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.
| 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 | EUCTR2019-004764-22-GB (EUCTR) | 23/12/2020 | 09/07/2020 | This is a randomized, double-blinded, Placebo controlled trial with a 3-part treatment period that will evaluate the efficacy and safety of up to 52 weeks of treatment with tildacerfont in subjects with classic Congenital adrenal hyperplasia (CAH) who have elevated blood hormones at baseline | A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia - Efficacy and Safety of Tildacerfont in Adult Subjects with Classic Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Tildacerfont Product Code: SPR001 INN or Proposed INN: TILDACERFONT | Spruce Biosciences, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;France;Spain;Poland;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 3 | 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 | ||
| 4 | EUCTR2019-004765-40-DE (EUCTR) | 20/11/2020 | 18/09/2020 | This is a randomized, double-blinded, Placebo controlled trial that will evaluate the potential of tildacerfont to reduce GC use in adult subjects with classic CAH who are on supraphysiologic doses of GC therapy. | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia - Efficacy and Safety of tildacerfont in Reducing Supraphysiologic GC doses in adult subjects with CAH | Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Tildacerfont Product Code: SPR001 INN or Proposed INN: TILDACERFONT | Spruce Biosciences, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;France;Spain;Poland;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 5 | EUCTR2019-004764-22-DK (EUCTR) | 17/11/2020 | 09/07/2020 | This is a randomized, double-blinded, Placebo controlled trial with a 3-part treatment period that will evaluate the efficacy and safety of up to 52 weeks of treatment with tildacerfont in subjects with classic Congenital adrenal hyperplasia (CAH) who have elevated blood hormones at baseline | A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia - Efficacy and Safety of Tildacerfont in Adult Subjects with Classic Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Tildacerfont Product Code: SPR001 INN or Proposed INN: TILDACERFONT | Spruce Biosciences, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;France;Spain;Poland;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 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 | EUCTR2019-004765-40-DK (EUCTR) | 17/11/2020 | 16/09/2020 | This is a randomized, double-blinded, Placebo controlled trial that will evaluate the potential of tildacerfont to reduce GC use in adult subjects with classic CAH who are on supraphysiologic doses of GC therapy. | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia - Efficacy and Safety of tildacerfont in Reducing Supraphysiologic GC doses in adult subjects with CAH | Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Tildacerfont Product Code: SPR001 INN or Proposed INN: TILDACERFONT | Spruce Biosciences, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;France;Spain;Poland;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 7 | EUCTR2019-004765-40-SE (EUCTR) | 03/11/2020 | 18/09/2020 | This is a randomized, double-blinded, Placebo controlled trial that will evaluate the potential of tildacerfont to reduce GC use in adult subjects with classic CAH who are on supraphysiologic doses of GC therapy. | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia - Efficacy and Safety of tildacerfont in Reducing Supraphysiologic GC doses in adult subjects with CAH | Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Tildacerfont Product Code: SPR001 INN or Proposed INN: TILDACERFONT | Spruce Biosciences, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;France;Spain;Poland;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 8 | EUCTR2019-004764-22-NL (EUCTR) | 15/10/2020 | 01/07/2020 | This is a randomized, double-blinded, Placebo controlled trial with a 3-part treatment period that will evaluate the efficacy and safety of up to 52 weeks of treatment with tildacerfont in subjects with classic Congenital adrenal hyperplasia (CAH) who have elevated blood hormones at baseline | A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia - Efficacy and Safety of Tildacerfont in Adult Subjects with Classic Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Tildacerfont Product Code: SPR001 INN or Proposed INN: TILDACERFONT | Spruce Biosciences, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | Poland;United States;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Denmark;Netherlands;Germany;Norway;Sweden | ||
