Clinical trials: CAH - 21-Hydroxylase Deficiency
Adrenal Hyperplasia, Congenital
Role of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal Hyperplasia
21-hydroxylase deficiency (21-OHD) is an inherited disorder that results from a mutation on the CYP21A2 gene. It affects the adrenal glands and is the most common cause of congenital adrenal hyperplasia (CAH).
- Trial name
- NCT00694525
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Office of Rare Diseases (ORD)
Congenital Adrenal Hyperplasia
Electronic Registry of Male Patients With Congenital Adrenal Hyperplasia 21-hydroxylase Deficiency
Observational, retrospective, prospective, single-center cohort study. Participation in the registry will be offered consecutively to any patient with the disease, newly diagnosed or with documented diagnosis made at another…
- Trial name
- NCT06754423
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Congenital Adrenal Hyperplasia
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due…
- Trial name
- NCT04806451
- Testing
- Crinecerfont
- Where
- 46 sites in United States, Belgium and 8 more
- Volunteers
- 103 people
- Sponsor
- Neurocrine Biosciences
21-hydroxylase Deficiency
A Study Examining Doses of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency
The purpose of this study is to determine the minimum dose of abiraterone acetate needed to decrease serum androstenedione to age-appropriate levels in premenopausal women on steroid replacement for classic 21-hydroxylase…
- Trial name
- NCT01495910
- Testing
- Abiraterone acetate
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Congenital Adrenal Hyperplasia
A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT02804178
- Testing
- ATR-101
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Millendo Therapeutics, Inc.
Congenital Adrenal Hyperplasia, Exercise Capacity
Exercise Capacity, Physical Fitness, and Physical Activity Levels in Children With CAH
Congenital Adrenal Hyperplesia (CAH) is a group of automosal recessive disorders that develop due to a deficiency of one of the five enyzmes necessary for cortisol synthesis in the adrenal cortex.
- Conditions
- Congenital Adrenal Hyperplasia, Exercise Capacity, Physical Activity, Quality of Life
- Trial name
- NCT06449456
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
CAH - Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia
This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a…
- Trial name
- NCT04045145
- Testing
- Crinecerfont
- Where
- 6 sites in United States
- Volunteers
- 8 people
- Sponsor
- Neurocrine Biosciences
Congenital Adrenal Hyperplasia
Final Height in Patients With CAH
Congenital adrenal hyperplasia (CAH) is the most common inherited disorder in the adrenal gland in children. Growth is usually affected in CAH patients either due to the disease itself or treatment consequences.
- Trial name
- NCT04293133
- Volunteers
- 30 people
- Sponsor
- Ain Shams University
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
The purpose of this study is to evaluate the efficacy, safety, PK, and PD of atumelnant in adults with classic CAH due to 21-OHD.
- Trial name
- NCT07144163
- Testing
- Atumelnant
- Where
- 75 sites in United States, Argentina and 13 more
- Volunteers
- 150 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)
The purpose of this Phase 2, open-label, sequential dose cohort study is to evaluate the safety, efficacy, and pharmacokinetics (PK) of atumelnant (CRN04894) in participants with classic congenital adrenal hyperplasia (CAH)…
- Trial name
- NCT05907291
- Testing
- atumelnant (CRN04894)
- Where
- 27 sites in United States, Argentina and 5 more
- Volunteers
- 38 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
Congenital Adrenal Hyperplasia
A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])
This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 [NCT04783181] gene therapy trial with BBP-631.
- Trial name
- NCT05101902
- Testing
- Pre-Screening
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Adrenas Therapeutics Inc
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
- Conditions
- Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
- Trial name
- NCT03687242
- Testing
- SPR001
- Where
- 8 sites in United States
- Volunteers
- 11 people
- Sponsor
- Spruce Biosciences
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia
This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT03257462
- Testing
- SPR001
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Spruce Biosciences
Adrenal Hyperplasia, Congenital
An Extension Trial of Asedebart in Participants With Congenital Adrenal Hyperplasia
The main goal of the trial is to study the long-term safety, how well people can handle the treatment, and how asedebart acts in the body and affects the body when used to treat classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT07859293
- Testing
- Asedebart
- Volunteers
- 45 people
- Sponsor
- H. Lundbeck A/S
Congenital Adrenal Hyperplasia
Effects and Importance of Epinephrine/Adrenalin Deficiency in CAH
Individuals with CAH produce lower levels of epinephrine (adrenalin) than controls. This can be correlated to the CYP21A2 genotype and is most pronounced in the classic forms.
- Trial name
- NCT05162950
- Testing
- physical exercise as a standardized high intensity exercise test, Stress vulnerability
- Where
- Sweden
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Region Stockholm
Endocrine Disease, Adrenal Insufficiency
Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH
The purpose of this study is to gather safety and effectiveness information about a new formulation of Hydrocortisone (Chronocort®) used to treat patients with a disease called congenital adrenal hyperplasia (CAH).
- Conditions
- Endocrine Disease, Adrenal Insufficiency, Congenital Adrenal Hyperplasia
- Trial name
- NCT01735617
- Testing
- Hydrocortisone Modified Release Capsules
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Neurocrine UK Limited
Congenital Adrenal Hyperplasia (CAH)
Salt Wasting, Hydro-sodium Balance and Fludrocortisone Requirement in Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) in its classic neonatal form with severe salt-wasting represents a challenge for pediatric endocrinologists in order to maintain sodium balance, especially as the physiopathology and optimal…
- Trial name
- NaCAH (NCT03550261)
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
CAH - 21-Hydroxylase Deficiency
Parenting and CAH - 21-hydroxylase Deficiency
Congenital adrenal hyperplasia (CAH) is a genetic disease with autosomal recessive transmission, which is defined by a deficiency of one of the steroidogenesis enzymes.
- Trial name
- PARENT-HCS (NCT06900153)
- Testing
- phone questionnaire
- Where
- 7 sites in France
- Volunteers
- 200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Classic Congenital Adrenal Hyperplasia, 21-Hydroxylase Deficiency
Pediatric Congenital Adrenal Hyperplasia Management: Regret and Long-term Outcomes
Congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency is a chronic endocrine disorder, often diagnosed in the neonatal period. Severe forms may lead to genital virilization in afected girls and adrenal crises.
- Trial name
- CAH - REC (NCT07781267)
- Testing
- questionnaire
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Lille
Adrenal Hyperplasia, Congenital, 21-hydroxylase Deficiency
Adrenocortical Functions in Women With Nonclassical 21-hydroxylase Deficiency.
The risk of adrenal insufficiency in patients with nonclassical congenital adrenal hyperplasia due to 21-hydroxylase deficiency is not well documented.
- Trial name
- EVAF-21 (NCT01862380)
- Testing
- Functional explorations
- Where
- France
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris