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Congenital Adrenal Hyperplasia clinical trials

View clinical trials related to Congenital Adrenal Hyperplasia.

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NCT ID: NCT05907291 Recruiting - Clinical trials for Congenital Adrenal Hyperplasia

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

Start date: July 3, 2023
Phase: Phase 2
Study type: Interventional

The purpose of this Phase 2, open-label, sequential dose cohort study is to evaluate the safety, efficacy, and pharmacokinetics (PK) of CRN04894 in participants with classic congenital adrenal hyperplasia (CAH) caused by 21-hydroxylase deficiency.

NCT ID: NCT05687474 Recruiting - Cystic Fibrosis Clinical Trials

Baby Detect : Genomic Newborn Screening

Start date: September 1, 2022
Phase:
Study type: Observational

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.

NCT ID: NCT05669950 Recruiting - Clinical trials for Congenital Adrenal Hyperplasia

Study of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

Start date: December 19, 2022
Phase: Phase 1
Study type: Interventional

This study will evaluate the effects of different doses of Lu AG13909 in adult participants with congenital adrenal hyperplasia, also called CAH. CAH is a rare genetic disorder that affects a person's ability to produce certain hormones. The main goals of this study are to learn about the safety and tolerability of Lu AG13909, how Lu AG13909 behaves in the body, and how the body responds to Lu AG13909.

NCT ID: NCT05663320 Recruiting - Clinical trials for Congenital Adrenal Hyperplasia

A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in Adolescent and Young Adults With Congenital Adrenal Hyperplasia

Start date: March 31, 2024
Phase: N/A
Study type: Interventional

Background: Children with congenital adrenal hyperplasia (CAH) can survive well into adulthood with proper treatment. But the change from pediatric to adult care can be challenging. Many people with CAH need extra support as they learn to take control of their own health care needs. Researchers have studied how people respond to different types of patient education. Now researchers want to find out if virtual education, via computer, is a good way to teach people how to manage CAH as they become adults. Objective: To test a virtual method of delivering patient education to adolescents and young adults with CAH. Eligibility: Adolescents and young adults aged 16 to 22 years who have CAH. They must already be enrolled in Natural History Study Protocol 06-CH-0011. Design: Participants may take part in the study remotely; they may also come to the clinic. They will have 3 visits in 1 year. Participants will complete questionnaires. Topics will include what they know about CAH; whether they remember to take their medications on their own; and whether they schedule their own appointments. They will be asked about their quality of life. They will be asked about their physical and emotional health. All participants will be taught how to care for themselves. The participants will be divided into 2 groups. Some will watch an 11-minute video on CAH that focuses on their goals as they become adults. The others will receive standard education. After 6 months, participants will receive CAH education again. After 12 months, participants will repeat the questionnaires from their first visit.

NCT ID: NCT05299554 Enrolling by invitation - Clinical trials for Congenital Adrenal Hyperplasia

Long-term Safety Study of Chronocort in the Treatment of Participants With Congenital Adrenal Hyperplasia

Start date: April 1, 2022
Phase: Phase 3
Study type: Interventional

This phase III study is an open-label extension study to be conducted at approximately 21 investigational sites across 3 countries. The study will evaluate the long-term safety and tolerability of Chronocort in participants aged 16 years and over when used as treatment for Congenital Adrenal Hyperplasia (CAH).

NCT ID: NCT05162950 Enrolling by invitation - Clinical trials for Congenital Adrenal Hyperplasia

Effects and Importance of Epinephrine/Adrenalin Deficiency in CAH

Start date: September 1, 2020
Phase:
Study type: Observational

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. Individuals with CAH have an increased risk of developing hypoglycemia because both cortisol and epinephrine are important counter regulatory hormones. Stress dosing is essential in situations of increased physical stress such as infections with fever for example. Glucocorticoid treatment and stress dosing cannot compensate fully during physical stress neither for the reaction to psychological stress. This may render various types of difficulties in the individual's life. We aim to investigate if the deficient epinephrine production can be confirmed and if it is related to the increased level of anxiety and vulnerability to stress that we observe in the patients. Specific aims of the study: - Analyse the epinephrine/adrenalin production in patients with CAH using measurements of epinephrine and metanephrine in blood, during an exercise test - Assess stress vulnerability and anxiety using validated questionnaires - Correlate the results to severity of disease, CYP21A2 genotype - Investigate if psychological and somatic stress symptoms are related to the epinephrine production capacity.

NCT ID: NCT05128942 Recruiting - Clinical trials for Congenital Adrenal Hyperplasia

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

Start date: December 10, 2021
Phase: Phase 2
Study type: Interventional

An investigation of the safety and efficacy of tildacerfont in pediatric subjects with CAH.

NCT ID: NCT05101902 Recruiting - Clinical trials for Congenital Adrenal Hyperplasia

A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

Start date: July 1, 2021
Phase:
Study type: Observational

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.

NCT ID: NCT05063994 Completed - Clinical trials for Congenital Adrenal Hyperplasia

Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia

CONnECT
Start date: December 13, 2021
Phase: Phase 3
Study type: Interventional

This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal Hyperplasia.

NCT ID: NCT04903587 Recruiting - Clinical trials for Congenital Adrenal Hyperplasia

Gonadal Changes In Congenital Adrenal Hyperplasia Patients

Start date: October 1, 2021
Phase:
Study type: Observational

To detect the prevalence of gonadal changes by US among the patients with CAH. - assess the patients' radiological findings in relation to their hormonal profile. - early management and prevention of complications resulting from possible gonadal dysfunction.