Active not recruitingPHASE3INTERVENTIONAL

Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1

This research study is investigating a new medication, encaleret, for individuals with a rare genetic condition called Autosomal Dominant Hypocalcemia Type 1 (ADH1). ADH1 affects how the body controls calcium levels. The main goal is to find out if encaleret is more effective and safer than the standard treatments currently used for ADH1. Researchers will carefully compare encaleret with the usual care to understand its benefits and any potential side effects. This is a Phase 3 study, which means it’s one of the final steps before a new medicine might become widely available, aiming to gather strong evidence about its performance.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Calcilytix Therapeutics, Inc., a BridgeBio company

Enrolment target

Start

06 Jan 2023

Estimated completion

01 Aug 2029

Autosomal Dominant Hypocalcemia (ADH)

What is this study about?

This research study is focused on a health condition called Autosomal Dominant Hypocalcemia Type 1, often shortened to ADH1. This is a rare genetic condition where the body has trouble managing calcium levels, leading to levels that are too low. These low calcium levels can cause various symptoms and health problems.

The study wants to see how a new medicine, encaleret, works compared to the treatments people usually receive for ADH1. The main idea is to find out if encaleret can help control calcium levels more effectively and safely than the current standard care. Researchers will be looking closely at how well encaleret works, and also tracking any side effects people might experience.

By comparing encaleret with standard care, the study hopes to gather important information. This information is key to understanding if encaleret could be a better treatment option in the future for people living with ADH1, potentially improving their quality of life. Participants will be carefully monitored throughout the study to ensure their safety and well-being.

Key takeaways

The study is testing a new medicine called encaleret for Autosomal Dominant Hypocalcemia Type 1 (ADH1).
It compares encaleret against the standard, usual treatments for ADH1.
The main goals are to check how well encaleret works and if it's safe.
Participants must be at least 16 years old and diagnosed with ADH1.
Certain medical conditions or other medications might prevent someone from joining.
Close medical supervision will be provided to all participants.

Who may be eligible?

To join this study, participants generally need to have been diagnosed with Autosomal Dominant Hypocalcemia Type 1 (ADH1) through genetic testing and show symptoms of the condition. They also need to be at least 16 years old. If you are aged 16 or 17, doctors will need to check that your bones are fully grown.

There are some medications that you would need to temporarily stop before or during parts of the study, such as certain water pills (diuretics), magnesium or potassium supplements, and some treatments for managing phosphate levels. Your study doctor would guide you through this process.

However, some people won't be able to join. This includes if you've had a seizure due to low calcium in the last three months, or if you've had surgery on your thyroid or parathyroid glands, or a kidney transplant. Pregnant or breastfeeding women cannot take part. There are also specific rules about recent treatments for parathyroid hormone and vitamin D levels that might prevent someone from joining.

Quick self-check

Do I have a confirmed diagnosis of Autosomal Dominant Hypocalcemia Type 1 (ADH1)?
Am I at least 16 years old?
Have I had a seizure due to low calcium in the last 3 months?
Am I pregnant or breastfeeding?
Have I had thyroid, parathyroid, or kidney transplant surgery?
Am I willing to adjust or stop certain other medications if needed for the study?

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

What does participation involve?

If you join this study, you will either receive the new medicine, encaleret, or continue with the standard care for ADH1. The study involves regular visits to the clinic, where doctors and nurses will check your health, take blood and urine samples, and ask about your symptoms. These checks help the researchers understand how well the treatment is working and if there are any side effects.

You would also need to follow specific instructions regarding any current medications, as some might need to be stopped or adjusted before or during the study, under the guidance of the study team. The total duration of your participation in the study is not specified in detail here, but these types of studies typically involve several months to over a year of follow-up to fully assess the treatment.

Potential risks and benefits

Taking part in a study like this could offer potential benefits, such as access to a new treatment (encaleret) that might be more effective than current options for ADH1. You would also receive close medical attention and monitoring from the study team. However, there are potential risks, including side effects from the study medicine, or the possibility that the new treatment may not work better than standard care. There's also the time commitment involved with clinic visits and following study procedures. It's important to remember that you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (25)

UCSF Benioff Children's Hospital, Oakland
Oakland, United States
Children's Hospital Colorado
Aurora, United States
Indiana University Health University Hospital
Indianapolis, United States
NIH
Bethesda, United States
Massachusetts General Hospital
Boston, United States
Mayo Clinic - Rochester
Rochester, United States
Columbia University Irving Medical Center
New York, United States
Physicians East
Greenville, United States
Ohio State University Medical Center
Columbus, United States
The Children's Hospital of Philadelphia
Philadelphia, United States
Houston Methodist Hospital
Houston, United States
Royal North Shore Hospital
Saint Leonards, Australia

+13 more sites — see the official record for the full list.

Common questions

What is ADH1?

ADH1 stands for Autosomal Dominant Hypocalcemia Type 1. It's a rare genetic condition that causes low calcium levels in the blood, leading to various health issues.

What is encaleret?

Encaleret is a new investigational medication being studied to see if it can help manage calcium levels in people with ADH1.

What does 'Standard of Care' mean?

Standard of Care refers to the usual treatments that doctors currently recommend and use for ADH1.

Will I know if I'm getting encaleret or the usual treatment?

In many studies like this, participants don't know which treatment they are receiving until after the study ends. This helps ensure fair results.

What if I experience side effects?

The study team will closely monitor you for any side effects. You should always report any new or worsening symptoms to them immediately.