Encaleret for Familial Hypocalciuric Hypercalcemia
(CALIBRATE-PEDS Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a treatment called encaleret for young people with a condition known as ADH1, which affects calcium levels in the body. The goal is to determine how the body processes encaleret and whether it safely improves symptoms. Children and teens up to 17 years old who have experienced symptoms of hypoparathyroidism (such as low calcium levels) and have been on treatment for at least a few months might be suitable candidates. As a Phase 3 trial, this is the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.
Is there any evidence suggesting that encaleret is likely to be safe for humans?
Research has shown that encaleret is generally safe for people. Studies have found that it helps maintain balanced calcium levels, which is important for health. In past studies, encaleret was well-tolerated, with most participants not experiencing significant side effects. Some mild issues were reported, but these were uncommon. As this trial is in a more advanced phase, evidence already suggests that encaleret is safe for humans.12345
Why do researchers think this study treatment might be promising?
Unlike the standard treatments for Familial Hypocalciuric Hypercalcemia, which typically focus on managing symptoms through dietary calcium restriction and monitoring, Encaleret offers a novel approach by directly targeting the calcium-sensing receptor. This receptor is pivotal in maintaining calcium balance in the body, and Encaleret's mechanism of action may provide more precise control over calcium levels. Researchers are excited about Encaleret because it has the potential to offer a more effective and targeted treatment option, possibly reducing the need for ongoing symptom management and improving quality of life for patients.
What evidence suggests that encaleret might be an effective treatment for familial hypocalciuric hypercalcemia?
Research has shown that encaleret can help control calcium levels in people with Autosomal Dominant Hypocalcemia Type 1 (ADH1). Studies have found that encaleret significantly lowers calcium in urine, with noticeable improvements over a few weeks. This helps maintain balanced calcium levels in the body. By doing so, encaleret may relieve symptoms like muscle cramps and tingling, which are common in ADH1. Overall, evidence supports encaleret as a promising option for managing calcium issues related to ADH1.12467
Who Is on the Research Team?
Calcilytix Medical Director
Principal Investigator
Calcilytix Therapeutics, Inc., a BridgeBio company
Are You a Good Fit for This Trial?
This trial is for children up to 17 years old with Autosomal Dominant Hypocalcemia Type 1 (ADH1). They must show symptoms or have a history of hypoparathyroidism, and carry a specific genetic variant linked to the condition. Participants need to be on ADH1 treatment for at least 3-6 months before joining.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Period 1
Participants receive encaleret dosing for initial pharmacokinetics evaluation
Treatment Period 2
Participants continue encaleret dosing to evaluate efficacy and safety
Treatment Period 3
Participants complete the final phase of encaleret dosing
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term extension
Participants may opt into continuation of encaleret treatment long-term
What Are the Treatments Tested in This Trial?
Interventions
- Encaleret
Trial Overview
The study tests Encaleret's effects in young patients with ADH1. It aims to understand how the body processes the drug (pharmacokinetics), its effectiveness in treating the condition, and any safety concerns that might arise during treatment.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants will receive encaleret dosing in Period 1 (6 days), Period 2 (20 weeks) and Period 3 (4 weeks). Following completion of Period 3, participants will have the option to enter a long-term extension (LTE) to continue encaleret for an additional approximately 24 months or until the sponsor decides to end the study, whichever occurs first.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Calcilytix Therapeutics, Inc., a BridgeBio company
Lead Sponsor
Citations
1.
investor.bridgebio.com
investor.bridgebio.com/news/news-details/2025/BridgeBio-Reports-Positive-Phase-3-Topline-Results-for-Encaleret-in-Patients-with-Autosomal-Dominant-Hypocalcemia-Type-1/default.aspxBridgeBio Reports Positive Phase 3 Topline Results for ...
Encaleret achieved a mean reduction in change from baseline of 200 mg/day in 24-hour urine calcium from Week 4 to Week 24 (p<0.0001); Encaleret ...
A Phase 2, Randomized, Placebo-Controlled, Dose-Ranging ...
Hypercalcemia resulted in reduction in MK-5442 dose in 0%, 7%, 11%, 16%, and 33% of patients in the MK-5442 2.5-, 5.0-, 7.5-, 10-, and 15-mg groups, ...
NCT05680818 | Efficacy and Safety of Encaleret ...
The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment.
Encaleret for Hypocalcemia · Info for Participants
Studies have found that encaleret helps maintain balanced calcium levels in individuals with Autosomal Dominant Hypocalcemia Type 1 (ADH1). In earlier research, ...
A novel chemoreactive calcilytic for the potential treatment ...
ADH causes hypocalcemia with reduced PTH secretion and normal or increased urinary Ca2+o excretion (hypercalciuria). ADH symptoms, including paraesthesia, ...
Autosomal Dominant Hypocalcemia Type 1 | What Is ADH1?
ADH1 is a common form of genetic hypoparathyroidism. Gain-of-function variants in the calcium-sensing receptor gene (CASR) are the root cause of ADH1.
Familial Hypocalciuric Hypercalcemia - StatPearls - NCBI - NIH
FHH is a genetic condition characterized by mild hypercalcemia, typically found in otherwise healthy and asymptomatic individuals.
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.