Crinecerfont for Adrenal Hyperplasia, Congenital

Phase-Based Estimates
Neurocrine Clinical Site, Barcelona, Spain
Adrenal Hyperplasia, Congenital+4 More
Crinecerfont - Drug
< 18
All Sexes
Eligible conditions
Adrenal Hyperplasia, Congenital

Study Summary

This study is evaluating whether a drug may help treat a rare condition in children.

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Eligible Conditions

  • Adrenal Hyperplasia, Congenital
  • Adrenocortical Hyperfunction
  • Adrenogenital Syndrome
  • Hyperplasia
  • Congenital Adrenal Hyperplasia (CAH)

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

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.

Baseline to Week 28
Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 28
Change from baseline in body mass index at Week 28
Change from baseline in salivary 17-OHP at Week 28
Change in bone age advancement at Week 28
Percent Change from Baseline in Glucocorticoid Daily Dose at Week 28
Baseline to Week 4
Change from Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
Change from Baseline in Serum Androstenedione (A4) at Week 4
Baseline to Week 52
Change from baseline in predicted adult height at Week 52

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

Placebo group

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.

Solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment for 24 weeks.
PlaceboSolution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment for 24 weeks.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline to week 52
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline to week 52 for reporting.

Closest Location

Neurocrine Clinical Site - Pittsburgh, PA

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. There are 5 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Have high levels of androgens. show original
Patients who may become pregnant must avoid sex or use birth control during the study. show original
Please be available to attend all study-related procedures and return for the follow-up visit. show original
You must have a medically confirmed diagnosis of 21-hydroxylase deficiency to be eligible for services. show original
If you have CAH, you should be on a stable regimen of steroidal treatment. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can adrenal hyperplasia, congenital be cured?

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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.

Unverified Answer

What causes adrenal hyperplasia, congenital?

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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.

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What are the signs of adrenal hyperplasia, congenital?

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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.

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What are common treatments for adrenal hyperplasia, congenital?

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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.

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How many people get adrenal hyperplasia, congenital a year in the United States?

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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.

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What is adrenal hyperplasia, congenital?

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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.

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Is crinecerfont safe for people?

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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.

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What are the latest developments in crinecerfont for therapeutic use?

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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.

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Who should consider clinical trials for adrenal hyperplasia, congenital?

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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.

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Have there been any new discoveries for treating adrenal hyperplasia, congenital?

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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].

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What does crinecerfont usually treat?

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[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”.

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Is crinecerfont typically used in combination with any other treatments?

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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.

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