Congenital Adrenal Hyperplasia (CAH) (DBCOND0033416)

Identifiers

Synonyms
CAH - Congenital Adrenal Hyperplasia / Congenital Adrenal Hyperplasia / Adrenal Hyperplasia Congenital / Adrenal Hyperplasia, Congenital / Congenital adrenogenital disorders associated with enzyme deficiency / Congenital adrenal hyperplasia (disorder) / Adrenogenital disorder, unspecified / Adrenal virilism / Adrenal virilism (disorder) / Androgenital syndrome / Adrenogenital syndrome NOS / Adrenogenital syndrome congenital / Adrenogenital disorder (disorder) / Adrenogenital Syndrome

Associated Data

Indicated Drugs and Targets
DrugDescriptionTargets
Betamethasone
A systemic corticosteroid used to relieve inflammation in various conditions, including but not limited to allergic states, dermatologic disorders, gastrointestinal diseases, and hematological disorders.
Bupivacaine
A local anesthetic used in a wide variety of superficial and invasive procedures.
Cortisone acetate
A steroid hormone used for the relief of the inflammatory and pruritic manifestations of corticosteroid-responsive dermatoses and endocrine disorders associated with inadequate production of steroid hormones.
Dexamethasone
A glucocorticoid available in various modes of administration that is used for the treatment of various inflammatory conditions, including bronchial asthma, as well as endocrine and rheumatic disorders.
Hydrocortisone
A glucocorticoid used to treat corticosteroid-responsive dermatoses, endocrine disorders, immune conditions, and allergic disorders.
Hydrocortisone phosphate
A corticosteroid used to treat congenital adrenal hyperplasia, for emergency asthma treatment, hypersensitivity, and inflammation.
Hydrocortisone succinate
A corticosteroid used to treat severe allergic reactions, dermatologic diseases, endocrine disorders, gastrointestinal diseases, hematological disorders, neoplastic diseases, nervous system conditions, ophthalmic diseases, renal diseases, respiratory diseases, and rheumatic disorders.
Methylprednisolone
A corticosteroid used to treat inflammation or immune reactions across a variety of organ systems, endocrine conditions, and neoplastic diseases.
Methylprednisolone hemisuccinate
A water soluble corticosteroid used to treat severe allergic reactions, dermatologic diseases, endocrine disorders, gastrointestinal diseases, hematological disorders, neoplastic diseases, nervous system conditions, ophthalmic diseases, renal diseases, respiratory diseases, and rheumatic disorders.
Prednisolone
A glucocorticoid used to treat adrenocortical insufficiency, inflammatory conditions, and some cancers.
Prednisone
A corticosteroid used to treat inflammation or immune-mediated reactions and to treat endocrine or neoplastic diseases.
Triamcinolone
A glucocorticoid used to treat a wide variety of inflammatory conditions of organ systems and tissues.
Clinical Trials
IdentifierTitleDrug(s)PurposePhaseStatus
NCT03687242
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal HyperplasiaNo drug interventionstreatment2completed
NCT03525886
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasiatreatment2completed
NCT03257462
Study of SPR001 in Adults With Classic Congenital Adrenal HyperplasiaNo drug interventionstreatment2completed
NCT04045145
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasiatreatment2completed
NCT00250159
Natural History Study of Patients With Excess AndrogenNo drug interventionsNot AvailableNot Availablerecruiting
NCT03550261
Salt Wasting, Hydro-sodium Balance and Fludrocortisone Requirement in Congenital Adrenal HyperplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT01859312
Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal Hyperplasiatreatment2completed
NCT00001521
Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasiatreatment4completed
NCT05663320
A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in Adolescent and Young Adults With Congenital Adrenal HyperplasiaNo drug interventionssupportive_careNot Availablerecruiting
NCT04783181
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)No drug interventionstreatment1 / 2active_not_recruiting
NCT05101902
A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])No drug interventionsNot AvailableNot Availablecompleted
NCT06573723
Institutional Registry of Rare DiseasesNo drug interventionsNot AvailableNot Availablerecruiting
NCT02349503
Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasiatreatment1withdrawn
NCT01807364
Cardiovascular Risk Profile in Patients With Congenital Adrenal HyperplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT01184651
Tweens to Teens Project at Penn StateNo drug interventionsNot AvailableNot Availablecompleted
NCT04806451
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)treatment3active_not_recruiting
NCT03051893
A Two-part, Study to Compare the Pharmacokinetics and Dose Proportionality of up to 6 Chronocort FormulationsNo drug interventionstreatment1completed
NCT00749593
Study of UK Adults With Congenital Adrenal Hyperplasia.No drug interventionsNot AvailableNot Availablecompleted
NCT02804178
A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasiatreatment2completed
NCT02934399
Dynamic Hormone Diagnostics in Endocrine DiseaseNo drug interventionsNot AvailableNot Availablecompleted
NCT00519818
Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasiatreatment1 / 2completed
NCT02716818
Comparison of Chronocort® With Standard Glucocorticoid Therapy in Patients With Congenital Adrenal Hyperplasiatreatment3completed
NCT01735617
Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAHtreatment2completed
NCT04087148
Linear Growth of Children With Congenital Adrenal HyperplasiaNo drug interventionsNot AvailableNot Availableunknown_status
NCT05063994
Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasiatreatment3completed
