Study on the Effects of Tildacerfont and Hydrocortisone in Adults with Classic Congenital Adrenal Hyperplasia

What is this study about?

This clinical trial is focused on studying a condition called (CAH), which is a genetic disorder affecting the adrenal glands. The trial will evaluate a new treatment using a medication called , also known by its code name . The purpose of the study is to assess how effective and safe Tildacerfont is for adults with CAH who have high levels of certain hormones in their blood.

Participants in the study will receive treatment over a period of up to 70 weeks. The study is designed to be “double-blind,” meaning neither the participants nor the researchers will know who is receiving Tildacerfont and who is receiving a placebo. This helps ensure the results are unbiased. The study will be conducted in three parts, with the main goal being to see if Tildacerfont can reduce levels of a hormone called androstenedione over 12 weeks.

In addition to Tildacerfont, some participants may continue their current treatment with , a medication commonly used to manage CAH. The trial will monitor changes in hormone levels and any side effects experienced by participants. The results will help determine if Tildacerfont is a beneficial treatment option for people with CAH.

1 initial assessment

Upon joining the study, an initial assessment will be conducted to confirm eligibility. This includes reviewing your medical history and current treatment for classic congenital adrenal hyperplasia.

You will need to provide information about your current medications, including hydrocortisone or other glucocorticoids, and ensure that your dosage has been stable for at least one month.

2 baseline evaluation

A baseline evaluation will be performed to measure your hormone levels, specifically androstenedione and 17-hydroxyprogesterone. These levels will be used to assess changes during the trial.

If you have the salt-wasting form of the condition, your mineralocorticoid replacement dose must also be stable for at least one month before this evaluation.

3 treatment period part 1

The first part of the treatment period involves taking the study medication, tildacerfont, in tablet form. This medication is taken orally.

The dosage and frequency will be determined by the study protocol, and you will be required to follow the instructions carefully for the duration of this part.

4 treatment period part 2

The second part of the treatment period continues with the administration of tildacerfont. Your response to the medication will be monitored through regular check-ups and hormone level assessments.

This part of the treatment aims to evaluate the effect of the medication on reducing androstenedione levels over a 12-week period.

5 treatment period part 3

The final part of the treatment period involves continued monitoring and assessment of your hormone levels and overall health.

The study will evaluate the proportion of participants who achieve normal hormone levels and any changes in testicular adrenal rest tumors, if applicable.

6 end of treatment evaluation

At the end of the treatment period, a comprehensive evaluation will be conducted to assess the overall impact of the study medication on your condition.

This includes measuring the percent change in androstenedione levels from the baseline and other secondary outcomes.

Who Can Join the Study?

Participants must be 18 years or older.
Participants must have a known childhood diagnosis of classic congenital adrenal hyperplasia due to a specific enzyme deficiency. This should be confirmed by a genetic test or past medical records showing high levels of a hormone called 17-hydroxyprogesterone. They should currently be treated with certain medications like hydrocortisone or similar drugs.
Participants must have been on a stable dose of their medication for at least 1 month before joining the study.
Participants must have levels of a hormone called androstenedione that are higher than normal at both the start of the study and at Week 4, depending on their medication dose.
If participants have a form of the condition that affects salt balance, they must be on a stable dose of a specific medication for at least 1 month before joining the study.
Participants must agree to follow specific guidelines for contraception during the study. Male participants must also agree not to donate sperm during the study and for 90 days after the last dose of the study drug.
Participants must be able to understand all study procedures and risks and provide written consent to show they agree to follow all study requirements.

Who Cannot Join the Study?

Patients who do not have Classic Congenital Adrenal Hyperplasia cannot participate.
Patients who are not within the specified age range cannot participate.
Patients who are not part of the specified clinical trial groups cannot participate.
Patients who are not female or male cannot participate.
Patients who are not considered part of a vulnerable population cannot participate.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
Ospedale San Raffaele S.r.l.	Milan	Italy
Karolinska University Hospital	Solna	Sweden
University Hospital Galway	Galway	Ireland
East Tallinn Central Hospital	Tallin	Estonia
Pauls Stradins Clinical University Hospital	Riga	Latvia
Virgen del Rocío University Hospital	Sevilla	Spain
Tartu University Hospital	Tartu	Estonia
Ludwig Maximilian University Of Munich	Munich	Germany
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie	Cracow	Poland
Spitalul Universitar De Urgenta Militar Central Dr. Carol Davila	Bucharest	Romania
Region Midtjylland	Aarhus	Denmark
Rigshospitalet	Copenhagen	Denmark
Azienda Ospedaliero-Universitaria Sant Andre	Rome	Italy
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Lietuvos sveikatos mokslu universiteto ligonine Kauno klinikos	Kaunas	Lithuania
Sana Monitoring S.R.L.	Bucharest	Romania
Hospital Universitari Joan XXIII De Tarragona	Tarragona	Spain
Region Dalarna	Falun	Sweden
National Institute Of Endocrinology C.I. Parhon	Bucharest	Romania
Beaumont Hospital	Dublin	Ireland
Svmvgosli Rmkbnpw Uqlezhleew Mgsehmr Cycxtq	Nijmegen	The Netherlands
Ea Gdigl Skr z oihw	Warsaw	Poland
Auwansn Obylaurmjvh Uhxmgsbvddofq Cnskhyrofkgd Dmqnv Sfwwmh E Dpviy Scrgvjx Dl Tcadet	Turin	Italy
Htuvqklw Vvxt ddphqjtp	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	01.07.2020
Estonia	Not recruiting	01.07.2020
Germany	Not recruiting	01.07.2020
Ireland	Not recruiting	01.07.2020
Italy	Not recruiting	01.07.2020
Latvia	Not recruiting	01.07.2020
Lithuania	Not recruiting	01.07.2020
Poland	Not recruiting	01.07.2020
Romania	Not recruiting	01.07.2020
Spain	Not recruiting	01.07.2020
Sweden	Not recruiting	01.07.2020
The Netherlands	Not recruiting	01.07.2020

Trial locations

Investigated drugs:

Tildacerfont is a medication being studied for its potential to help people with a condition called congenital adrenal hyperplasia. This condition affects the adrenal glands, which are small organs on top of the kidneys that produce important hormones. In people with this condition, the adrenal glands produce too much of a hormone called androstenedione. Tildacerfont is being tested to see if it can help reduce the levels of this hormone in the body, which might help manage the symptoms of the condition. The study aims to see how well this medication works and if it is safe for people to use over a period of 12 weeks.

Classic Congenital Adrenal Hyperplasia – Classic Congenital Adrenal Hyperplasia is a genetic disorder affecting the adrenal glands, which are responsible for producing certain hormones. This condition leads to a deficiency in enzymes needed for hormone production, resulting in an imbalance of hormones such as cortisol and aldosterone. As a result, the body produces excess androgens, which are male sex hormones. The disease often manifests in early childhood and can cause symptoms such as early puberty, rapid growth during childhood but shorter than average final height, and ambiguous genitalia in females. Over time, individuals may experience issues with salt balance, blood pressure, and energy levels. The progression of the disease can vary, with some individuals experiencing more severe symptoms than others.

Trial ID:

2023-503770-21-00

Protocol code:

SPR001-203

NCT ID:

NCT04457336

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effects of Tildacerfont and Hydrocortisone in Adults with Classic Congenital Adrenal Hyperplasia

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?