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Encaleret Sulfate

This article explores the ongoing clinical trials of Encaleret Sulfate, a promising drug being studied for the treatment of Autosomal Dominant Hypocalcemia Type 1 (ADH1). ADH1 is a rare genetic disorder that affects calcium levels in the body, leading to various symptoms and complications. The trials aim to assess the efficacy and safety of Encaleret Sulfate compared to standard care in managing this condition.

Table of Contents

What is Encaleret?

Encaleret sulfate, also known as CLTX-305 (previously JTT-305 and MK-5442), is a new medication being developed to treat a rare genetic condition called Autosomal Dominant Hypocalcemia Type 1 (ADH1)[1]. It comes in the form of a film-coated tablet that is taken by mouth[1].

What is Autosomal Dominant Hypocalcemia Type 1 (ADH1)?

Autosomal Dominant Hypocalcemia Type 1 (ADH1) is a rare genetic disorder that affects the body’s calcium balance[1]. People with ADH1 have low levels of calcium in their blood (hypocalcemia) and often experience symptoms such as:

  • Muscle cramps and spasms
  • Tingling sensations (paresthesia)
  • Seizures
  • Cognitive issues (like difficulty concentrating)
  • Fatigue
  • Headaches
  • Anxiety

ADH1 is caused by mutations in the CASR gene, which controls how the body regulates calcium levels[1].

How Encaleret Works

Encaleret is designed to target the underlying cause of ADH1. It works by interacting with the calcium-sensing receptor (CaSR) in the body, which is overactive in people with ADH1[1]. By modulating the activity of this receptor, Encaleret aims to help normalize calcium levels in the blood and reduce the symptoms associated with ADH1.

Clinical Trial Information

A Phase 3 clinical trial called CALIBRATE is currently underway to evaluate the effectiveness and safety of Encaleret compared to standard treatment for ADH1[1]. The trial aims to:

  1. Assess how well Encaleret corrects blood calcium levels and reduces calcium excretion in urine
  2. Evaluate its effects on parathyroid hormone levels
  3. Measure improvements in quality of life for patients
  4. Monitor for any side effects or safety concerns

The trial is open to adults (18 years and older) with a confirmed diagnosis of ADH1 and specific symptoms or signs of the condition[1].

Potential Benefits of Encaleret

If successful, Encaleret could offer several benefits for people with ADH1:

  • Improved calcium balance in the body
  • Reduction in symptoms associated with low calcium levels
  • Potential decrease in the need for calcium and vitamin D supplements
  • Better quality of life

Possible Side Effects and Precautions

As Encaleret is still being studied, all potential side effects are not yet known. The clinical trial will closely monitor participants for any adverse events[1]. Some precautions and considerations include:

  • Patients with severe kidney problems (eGFR <30 mL/min/1.73 m2) are not eligible for the trial
  • The medication may interact with certain other drugs, such as digoxin
  • Pregnant or nursing women are excluded from the trial
  • Patients with a history of certain cancers or recent use of specific medications may not be eligible

Conclusion

Encaleret sulfate represents a promising new approach to treating Autosomal Dominant Hypocalcemia Type 1. By targeting the underlying cause of the condition, it has the potential to improve calcium balance and quality of life for people with ADH1. However, as the medication is still in clinical trials, more research is needed to fully understand its effectiveness and safety profile. Patients with ADH1 who are interested in learning more about Encaleret should consult with their healthcare providers and consider whether participating in clinical trials might be appropriate for their individual situations.

Aspect Details
Study Type Phase 3, Randomized, Open-Label
Primary Objective Evaluate efficacy of Encaleret vs. standard care in managing blood calcium and urinary calcium excretion
Key Inclusion Criteria Adults with ADH1, documented CASR gene variant, history of ADH1 symptoms
Key Exclusion Criteria Recent hypocalcemic seizures, certain cancers, renal transplantation, pregnancy
Primary Endpoint Responder status based on blood calcium and 24-hr urinary calcium levels
Secondary Endpoints Effects on iPTH, phosphate, magnesium levels; QTcF duration; patient-reported outcomes; safety and tolerability
Treatment Duration 24 weeks for primary endpoint assessment

FAQ

  • What is Autosomal Dominant Hypocalcemia Type 1 (ADH1)? ADH1 is a rare genetic disorder characterized by low calcium levels in the blood. It is caused by mutations in the CASR gene, which regulates calcium metabolism in the body.
  • What is the main goal of the Encaleret Sulfate clinical trial? The main goal is to demonstrate the efficacy of Encaleret compared to standard care treatment in managing blood calcium levels and 24-hour urinary calcium excretion in patients with ADH1.
  • Who can participate in the Encaleret clinical trial? Participants must be at least 18 years old, have a documented pathogenic variant of the CASR gene, and a history of ADH1 symptoms. They must also meet specific criteria regarding calcium and parathyroid hormone levels.
  • What are some of the exclusion criteria for the trial? Exclusion criteria include a history of hypocalcemic seizures within the past 3 months, certain types of cancer, renal transplantation, pregnancy, and severe kidney dysfunction.
  • How long does the clinical trial last? The trial includes multiple periods, with the primary endpoint being assessed at the completion of the maintenance period (Period 3), which is 24 weeks into the study.

Ongoing Clinical Trials on Encaleret Sulfate

  • Study of encaleret compared to standard care in patients with autosomal dominant hypocalcemia type 1

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia Denmark France Italy The Netherlands

Glossary

  • Autosomal Dominant Hypocalcemia Type 1 (ADH1): A rare genetic disorder characterized by low calcium levels in the blood, caused by mutations in the CASR gene.
  • CASR gene: A gene that provides instructions for making the calcium-sensing receptor protein, which helps regulate calcium levels in the body.
  • Encaleret Sulfate: The investigational drug being studied for the treatment of ADH1, also known as CLTX-305.
  • Albumin-corrected blood calcium (cCa): A measurement of calcium in the blood that takes into account the levels of albumin, a protein that binds to calcium.
  • 24-hr urinary calcium (UCa) excretion: The amount of calcium excreted in urine over a 24-hour period, used to assess calcium metabolism.
  • Intact parathyroid hormone (iPTH): A hormone produced by the parathyroid glands that helps regulate calcium levels in the blood.
  • QTcF: The QT interval on an electrocardiogram corrected for heart rate using the Fridericia formula, used to assess cardiac electrical activity.
  • Nephrocalcinosis: A condition where there is too much calcium deposited in the kidneys.
  • Nephrolithiasis: The formation of kidney stones, often related to calcium metabolism disorders.
  • Standard of Care (SoC): The current best practice treatment for a condition, used as a comparison for new treatments in clinical trials.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-encaleret-for-patients-with-autosomal-dominant-hypocalcemia-type-1-adh1-compared-to-standard-treatment/