Study on the Safety and Effectiveness of Crinecerfont for Adults with Classic Congenital Adrenal Hyperplasia

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What is this study about?

This clinical trial is focused on studying a condition known as Classic Congenital Adrenal Hyperplasia (CAH). This is a genetic disorder that affects the adrenal glands, which are responsible for producing important hormones. The study is testing a new treatment called Crinecerfont, also known by its code name NBI-74788. This medication is being compared to a placebo to see how effective it is in managing the symptoms of CAH.

The purpose of the study is to evaluate how well Crinecerfont works in reducing the need for other medications called glucocorticoids, which are often used to treat CAH. The study will also look at how Crinecerfont affects hormone levels in the body and its overall safety. Participants will take the medication in capsule form, twice a day, for a period of time. The study will start with a four-week treatment period, followed by additional assessments to monitor changes in hormone levels and any side effects.

Throughout the study, researchers will collect information on how Crinecerfont affects the body, including its impact on hormone levels and any potential side effects. The study aims to provide a better understanding of how this new treatment can help manage Classic Congenital Adrenal Hyperplasia and improve the quality of life for those affected by this condition.

1 initial treatment phase

The study begins with a randomized, double-blind, placebo-controlled phase. Participants receive either Crinecerfont (NBI-74788) or a placebo.

The medication is administered in the form of oral capsules. The dosage for Crinecerfont is 100 mg taken twice daily.

This phase aims to evaluate the reduction in daily glucocorticoid dosage while maintaining control over adrenal androgens.

2 evaluation period

After an initial 4-week treatment period, the study evaluates the reduction in adrenal steroid levels.

The effect of Crinecerfont on clinical endpoints related to high glucocorticoid dosing is assessed.

Plasma concentrations of Crinecerfont and its metabolites are measured to ensure safety and tolerability.

3 adjustment phase

Participants who have not reduced their glucocorticoid dose by Month 12 may be evaluated for an alternate dosing regimen of Crinecerfont.

The goal is to achieve a reduction in glucocorticoid daily dose to physiologic levels by Week 24.

4 open-label treatment

Following the double-blind phase, participants may enter an open-label treatment phase where all receive Crinecerfont.

This phase continues to monitor safety, efficacy, and any changes in health markers such as weight and fat mass.

5 final assessment

The study concludes with a final assessment of the primary and secondary endpoints.

The primary endpoint is the percent change from baseline in glucocorticoid daily dose at Week 24.

Secondary endpoints include changes in serum androstenedione, HOMA-IR, weight, and fat mass.

Who Can Join the Study?

Subjects must provide written informed consent. This means you agree to participate in the study and understand what it involves.
Be a female or male at least 18 years of age.
Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency. This is a specific type of condition related to the adrenal glands.
Be on a stable, supraphysiologic glucocorticoid dose regimen that has been stable for at least 1 month prior to screening. This means you are taking a higher than normal dose of a specific medication for at least a month before the study starts.
If treated with fludrocortisone, the dose should be stable for at least 1 month prior to screening. During screening, your plasma renin activity (PRA) should not be greater than the upper limit of normal (ULN) on your usual salt intake. If PRA is higher than normal, your blood pressure should be over 100 mmHg, without a drop in blood pressure when standing up, and your blood sodium and potassium levels should be normal.
Female subjects of childbearing potential with fertile male partners must agree to use contraception consistently from screening until the final study visit or 30 days after the last dose of study drug, whichever is longer. This means using birth control to prevent pregnancy during the study and for a short time after.

Who Cannot Join the Study?