| 9 | 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 | ||
| 10 | 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 | ||
| 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 |
| 11 | EUCTR2019-004764-22-DE (EUCTR) | 08/10/2020 | 08/07/2020 | This is a randomized, double-blinded, Placebo controlled trial with a 3-part treatment period that will evaluate the efficacy and safety of up to 52 weeks of treatment with tildacerfont in subjects with classic Congenital adrenal hyperplasia (CAH) who have elevated blood hormones at baseline | A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia - Efficacy and Safety of Tildacerfont in Adult Subjects with Classic Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Tildacerfont Product Code: SPR001 INN or Proposed INN: TILDACERFONT | Spruce Biosciences, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;France;Spain;Poland;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 12 | NCT04536662 (ClinicalTrials.gov) | October 1, 2020 | 23/8/2020 | Comparisons of Different Forms of Glucocorticoid on the Recovery of Reproductive Function in Patients With 21a-hydroxylase Deficiency | Comparisons of Different Forms of Glucocorticoid on the Recovery of Reproductive Function in Patients With 21a-hydroxylase Deficiency | Congenital Adrenal Hyperplasia | Drug: Hydrocortisone;Drug: Prednisone;Drug: Dexamethasone | Shanghai Jiao Tong University School of Medicine | NULL | Not yet recruiting | 14 Years | 45 Years | All | 120 | Phase 4 | China |
| 13 | NCT04544410 (ClinicalTrials.gov) | September 29, 2020 | 30/8/2020 | A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects With Classic Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | Drug: Tildacerfont/Placebo | Spruce Biosciences | NULL | Recruiting | 18 Years | 55 Years | All | 60 | Phase 2 | United States |
| 14 | EUCTR2019-004764-22-SE (EUCTR) | 26/08/2020 | 08/07/2020 | This is a randomized, double-blinded, Placebo controlled trial with a 3-part treatment period that will evaluate the efficacy and safety of up to 52 weeks of treatment with tildacerfont in subjects with classic Congenital adrenal hyperplasia (CAH) who have elevated blood hormones at baseline | A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia - Efficacy and Safety of Tildacerfont in Adult Subjects with Classic Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Tildacerfont Product Code: SPR001 INN or Proposed INN: TILDACERFONT | Spruce Biosciences, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;France;Spain;Poland;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 15 | NCT04457336 (ClinicalTrials.gov) | August 26, 2020 | 25/6/2020 | A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH | A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects With Classic Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | Drug: Tildacerfont/Placebo | Spruce Biosciences | NULL | Recruiting | 18 Years | 55 Years | All | 72 | 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 |
| 16 | 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 | ||
| 17 | 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 |
| 18 | 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 |
