Clinical trials: Congenital Adrenal Hyperplasia, open to volunteers
Mild Autonomous Cortisol Secretion (MACS)
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
To characterize the impact of Isturisa on clinical features and comorbidities associated with MACS. The investigators hypothesize that patients treated with Isturisa will exhibit significantly better metabolic indicators (such as…
- Trial name
- NCT07247058
- Testing
- Osilodrostat (Isturisa)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Johns Hopkins 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
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
Development of Healthcare Transition for Patients With Congenital Adrenal Hyperplasia
The purpose of this study is to implement and evaluate the feasibility and acceptability of a structured healthcare transition program for adolescents and young adults with congenital adrenal hyperplasia (CAH).
- Trial name
- NCT07611786
- Testing
- exposure to healthcare transition protocol
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
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
Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adult Participants With Classic Congenital Adrenal Hyperplasia (CAH)
The main objective of this study is to evaluate the efficacy of different dose levels of NBIP-01435 on reducing elevated androstenedione (A4) levels in adults with classic CAH.
- Trial name
- NCT07850739
- Testing
- NBIP-01435
- Volunteers
- 36 people
- Sponsor
- Neurocrine Biosciences
Mild Autonomous Cortisol Secretion (MACS), Autonomous Cortisol Secretion (ACS)
Metyrapone Study for Patients Diagnosed With Mild Autonomous Cortisol Secretion - MACS
single-center, randomized, double-blind, placebo-controlled Phase 2 study to evaluate safety and efficacy of overnight metyrapone in patients with MACS.
- Trial name
- NCT07138274
- Testing
- Metyrapone 250 mg Oral Tablets
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Mayo Clinic
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
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 (CAH), Familial Male-Limited Precocious Puberty (FMPP)
Natural History Study of Patients With Excess Androgen
This study will evaluate and gather information in patients with genetic causes of too much androgen (male-like hormone) in order to better understand the effects of too much androgen and describe problems associated with it.
- Trial name
- NCT00250159
- Where
- 2 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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
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
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
An Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal Hyperplasia
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of atumelnant (CRN04894).
- Trial name
- CALM2-CAH (NCT06712823)
- Testing
- atumelnant (CRN04894)
- Where
- 13 sites in United States, Argentina and 4 more
- Volunteers
- 200 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
CAH - Congenital Adrenal Hyperplasia
Hydrocortisone Modified-release in Adults: Real-world Monitoring of Longitudinal Outcomes in coNgenital Adrenal hYperplasia
Classic congenital adrenal hyperplasia (CAH) is an autosomal recessive genetic disorder caused by a defect in the enzyme cascade regulating adrenal steroidogenesis; in approximately 95% of cases the defect is located in CYP21A2…
- Trial name
- HARMONY (NCT07622030)
- Volunteers
- 100 people
- Sponsor
- IRCCS San Raffaele
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
Congenital Adrenal Hyperplasia
A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia
This trial will evaluate the effects of different doses of Lu AG13909 in adult participants with congenital adrenal hyperplasia, also called CAH.
- Trial name
- NCT05669950
- Testing
- Lu AG13909
- Where
- 17 sites in United States, Denmark and 7 more
- Volunteers
- 42 people
- Sponsor
- H. Lundbeck A/S
Mild Autonomous Cortisol Secretion, Autonomous Cortisol Secretion (ACS)
Impact of 1 mg Osilodrostat Therapy on Mild Autonomous Cortisol Secretion (MACS)
The purpose of this study is to evaluate the safety and tolerability of 1 mg osilodrostat therapy in patients with mild autonomous cortisol secretion (MACS), and to determine the impact on 24h urine steroid metabolome and…
- Trial name
- NCT07104812
- Testing
- Osilodrostat 1 MG
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Mayo Clinic
Rare Diseases, Amyloidosis
Institutional Registry of Rare Diseases
The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD).
- Conditions
- Rare Diseases, Amyloidosis, Sarcoidosis, Phacomatosis
- Trial name
- NCT06573723
- Where
- Argentina
- Volunteers
- 380 people
- Sponsor
- Hospital Italiano de Buenos Aires
Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatment
This is a controlled, open study designed to compare the effects of dual-release hydrocortisone preparations versus conventional glucocorticoid therapy on clinical, anthropometric parameters, metabolic syndrome, hormonal profile…
- Trial name
- CareOnTIME (NCT03760835)
- Testing
- Conventional Glucocorticoids (immediate release hydrocortisone, cortisone acetate, prednisone, prednisolone, dexamethasone), Dual release hydrocortisone (plenadren)
- Where
- Italy
- Volunteers
- 150 people
- Sponsor
- Federico II University