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Home › Conditions › Congenital Adrenal Hyperplasia

Congenital Adrenal Hyperplasia

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Congenital Adrenal Hyperplasia

10 trials tracked for Congenital Adrenal Hyperplasia: 3 in phase 3 or 4 and 2 with published results. The most-cited published study has 83 citations.

10Trials tracked
3Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 3 3 Phase 2 6 Phase 1 1
  1. Phase 3 Comparison of Chronocort® With Standard Glucocorticoid Therapy in Patients With Congenital Adrenal Hyperplasia Completed · 83 cited
  2. Phase 3 A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH Completed · 83 cited
  3. Phase 3 Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia Completed
  4. Phase 2 Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia Completed
  5. Phase 2 A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia Completed
  6. Phase 2 Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH Completed
Show 4 more trials
  1. Phase 2 Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia Completed
  2. Phase 2 Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasia Completed
  3. Phase 2 Dexamethasone Treatment of Congenital Adrenal Hyperplasia Completed
  4. Phase 1 Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia Completed

Showing the 10 most-cited and recently-updated of 10 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Congenital Adrenal Hyperplasia: what the trials found

Established evidence from multiple phase 3 trials demonstrates that Chronocort (hydrocortisone) is associated with significant biochemical improvements. Specifically, patients treated with Chronocort showed a significantly higher percentage of biochemical responders at week 28 compared to standard therapy (40.0% vs. 14.3%; p=0.0003) and a higher percentage of dose responders (36.0% vs. 10.7%; p=0.012). Additionally, the total daily dose of hydrocortisone was significantly lower in the Chronocort group at week 28 (20.2 mg vs. 26.0 mg; p=0.0005).

Clinical data from phase 3 trials also indicate improvements in quality of life scores (SF-36) over 12 to 18 months 1. Other studies evaluated the impact of hydrocortisone and Chronocort on 17-hydroxyprogesterone (17-OHP) and androstenedione (A4) levels; while some trials showed specific counts for disease control at various times of day 1, others noted that changes in mean standard deviation scores for these markers did not reach statistical significance 2. Early phase studies also evaluated the safety and efficacy of SPR001 [4, 5] and Chronocort's impact on morning 17-OHP levels 8.

Recent results — preliminary, needs further review

  • * ATR-101 showed a reduction of 17-hydroxyprogesterone to ≤ 2 times the upper limit of normal in 2 participants, but this finding is not yet corroborated 6. * Hydrocortisone modified release capsules showed an 81.3% success rate for reaching optimal ranges for 17-OHP and androstenedione at 0700h compared to 56.3% on standard therapy, though this is not yet corroborated 7. * Dexamethasone showed a mean log transformed area under the curve of 17-hydroxyprogesterone with a p-value of 0.09, which is not yet corroborated 9. * Subcutaneous hydrocortisone was evaluated in a phase 1 trial but results are not yet corroborated 10.

For the clinician treating this condition

  • Chronocort demonstrates superior biochemical response rates and lower total daily hydrocortisone requirements compared to standard therapy at week 28.
  • Clinical data suggests improvements in patient quality of life (SF-36) over a 12-18 month period with hydrocortisone treatment.

AI synthesis of 7 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
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