#1 Clinical Trials Search in Europe

Tildacerfont

Tildacerfont, also known as SPR001, is an investigational drug being studied in clinical trials for the treatment of Congenital Adrenal Hyperplasia (CAH) and Polycystic Ovary Syndrome (PCOS). These trials aim to evaluate the safety, efficacy, and potential benefits of Tildacerfont in managing hormone imbalances and reducing the need for high doses of glucocorticoids in patients with these conditions. The studies involve both adults and children, exploring various dosing regimens and treatment durations to determine the optimal use of this promising medication.

Table of Contents

What is Tildacerfont?

Tildacerfont, also known by its research name SPR001, is an investigational drug currently being studied for its potential to treat certain hormonal disorders[1][2]. It is a new type of medication that aims to help regulate hormone production in the body, particularly in conditions where hormone levels are imbalanced.

What Conditions Does Tildacerfont Treat?

Tildacerfont is being investigated primarily for two conditions:

  1. Congenital Adrenal Hyperplasia (CAH): This is a genetic disorder that affects the adrenal glands, which are responsible for producing various hormones. In CAH, the body cannot produce enough of a hormone called cortisol, leading to an overproduction of male hormones (androgens)[1][2][3].
  2. Polycystic Ovary Syndrome (PCOS): This is a hormonal disorder common among women of reproductive age. It can cause irregular menstrual periods, excess male hormone levels, and small cysts on the ovaries[4].

How Does Tildacerfont Work?

While the exact mechanism of action is not fully explained in the provided clinical trial information, we can infer that Tildacerfont works by helping to regulate hormone production in the body. In the case of CAH, it appears to help reduce the overproduction of androgens (male hormones) and potentially allow for a reduction in glucocorticoid medication (a type of steroid hormone) that patients typically need to take[3]. For PCOS, it seems to target elevated levels of adrenal androgens, specifically a hormone called DHEAS[4].

Current Clinical Trials

Tildacerfont is currently being studied in several clinical trials:

  • A Phase 2 study in children aged 2 to 17 years with CAH, evaluating safety, efficacy, and how the drug is processed in the body (pharmacokinetics)[1].
  • A Phase 2b study in adults with classic CAH, looking at the drug’s effectiveness and safety over 70 weeks of treatment[2].
  • Another Phase 2b study in adults with classic CAH, specifically examining if Tildacerfont can help reduce the need for high doses of glucocorticoid steroids[3].
  • A Phase 2 study in adult women with PCOS and elevated adrenal androgens, evaluating safety and efficacy[4].

How is Tildacerfont Administered?

Tildacerfont is administered as an oral tablet, typically taken once daily[1][2][3][4]. In some studies, it is taken in combination with glucocorticoid therapy, which is a standard treatment for CAH[1]. The dosage and duration of treatment vary depending on the specific study and condition being treated.

Safety and Efficacy

The safety and efficacy of Tildacerfont are primary focuses of the ongoing clinical trials. Researchers are monitoring for any treatment-emergent adverse events (side effects) using standardized criteria[1][4]. The efficacy is being measured through various outcomes, including:

  • Reduction in androgen levels (such as androstenedione and 17-OHP)[2]
  • Ability to reduce glucocorticoid dosing in CAH patients[3]
  • Reduction in DHEAS levels in PCOS patients[4]
  • Improvement in cardiovascular risk factors in CAH patients[3]
  • In male CAH patients, reduction in the size of testicular adrenal rest tumors (TARTs)[2]

Future Prospects

If the ongoing clinical trials show positive results, Tildacerfont could potentially become an important treatment option for people with CAH and certain cases of PCOS. For CAH patients, it might offer a way to better control hormone levels while reducing the need for high doses of steroid medications, which can have significant side effects[3]. For women with PCOS and elevated adrenal androgens, it could provide a targeted approach to managing hormone imbalances[4].

It’s important to note that Tildacerfont is still an investigational drug, and more research is needed to fully understand its benefits and potential risks. Patients interested in this treatment should discuss it with their healthcare providers and consider participating in clinical trials if appropriate.

