Enrolling by invitationOBSERVATIONAL

Zoledronate In the Prevention of Paget's Disease: Long Term Extension

This research is an extension of a previous study called ZiPP, looking at people who have a higher chance of developing Paget's disease because of a specific gene. Paget's disease can affect bones, sometimes causing pain or other problems, but often has no symptoms. The original ZiPP study investigated whether an early treatment called zoledronic acid (ZA) could prevent or delay the signs of Paget's on bone scans compared to a dummy treatment. This extension study will follow these participants for another five years. The aim is to see if they develop any symptoms of Paget's disease and to check if the bone changes have progressed at the end of this period. This will help doctors understand the long-term effects of early treatment.

At a glance

Status

Enrolling by invitation

Sponsor

University of Edinburgh

Enrolment target

287

Start

05 Apr 2019

Estimated completion

01 May 2027

Paget Disease

What is this study about?

This study is an important continuation of earlier research focused on Paget's disease, a common bone condition. It primarily affects older people and causes bones to grow abnormally. While many people with Paget's disease don't experience any issues, for others, it can lead to problems like bone pain, unusual bone shapes, arthritis, or even hearing difficulties. We know that genetics play a big role in who gets Paget's disease, and there's a particular gene called SQSTM1 that significantly increases a person's risk.

The original study, called ZiPP, looked at people who had this specific gene. Its main question was whether giving an early treatment called zoledronic acid (ZA) could stop or delay the first signs of Paget's disease from appearing on bone scans, compared to giving a dummy treatment (a placebo). While the ZiPP study is expected to tell us if ZA can help prevent these bone changes, we don't yet know if this will actually make a difference to a person's symptoms or quality of life.

That's where this new extension study comes in. We are inviting people who took part in the original ZiPP study to continue being monitored for another five years. During this time, the research team will keep a close eye on whether any participants develop symptoms or signs of Paget's disease. At the end of the five years, a bone scan will be done to see if there have been any changes or worsening of the Paget's-like bone changes. This will help doctors understand much better if early treatment truly benefits patients in the long run.

Key takeaways

This study is for people who were in the original ZiPP study on Paget's disease.
It aims to understand if early treatment helps prevent symptoms or slow down the disease over five years.
Participants will be monitored for signs and symptoms of Paget's disease.
Treatment will be offered if Paget's symptoms develop during the study.
The study helps doctors understand the long-term benefits of early intervention.

Who may be eligible?

To be part of this extended study, you must have already taken part in the original ZiPP study. This is because the extension builds directly on the information gathered from that initial research.

You also need to be willing and able to understand what the study involves and agree to take part. This includes being able to follow the study's instructions and attend any necessary appointments.

If you are unable or unwilling to give your full agreement to participate, you won't be able to join this study.

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.

Did I participate in the previous ZiPP study?
Am I able to understand what the study involves?
Am I willing to agree to the study rules and attend appointments?
Do I want to contribute to research on Paget's disease?

Answer every question to see your result.

What does participation involve?

If you decide to join this extension study, you will be monitored over a five-year period. This is an 'observational' study, meaning that while you are being carefully watched, there isn't a specific study medicine or treatment being given to everyone. However, if you do develop symptoms or signs of Paget's disease during these five years, you will be offered treatment, just as would happen in standard medical care.

For those who received zoledronic acid (ZA) in the previous ZiPP study and later show signs of increased activity of Paget's (even without symptoms), you might be offered ZA again or another similar approved medication. This is because those who have had ZA before tend to tolerate it better. The study team will be looking at how people feel, how their quality of life is, whether new bone changes occur, and if there are any side effects. You'll need to attend regular check-ups and may have bone scans at the end of the five years. The total time you'll be involved is five years.

Potential risks and benefits

This study aims to provide valuable information about the long-term effects of early treatment for Paget's disease and how the condition develops over time. By taking part, you'll be contributing to medical knowledge that could help future patients. You will also receive regular monitoring for Paget's disease over five years, and if symptoms develop, you will be offered treatment. Potential risks are generally low, as it's mainly an observational study with standard medical care offered if needed. For those who might receive further medication, there could be side effects, which your doctor will discuss with you. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time without affecting your usual medical care.

Locations (21)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University Hospital Geelong
Verified postcode
Geelong, Australia
Sir Charles Gardner Hospital
Verified postcode
Nedlands, Australia
Royal Newcastle Centre
Verified postcode
Newcastle, Australia
University of Sydney
Verified postcode
Sydney, Australia
University of Queensland
Verified postcode
Toowoomba, Australia
University Hospital Saint-Luc
Verified postcode
Brussels, Belgium
St. Vincent's University Hospital
Verified postcode
Dublin, Ireland
University Hospital of Careggi
Verified postcode
Florence, Italy
University of Siena
Verified postcode
Siena, Italy
University of Turin
Verified postcode
Turin, Italy
University of Auckland
Verified postcode
Auckland, New Zealand
The Princess Margaret Hospital
Verified postcode
Christchurch, New Zealand

Common questions

What is Paget's disease?

Paget's disease is a condition where your bones grow unusually, which can sometimes lead to pain or other problems, but often has no symptoms.

What was the original ZiPP study about?

The original ZiPP study looked at whether early treatment with a medicine called zoledronic acid could prevent the first signs of Paget's disease in people at high genetic risk.

Why is this a 'long-term extension' study?

It's an extension because it continues to follow people from the original study for an additional five years to see the long-term impact of early detection or treatment on their health.

Will I receive treatment in this study?

This is mainly an observational study, but if you develop symptoms or signs of Paget's disease during the five years, you will be offered appropriate treatment based on standard medical care.

How long will I be involved?

You will be involved in this extension study for a period of five years.

How to find out more

Official trial record

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