A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
This study is testing a new combination treatment, cipaglucosidase alfa and miglustat, for children from birth up to 17 years old who have a serious genetic condition called Infantile Onset Pompe Disease (IOPD). This is a 'Phase 3' study, which means it's a critical step in checking if the treatment is safe and effective before it can be widely used. Researchers will observe how well the treatment works, how the children's bodies react to it, and if there are any side effects. The study includes children who have previously received treatment for IOPD and those who are new to treatment. The main goal is to find out if this new treatment can make a positive difference for children with IOPD.
At a glance
What is this study about?
This study is investigating a new treatment for a serious genetic condition called Infantile Onset Pompe Disease (IOPD). IOPD is a very rare and severe illness that affects how the body uses sugar for energy. It can lead to problems with muscles, especially the heart. The new treatment being studied is a combination of two medicines, cipaglucosidase alfa and miglustat. Think of cipaglucosidase alfa as a replacement enzyme that the body with IOPD is missing, and miglustat helps this enzyme work better.
This is a 'Phase 3' study, which is an important stage of research. It means that previous studies have shown some promise, and now researchers need to gather more information in a larger group of patients. The main goals are to find out if this new treatment is safe for children, how well it works to improve their health, and how their bodies handle the medication. They will also look at how the body's immune system reacts to the treatment.
This research is really important because it could offer a new and potentially better way to manage IOPD for children. By studying how the treatment affects different children, both those new to treatment and those who have tried other therapies, the researchers hope to understand its full potential and bring new hope to families affected by this condition.
Key takeaways
- This study is testing a new combined treatment for Infantile Onset Pompe Disease (IOPD) in children.
- It aims to check the safety and how well the treatment works.
- The study includes babies and children up to 17 years old, both those new to treatment and those currently on other treatments.
- Regular hospital visits and assessments will be part of the study participation.
- The main treatment period is for about two years, with potential for longer follow-up.
- The research could lead to a new treatment option for children with IOPD.
Who may be eligible?
The study is looking for children with Infantile Onset Pompe Disease (IOPD). There are two main groups being looked at. The first group includes children aged from 6 months up to 17 years who have already been receiving a different enzyme replacement therapy (ERT) for at least six months.
To be in this first group, children must have been diagnosed with IOPD, have had heart enlargement (hypertrophic cardiomyopathy) at diagnosis, and have shown signs that their current treatment isn't working as well as it used to. They also need to be able to complete a walking test over a certain distance, depending on their age. The second group is for very young babies, from birth up to 6 months old, who have IOPD, had heart enlargement at diagnosis, and have not yet received any enzyme replacement therapy.
There are also some things that would prevent a child from joining the study. For example, if a child needs a breathing tube (like a tracheostomy) or has previously had a very serious allergic reaction to similar medicines, they would not be able to take part. Also, if a child specific immune system reaction which means they might not respond well to the treatment without a special additional treatment.
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.
- Is your child diagnosed with Infantile Onset Pompe Disease (IOPD)?
- Did your child have an enlarged heart (hypertrophic cardiomyopathy) when first diagnosed?
- If your child is 6 months to 17 years old, have they been on another Pompe treatment for at least 6 months?
- If your child is under 6 months old, have they NOT had any Pompe treatment yet?
- Does your child NOT need permanent breathing support (like a tracheostomy)?
- Has your child NOT had a very serious allergic reaction to similar medicines before?
What does participation involve?
Taking part in this study involves regular visits to the hospital or clinic over a long period. During these visits, doctors and nurses will carry out various checks and tests. These will include blood tests, physical examinations, and assessments to see how well the child is moving and breathing. For older children, walking tests will be a regular part of the assessment. The new treatment, cipaglucosidase alfa/miglustat, will be given regularly, and the study team will explain exactly how and when this will happen.
Initially, the main treatment period will last for about two years (104 weeks). After this, if the doctors think the child is benefiting from the treatment and there are no safety concerns, they might be invited to continue receiving the treatment in a follow-up phase. The total duration of a child's involvement in the study could therefore be quite long, potentially several years.
Potential risks and benefits
Locations (14)
- University of Florida Clinical Research CenterVerified postcodeGainesville, United States· Recruiting
- The Emory ClinicVerified postcodeAtlanta, United States· Recruiting
- Duke University Early Phase Research UnitVerified postcodeDurham, United States· Recruiting
- Cincinnati Children's Hospital Medical CenterVerified postcodeCincinnati, United States· Recruiting
- UPMC Hospital of PittsburghVerified postcodePittsburgh, United States· Recruiting
- University of Utah, Clinical and Translational Sciences InstituteVerified postcodeSalt Lake City, United States· Recruiting
- Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und EpileptologieVerified postcodeGiessen, Germany· Recruiting
- Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)Verified postcodeHeidelberg, Germany· Recruiting
- SphinCS GmbHVerified postcodeHöchheim, Germany· Recruiting
- Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1Verified postcodeMünster, Germany· Recruiting
- AOU Federico IIVerified postcodeNaples, Italy· Recruiting
- Erasmus MC, Sophia KinderziekenhuisVerified postcodeRotterdam, Netherlands· Recruiting
Common questions
What is Infantile Onset Pompe Disease (IOPD)?
IOPD is a rare genetic condition where the body can't properly break down a type of sugar, leading to muscle weakness, especially in the heart.
What does 'Phase 3 study' mean?
It means this is a major study to see if a new treatment is safe and effective in a larger group of patients, after earlier studies have shown some promise.
Will my child definitely get the new treatment?
Yes, this is an 'open-label' study, which means all participants will receive the active treatment, cipaglucosidase alfa/miglustat.
What kind of tests will my child have?
Tests may include blood tests, physical exams, and walking tests, depending on your child's age and condition.
How long will my child be in the study?
The main treatment period is about two years, but some children may be able to continue in a longer follow-up period if they are benefiting.
How to find out more
For Site
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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