RecruitingPHASE2, PHASE3INTERVENTIONAL

Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)

This research study is investigating a new medication, Encaleret, for children and teenagers from birth to 17 years old who have a genetic condition called Autosomal Dominant Hypocalcemia Type 1, or ADH1. ADH1 causes low calcium levels in the blood. The main goals of the study are to understand how Encaleret is processed by the body in young people, to check if it effectively improves their symptoms, and to make sure it is safe for them to use. This is a crucial step to potentially offer new treatment options for children living with ADH1. It's a late-stage study, meaning it has already shown some promise in earlier tests.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

Calcilytix Therapeutics, Inc., a BridgeBio company

Enrolment target

Start

30 Jan 2026

Estimated completion

01 Dec 2030

Autosomal Dominant Hypocalcemia Type 1 (ADH1)

What is this study about?

This study is focused on a genetic condition called Autosomal Dominant Hypocalcemia Type 1, or ADH1. People with ADH1 have a problem with how their bodies control calcium levels, often leading to low calcium in their blood. This can cause various health issues.

Researchers are testing a new medicine called Encaleret. They want to find out how this medicine works in children and teenagers (from newborns up to 17 years old) who have ADH1. They will be looking at several things: how the body handles the medicine (like how much is absorbed and how long it stays in the system), if it helps to improve the symptoms of ADH1, and if it's safe to use in this age group. This type of research is vital because it could lead to new and better treatments for children with this rare condition.

This study is in its later stages (Phase 2 and Phase 3), which means it has already shown some positive results in earlier tests. The goal now is to gather more detailed information to determine if Encaleret could become a widely available treatment. It's important to understand that participating in a study helps everyone learn more about medical conditions and potential new ways to treat them.

Key takeaways

This study is testing a new medicine called Encaleret for children aged 0-17 with ADH1.
The main goals are to check how the medicine works, if it helps symptoms, and if it's safe.
Participation involves regular clinic visits for medical checks and blood tests.
There's a chance your child could benefit from a new treatment, but also a risk of side effects.
You can withdraw your child from the study at any time without affecting their care.

Who may be eligible?

To join this study, children and teenagers need to be between birth and 17 years old. They must have a confirmed diagnosis of Autosomal Dominant Hypocalcemia Type 1 (ADH1), or have test results that strongly suggest it, along with symptoms related to low calcium levels.

Participants should also have been receiving their usual ADH1 treatment for at least three to six months before starting the study, depending on the specific group they might be in. If a child is legally able, they will be asked for their agreement to take part. For younger children, a parent or legal guardian will need to give their permission.

There are also some reasons why a child might not be able to join. For example, if they've had certain surgeries like on the thyroid or parathyroid glands, or if they've had specific types of cancer or kidney transplants. Also, if they've recently taken part in other clinical studies or are on certain medications that could interact with the study drug, they might not be eligible. The study team will review each child's medical history carefully to make sure it's safe for them to participate.

Quick self-check

Is my child between newborn and 17 years old?
Does my child have a confirmed diagnosis of ADH1 or strong evidence of it?
Does my child experience symptoms related to low calcium levels from ADH1?
Has my child been on their usual ADH1 treatment for at least 3-6 months?
Has my child NOT had a thyroid or parathyroid surgery?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your child takes part in this study, they will receive the study medication, Encaleret. They will have regular visits to the clinic so the study team can monitor their health, take blood samples to check calcium levels and how their body is handling the medicine, and assess any changes in their ADH1 symptoms. These visits will help doctors understand how well Encaleret is working and if it's safe.

The study involves different groups of participants, and the exact schedule of visits and tests will vary slightly depending on which group your child is in. There will be initial screening tests to ensure your child is suitable for the study, followed by treatment and regular check-ups. The total length of time your child would be involved in the study will be explained by the study team, but it's designed to gather enough information about the long-term effects of the medication.

Potential risks and benefits

Participating in a clinical study means your child could potentially benefit from a new treatment for ADH1 before it's widely available. However, there's no guarantee that Encaleret will work for your child, or that it will be better than their current treatment. There may also be side effects from the medication, some of which might not be known yet. The study team will closely monitor your child for any problems, and you will be fully informed of any known risks. You can withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (6)

Yale University
New Haven, United States· Recruiting
Children's National Medical Center
Washington D.C., United States· Recruiting
Nemours Children's Health
Jacksonville, United States· Recruiting
The Children's Hospital of Philadelphia
Philadelphia, United States· Recruiting
Royal London Hospital
London, United Kingdom· Recruiting
Royal Manchester Children's Hospital
Manchester, United Kingdom· Recruiting

Common questions

What is Autosomal Dominant Hypocalcemia Type 1 (ADH1)?

ADH1 is a rare genetic condition where the body has trouble controlling calcium levels, usually resulting in low calcium in the blood.

What is Encaleret?

Encaleret is a new investigational medicine being tested to see if it can help improve calcium levels and symptoms in people with ADH1.

Which age group is this study for?

This study is specifically for children and teenagers, from birth (newborns) up to 17 years old.

Will my child definitely get the new medicine?

Yes, if your child is eligible and joins the study, they will receive Encaleret as the study medication.

Can we stop participating at any time?

Yes, you have the right to withdraw your child from the study at any point, and it will not affect their future medical treatment.

How to find out more

Medical Information

MedInfo@bridgebio.com 650-600-3610 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.