An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia (RESTORE)
This study, called RESTORE, is for people with a rare bone condition called Hypophosphatasia. The main medicine for this condition, Asfotase Alfa (STRENSIQ®), sometimes faces a challenge: the body can develop an immune response that makes the medicine less effective over time. This trial is investigating different additional treatments given alongside Asfotase Alfa. The aim is to see if these extra treatments can stop the body from fighting the medicine, helping Asfotase Alfa to work better and improve bone health. Researchers will check people over 100 weeks to see if the combined treatments successfully reduce the immune response and show improvements in their bones, which will be checked with X-rays.
At a glance
What is this study about?
This study is called RESTORE, and it's looking into ways to improve treatment for a condition called Hypophosphatasia. Hypophosphatasia is a rare disease that affects the bones and teeth, making them weak or soft. The main medicine for this condition is called Asfotase Alfa (you might know it by its brand name, STRENSIQ®), and it helps to strengthen bones.
Sometimes, when people take medicines like Asfotase Alfa, their body's immune system (which normally fights off infections) can start to see the medicine as something foreign and try to get rid of it. This can make the medicine less effective over time. This study wants to find out if giving other medicines alongside Asfotase Alfa can stop this immune reaction from happening or make it less severe. The goal is to help Asfotase Alfa work as well as possible for patients.
The researchers will be carefully checking how participants' bodies react to Asfotase Alfa over about two years. They will also look at X-rays to see if the bones are getting stronger. This study is important because it could lead to better ways to manage Hypophosphatasia and help people get the most benefit from their treatment.
Key takeaways
- This study is for people with Hypophosphatasia.
- It aims to improve how well the medicine Asfotase Alfa works.
- Additional treatments are given to reduce the body's immune response to the main medicine.
- The study lasts for 100 weeks (about two years) with regular check-ups.
- It's open to all ages and sexes with Hypophosphatasia.
- Participation includes blood tests, X-rays, and other health checks.
Who may be eligible?
This study is open to anyone with Hypophosphatasia, regardless of their age or sex. The researchers welcome both men and women, and patients of all ages, to take part.
However, it's important to remember that even if you have Hypophosphatasia, there might be other medical reasons why this particular study isn't suitable for you. For example, your other health conditions or current medications could affect whether you can safely participate.
The best way to find out if this study is right for you is to talk to your doctor. They can review all the detailed requirements and discuss if you meet the specific criteria to join.
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.
- Do I have Hypophosphatasia?
- Am I currently being treated with Asfotase Alfa (STRENSIQ®)?
- Am I comfortable with receiving additional medicines as part of treatment?
- Can I attend regular appointments, including for blood tests and X-rays, over two years?
- Have I discussed this study thoroughly with my doctor?
What does participation involve?
If you decide to join this study, you will continue to receive your usual Asfotase Alfa treatment. In addition, you will be given one or more extra medicines aimed at helping Asfotase Alfa work better. These extra medicines might be given as injections, infusions (drips), or tablets.
Throughout the study, which will last for 100 weeks (about two years), you'll have regular appointments. At these appointments, the study team will take blood and urine samples, carry out physical examinations, measure your vital signs (like blood pressure and heart rate), and perform ECGs (heart tracings). You'll also have X-rays to check on your bone health. If you are given Bortezomib, you may also have special heart scans (echocardiograms) and nerve checks.
Potential risks and benefits
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Common questions
What is Hypophosphatasia?
Hypophosphatasia is a rare genetic condition that affects the development of bones and teeth, making them weaker or softer than they should be.
What is Asfotase Alfa (STRENSIQ®)?
It's a medicine used to treat Hypophosphatasia by helping to correct the underlying problem and improve bone strength.
Why would my body fight the medicine?
Sometimes, the body's immune system, which protects against germs, can mistakenly see some medicines as foreign and try to remove them, making the medicine less effective.
How long will the study last?
The main part of the study will last for 100 weeks, which is about two years.
Will I get my usual medicine during the study?
Yes, you will continue to receive Asfotase Alfa, and the study will add other treatments to see if they help it work better.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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