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Crinecerfont

Crinecerfont, also known as NBI-74788 or Crenessity, is an investigational drug being studied for the treatment of Congenital Adrenal Hyperplasia (CAH). This article explores the ongoing clinical trials evaluating the safety, efficacy, and tolerability of Crinecerfont in both adult and pediatric patients with classic CAH due to 21-hydroxylase deficiency. These trials aim to assess the drug’s potential in reducing glucocorticoid doses and improving hormone balance in CAH patients.

Table of Contents

What is Crinecerfont?

Crinecerfont, also known by its research name NBI-74788, is a new medication being developed to treat Congenital Adrenal Hyperplasia (CAH). It is currently undergoing clinical trials to evaluate its safety and effectiveness in both adults and children with this condition[1][2][3].

What Condition Does Crinecerfont Treat?

Crinecerfont is specifically designed to treat Congenital Adrenal Hyperplasia (CAH). CAH is a genetic disorder that affects the adrenal glands, which are small glands located on top of the kidneys. In people with CAH, the adrenal glands cannot produce certain hormones properly, particularly cortisol. This leads to an overproduction of male hormones (androgens) and can cause various health issues[1][2][3].

How Does Crinecerfont Work?

Crinecerfont is a CRF1-receptor antagonist. This means it blocks a specific receptor in the body that is involved in the production of certain hormones. By doing so, Crinecerfont aims to reduce the overproduction of androgens (male hormones) in people with CAH. This could potentially help manage the symptoms of the condition and reduce the need for high doses of other medications currently used to treat CAH[2][3].

Clinical Trials for Crinecerfont

Crinecerfont is currently being studied in several clinical trials:

  • A Phase 2 trial in adolescents (14-17 years old) with CAH[1]
  • A Phase 3 trial in adults with classic CAH[2]
  • A Phase 3 trial in children with classic CAH[3]

These trials are designed to evaluate the safety, effectiveness, and proper dosing of Crinecerfont in different age groups. They involve both short-term and long-term treatment periods to gather comprehensive data on the medication’s effects[1][2][3].

Potential Benefits of Crinecerfont

Based on the clinical trials, researchers are investigating several potential benefits of Crinecerfont for people with CAH:

  • Reduction in glucocorticoid dose: Crinecerfont may allow patients to reduce their daily dose of glucocorticoids, which are currently used to treat CAH but can have significant side effects when used long-term[2][3]
  • Improved hormone balance: The medication aims to reduce levels of certain hormones that are typically elevated in CAH, such as androstenedione and 17-hydroxyprogesterone[1][2][3]
  • Metabolic improvements: Researchers are studying whether Crinecerfont can help improve various metabolic factors, including insulin resistance, body weight, and fat mass[2]
  • Other potential benefits: The trials are also investigating effects on blood pressure, glucose tolerance, menstrual regularity in females, and testicular adrenal rest tumors in males[2]

How is Crinecerfont Administered?

In the clinical trials, Crinecerfont is being administered orally (by mouth). It is given twice daily, either as a capsule or as a solution. The exact dosage and formulation may vary depending on the age of the patient and the specific trial[1][2][3].

It’s important to note that Crinecerfont is still an investigational drug. This means it has not yet been approved by regulatory agencies like the FDA for general use. Its safety and effectiveness are still being studied, and it is only available to patients participating in clinical trials[1][2][3].

Aspect Details
Drug Name Crinecerfont (NBI-74788, Crenessity)
Condition Studied Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency
Study Phases Phase 2 and Phase 3
Age Groups Adults and Pediatric (14-17 years)
Administration Oral (capsule or solution), twice daily
Primary Outcomes Changes in hormone levels (17-OHP, androstenedione), glucocorticoid dose reduction
Secondary Outcomes Body composition changes, metabolic parameters, menstrual regularity, TART volume
Study Duration Core studies: 14-20 months; Open-label extension: ~3 years
Mechanism of Action CRF type 1 receptor antagonist

FAQ

  • What is Crinecerfont and how does it work? Crinecerfont is a CRF type 1 receptor antagonist being studied for the treatment of Congenital Adrenal Hyperplasia (CAH). It works by targeting the underlying mechanism of CAH to potentially reduce the need for high doses of glucocorticoids and improve hormone balance.
  • What age groups are being studied in the Crinecerfont clinical trials? The clinical trials are studying both adult and pediatric patients with CAH. One study focuses on adults, while another specifically targets pediatric participants aged 14 to 17 years.
  • How long do the clinical trials for Crinecerfont last? The duration varies depending on the trial. The adult study lasts approximately 20 months for the core study, with an additional open-label extension estimated to last about 3 years. The pediatric study has a core duration of about 14 months, also with an open-label extension of approximately 3 years.
  • What are the main outcomes being measured in these trials? The trials are measuring several outcomes, including changes in hormone levels (such as 17-OHP, androstenedione, and ACTH), changes in glucocorticoid daily dose, body composition changes, and various metabolic parameters like insulin resistance and glucose tolerance.
  • How is Crinecerfont administered in these trials? Crinecerfont is administered orally, either as a capsule or solution, typically twice daily. The exact dosage and formulation may vary depending on the specific trial and participant age group.

Ongoing Clinical Trials on Crinecerfont

  • Study of crinecerfont oral solution in children under 2 years old with congenital adrenal hyperplasia to evaluate safety and how the drug works in the body

    Recruiting

    1 1
    Investigated drugs:
    Germany

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

    Not recruiting

    1 1
    Investigated drugs:
    Belgium France Germany Greece Italy Poland +1

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

    Not recruiting

    1 1
    Investigated drugs:
    Austria Belgium Bulgaria Czechia France Germany +6

Glossary

  • Congenital Adrenal Hyperplasia (CAH): A group of genetic disorders affecting the adrenal glands, most commonly due to 21-hydroxylase deficiency, leading to imbalances in hormone production.
  • 21-hydroxylase deficiency: The most common form of CAH, caused by a deficiency in the enzyme 21-hydroxylase, which is necessary for cortisol and aldosterone production.
  • Glucocorticoid: A class of steroid hormones that includes cortisol, often used as replacement therapy in CAH patients.
  • 17-hydroxyprogesterone (17-OHP): A hormone that is typically elevated in patients with CAH due to 21-hydroxylase deficiency.
  • Androstenedione: An androgen hormone that is often elevated in CAH patients and used as a marker of disease control.
  • Adrenocorticotropic Hormone (ACTH): A hormone produced by the pituitary gland that stimulates the adrenal glands and is often elevated in CAH.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Pharmacodynamics (PD): The study of a drug's effects on the body, including its mechanism of action and relationship between drug concentration and effect.
  • Open-label extension (OLE): A phase of a clinical trial where all participants receive the active drug, often following a placebo-controlled period.
  • CRF type 1 receptor antagonist: A type of drug that blocks the action of corticotropin-releasing factor (CRF) at its receptor, which is the mechanism of action for Crinecerfont.

References