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	EUCTR2019-004873-17-PL (EUCTR)	01/12/2020	03/07/2020	A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia	A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment	Classic Congenital Adrenal Hyperplasia (CAH) MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19]	Product Name: Crinecerfont Product Code: NBI-74788 INN or Proposed INN: Crinecerfont	Neurocrine Biosciences, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	165	Phase 3	United States;Portugal;Czechia;Greece;Spain;Austria;United Kingdom;Italy;France;Poland;Belgium;Bulgaria;Germany;Netherlands;Sweden
2	EUCTR2019-004873-17-GB (EUCTR)	15/10/2020	30/04/2020	A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia	A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment	Classic Congenital Adrenal Hyperplasia (CAH) MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19]	Product Name: Crinecerfont Product Code: NBI-74788 INN or Proposed INN: Crinecerfont	Neurocrine Biosciences, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	165	Phase 3	United States;Portugal;Greece;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Belgium;Poland;Bulgaria;Germany;Netherlands;Sweden
3	EUCTR2019-004873-17-PT (EUCTR)	12/10/2020	30/06/2020	A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia	A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment	Classic Congenital Adrenal Hyperplasia (CAH) MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19]	Product Name: Crinecerfont Product Code: NBI-74788 INN or Proposed INN: Crinecerfont	Neurocrine Biosciences, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	165	Phase 3	Italy;France;Czech Republic;Poland;Belgium;Bulgaria;Netherlands;Germany;Sweden;Portugal;United States;Greece;Spain;Austria;United Kingdom
4	EUCTR2019-004873-17-GR (EUCTR)	29/07/2020	30/07/2020	A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia	A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment	Classic Congenital Adrenal Hyperplasia (CAH) MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19]	Product Name: Crinecerfont Product Code: NBI-74788 INN or Proposed INN: Crinecerfont	Neurocrine Biosciences, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	165	Phase 3	Portugal;United States;Greece;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Poland;Belgium;Bulgaria;Germany;Netherlands;Sweden
5	NCT04490915 (ClinicalTrials.gov)	July 23, 2020	24/7/2020	Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia	A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment	Congenital Adrenal Hyperplasia	Drug: Crinecerfont;Drug: Placebo	Neurocrine Biosciences	NULL	Recruiting	18 Years	N/A	All	165	Phase 3	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
6	NCT04045145 (ClinicalTrials.gov)	September 11, 2019	31/7/2019	Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia	A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia	CAH - Congenital Adrenal Hyperplasia	Drug: NBI-74788	Neurocrine Biosciences	NULL	Recruiting	14 Years	17 Years	All	12	Phase 2	United States
7	NCT03525886 (ClinicalTrials.gov)	April 10, 2018	2/5/2018	Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia	A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adult Subjects With Congenital Adrenal Hyperplasia	CAH - Congenital Adrenal Hyperplasia	Drug: NBI-74788	Neurocrine Biosciences	NULL	Completed	18 Years	50 Years	All	18	Phase 2	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

EUCTR2019-004873-17-PL
(EUCTR)

01/12/2020

03/07/2020

A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

Classic Congenital Adrenal Hyperplasia (CAH)
MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Product Name: Crinecerfont
Product Code: NBI-74788
INN or Proposed INN: Crinecerfont

Neurocrine Biosciences, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

165

Phase 3

United States;Portugal;Czechia;Greece;Spain;Austria;United Kingdom;Italy;France;Poland;Belgium;Bulgaria;Germany;Netherlands;Sweden

EUCTR2019-004873-17-GB
(EUCTR)

15/10/2020

30/04/2020

A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia

Product Name: Crinecerfont
Product Code: NBI-74788
INN or Proposed INN: Crinecerfont

Neurocrine Biosciences, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

165

Phase 3

United States;Portugal;Greece;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Belgium;Poland;Bulgaria;Germany;Netherlands;Sweden

EUCTR2019-004873-17-PT
(EUCTR)

12/10/2020

30/06/2020

A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia

Product Name: Crinecerfont
Product Code: NBI-74788
INN or Proposed INN: Crinecerfont

Neurocrine Biosciences, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

165

Phase 3

Italy;France;Czech Republic;Poland;Belgium;Bulgaria;Netherlands;Germany;Sweden;Portugal;United States;Greece;Spain;Austria;United Kingdom

EUCTR2019-004873-17-GR
(EUCTR)

29/07/2020

30/07/2020

A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia

Product Name: Crinecerfont
Product Code: NBI-74788
INN or Proposed INN: Crinecerfont

Neurocrine Biosciences, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

165

Phase 3

Portugal;United States;Greece;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Poland;Belgium;Bulgaria;Germany;Netherlands;Sweden

NCT04490915
(ClinicalTrials.gov)

July 23, 2020

24/7/2020

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

Congenital Adrenal Hyperplasia

Drug: Crinecerfont;Drug: Placebo

Neurocrine Biosciences

NULL

Recruiting

18 Years

N/A

All

165

Phase 3

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

NCT04045145
(ClinicalTrials.gov)

September 11, 2019

31/7/2019

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia

A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia

CAH - Congenital Adrenal Hyperplasia

Drug: NBI-74788

Neurocrine Biosciences

NULL

Recruiting

14 Years

17 Years

All

Phase 2

United States

NCT03525886
(ClinicalTrials.gov)

April 10, 2018

2/5/2018

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia

A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adult Subjects With Congenital Adrenal Hyperplasia

CAH - Congenital Adrenal Hyperplasia

Drug: NBI-74788

Neurocrine Biosciences

NULL

Completed

18 Years

50 Years

All

Phase 2

United States