DDrare: Database of Drug Development for Rare Diseases

81. 先天性副腎皮質酵素欠損症
［臨床試験数：65，薬物数：77（DrugBank：20），標的遺伝子数：11，標的パスウェイ数：65］

Searched query = "Congenital adrenal hyperplasia", "Congenital adrenal enzyme deficiency", "Congenial adrenal cortex enzyme deficiency", "Congenital Lipoid Adrenal Hyperplasia", "3β-Hydroxysteroid Dehydrogenase Deficiency", "21-Hydroxylase deficiency", "11β-Hydroxylase deficiency", "17α-Hydroxylase deficiency", "P450 oxidoreductase deficiency", "Aldosterone synthase deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = ATR-101; Abiraterone; Abiraterone acetate; Acetate; Balance; Behavioral: quality of life assessment; Bone balance measurements; CI-984, 17AA70, PD 132301-2; Chronocort; Chronocort®; Chronocort® 10 mg; Chronocort® 20 mg; Chronocort® 5 mg; Conventional Glucocorticoids (immediate release hydrocortisone, cortisone acetate, prednisone, prednisolone, dexamethasone); Cortef; Cortisol; Cortison; Cortisone; Cortisone acetate; Crinecerfont; DEXAMETHASONE; DIURF-006; Dectancyl; Device: Insulin pump (Medtronic); Dexamethasone; Dexamethasone 0,1 mg Tablets; Dexamethasone 0.5mg Tablet; Dexamethasone 1,5 mg Tablets; Dexamethasone 2mg Tablets; Dual release hydrocortisone (plenadren); Fludrocortisone; Flutamide; HYDROCORTISONE; HYDROCORTISONE SODIUM SUCCINATE; Hormonal balance measurements; Hydrocortisone; Hydrocortisone (Solucortef); Hydrocortisone 10mg Tablet; Hydrocortisone 10mg Tablets; Hydrocortisone 20mg Tablets; Hydrocortisone Modified Release Capsules; Hydrocortisone sodium acetate; Hydrocortisone sodium phosphate; Hydrocortisone tablet 2.5 mg; Infacort; Infacort®; Letrozole; METFORMIN HYDROCHLORIDE; Metabolic balance measurements; Metformin; Metformine; Metformine Sandoz 850 mg filmomhulde tabletten; NBI-74788; NBI-77860; Nevanimibe; Nevanimibe Hydrochloride; Nevanimibe hydrochloride; Nifedipine; PREDNISOLONE; Phosphate; Pioglitazone; Prednisolone; Prednisolone 1mg Tablets; Prednisolone 2,5 mg Tablets; Prednisolone 5 mg Tablets; Prednisolone 5mg Tablet; Prednisone; Procedure: Hydrocortisone withdrawal; SPR001; Sodium acetate; Solu-Cortef; Solu-cortef; Standard Care; Standard glucocorticoid therapy; Subcutaneous hydrocortisone; TILDACERFONT; Tildacerfont

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	NCT02349503 (ClinicalTrials.gov)	February 2015	20/1/2015	Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia	A Phase 1, Open-Label, Single-Dose, Sequential Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia	Congenital Adrenal Hyperplasia	Drug: NBI-77860	Neurocrine Biosciences	NULL	Withdrawn	12 Years	18 Years	Female	0	Phase 1	United States

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

NCT02349503
(ClinicalTrials.gov)

February 2015

20/1/2015

Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia

A Phase 1, Open-Label, Single-Dose, Sequential Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia

Congenital Adrenal Hyperplasia

Drug: NBI-77860

Neurocrine Biosciences

NULL

Withdrawn

12 Years

18 Years

Female

Phase 1

United States