncNULLRecruiting4 Years17 YearsAll83Phase 1/Phase 2United States;Canada;United Kingdom