| 19 | EUCTR2018-004802-24-NL (EUCTR) | 20/05/2019 | 13/05/2019 | Avoiding over and undertreatment by optimizing of the timing ofglucocorticoïd treatment in children with cnogenital adrenal hyperplasia | Optimizing timing of glucocorticoid treatment in children with congenitaladrenal hyperplasia - OPTIMED study | Congenital adrenal hyperplasia (CAH) is a disorder of adrenal steroidsynthesis leading to cortisol deficiency and an increase in androgenproduction.Treatment in children consists of hydrocortisone substitutionthereby also restoring the negative feedback on the pituitary gland andconsequently normalize androgen production. There is still no evidenceabout the best timing of hydrocortisone use: The highest dosage ofhydrocortisone in the morning or the highest dosage at night;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Product Name: Hydrocortisone INN or Proposed INN: Hydrocortisone Other descriptive name: HYDROCORTISONE | Radboud University Nijmegen Medical Centre | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | Netherlands | ||
| 20 | NCT03718234 (ClinicalTrials.gov) | January 1, 2019 | 11/10/2018 | Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia | Interval Bolus Delivery of Subcutaneous Hydrocortisone Via Infusion Pump in Children With Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia;Hyperplasia;Adrenal Hyperplasia;Congenital Disorders;Adrenocortical Hyperfunction;Disorders of Sex Development;Urogenital Abnormalities;Genetic Diseases, Inborn;Steroid Metabolic Diseases, Inborn;Adrenal Gland Disease;Hydrocortisone | Drug: Subcutaneous hydrocortisone;Drug: Standard glucocorticoid therapy | University of Minnesota | NULL | Recruiting | 4 Years | 18 Years | All | 8 | 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 |
| 21 | NCT03532022 (ClinicalTrials.gov) | October 4, 2018 | 18/4/2018 | Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy | An Open-label, Randomized, Titration-blinded, Phase III Study of Efficacy, Safety and Tolerability Of Chronocort® Compared With Standard Glucocorticoid REeplacement Therapy in the Treatment of Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | Drug: Chronocort®;Drug: Standard Care | Diurnal Limited | National Institutes of Health (NIH) | Suspended | 16 Years | N/A | All | 132 | Phase 3 | United States |
| 22 | EUCTR2017-004878-34-FR (EUCTR) | 18/09/2018 | 24/04/2018 | A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia | A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia 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: Nevanimibe Hydrochloride Product Code: ATR-101 INN or Proposed INN: Nevanimibe hydrochloride Other descriptive name: CI-984, 17AA70, PD 132301-2 | Millendo Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | France;Spain;Brazil;Israel | ||
| 23 | NCT03687242 (ClinicalTrials.gov) | September 6, 2018 | 11/9/2018 | Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia | A 3-Month Phase 2 Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia;CAH - Congenital Adrenal Hyperplasia;CAH - 21-Hydroxylase Deficiency | Drug: SPR001 | Spruce Biosciences | NULL | Completed | 18 Years | N/A | All | 11 | Phase 2 | United States |
| 24 | EUCTR2017-004878-34-ES (EUCTR) | 01/08/2018 | 21/05/2018 | A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia | A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia 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: Nevanimibe Hydrochloride Product Code: ATR-101 INN or Proposed INN: Nevanimibe hydrochloride Other descriptive name: CI-984, 17AA70, PD 132301-2 | Millendo Therapeutics US, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | France;Brazil;Spain;Israel | ||
| 25 | NCT03669549 (ClinicalTrials.gov) | July 11, 2018 | 10/9/2018 | Nevanimibe HCl for the Treatment of Classic CAH | A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | Drug: Nevanimibe hydrochloride | Millendo Therapeutics US, Inc. | NULL | Terminated | 18 Years | 80 Years | All | 15 | Phase 2 | Brazil;Czechia;France;Israel;Spain |
| 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 |
| 26 | 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 |