Patients who are not diagnosed with Classic Congenital Adrenal Hyperplasia (CAH) cannot participate. This is a condition where the body has trouble making certain hormones.
Patients who are unable to reduce their daily glucocorticoid dosage while maintaining control of adrenal androgens are excluded. Glucocorticoids are a type of steroid medication, and adrenal androgens are hormones produced by the adrenal glands.
Patients who cannot reduce adrenal steroid levels after an initial 4-week treatment period are not eligible. Adrenal steroids are hormones produced by the adrenal glands.
Patients who have not reduced their glucocorticoid dose by Month 12 are excluded.
Patients who are not within the specified age range for the study cannot participate. The study includes specific age groups.
Both male and female patients are eligible, but those who do not meet gender criteria are excluded.
Patients who are part of a vulnerable population, which may include groups like children or those with certain disabilities, are not eligible.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country
Medical University Of Vienna	Vienna	Austria
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy
Szpital Bielanski Im.Ks.Jerzego Popieluszki samodzielny publiczny zakład opieki zdrowotnej	Warsaw	Poland
Medical University Of Graz	Graz	Austria
Centro Hospitalar Universitario Sao Joao E.P.E.	Porto	Portugal
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Katholieke Universiteit te Leuven	Leuven	Belgium
CHU Grenoble Alpes	La Tronche	France

Other Sites

Site Name	City	Country
Ippokratio General Hospital Of Thessaloniki	Thessaloniki	Greece
Centrum Nowoczesnych Terapii Dobry Lekarz Sp. z o.o.	Cracow	Poland
Azienda Ospedaliera di Padova	Padua	Italy
Evangelismos S.A.	Athens	Greece
Virgen del Rocío University Hospital	Sevilla	Spain
Ospedale San Raffaele S.r.l.	Milan	Italy
Azienda Unita Sanitaria Locale Di Bologna	Bologna	Italy
Fakultni Nemocnice Hradec Kralove	Novy Hradec Kralove	Czechia
Karolinska University Hospital	Solna	Sweden
Universita’ Politecnica Delle Marche	Ancona	Italy
Centre Hospitalier Universitaire De Nantes	Nantes	France
Uniwersytecki Szpital Kliniczny W Poznaniu	Poznan	Poland
Hopital Beaujon	Clichy	France
Cqapew Huzdotwhgro Rvqoudzz Dgvjqsklryxmto	Angers	France
Ltbzx Glhymah Hzqmbyrz Ow Ahhcvh	Athens	Greece
Giixlp Uzkcgakcty Fppiusuur	Frankfurt	Germany
Kwbdjekz dxm Uquxifhcaxnh Maxcieha Asv	Munich	Germany
Upykglzwtb Dwkya Scfrw Di Rdgh Lk Sozflhln	Rome	Italy
Mtwhvgg Cuvdyf Afwgm Inu Pcarkkf Eszi	Sofia	Bulgaria
Isdwa Otbxzdcb Afqhuuswzi Sra Lzfm	Milan	Italy

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	23.12.2020
Belgium	Not recruiting	23.12.2020
Bulgaria	Not recruiting	23.12.2020
Czechia	Not recruiting	23.12.2020
France	Not recruiting	23.12.2020
Germany	Not recruiting	23.12.2020
Greece	Not recruiting	23.12.2020
Italy	Not recruiting	23.12.2020
Poland	Not recruiting	23.12.2020
Portugal	Not recruiting	23.12.2020
Spain	Not recruiting	23.12.2020
Sweden	Not recruiting	23.12.2020

Trial locations

Investigated drugs:

Crinecerfont

Crinecerfont (NBI-74788) is a medication being studied for its potential to help adults with Classic Congenital Adrenal Hyperplasia. This condition affects the adrenal glands, which produce important hormones. The trial is testing if Crinecerfont can reduce the need for other medications called glucocorticoids, which are often used to manage this condition. The study also looks at how well Crinecerfont can control hormone levels and its overall safety for patients.

Classic Congenital Adrenal Hyperplasia (CAH) – This is a genetic disorder affecting the adrenal glands, which are responsible for producing certain hormones. In CAH, the body lacks an enzyme needed to make cortisol, a hormone crucial for stress response, blood pressure, and other vital functions. As a result, the adrenal glands produce excess androgens, which are male sex hormones. This hormonal imbalance can lead to early signs of puberty in children and affect growth and development. Symptoms may include ambiguous genitalia in newborns, rapid growth during childhood, and early or excessive hair growth. The condition is present from birth and requires lifelong management.

Trial ID:

2023-509171-16-00

Protocol code:

NBI-74788-CAH3003

NCT ID:

NCT04490915

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Effectiveness of Crinecerfont for Adults with Classic Congenital Adrenal Hyperplasia

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