NCT05907291
Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)No drug interventionstreatment2recruiting
NCT03062280
A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAHtreatment3completed
NCT04890444
China Adrenal Disease RegistryNo drug interventionsNot AvailableNot Availablerecruiting
NCT03897504
Surgical Evaluation of Using the Prepuce in Feminizing GenitoplastyNo drug interventionstreatmentNot Availableunknown_status
NCT03548246
Androgen Reduction in Congenital Adrenal Hyperplasiatreatment2withdrawn
NCT04544410
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAHNo drug interventionstreatment2active_not_recruiting
NCT00151710
Effects of Pioglitazone in Congenital Adrenal HyperplasiaNot AvailableNot Availablecompleted
NCT00172510
Mutation Analysis of 17α-HydroxylaseNo drug interventionsNot AvailableNot Availableunknown_status
NCT03019614
An Open Label Study in Healthy Volunteers to Compare Chronocort® to Hydrocortisonetreatment1completed
NCT05162950
Effects and Importance of Epinephrine/Adrenalin Deficiency in CAHNo drug interventionsNot AvailableNot Availableunknown_status
NCT00000102
Congenital Adrenal Hyperplasia: Calcium Channels as Therapeutic Targetstreatment1 / 2completed
NCT04903587
Gonadal Changes In Congenital Adrenal Hyperplasia PatientsNo drug interventionsNot AvailableNot Availableunknown_status
NCT04293133
Final Height in Patients With CAHNo drug interventionsNot AvailableNot Availableunknown_status
NCT05299554
Long-term Safety Study of Chronocort in the Treatment of Participants With Congenital Adrenal Hyperplasiatreatment3enrolling_by_invitation
NCT02552251
COrticosteroid in Congenital Adrenal HyperplasiaNo drug interventionsbasic_science2 / 3unknown_status
NCT02795871
Prenatal Dex StudyNo drug interventionsotherNot Availableunknown_status
NCT00559078
Qualitative Research on Women With Congenital Adrenal HyperplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT06449456
Exercise Capacity, Physical Fitness, and Physical Activity Levels in Children With CAHNo drug interventionsNot AvailableNot Availablerecruiting
NCT04490915
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasiatreatment3active_not_recruiting
NCT03162159
Adult Height Prediction in Congenital Adrenal HyperplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT05128942
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children With CAHtreatment2recruiting
NCT03669549
Nevanimibe HCl for the Treatment of Classic CAHtreatment2terminated
NCT04457336
A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAHNo drug interventionstreatment2terminated
NCT03718234
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasiatreatment1completed
NCT04463316
GROWing Up With Rare GENEtic SyndromesNo drug interventionsNot AvailableNot Availablerecruiting
NCT03760835
Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatmenttreatment4unknown_status
NCT00485186
Gene Polymorphisms Influencing Steroid Synthesis and ActionNo drug interventionsNot AvailableNot Availablewithdrawn
NCT03532022
Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapytreatment3withdrawn
NCT05687474
Baby Detect : Genomic Newborn ScreeningNo drug interventionsNot AvailableNot Availablerecruiting
NCT06564220
Testicular Adrenal Rest Tumor in Congenital Adrenal Hyperplasia Patients Attending Assuit University Children HospitalNo drug interventionsNot AvailableNot Availablenot_yet_recruiting
NCT00011791
Catecholamine Reserve and Exercise Tolerance in Healthy Volunteers and Patients With Congenital Adrenal HyperplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT04536662
Comparisons of Different Forms of Glucocorticoid on the Recovery of Reproductive Function in Patients With 21α-hydroxylase Deficiencytreatment4unknown_status
NCT01875640
Decision Support for Parents Receiving Information About Child's Rare DiseaseNo drug interventionsNot AvailableNot Availablecompleted
NCT04252001
Growing up With the Young Endocrine Support System (YESS!)No drug interventionssupportive_careNot Availablenot_yet_recruiting
NCT02574910
Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1treatment1suspended
NCT05669950
Study of Lu AG13909 in Participants With Congenital Adrenal HyperplasiaNo drug interventionstreatment1recruiting
NCT02096510
Ultradian Subcutaneous Hydrocortisone Infusion in Addison Disease and Congenital Adrenal Hyperplasiatreatment1 / 2completed
NCT01771328
Continuous Subcutaneous Hydrocortisone Infusion in Congenital Adrenal Hyperplasiatreatment2unknown_status
NCT00621985
Dexamethasone Treatment of Congenital Adrenal Hyperplasiatreatment2completed
NCT00529841
Research Study for Children With Salt Wasting Congenital Adrenal HyperplasiatreatmentNot Availablecompleted
NCT00694525
Role of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal HyperplasiaNo drug interventionsNot AvailableNot Availableunknown_status
NCT01488721
Clinical Evaluation of NeoPlex4 Assay and NeoPlex SystemNo drug interventionsNot AvailableNot Availablecompleted
NCT05228652
Questionnaire Study to Assess the Outcomes of the Management of Congenital Adrenal Hyperplasia IndividualsNo drug interventionsNot AvailableNot Availablerecruiting
NCT01862380
Adrenocortical Functions in Women With Nonclassical 21-hydroxylase Deficiency.No drug interventionsdiagnosticNot Availableunknown_status
NCT00617292
Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their MothersNo drug interventionsNot AvailableNot Availableunknown_status
NCT03589144
LC-MS / MS Adrenal Steroids Assayed on Dried Blot Spot for the Congenital Adrenal Hyperplasia Neonatal Screening (SPECTROSPOT)No drug interventionsNot AvailableNot Availablecompleted
NCT03162172
Growth Hormone (GH) in Congenital Adrenal HyperplasiaNo drug interventionsNot AvailableNot Availablecompleted