| 27 | EUCTR2015-005448-32-DK (EUCTR) | 01/09/2017 | 28/06/2017 | The proposed study aims to build on the results of clinical studies DIUR-003 and DIUR-005 and evaluate the long-term safety of Chronocort® and also its long term efficacy. | A Phase III extension study of efficacy, safety and tolerability of Chronocort® in the treatment of congenital adrenal hyperplasia (CAH) | 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: 20.0;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 | Diurnal Ltd | NULL | Not Recruiting | Female: yes Male: yes | 136 | Phase 3 | France;United States;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 28 | 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 |
| 29 | NCT03257462 (ClinicalTrials.gov) | July 26, 2017 | 15/8/2017 | Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia | A Phase 2, Multiple-Dose, Dose-Escalation Study to Evaluate the Safety and Efficacy of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia (CAH) | Congenital Adrenal Hyperplasia;CAH - Congenital Adrenal Hyperplasia | Drug: SPR001 | Spruce Biosciences | NULL | Completed | 18 Years | N/A | All | 26 | Phase 2 | United States |
| 30 | EUCTR2015-005448-32-SE (EUCTR) | 09/02/2017 | 06/09/2016 | The proposed study aims to build on the results of clinical studies DIUR-003 and DIUR-005 and evaluate the long-term safety of Chronocort® and also its long term efficacy. | A Phase III extension study of efficacy, safety and tolerability of Chronocort® in the treatment of congenital adrenal hyperplasia (CAH) | 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: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Diurnal Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 138 | Phase 3 | France;United States;Denmark;Germany;United Kingdom;Sweden | |||
| 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 |
| 31 | EUCTR2015-005448-32-DE (EUCTR) | 31/01/2017 | 02/11/2016 | The proposed study aims to build on the results of clinical studies DIUR-003 and DIUR-005 and evaluate the long-term safety of Chronocort® and also its long term efficacy. | A Phase III extension study of efficacy, safety and tolerability of Chronocort® in the treatment of congenital adrenal hyperplasia (CAH) | 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: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Diurnal Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 3 | France;United States;Denmark;Netherlands;Germany;United Kingdom;Sweden | |||
| 32 | EUCTR2015-000711-40-DK (EUCTR) | 08/09/2016 | 11/07/2016 | 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: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000012082;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 10mg Tablets INN or Proposed INN: Hydrocortisone Other descriptive name: HYDROCORTISONE Trade Name: Dexamethasone 0,1 mg Tablets INN or Proposed INN: DEXAMETHASONE Trade Name: Prednisolone 2,5 mg Tablets INN or Proposed INN: Prednisolone Other descriptive name: PREDNISOLONE Product Name: Hydrocortisone tablet 2.5 mg | Diurnal Ltd | NULL | Not Recruiting | Female: yes Male: yes | 120 | Phase 3 | France;United States;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 33 | NCT03062280 (ClinicalTrials.gov) | August 18, 2016 | 22/8/2016 | A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH | A Phase III Extension Study of Efficacy, Safety and Tolerability of Chronocort® in the Treatment of Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | Drug: Hydrocortisone | Diurnal Limited | NULL | Active, not recruiting | 18 Years | N/A | All | 92 | Phase 3 | United States |
| 34 | NCT03760835 (ClinicalTrials.gov) | August 11, 2016 | 9/11/2018 | Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatment | Congenital Adrenal Hyperplasia: Innovative Once Daily Dual Release Hydrocortisone Treatment | Congenital Adrenal Hyperplasia | Drug: Conventional Glucocorticoids (immediate release hydrocortisone, cortisone acetate, prednisone, prednisolone, dexamethasone);Drug: Dual release hydrocortisone (plenadren) | Federico II University | NULL | Recruiting | 18 Years | N/A | All | 150 | Phase 4 | Italy |
