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Adrenal Hyperplasia, Congenital clinical trials

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NCT ID: NCT03550261 Completed - Clinical trials for Congenital Adrenal Hyperplasia (CAH)

Salt Wasting, Hydro-sodium Balance and Fludrocortisone Requirement in Congenital Adrenal Hyperplasia

NaCAH
Start date: May 17, 2018
Phase:
Study type: Observational

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 therapeutic management of this urinary salt loss remain poorly studied, particularly during the neonatal period. The human kidney presents the characteristic of being immature at birth with a functional tubulopathy associating sodium wasting and difficulty to concentrate urine, in connection with a transient renal resistance to aldosterone action, which is exacerbated in case of CAH by insufficiency of aldosterone production. The objective of project is therefore to study the secretion profiles of plasma and urinary steroids in neonates with classical salt-wasting form of CAH before treatment and under treatment with Fludrocortisone and Hydrocortisone during the first months of life, using an advanced technology: LC-MSMS (Liquid chromatography coupled with tandem mass spectrometry). The study of the existence of a correlation between plasma and urinary steroid profiles will also make it possible to subsequently consider simplified medical follow-up for these patients. This project will lead to a better understanding of sodium handling and steroid secretion and excretion profiles in CAH neonates, in order to improve the therapeutic management of mineralocorticoid replacement in these patients.

NCT ID: NCT03525886 Completed - Clinical trials for CAH - Congenital Adrenal Hyperplasia

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia

Start date: April 10, 2018
Phase: Phase 2
Study type: Interventional

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 age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH). The study will include a sequential-cohort design with four NBI-74788 dosing regimens, with each regimen administered for 14 days.

NCT ID: NCT03257462 Completed - Clinical trials for Congenital Adrenal Hyperplasia

Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

Start date: July 26, 2017
Phase: Phase 2
Study type: Interventional

This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH).

NCT ID: NCT03174847 Completed - Clinical trials for Primary Aldosteronism

Prospective Study Assessing Blood Pressure and Other Outcomes Post-treatment in Patients With Primary Aldosteronism

PA_PACES
Start date: February 20, 2017
Phase:
Study type: Observational

Majority of patients with hypertension have primary hypertension (without an underlying cause). Secondary hypertension (due to an underlying disease) is important to recognize, as treatment can lead to cure of hypertension. Primary aldosteronism (PA) is the most common cause of secondary hypertension, and can be found in 5-10% of patients locally. PA is caused by excessive release of a hormone (aldosterone) from the adrenal glands, which can be unilateral (one gland) or bilateral (both glands). Distinction between two is crucial as unilateral disease is treated with the aim of cure by surgery, and bilateral disease is treated by medication. It has been shown that excess aldosterone has other harmful effects in addition to hypertension, such as directly affecting the heart, blood vessels, kidneys, diabetes and quality of life. This is supported by studies showing reversal of these effects after treatment for PA. In addition, improvements after surgery appears to be superior to medical treatment, although studies have found variable results. Hence, the investigators aim to accurately subtype patients with PA into unilateral or bilateral disease and study the post-treatment response after both surgery and medicine with regards to the effects on blood pressure, cardiovascular, renal, metabolic and quality of life.

NCT ID: NCT03162172 Completed - Clinical trials for Adrenal Hyperplasia, Congenital

Growth Hormone (GH) in Congenital Adrenal Hyperplasia

OPALE GH
Start date: September 15, 2015
Phase: N/A
Study type: Observational

Congenital adrenal hyperplasia (CAH) is a genetic rare disease, which alters the adrenal production of gluco and mineralo corticoids. The treatment consists in supplementing children using hydrocortisone. Despite care for these children has improve substantially across decades, short adult height still remains an important consequence of the disease. About 20 % of patients have an AH below 2 standard deviations compared to their expected height. In the OPALE model study, the investigators have collected data from a cohort of 496 French patients, born between 1970 and 1991 and with a known genotype. Using their age, sex, growth, disease, bone maturation and pubertal data, they have built a model which allows to predict their AH using data available at 8 years of age. This model has shown that the currently used formula to calculate the predicted AH (Bayley Pineau's method) is not applicable to children with CAH. In this project, the investigators plan to use the prediction model to compare the AH in patients who have received GH treatment to their predicted AH using the model. The hypothesis is that GH improves the AH in such patients. Existing cohorts have shown improved growth celerity, and growth expectation using the Bayley-Pineau formula), but this has not been shown on the actual AH. This study will allow to reinforce the investigators' hypothesis.

