This trial is evaluating whether Crinecerfont will improve 1 primary outcome and 7 secondary outcomes in patients with Adrenal Hyperplasia, Congenital. Measurement will happen over the course of Baseline to Week 4.
This trial requires 81 total participants across 2 different treatment groups
This trial involves 2 different treatments. Crinecerfont is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.
It can be cured with surgery in very few cases but remains as a progressive disease unless treated properly. It is best diagnosed as early as possible in order to give the child and family the best prognosis by the end of its treatment.
Findings from a recent study, we confirm that the commonest cause of adrenal stenosis and hypertrophic adrenal hyperplasia is a result of inadequate enzymatic activity of cortisol 17 alpha-hydroxylase and 21-hydroxylase.
It is the most common endocrine disorder in males. Adrenal insufficiency or excess is characterized by hypoglycaemic symptoms in young and middle-aged adults. In children, adrenal insufficiency can develop at any age but is more likely to occur in early life. In adults, adrenal insufficiency usually has multiple causes and is often under-reported. It is more common in women with polycystic ovarian syndrome and infertility. Diagnosis of CAH relies on biochemical markers. These include the 21-hydroxylase assay and the 17α-hydroxyprogesterone measurement. Imaging of the adrenal glands may also be required.
The cause of adrenal hyperplasia, congenital adrenal hyperplasia, seems to be multifactorial. There are genetic, endocrine, and other causes that may necessitate an interdisciplinary care approach. These conditions can be treated by medications, dietary and behavioral changes, and surgery/excision. The prognosis and outcome may depend on underlying factors and the extent of disease. Patients seeking treatment must be carefully screened and monitored, based on severity, to best manage complications.
Since adrenal hyperplasia is a very uncommon condition, it is impossible to estimate how many people are affected in a year in the United States.
Adrenal hyperplasia is a very rare malformation of the adrenal gland that most commonly arises from an abnormal embryological process and presents as the first sign of the disease before the onset of clinically apparent symptoms. Patients typically present with the classic triad of features: 1) signs and symptoms of hypoglycemia (hypoglycemia due to the ACTH response to hypoglycemia is usually undetected), 2) growth retardation or short stature (if not growth hormone deficiency), and 3) impaired adrenal gland function (secondary adrenal insufficiency). Patients are typically diagnosed at an immature age, ≤5 years of age.
Results from a recent clinical trial, we found that crinecerfont was safe during 24-h post-dose pharmacokinetic and pharmacodynamic evaluation in healthy volunteers. We also found that the results of the pharmacokinetic analysis of the plasma concentrations were comparable among study subjects of different age groups, indicating that the safety of crinecerfont is comparable across patients of different age groups.
New synthetic organic drugs seem to be a better alternative to synthetic steroids since they have an even wider therapeutic window, lower incidence of gynecomastia and lower risk of osteoporosis and osteomalacia.
Only a small proportion of patients with congenital adrenal hyperplasia have been subjected to clinical trials to date, and such trials have been less successful than trials in non-CAH patients. This has prompted renewed interest in the benefits of clinical trials for these patients. Data from a recent study suggests that patients who are older and/or have congenital adrenal hyperplasia should be offered clinical trials for phentolamine in the context of a rigorous clinical trial protocol.
Adrenal insufficiency in children is very hard to treat because it is a chronic condition. Although new drugs have been discovered for the treatment of adrenocortical insufficiency, they have not proven effective enough to be universally recommended. It is crucial that all affected individuals are treated in a timely manner, as soon as they're diagnosed with the disorder because the chances of a positive response for all the drugs known to be effective are very slim. To find out about adrenal insufficiency and any other forms of pediatric adrenocortical insufficiency you can check the [Emedicine website].
[As most individuals with congenital adrenal hyperplasia (CAH), particularly in the United States, will be exposed to increased hormonal stress from the premature adrenarche (larval stage of development of the human adrenal gland) during childhood and adolescence, such individuals are sometimes referred to as having an “adrenal crisis”.
Crinecerfont is commonly used in combination with other nephroprotective agents in the treatment of adenocarcinoma. However, a randomized trial needs to be performed in the future.