| 35 | EUCTR2015-005448-32-GB (EUCTR) | 20/07/2016 | 05/07/2016 | The proposed study aims to build on the results of clinical studies DIUR-003 and DIUR-005 and evaluate the long-term safety of Chronocort® and also its long term efficacy. | A Phase III extension study of efficacy, safety and tolerability of Chronocort® in the treatment of congenital adrenal hyperplasia (CAH) | 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: 20.0;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 | Diurnal Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 3 | France;United States;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 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 |
| 36 | EUCTR2015-000711-40-DE (EUCTR) | 23/05/2016 | 06/10/2015 | 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: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Diurnal Ltd | NULL | Not Recruiting | Female: yes Male: yes | 120 | Phase 3 | France;United States;Denmark;Netherlands;Germany;United Kingdom;Sweden | |||
| 37 | NCT02804178 (ClinicalTrials.gov) | May 18, 2016 | 1/6/2016 | A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia | A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | Drug: ATR-101 | Millendo Therapeutics, Inc. | NULL | Completed | 18 Years | 80 Years | All | 10 | Phase 2 | United States |
| 38 | 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 | ||
| 39 | NCT02733367 (ClinicalTrials.gov) | March 4, 2016 | 16/3/2016 | Extension Study for Patients Entered Into Study Infacort 003 | Open-label, Long-term Follow-up of Safety and Biochemical Disease Control of Infacort® in Neonates, Infants and Children With Congenital Adrenal Hyperplasia and Adrenal Insufficiency Previously Enrolled in the Infacort 003 Study | Adrenal Insufficiency | Drug: Infacort® | Diurnal Limited | NULL | Completed | 1 Month | 6 Years | All | 18 | Phase 3 | Germany |
| 40 | NCT02716818 (ClinicalTrials.gov) | February 22, 2016 | 26/2/2016 | Comparison of Chronocort® With Standard Glucocorticoid Therapy in Patients With Congenital Adrenal Hyperplasia | 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 | Drug: Chronocort®;Drug: standard glucocorticoid therapy | Diurnal Limited | NULL | Completed | 18 Years | N/A | All | 122 | 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 |
| 41 | EUCTR2015-000711-40-SE (EUCTR) | 11/01/2016 | 13/10/2015 | 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: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000012082;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 2mg Tablets INN or Proposed INN: DEXAMETHASONE Trade Name: Prednisolone 1mg Tablets INN or Proposed INN: Prednisolone Other descriptive name: PREDNISOLONE | Diurnal Ltd | NULL | Not Recruiting | Female: yes Male: yes | 120 | Phase 3 | France;United States;Denmark;Netherlands;Germany;United Kingdom;Sweden | ||
| 42 | EUCTR2015-000711-40-GB (EUCTR) | 19/11/2015 | 28/07/2015 | 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: 20.0;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Diurnal Ltd | NULL | Not Recruiting | Female: yes Male: yes | 120 | Phase 3 | France;United States;Denmark;Netherlands;Germany;United Kingdom;Sweden | |||
| 43 | EUCTR2015-003996-32-FR (EUCTR) | 03/11/2015 | 04/12/2015 | Multicentric evaluation of in utero dexamethasone (DEX) on the cognitive development of children at risk of Congenital Adrenal Hyperplasia - PRENATAL DEX Study | Multicentric evaluation of in utero dexamethasone (DEX) on the cognitive development of children at risk of Congenital Adrenal Hyperplasia - PRENATAL DEX Study - PRENATAL DEX | Patient with Congenital Adrenal Hyperplasia (CAH) or sibling of a CAH patient . Patient no connection with Congenital Adrenal Hyperplasia MedDRA version: 18.1;Level: PT;Classification code 10061630;Term: Adrenogenital syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Dectancyl | Hospices Civils de Lyon | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | France | ||||
