Congenital Adrenal Hyperplasia
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2025-07-08Clinical Trial Update: A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH [Phase 2]
Phase 2b trial evaluating the efficacy and safety of tildacerfont in adults with congenital adrenal hyperplasia was terminated. Results for …
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CLINICAL_TRIALS trial_result · 2025-07-08Clinical Trial Update: Exercise Capacity, Physical Fitness, and Physical Activity Levels in Children With CAH
A completed clinical trial update evaluating exercise capacity, physical fitness, and physical activity levels in children with congenital a…
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CLINICAL_TRIALS trial_result · 2025-06-25Clinical Trial Update: Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia [Phase 1]
Phase 1 trial evaluating subcutaneous hydrocortisone in children with congenital adrenal hyperplasia. The study is completed and results wer…
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CLINICAL_TRIALS trial_result · 2025-06-12Clinical Trial Update: Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia [Phase 3]
A Phase 3 clinical trial comparing a three-drug combination therapy against conventional treatment in children with congenital adrenal hyper…
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FDA approval · 2025-05-28FDA Approved: KHINDIVI — Original Application (Eton Pharmaceuticals, Inc.)
FDA approved KHINDIVI (hydrocortisone) through an original NDA submitted by Eton Pharmaceuticals, Inc.
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CLINICAL_TRIALS trial_result · 2025-04-01Clinical Trial Update: Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia [Phase 2]
Phase 2 study evaluating SPR001 for classic congenital adrenal hyperplasia has completed, with results posted.
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CLINICAL_TRIALS trial_result · 2025-02-24Clinical Trial Update: Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia [Phase 3]
Phase 3 trial comparing Chronocort versus standard hydrocortisone replacement therapy in participants aged 16 years and older with congenita…
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CLINICAL_TRIALS trial_result · 2025-02-05Clinical Trial Update: Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia [Phase 3]
Phase 3 global safety and efficacy trial of crinecerfont for congenital adrenal hyperplasia has posted results; the study is currently not r…
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CLINICAL_TRIALS trial_result · 2025-02-05Clinical Trial Update: Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study) [Phase 3]
Phase 3 registration study evaluating Crinecerfont in pediatric participants with classic congenital adrenal hyperplasia has posted results.…
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CLINICAL_TRIALS research · 2024-12-31Clinical Trial Update: Electronic Registry of Male Patients With Congenital Adrenal Hyperplasia 21-hydroxylase Deficiency
Clinical trial registry update indicating ongoing recruitment for male patients with congenital adrenal hyperplasia due to 21-hydroxylase de…
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FDA approval · 2024-12-13FDA Approved: CRENESSITY — Original Application (Neurocrine Biosciences, Inc.)
FDA approved CRENESSITY (crinecerfont) from Neurocrine Biosciences, granting its first approval for congenital adrenal hyperplasia.
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CLINICAL_TRIALS trial_result · 2024-10-24Clinical Trial Update: A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH [Phase 3]
Phase 3 trial evaluating Chronocort for congenital adrenal hyperplasia has completed with posted results assessing efficacy, safety and tole…
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FDA approval · 2024-10-16FDA Approved: FLUDROCORTISONE ACETATE — Original Application (Viona Pharmaceuticals Inc)
The FDA approved the original application for Fludrocortisone acetate (generic) from Viona Pharmaceuticals Inc. for use as a mineralocortico…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])
A completed study assessed eligibility criteria for the CAH-301 gene therapy in classic congenital adrenal hyperplasia.
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CLINICAL_TRIALS research · 2024-08-21Clinical Trial Update: Testicular Adrenal Rest Tumor in Congenital Adrenal Hyperplasia Patients Attending Assuit University Children Hospital
A clinical trial at Assiut University investigates testicular adrenal rest tumor in patients with congenital adrenal hyperplasia. The study …
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CLINICAL_TRIALS trial_result · 2024-07-18Clinical Trial Update: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia [Phase 2]
This Phase 2 clinical trial update reports safety, tolerability, and pharmacokinetic/pharmacodynamic data for crinecerfont (NBI-74788) in pe…
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CLINICAL_TRIALS trial_result · 2024-06-07Clinical Trial Update: Ultradian Subcutaneous Hydrocortisone Infusion in Addison Disease and Congenital Adrenal Hyperplasia [Phase 1, Phase 2]
A phase 1/2 clinical trial evaluating an ultradian subcutaneous hydrocortisone infusion for Addison's disease and congenital adrenal hyperpl…
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FDA approval · 2024-06-05FDA Approved: ALKINDI SPRINKLE — Supplemental Application (Eton Pharmaceuticals, Inc.)
The FDA approved a supplemental application for ALKINDI SPRINKLE (hydrocortisone) from Eton Pharmaceuticals, expanding or updating its label…
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CLINICAL_TRIALS trial_result · 2024-06-04Clinical Trial Update: Study of UK Adults With Congenital Adrenal Hyperplasia.
This is a clinical trial update about studying congenital adrenal hyperplasia in UK adults. The study status is completed.
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CLINICAL_TRIALS trial_result · 2024-05-08Clinical Trial Update: Decision Support for Parents Receiving Information About Child's Rare Disease
This clinical trial update reports on a completed study from the University of Michigan that explores decision-support for parents receiving…
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FDA approval · 2022-12-23FDA Approved: ALKINDI SPRINKLE — Supplemental Application (Eton Pharmaceuticals, Inc.)
FDA approved a supplemental application for ALKINDI SPRINKLE (hydrocortisone) from Eton Pharmaceuticals. The approval updates its labeling a…
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CLINICAL_TRIALS trial_result · 2022-05-03Clinical Trial Update: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia [Phase 2]
Phase 2 clinical trial assessing safety, tolerability, pharmacokinetics, and pharmacodynamics of NBI-74788 in adults with congenital adrenal…
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CLINICAL_TRIALS withdrawal · 2022-04-08Clinical Trial Update: Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy [Phase 3]
Phase 3 open-label comparison trial of Chronocort versus standard glucocorticoid therapy for congenital adrenal hyperplasia was withdrawn.
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FDA approval · 2022-02-03FDA Approved: ALKINDI SPRINKLE — Supplemental Application (Eton Pharmaceuticals, Inc.)
FDA approved a supplemental application for ALKINDI SPRINKLE (hydrocortisone) from Eton Pharmaceuticals. The action represents an approval o…
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CLINICAL_TRIALS trial_result · 2021-03-04Clinical Trial Update: Nevanimibe HCl for the Treatment of Classic CAH [Phase 2]
A Phase 2 trial of nevanimibe HCl for congenital adrenal hyperplasia was terminated; results were posted.