Clinical trials: CAH - 21-Hydroxylase Deficiency
Congenital Adrenal Hyperplasia
Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height.
- Trial name
- NCT02574910
- Testing
- Abiraterone acetate
- Where
- 4 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
Congenital Adrenal Hyperplasia (CAH)
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of OMS1620
The goal of this study is to explore the safety, drug concentrations and effects on adrenal gland hormones after a single dose or multiple doses of OMS1620 in healthy participants.
- Trial name
- NCT07811089
- Testing
- OMS1620
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- OMass Therapeutics Australia Proprietary Ltd
Congenital Adrenal Hyperplasia, Adrenal Insufficiency
Evaluation of Circadian Hormonal Balance: LC-MS/MS Measurement of Salivary and Serum Steroids in Patients With Congenital Adrenal Hyperplasia
In congenital adrenal hyperplasia (CAH), lifelong hormone replacement therapy is required to treat adrenal insufficiency and to reduce elevated androgen levels. This is essential to ensure "normal" growth and puberty.
- Conditions
- Congenital Adrenal Hyperplasia, Adrenal Insufficiency, Hyperandrogenism; 21-hydroxylase Deficiency
- Trial name
- MASSALIVE (NCT07718230)
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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)
CAH - Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia
This is a Phase 2, open-label, multiple-dose, dose-escalation study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 in up to 30 adult female and male subjects (18 to 50 years of…
- Trial name
- NCT03525886
- Testing
- NBI-74788
- Where
- 6 sites in United States
- Volunteers
- 18 people
- Sponsor
- Neurocrine Biosciences
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT07159841
- Testing
- Atumelnant
- Where
- 51 sites in United States, Argentina and 11 more
- Volunteers
- 153 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
Congenital Adrenal Hyperplasia
Cardiovascular Risk Profile in Patients With Congenital Adrenal Hyperplasia
Treatment with glucocorticoids and mineralocorticoids has changed congenital adrenal hyperplasia (CAH) from a fatal to a chronic lifelong disease.
- Trial name
- cardiohcs (NCT01807364)
- Where
- France
- Volunteers
- 170 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Congenital Adrenal Hyperplasia, 21-OHD
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH
An investigation of the safety and efficacy of tildacerfont in participants with CAH.
- Trial name
- NCT05128942
- Testing
- Tildacerfont
- Where
- 13 sites in United States
- Volunteers
- 67 people
- Sponsor
- Spruce Biosciences
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
Congenital Adrenal Hyperplasia
Androgen Reduction in Congenital Adrenal Hyperplasia
Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height.
- Trial name
- ARCH (NCT03548246)
- Testing
- Abiraterone acetate, Hydrocortisone, Fludrocortisone
- Where
- 4 sites in United States
- Sponsor
- University of Texas Southwestern Medical Center
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
Neonatal Salt Loss, Congenital Adrenal Hyperplasia (CAH)
Syndromes With Neonatal Salt Loss: Not Only Congenital Adrenal Hyperplasia Due to 21-hydroxylase Deficiency (21OH-ISC)
Neonatal salt loss can be caused not only by infections but also by rare endocrine disorders that resemble 21-hydroxylase deficiency but are not detected by neonatal screening.
- Trial name
- NCT07473804
- Where
- Italy
- Volunteers
- 25 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
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
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
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old
The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to <4 years of age with CAH.
- Trial name
- NCT07536269
- Testing
- Crinecerfont
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Neurocrine Switzerland GmbH
Adrenal Hyperplasia, Congenital
Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their Mothers
Congenital adrenal hyperplasia (CAH) is a genetic disorder that affects the amount of steroids that the body forms. The most common form of CAH is 21-hydroxylase deficiency (21OHD), which leads to cortisol deficiency and causes…
- Trial name
- NCT00617292
- Where
- 4 sites in United States, Brazil and 1 more
- Volunteers
- 233 people, healthy volunteers welcome
- Sponsor
- Office of Rare Diseases (ORD)
Congenital Adrenal Hyperplasia, Healthy
Catecholamine Reserve and Exercise Tolerance in Healthy Volunteers and Patients With Congenital Adrenal Hyperplasia
This study will examine and compare the effects of intense exercise on the release of catecholamines in patients with congenital adrenal hyperplasia (CAH) and in healthy persons.
- Trial name
- NCT00011791
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
21-hydroxylase Deficiency
Examining Genetic Differences Among People With 21-Hydroxylase Deficiency
Congenital adrenal hyperplasia (CAH) is a genetic disorder that affects the amount of steroids that the body forms. The most common form of CAH is 21-hydroxylase deficiency (21OHD), which leads to cortisol deficiency.
- Trial name
- NCT00542841
- Testing
- Hydrocortisone withdrawal
- Where
- 3 sites in United States, Brazil
- Volunteers
- 99 people
- Sponsor
- Maria I. New
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
Qualitative Research on Women With Congenital Adrenal Hyperplasia
This qualitative interview-based study will investigate the impact that living with congenital adrenal hyperplasia has for women in the following areas: health-related quality of life (HRQL), psychological health, and…
- Trial name
- CAH (NCT00559078)
- Testing
- Interview
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Lehigh University