Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital...
Congenital Adrenal HyperplasiaClassic Congenital Adrenal HyperplasiaThe 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.
Study of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia
Congenital Adrenal HyperplasiaThis 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.
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
Congenital Adrenal HyperplasiaAn investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic CAH subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.
Calcium Channel Blockade in Primary Aldosteronism
Primary AldosteronismPrimary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)Primary aldosteronism is a common cause of hypertension. Recent evidence suggests that many patients with bilateral idiopathic hyperaldosteronism harbor gain-of-function somatic mutations in zona glomerulosa calcium channels that results in aldosterone production. This finding raises the possibility that calcium channel antagonists may be a targeted therapy to reduce aldosterone production in patients who harbor these mutations.
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children With CAH
Congenital Adrenal Hyperplasia21-OHDAn investigation of the safety and efficacy of tildacerfont in pediatric subjects with CAH.
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Congenital Adrenal HyperplasiaThis study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic congenital adrenal hyperplasia.
Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatment
Congenital Adrenal HyperplasiaThis 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, bone status, quality of life, reproductive, sexual and psychological functions and treatment compliance in patients affected by congenital adrenal hyperplasia due to 21 OH deficiency.
Superselective Adrenal Arterial Embolization for Bilateral Idiopathic Hyperaldosteronism: A Prospective...
Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)The most common two subtypes of primary aldosteronism (PA ) are aldosterone producing adenoma (APA) and bilateral idiopathic hyperaldosteronism (IHA). Mineralocorticoid receptor (MR) antagonists is the main treatment for bilateral IHA, because of its side effects, the treatment compliance of those patients is poor. Hence, an alternative therapy is needed in such cases. We hypothesized that superselective adrenal artery embolization (SAAE) could be a suitable alternative approach. To our knowledge, SAAE has so far not been applied to treat bilateral IHA. This study aimed to evaluate the efficacy and safety of SAAE in the treatment of PA patients with bilateral IHA.
Long-term Safety Study of Chronocort in the Treatment of Participants With Congenital Adrenal Hyperplasia...
Congenital Adrenal HyperplasiaThis phase III study is an open-label extension study to be conducted at approximately 36 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).
A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in...
Congenital Adrenal HyperplasiaBackground: 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.