Congenital Adrenal Hyperplasia

Nephrology
9
Pipeline Programs
5
Companies
10
Clinical Trials
3 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
7
1
1
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Crinetics Pharmaceuticals
4 programs
2
1
1
AtumelnantPhase 31 trial
AtumelnantPhase 2/31 trial
atumelnantPhase 21 trial
atumelnantPhase 21 trial
Active Trials
NCT06712823Recruiting150Est. Nov 2027
NCT05907291Completed38Est. Aug 2025
NCT07159841Recruiting153Est. Mar 2030
+1 more trials
Spruce Biosciences
Spruce BiosciencesSOUTH SAN FRANCISCO, CA
5 programs
5
SPR001Phase 21 trial
SPR001Phase 21 trial
TildacerfontPhase 21 trial
Tildacerfont/PlaceboPhase 21 trial
Tildacerfont/PlaceboPhase 21 trial
Active Trials
NCT03687242Completed11Est. Aug 2019
NCT03257462Completed24Est. Mar 2019
NCT05128942Terminated67Est. Jan 2025
+2 more trials
Orchard Therapeutics
1 program
Baby Detect : Genomic Newborn ScreeningN/A
Sanofi
SanofiPARIS, France
1 program
Baby Detect : Genomic Newborn ScreeningN/A
Takeda
TakedaTOKYO, Japan
1 program
Baby Detect : Genomic Newborn ScreeningN/A1 trial
Active Trials
NCT05687474Completed6,824Est. Jun 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Crinetics PharmaceuticalsAtumelnant
Crinetics PharmaceuticalsAtumelnant
Crinetics Pharmaceuticalsatumelnant
Crinetics Pharmaceuticalsatumelnant
Spruce BiosciencesTildacerfont
Spruce BiosciencesTildacerfont/Placebo
Spruce BiosciencesTildacerfont/Placebo
Spruce BiosciencesSPR001
Spruce BiosciencesSPR001
TakedaBaby Detect : Genomic Newborn Screening

Clinical Trials (10)

Total enrollment: 7,613 patients across 10 trials

NCT07144163Crinetics PharmaceuticalsAtumelnant

A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia

Start: Dec 2025Est. completion: May 2027150 patients
Phase 3Recruiting
NCT07159841Crinetics PharmaceuticalsAtumelnant

A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

Start: Jan 2026Est. completion: Mar 2030153 patients
Phase 2/3Recruiting
NCT06712823Crinetics Pharmaceuticalsatumelnant

An Extension Study to Evaluate Safety and Efficacy in Participants Treated With CRN04894

Start: Feb 2025Est. completion: Nov 2027150 patients
Phase 2Recruiting
NCT05907291Crinetics Pharmaceuticalsatumelnant

Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)

Start: Jul 2023Est. completion: Aug 202538 patients
Phase 2Completed
NCT05128942Spruce BiosciencesTildacerfont

A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH

Start: Dec 2021Est. completion: Jan 202567 patients
Phase 2Terminated
NCT04544410Spruce BiosciencesTildacerfont/Placebo

A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH

Start: Feb 2021Est. completion: Jan 2025100 patients
Phase 2Terminated
NCT04457336Spruce BiosciencesTildacerfont/Placebo

A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

Start: Aug 2020Est. completion: May 202496 patients
Phase 2Terminated
NCT03687242Spruce BiosciencesSPR001

Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia

Start: Sep 2018Est. completion: Aug 201911 patients
Phase 2Completed
NCT03257462Spruce BiosciencesSPR001

Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

Start: Jul 2017Est. completion: Mar 201924 patients
Phase 2Completed
NCT05687474TakedaBaby Detect : Genomic Newborn Screening

Baby Detect : Genomic Newborn Screening

Start: Sep 2022Est. completion: Jun 20256,824 patients
N/ACompleted

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
3 actively recruiting trials targeting 7,613 patients
5 companies competing in this space

Related Indications

Chronic Kidney Disease(86)Renal Impairment(67)End Stage Renal Disease(56)Kidney Transplantation(49)Renal Insufficiency(27)Chronic Kidney Diseases(25)Kidney Transplant(19)Diabetic Nephropathies(18)Kidney Diseases(17)Kidney Transplant Rejection(16)