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	NCT03548246 (ClinicalTrials.gov)	January 2022	24/4/2015	Androgen Reduction in Congenital Adrenal Hyperplasia	A Phase 1-2 Multi-Center Study to Assess the Efficacy and Safety of Abiraterone Acetate as Adjunctive Therapy in Pre-Pubescent Children With Classic 21-Hydroxylase Deficiency	Congenital Adrenal Hyperplasia	Drug: Abiraterone acetate;Drug: Placebo;Drug: Hydrocortisone;Drug: Fludrocortisone	University of Texas Southwestern Medical Center	National Institutes of Health Clinical Center (CC);University of Michigan;Children's Hospital Los Angeles;Feinstein Institute for Medical Research	Not yet recruiting	2 Years	9 Years	All	54	Phase 2	United States
2	NCT02574910 (ClinicalTrials.gov)	August 1, 2017	24/4/2015	Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1	A Phase 1 Multi-Center Study to Assess the Efficacy and Safety of Abiraterone Acetate as Adjunctive Therapy in Pre-Pubescent Children With Classic 21-Hydroxylase Deficiency	Congenital Adrenal Hyperplasia	Drug: Abiraterone acetate	University of Texas Southwestern Medical Center	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);University of Michigan;Children's Hospital Los Angeles	Active, not recruiting	2 Years	9 Years	All	36	Phase 1	United States
3	NCT01495910 (ClinicalTrials.gov)	December 2011	23/11/2011	A Study Examining Doses of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency	An Open-Label, Multiple-Dose, Dose-Finding Study of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency	21-hydroxylase Deficiency	Drug: Abiraterone acetate	Johnson & Johnson Pharmaceutical Research & Development, L.L.C.	NULL	Completed	18 Years	N/A	Female	6	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

NCT03548246
(ClinicalTrials.gov)

January 2022

24/4/2015

Androgen Reduction in Congenital Adrenal Hyperplasia

A Phase 1-2 Multi-Center Study to Assess the Efficacy and Safety of Abiraterone Acetate as Adjunctive Therapy in Pre-Pubescent Children With Classic 21-Hydroxylase Deficiency

Congenital Adrenal Hyperplasia

Drug: Abiraterone acetate;Drug: Placebo;Drug: Hydrocortisone;Drug: Fludrocortisone

University of Texas Southwestern Medical Center

National Institutes of Health Clinical Center (CC);University of Michigan;Children's Hospital Los Angeles;Feinstein Institute for Medical Research

Not yet recruiting

2 Years

9 Years

All

Phase 2

United States

NCT02574910
(ClinicalTrials.gov)

August 1, 2017

24/4/2015

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

A Phase 1 Multi-Center Study to Assess the Efficacy and Safety of Abiraterone Acetate as Adjunctive Therapy in Pre-Pubescent Children With Classic 21-Hydroxylase Deficiency

Congenital Adrenal Hyperplasia

Drug: Abiraterone acetate

University of Texas Southwestern Medical Center

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);University of Michigan;Children's Hospital Los Angeles

Active, not recruiting

2 Years

9 Years

All

Phase 1

United States

NCT01495910
(ClinicalTrials.gov)

December 2011

23/11/2011

A Study Examining Doses of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency

An Open-Label, Multiple-Dose, Dose-Finding Study of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency

21-hydroxylase Deficiency

Drug: Abiraterone acetate

Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

NULL

Completed

18 Years

N/A

Female

Phase 1

United States