Ex-vivo approach concerning 25 patients ( DrugBank: - )


2 diseases
IDDisease name (Link within this page)Number of trials
102Rubinstein-Taybi syndrome1
299Cystic fibrosis1

102. Rubinstein-Taybi syndrome


Clinical trials : 3 Drugs : 5 - (DrugBank : 1) / Drug target genes : 7 - Drug target pathways : 17
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05696912
(ClinicalTrials.gov)
January 30, 20237/12/2022Functional Tests to Resolve Unsolved Rare Diseases. Rares.Resolving Unsolved Rare Diseases : Functional Tests and New Diagnosis Strategy to Study Genetic Variants From High-throughput Sequencing (RID)Intellectual Disability;Rubinstein-Taybi Syndrome;Cystic Fibrosis;Congenital Heart Defect;Periventricular Nodular Heterotopia;Neurodegeneration With Brain Iron Accumulation (NBIA);AlbinismGenetic: Ex-vivo approach concerning 25 patients;Genetic: In-vitro approach concerning 25 patientsUniversity Hospital, BordeauxNULLRecruitingN/AN/AAll50N/AFrance

299. Cystic fibrosis


Clinical trials : 1,695 Drugs : 1,527 - (DrugBank : 268) / Drug target genes : 111 - Drug target pathways : 174
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05696912
(ClinicalTrials.gov)
January 30, 20237/12/2022Functional Tests to Resolve Unsolved Rare Diseases. Rares.Resolving Unsolved Rare Diseases : Functional Tests and New Diagnosis Strategy to Study Genetic Variants From High-throughput Sequencing (RID)Intellectual Disability;Rubinstein-Taybi Syndrome;Cystic Fibrosis;Congenital Heart Defect;Periventricular Nodular Heterotopia;Neurodegeneration With Brain Iron Accumulation (NBIA);AlbinismGenetic: Ex-vivo approach concerning 25 patients;Genetic: In-vitro approach concerning 25 patientsUniversity Hospital, BordeauxNULLRecruitingN/AN/AAll50N/AFrance