congenital-adrenal-hyperplasia Clinical Trials
A listing of congenital-adrenal-hyperplasia medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 24 clinical trials
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 CRN04894 in participants with classic congenital adrenal hyperplasia (CAH) caused by 21-hydroxylase deficiency.
Study of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia
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 …
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
This study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic 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
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 …
Fertility and Pregnancy in Patients With Classic Congenital Adrenal Hyperplasia
Classic congenital adrenal hyperplasia (CAH) is a rare genetic endocrine disorder. Its prevalence is around 1/15.000. It results from a deficiency in 21-hydroxylase, an adrenal steroidogenic enzyme involved in the biosynthesis of cortisol and aldosterone. Enzyme deficiency in the steroideogenesis pathway leads to cortisol and aldosterone deficiency of varying severity, …
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. Too much androgen in childhood, if untreated, results in rapid growth and early puberty with …
China Adrenal Disease Registry
Epidemiologic studies have revealed a tremendous increase in the prevalence of adrenal associated disease and related mortality worldwide. In order to meet all the therapeutic challenges in adrenal disease in China, CASE was founded in 2020. The objective of CASE is to launch an adrenal disease management model based on …
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome. During childhood, patients with rare genetic syndromes receive multidisciplinary and specialized medical care; they usually receive medical care from 3-4 medical specialists. …
Baby Detect : Genomic Newborn Screening
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 …
Questionnaire Study to Assess the Outcomes of the Management of Congenital Adrenal Hyperplasia Individuals
This is a questionnaire study involving women and young girls affected by Congenital Adrenal Hyperplasia (CAH) and their parents. The aim is to obtain information regarding the outcomes of conservative or surgical management of CAH at the Royal Manchester Children's Hospital (RMCH) in the last 50 years, with specific reference …