NCT ID: NCT03162159 Completed - Clinical trials for Congenital Adrenal Hyperplasia

Adult Height Prediction in Congenital Adrenal Hyperplasia

OPALE Model
Start date: September 2010
Phase:
Study type: Observational

Congenital Adrenal Hyperplasia (CAH) is a genetic rare disease, which alters the adrenal production of gluco and mineralo corticoïds. The treatment consists in supplementing children with hydrocortisone. Despite care for these children has improved substantially across decades, short adult height (AH) still remains an important consequence of the disease. About 20% of patients have an AH below 2 standard deviations compared to their expected AH. In the OPALE-Model study, the investigators want to collect data from a cohort of 496 CAH French patients, born between 1970 and 1991 with a known genotype. Using their age, sex, growth, disease, bone maturation and pubertal data, the investigators will build a model which allows to predict their AH using data available at 8 years of age. The growth charts built from this cohort have shown that currently used formula to calculate the predicted AH (Bayley-Pineau's formula) is not applicable to children with CAH. In this project, the investigators plan to compute an AH prediction model using data from children born between 1970 and 1993, and to validate the model using data from a different cohort (i.e. children born between 1994 and 1998). this choice was due to availability of data for computing the model first, and in a second stage, data from more recently born patients.

NCT ID: NCT03062280 Completed - Clinical trials for Congenital Adrenal Hyperplasia

A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH

Start date: August 18, 2016
Phase: Phase 3
Study type: Interventional

Subjects completing study DIUR-005 and those who have already completed study DIUR-003 will be offered the opportunity either to continue Chronocort® therapy or to switch from their current glucocorticoid therapy to Chronocort® in this open-label study.

NCT ID: NCT03051893 Completed - Clinical trials for Adrenal Insufficiency

A Two-part, Study to Compare the Pharmacokinetics and Dose Proportionality of up to 6 Chronocort Formulations

Start date: February 2011
Phase: Phase 1
Study type: Interventional

This was an open label, randomised, single dose study, comprising Part A (undertaken in two separate three-period crossover cohorts denoted as A1 and A2) and Part B (undertaken in one four-period crossover cohort), to evaluate the PK of Chronocort® in healthy male volunteers. The washout interval in both Part A and Part B was 1-week in between each treatment period.

NCT ID: NCT03019614 Completed - Clinical trials for Adrenal Insufficiency

An Open Label Study in Healthy Volunteers to Compare Chronocort® to Hydrocortisone

Start date: March 2010
Phase: Phase 1
Study type: Interventional

This was an open label, randomized, single dose, three period crossover pharmacokinetic study of Chronocort® in 30 healthy male volunteers. The study was conducted in smaller sub groups (Group 1, n=18 and Group 2, n=12).

NCT ID: NCT02934399 Completed - Acromegaly Clinical Trials

Dynamic Hormone Diagnostics in Endocrine Disease

ultradian
Start date: October 2016
Phase:
Study type: Observational

The study will investigate 27 hour profiles of hormones in the subcutaneous tissue of healthy subjects and patients with Addison's, Congenital Adrenal Hyperplasia, Growth Hormone Deficiency, acromegaly, Cushings and Primary Hyperaldosteronism during conventional diagnostic and therapeutic follow-up. The 27 hour monitoring by ULTRADIAN takes into account the rhythm of hormones throughout the day. It is hoped that this information may in the future improve and simplify diagnostic procedures. Follow-up of patients in endocrinology still remains difficult including clinical signs of over and under-treatment, questionnaires of quality of life and blood testing necessitating often retesting. Simplification of the diagnostic procedure by obtaining detailed knowledge about the rhythm of hormones may contribute to the improvement and individualization of treatment and may decrease morbidity and mortality of endocrine patients.