Aspect Details
Drug Name Tildacerfont (SPR001)
Conditions Studied Congenital Adrenal Hyperplasia (CAH), Polycystic Ovary Syndrome (PCOS)
Age Groups Children (2-17 years), Adults
Administration Oral tablet, daily dosing
Study Durations 4-12 weeks (short-term), Up to 70 weeks (long-term), Optional extensions up to 240 weeks
Primary Outcomes Safety assessment, Changes in hormone levels (A4, DHEAS), Reduction in glucocorticoid use
Secondary Outcomes Normalization of hormone levels, Reduction in TART volume, Improvement in cardiovascular risk factors
Study Designs Randomized, Double-blind, Placebo-controlled, Dose-ranging

FAQ

  • What is Tildacerfont and what conditions is it being studied for? Tildacerfont, also known as SPR001, is an investigational drug being studied in clinical trials for the treatment of Congenital Adrenal Hyperplasia (CAH) and Polycystic Ovary Syndrome (PCOS). It is taken as an oral tablet and aims to help manage hormone imbalances associated with these conditions.
  • How long do the clinical trials for Tildacerfont typically last? The duration of Tildacerfont clinical trials varies depending on the study. Some trials last for 4 to 12 weeks, while others can extend up to 70 weeks or even longer. For example, one study includes an optional open-label treatment extension period of up to 240 weeks.
  • What are the main goals of the Tildacerfont clinical trials? The main goals of the Tildacerfont clinical trials are to evaluate its safety, efficacy, and tolerability in patients with CAH and PCOS. Researchers are looking at its ability to reduce androgen levels, decrease the need for glucocorticoid use in CAH patients, and improve hormone balance in PCOS patients with elevated adrenal androgens.
  • Are there any studies involving children with CAH? Yes, there is a Phase 2 study evaluating the safety, efficacy, and pharmacokinetics of Tildacerfont in children aged 2 to 17 years with Congenital Adrenal Hyperplasia. This study involves different age cohorts and treatment durations to assess the drug's effects in pediatric patients.
  • How is the effectiveness of Tildacerfont measured in these trials? The effectiveness of Tildacerfont is measured through various outcomes, including changes in hormone levels (such as androstenedione, 17-OHP, and DHEAS), reduction in glucocorticoid doses, normalization of adrenal androgen levels, and improvements in clinical symptoms. Researchers also assess safety by monitoring adverse events and changes in cardiovascular risk factors.

Ongoing Clinical Trials on Tildacerfont

  • Study on Tildacerfont to Reduce Glucocorticoid Use in Adults with Classic Congenital Adrenal Hyperplasia

    Not recruiting

    2 1 1
    Investigated drugs:
    Denmark Estonia Germany Ireland Italy Latvia +6

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

    Not recruiting

    2 1
    Investigated drugs:
    Denmark Estonia Germany Ireland Italy Latvia +6

Glossary

  • Congenital Adrenal Hyperplasia (CAH): A group of genetic disorders affecting the adrenal glands, causing hormone imbalances and various health issues.
  • Polycystic Ovary Syndrome (PCOS): A hormonal disorder in women that can cause irregular periods, excess androgen levels, and small cysts on the ovaries.
  • Tildacerfont: An investigational drug (also known as SPR001) being studied for the treatment of CAH and PCOS, taken as an oral tablet.
  • Androstenedione (A4): A hormone produced by the adrenal glands and gonads, which can be elevated in CAH and PCOS.
  • Glucocorticoid (GC): A class of steroid hormones used to treat CAH, but long-term use can have side effects.
  • 17-OHP: 17-hydroxyprogesterone, a hormone that can be elevated in CAH patients.
  • DHEAS: Dehydroepiandrosterone sulfate, an androgen produced by the adrenal glands that can be elevated in PCOS.
  • TART: Testicular Adrenal Rest Tumors, benign growths that can occur in male CAH patients.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • PBPK model: Physiologically Based Pharmacokinetic model, a mathematical model used to predict drug behavior in the body.
  • HCe: Hydrocortisone equivalent, a way to compare different glucocorticoid doses.
  • ULN: Upper Limit of Normal, the highest value of a measurement considered within the normal range.
  • TEAE: Treatment-Emergent Adverse Event, any unfavorable medical occurrence that appears or worsens after starting a treatment.
  • CTCAE: Common Terminology Criteria for Adverse Events, a standardized method for classifying the severity of side effects in clinical trials.

References