A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
This research study is investigating a new treatment called OTL-203 for a rare genetic condition in children known as Hurler Syndrome (MPS-IH). Hurler Syndrome can cause serious health problems. The study aims to find out if OTL-203, which is a type of gene therapy, is as good as or better than the treatment currently used, which is a stem cell transplant. Researchers will compare how safe and effective both treatments are in young children with Hurler Syndrome. The study will involve a number of medical checks and follow participants for several years to understand the long-term effects of the treatments.
At a glance
What is this study about?
This study is about a condition called Hurler Syndrome, also known as MPS-IH. It's a rare genetic disorder that affects how the body breaks down certain sugars, leading to various health issues in children. Currently, a stem cell transplant is the main treatment, but doctors are always looking for better options.
This research is testing a new treatment called OTL-203. It's a type of 'gene therapy,' which means it aims to correct the underlying genetic problem causing Hurler Syndrome. The study wants to compare this new gene therapy directly with the standard stem cell transplant. This will help doctors understand if OTL-203 is a safe and effective alternative, or even an improvement, for children with this condition.
About 40 children with Hurler Syndrome will be part of this study. They will be randomly assigned to receive either the new gene therapy (OTL-203) or the standard stem cell transplant. Researchers will carefully monitor their health and development for up to 5 years after treatment to see how well each option works.
Key takeaways
- Tests a new gene therapy (OTL-203) for Hurler Syndrome.
- Compares the new gene therapy to standard stem cell transplant.
- Specifically for young children (28 days to 30 months old).
- Participation involves random assignment to one of two treatments.
- Long-term follow-up for 5 years after treatment.
- Aims to find safer and more effective treatment options.
Who may be eligible?
To be part of this study, children need to be diagnosed with Hurler Syndrome, which will be confirmed by special tests and a review committee. They also need to be very young, between 28 days and 30 months old, and have certain results on developmental tests that show their thinking and learning skills are within a specific range.
There are also some reasons why a child might not be able to join. For example, if they have already had a stem cell transplant or gene therapy, or if they are currently taking part in another experimental study. Children with HIV, Hepatitis, certain cancers, uncontrolled seizures, or active infections that aren't getting better with treatment would also not be able to participate. The doctors will make sure that taking part in the study is safe for your child and that they can follow all the necessary steps.
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.
- Does my child have a confirmed diagnosis of Hurler Syndrome?
- Is my child between 28 days and 30 months old?
- Has my child NOT had a stem cell transplant or gene therapy before?
- Does my child meet certain developmental scores for their age?
- Does my child have any serious active infections or other specified health problems?
What does participation involve?
If your child takes part, they will be randomly assigned to receive either the new gene therapy (OTL-203) or the standard stem cell transplant. This means neither you nor your doctor will get to choose which treatment your child receives. Before treatment, your child will have initial checks, called a 'screening' period, and then a 'baseline' assessment. Following treatment, there will be regular check-ups and assessments for 5 years. The main part of the analysis will look at results after 2 years. These visits will involve various medical tests and examinations to monitor your child's health and how they are responding to the treatment. The total duration of active follow-up for your child will be 5 years.
Potential risks and benefits
Locations (5)
- University of Minnesota, PediatricsVerified postcodeMinneapolis, United States
- Ospedale San RaffaeleVerified postcodeMilan, Italy
- Princess Maxima CenterVerified postcodeUtrecht, Netherlands
- UMC UtrechtVerified postcodeUtrecht, Netherlands
- Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's HospitalVerified postcodeManchester, United Kingdom
Common questions
What is Hurler Syndrome?
Hurler Syndrome is a rare genetic condition where the body can't properly break down certain complex sugars, leading to various health problems affecting many parts of the body.
What is gene therapy?
Gene therapy is a new type of treatment that aims to correct the genetic problem causing a disease by introducing healthy genes into the body.
Why is this study important?
This study is important because it compares a new gene therapy with the current standard treatment, hoping to find a safer or more effective option for children with Hurler Syndrome.
Will my child get to choose which treatment they receive?
No, children in this study will be randomly assigned to either the new gene therapy or the standard stem cell transplant, like flipping a coin. Neither you nor the doctors can choose.
How long will my child be followed after treatment?
Your child will have regular check-ups and medical monitoring for up to 5 years after receiving their treatment.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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