| 44 | EUCTR2015-000458-40-DE (EUCTR) | 20/10/2015 | 21/08/2015 | Open-label, long-term follow-up of safety and biochemical disease control of Infacort® in neonates, infants and children with congenital adrenal hyperplasia and adrenal insufficiency previously enrolled in the Infacort 003 study | Open-label, long-term follow-up of safety and biochemical disease control of Infacort® in neonates, infants and children with congenital adrenal hyperplasia and adrenal insufficiency previously enrolled in the Infacort 003 study - Infacort004 | Adrenal Insufficiency (AI) in children is most commonly due to Congenital Adrenal Hyperplasia (CAH) and results in cortisol deficiency with or without aldosterone deficiency and androgen excess. Current standard treatment in neonates is unsatisfactory, as unlicensed and crushed adult dosage formulations (Hydrocortisone tablets,10 mg) are used. Infacort® is a new paediatric and neonatal formulation of hydrocortisone that is provided in appropriate unit dosage (0.5mg, 1.0mg, 2.0mg and 5mg);Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Diurnal Limited | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 3 | Germany | |||
| 45 | EUCTR2014-002265-30-DE (EUCTR) | 19/02/2015 | 23/12/2014 | A Phase 3 open-label study of Infacort® in neonates, infants and children less than 6 years of age with adrenal insufficiency. | A Phase 3 open-label study of Infacort® in neonates, infants and children less than 6 years of age with adrenal insufficiency. | Adrenal Insufficiency (AI) in children is most commonly due to Congenital Adrenal Hyperplasia (CAH) and results in cortisol deficiency with or without aldosterone deficiency and androgen excess. Current standard treatment in neonates is unsatisfactory, as unlicensed and crushed adult dosage formulations (Hydrocortisone tablets, 10 mg) are used. Infacort® is a new paediatric and neonatal formulation of hydrocortisone that is provided in appropriate unit dosage (0.5mg, 1.0mg, 2.0mg and 5mg).;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Product Name: infacort INN or Proposed INN: infacort Other descriptive name: HYDROCORTISONE Product Name: infacort INN or Proposed INN: infacort Other descriptive name: HYDROCORTISONE Product Name: infacort INN or Proposed INN: infacort Other descriptive name: HYDROCORTISONE Product Name: infacort INN or Proposed INN: infacort Other descriptive name: HYDROCORTISONE | Diurnal Limited | NULL | Not Recruiting | Female: yes Male: yes | Phase 3 | Germany | |||
| 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 |
| 46 | 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 |
| 47 | EUCTR2011-005822-23-SE (EUCTR) | 18/11/2014 | 20/09/2013 | A trial comparing continuous subcutaneous hydrocortisone infusion (CSHI) therapy with conventional oral glucocorticoid therapy in patients with Congenital Andrenal Hyperplasia (CAH) | CONTINUOUS SUBCUTANEOUS HYDROCORTISONE INFUSION IN CONGENITAL ADRENAL HYPERPLASIA | Congenital Adrenal Hyperplasia MedDRA version: 14.1;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Trade Name: Solu-Cortef INN or Proposed INN: HYDROCORTISONE SODIUM SUCCINATE Other descriptive name: HYDROCORTISONE SODIUM SUCCINATE INN or Proposed INN: Prednisolone INN or Proposed INN: Hydrocortisone | Haukeland University Hospital | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Norway;Sweden | ||
| 48 | EUCTR2012-001104-37-GB (EUCTR) | 06/10/2014 | 24/07/2014 | pulses study | Pulsed glucocorticoid replacement therapy for patients with adrenocortical insufficiency secondary to Addison’s disease and congenital adrenal hyperplasia - the pulses study | Addison's disease and Congenital Adrenal Hyperplasia MedDRA version: 17.0;Level: LLT;Classification code 10011195;Term: Cortisol;System Organ Class: 100000004848 MedDRA version: 17.0;Classification code 10020518;Term: Hydrocortisone;System Organ Class: 100000004848;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Trade Name: hydrocortisone sodium phosphate Product Name: hydrocortisone sodium phosphate INN or Proposed INN: hydrocortisone sodium phosphate Trade Name: hydrocortisone Product Name: hydrocortisone INN or Proposed INN: hydrocortisone | University Hospitals Bristol NHS Foundation Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | United Kingdom | ||||
