DDrare: Database of Drug Development for Rare Diseases

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	EUCTR2015-000711-40-NL (EUCTR)	28/04/2016	17/11/2015	Chronocort®, a slow release medicinal preparation of hydrocortisone, will be compared with currently used glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia seeking to assess its safety, tolerability and effectiveness.	A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia.	Congenital adrenal hyperplasia (CAH); is generally due to 21-hydroxylase deficiency, is a disease of the adrenal cortex characterised by cortisol deficiency with or without aldosterone deficiency, and androgen excess. Subjects with CAH are at risk of developing a number of clinical manifestations, such as obesity in children, insulin resistance, and polycystic ovaries, which may contribute to infertility in women with CAH. Oligomenorrhoea or amenorrhoea may be present in adolescence. MedDRA version: 18.1;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19]	Product Name: Chronocort® Product Code: DIURF-006 INN or Proposed INN: HYDROCORTISONE Other descriptive name: Cortisol Trade Name: Hydrocortisone 20mg Tablets INN or Proposed INN: Hydrocortisone Other descriptive name: HYDROCORTISONE Trade Name: Dexamethasone 1,5 mg Tablets INN or Proposed INN: DEXAMETHASONE Trade Name: Prednisolone 5 mg Tablets INN or Proposed INN: Prednisolone Other descriptive name: PREDNISOLONE	Diurnal Ltd	NULL	Not Recruiting			Female: yes Male: yes	110	Phase 3	United States;Denmark;Germany;Netherlands;United Kingdom;Sweden

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Public_title

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Condition

Intervention

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Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

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EUCTR2015-000711-40-NL
(EUCTR)

28/04/2016

17/11/2015

Chronocort®, a slow release medicinal preparation of hydrocortisone, will be compared with currently used glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia seeking to assess its safety, tolerability and effectiveness.

A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia.

Congenital adrenal hyperplasia (CAH); is generally due to 21-hydroxylase deficiency, is a disease of the adrenal cortex characterised by cortisol deficiency with or without aldosterone deficiency, and androgen excess. Subjects with CAH are at risk of developing a number of clinical manifestations, such as obesity in children, insulin resistance, and polycystic ovaries, which may contribute to infertility in women with CAH. Oligomenorrhoea or amenorrhoea may be present in adolescence.
MedDRA version: 18.1;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Product Name: Chronocort®
Product Code: DIURF-006
INN or Proposed INN: HYDROCORTISONE
Other descriptive name: Cortisol
Trade Name: Hydrocortisone 20mg Tablets
INN or Proposed INN: Hydrocortisone
Other descriptive name: HYDROCORTISONE
Trade Name: Dexamethasone 1,5 mg Tablets
INN or Proposed INN: DEXAMETHASONE
Trade Name: Prednisolone 5 mg Tablets
INN or Proposed INN: Prednisolone
Other descriptive name: PREDNISOLONE

Diurnal Ltd

NULL

Not Recruiting

Female: yes
Male: yes

110

Phase 3

United States;Denmark;Germany;Netherlands;United Kingdom;Sweden