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Active not recruitingOBSERVATIONAL

ADH1 and ADH2 Disease Monitoring Study (DMS)

This study is called the ADH1 and ADH2 Disease Monitoring Study. It's for people with a rare condition called Autosomal Dominant Hypocalcemia (ADH), either type 1 or type 2. Researchers want to better understand what it's like to live with ADH, how it changes over time, and how it's currently treated by doctors in everyday practice. They will collect information about people's past health and continue to gather data into the future for up to five years. This will help them learn more about the condition's symptoms and how standard care affects them. Adults and children with a confirmed diagnosis of ADH are welcome to take part.

At a glance

Status
Active not recruiting
Sponsor
Calcilytix Therapeutics, Inc., a BridgeBio company
Enrolment target
95
Start
20 Jan 2022
Estimated completion
01 Dec 2028

What is this study about?

This study is designed to help us learn more about a rare genetic condition called Autosomal Dominant Hypocalcemia (ADH), specifically types 1 and 2. For people with ADH, their bodies have trouble managing calcium levels, which can lead to various symptoms.

Researchers want to get a clearer picture of what it's like to live with ADH. They will be looking at how the condition shows up in people, how it changes and progresses over time, and how doctors currently manage it in their clinics. This includes understanding how the treatments people are already receiving affect their symptoms and their overall health day-to-day.

The information gathered from this study will be really valuable in helping the medical community better understand ADH. This improved understanding could potentially lead to better ways to support and treat people with this condition in the future.

Key takeaways

  • This study helps doctors learn more about rare calcium conditions.
  • It's for people with Autosomal Dominant Hypocalcemia (ADH1 or ADH2).
  • No new medicines are given; it observes your existing care.
  • Information will be collected for up to five years.
  • Both children and adults can take part.
  • Your participation helps to improve care for others with ADH.

Who may be eligible?

To join this study, you need to have a confirmed diagnosis of Autosomal Dominant Hypocalcemia (ADH), either type 1 or type 2. This diagnosis should be based on a genetic test showing a change (variant) in a specific gene called CASR (for ADH1) or GNA11 (for ADH2). If you don't have this genetic test done already, the study team can arrange it for you.

Both adults and children can take part. It's important that you or your parent/guardian are willing to understand and agree to participate the study. You also need to be able to provide access to your past medical records, like blood test results or scans, if they are available.

Finally, you should be able to attend study visits as scheduled and follow the study's instructions. You would not be able to join if you have other serious health problems that the study doctor thinks would make it unsafe for you to participate or make it hard to follow the study plan. Also, if you are currently taking part in another study that involves new treatments, you wouldn't be able to join this one.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Do I have a confirmed diagnosis of ADH Type 1 or Type 2?
  2. Am I willing to share my past health records?
  3. Can I attend study appointments if needed?
  4. Am I currently not taking part in an experimental treatment study?
Answer every question to see your result.

What does participation involve?

If you join this study, the main thing you'll be doing is sharing your health information. The study will look at your past medical records, and then continue to collect information about your health over a period of up to five years. This includes details about your diagnosis, symptoms, and the treatments you're already receiving as part of your usual care. There are no new medications or experimental treatments involved in this study. The researchers are simply observing and collecting data about your condition and how it's managed in everyday life. Your doctor will continue to manage your care as normal.

Potential risks and benefits

The main benefit of taking part in this study is contributing to a better understanding of Autosomal Dominant Hypocalcemia. This could help improve care for future patients. As this is an observational study, meaning you won't be given any new medications or treatments, the physical risks are expected to be very low. The main 'risk' might be the time commitment for providing information and attending any scheduled study visits. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (27)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland
    Verified postcode
    Oakland, United States
  • Children's Hospital Colorado
    Verified postcode
    Aurora, United States
  • Nemours Children's Clinic
    Verified postcode
    Jacksonville, United States
  • Indiana University (IU) School of Medicine - University Hospital
    Verified postcode
    Indianapolis, United States
  • Boston Children's Hospital
    Verified postcode
    Boston, United States
  • Mayo Clinic
    Verified postcode
    Rochester, United States
  • Physician's East Endocrinology
    Verified postcode
    Greenville, United States
  • Cincinnati Children's Hospital Medical Center
    Verified postcode
    Cincinnati, United States
  • Ohio State University Medical Center (OSUMC)
    Verified postcode
    Columbus, United States
  • Royal North Shore Hospital
    Unverified
    Saint Leonards, Australia
  • Universitaire Ziekenhuizen Leuven
    Verified postcode
    Leuven, Belgium
  • Bone Research and Education Centre
    Verified postcode
    Oakville, Canada

Common questions

What is Autosomal Dominant Hypocalcemia (ADH)?

ADH is a rare genetic condition where your body has trouble controlling its calcium levels, often leading to lower-than-normal calcium in your blood.

Do I have to take new medicine in this study?

No, this study doesn't involve any new medicines or experimental treatments. The researchers are just observing and collecting information about your condition and current care.

How long will I be in the study?

If you join, the study will collect information about your health for up to five years.

What kind of information will the study collect?

The study will collect details about your diagnosis, symptoms, and how your condition is currently being managed by your doctors.

Can children participate in this study?

Yes, both adults and children with a confirmed diagnosis of ADH can take part in this study.

How to find out more

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

Discussion

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