| 49 | NCT02096510 (ClinicalTrials.gov) | August 2014 | 28/11/2013 | Ultradian Subcutaneous Hydrocortisone Infusion in Addison Disease and Congenital Adrenal Hyperplasia | Ultradian Subcutaneous Hydrocortisone Infusion in Addison Disease and Congenital Adrenal Hyperplasia | Addison Disease;Adrenal Hyperplasia Congenital | Drug: Solu-Cortef;Drug: Cortef | Haukeland University Hospital | NULL | Recruiting | 18 Years | 65 Years | Both | 10 | Phase 1;Phase 2 | Norway |
| 50 | EUCTR2013-002395-40-BE (EUCTR) | 24/03/2014 | 19/11/2013 | Prospective intervention trial with adjuvant metformin in girls and boys with classic congenital adrenal hyperplasia. | Prospective intervention trial with adjuvant metformin in girls and boys with classic CAH (METFOR CAH). - METFOR CAH | congenital adrenal hyperplasia (CAH) MedDRA version: 16.1;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Metformine Sandoz 850 mg filmomhulde tabletten Product Name: Metformine INN or Proposed INN: METFORMIN HYDROCHLORIDE Other descriptive name: METFORMIN HYDROCHLORIDE | Ghent University Hospital | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | Belgium | ||
| 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 |
| 51 | NCT01859312 (ClinicalTrials.gov) | May 6, 2013 | 17/5/2013 | Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal Hyperplasia | A Pilot Study Assessing the Use of Continuous Subcutaneous Hydrocortisone Infusion in the Treatment of Congenital Adrenal Hyperplasia | Adrenal Insufficiency;Excess Androgen;Congenital Adrenal Hyperplasia (CAH) | Drug: Hydrocortisone (Solucortef);Device: Insulin pump (Medtronic) | National Institutes of Health Clinical Center (CC) | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | 18 Years | 99 Years | All | 8 | Phase 2 | United States |
| 52 | NCT01771328 (ClinicalTrials.gov) | February 2013 | 10/1/2013 | Continuous Subcutaneous Hydrocortisone Infusion in Congenital Adrenal Hyperplasia | Continuous Subcutaneous Hydrocortisone Infusion in Congenital Adrenal Hyperplasia | Adrenal Hyperplasia, Congenital | Drug: Hydrocortisone;Drug: Cortisone acetate | Haukeland University Hospital | NULL | Recruiting | 18 Years | 60 Years | Both | 20 | Phase 2 | Norway |
| 53 | NCT01735617 (ClinicalTrials.gov) | December 2012 | 20/11/2012 | Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH | A Phase 2 Pilot Study to Characterize and Examine the Pharmacokinetics and Disease Bio-marker Response of Chronocort® in Adults With Congenital Adrenal Hyperplasia | Endocrine Disease;Adrenal Insufficiency;Congenital Adrenal Hyperplasia | Drug: Hydrocortisone Modified Release Capsules | Diurnal Limited | National Institutes of Health (NIH) | Completed | 18 Years | N/A | All | 16 | Phase 2 | United States |
| 54 | EUCTR2011-005822-23-NO (EUCTR) | 16/10/2012 | 25/02/2013 | A trial comparing continuous subcutaneous hydrocortisone therapy with conventional oral glucocorticoid therapy in congenital adrenal hyperplasia | CONTINUOUS SUBCUTANEOUS HYDROCORTISONE INFUSION IN CONGENITAL ADRENAL HYPERPLASIA | Congenital adrenal hyperplasia (CAH) MedDRA version: 14.1;Level: LLT;Classification code 10010323;Term: Congenital adrenal hyperplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Trade Name: Solu-Cortef Trade Name: Cortison Trade Name: Prednisolone | Haukeland University Hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Norway | ||||
| 55 | NCT02552251 (ClinicalTrials.gov) | August 2012 | 27/7/2015 | COrticosteroid in Congenital Adrenal Hyperplasia | Comparative Study of the Use of Glucocorticoids in the Treatment of Congenital Adrenal Hyperplasia in Its Classical Form | Congenital Adrenal Hyperplasia | Biological: Hormonal balance measurements;Biological: metabolic balance measurements;Biological: bone balance measurements;Behavioral: quality of life assessment | University Hospital, Caen | NULL | Recruiting | 18 Years | 55 Years | Female | 40 | Phase 2;Phase 3 | France |
| 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 |
| 56 | 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 |
| 57 | NCT03051893 (ClinicalTrials.gov) | February 2011 | 11/1/2017 | A Two-part, Study to Compare the Pharmacokinetics and Dose Proportionality of up to 6 Chronocort Formulations | A Two-part Open Label, Randomised, Single Dose, Crossover Study in Healthy Volunteers to: (Part A) Compare the Pharmacokinetics of up to 6 Chronocort® Formulations, and (Part B) Determine the Dose Proportionality of a Selected Chronocort® Formulation at Three Dose Levels With an Additional Comparison With the Selected Formulation Dosed on Two Occasions Over a 24 Hour Period | Adrenal Insufficiency;Congenital Adrenal Hyperplasia | Drug: Chronocort | Diurnal Limited | Simbec Research | Completed | 18 Years | 60 Years | Male | 28 | Phase 1 | NULL |
| 58 | NCT03019614 (ClinicalTrials.gov) | March 2010 | 11/1/2017 | An Open Label Study in Healthy Volunteers to Compare Chronocort® to Hydrocortisone | An Open Label, Randomised, Single Dose, 3-period Crossover Study in Healthy Volunteers to: a) Compare the Pharmacokinetics of Chronocort® Formulations Versus Immediate Release Hydrocortisone, and (b) Determine the Dose Proportionality of Chronocort® Formulations | Congenital Adrenal Hyperplasia;Adrenal Insufficiency | Drug: Hydrocortisone;Drug: Chronocort | Diurnal Limited | Simbec Research | Completed | 18 Years | 60 Years | Male | 30 | Phase 1 | NULL |
| 59 | NCT00621985 (ClinicalTrials.gov) | April 2008 | 11/2/2008 | Dexamethasone Treatment of Congenital Adrenal Hyperplasia | Dexamethasone Treatment of Congenital Adrenal Hyperplasia | Adrenal Hyperplasia, Congenital | Drug: dexamethasone;Drug: Hydrocortisone | Boston Children’s Hospital | NULL | Completed | 2 Years | 9 Years | All | 5 | Phase 2 | United States |
| 60 | NCT00542841 (ClinicalTrials.gov) | August 2007 | 10/10/2007 | Examining Genetic Differences Among People With 21-Hydroxylase Deficiency | Modifier Genes in 21-Hydroxylase Deficiency | 21-hydroxylase Deficiency | Procedure: Hydrocortisone withdrawal | Maria I. New | Office of Rare Diseases (ORD);National Center for Research Resources (NCRR) | Completed | 18 Years | 50 Years | Both | 99 | N/A | United States;Brazil;France |
| 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 |
| 61 | NCT00519818 (ClinicalTrials.gov) | August 2007 | 22/8/2007 | Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasia | A Phase 2, Open Label, Crossover Pharmacokinetic and Pharmacodynamic Study to Compare Chronocort Versus Cortef in Patients With CAH | Congenital Adrenal Hyperplasia;21-Hydroxylase Deficiency;Adrenogenital Syndrome | Drug: Chronocort;Drug: Cortef | Diurnal Limited | National Institutes of Health Clinical Center (CC) | Completed | 16 Years | 60 Years | All | 20 | Phase 1;Phase 2 | United States |
| 62 | NCT00529841 (ClinicalTrials.gov) | January 2007 | 12/9/2007 | Research Study for Children With Salt Wasting Congenital Adrenal Hyperplasia | A Novel Therapeutic Modality for Congenital Adrenal Hyperplasia | Adrenal Hyperplasia, Congenital | Drug: Hydrocortisone sodium acetate | Baylor College of Medicine | NULL | Completed | 3 Years | 18 Years | Both | 7 | N/A | United States |
| 63 | NCT00151710 (ClinicalTrials.gov) | May 2005 | 8/9/2005 | Effects of Pioglitazone in Congenital Adrenal Hyperplasia | Effects of Pioglitazone in Glucocorticoid-Induced Insulin Resistance. Studies in Congenital Adrenal Hyperplasia. | Congenital Adrenal Hyperplasia | Drug: Pioglitazone | Radboud University | NULL | Completed | 18 Years | N/A | Both | N/A | Netherlands | |
| 64 | NCT00001521 (ClinicalTrials.gov) | June 8, 1995 | 3/11/1999 | Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia | An Open, Randomized, Long-Term Clinical Trial of Flutamide, Testolactone, and Reduced Hydrocortisone Dose vs. Conventional Treatment of Children With Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia (CAH) | Drug: Flutamide;Drug: Letrozole;Drug: Hydrocortisone;Drug: Fludrocortisone | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | NULL | Active, not recruiting | 2 Years | 18 Years | All | 62 | Phase 2 | United States |
| 65 | NCT00000102 (ClinicalTrials.gov) | 3/11/1999 | Congenital Adrenal Hyperplasia: Calcium Channels as Therapeutic Targets | Congenital Adrenal Hyperplasia | Drug: Nifedipine | National Center for Research Resources (NCRR) | NULL | Completed | 14 Years | 35 Years | Both | Phase 1;Phase 2 | United States |