RecruitingOBSERVATIONAL

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

This study is a long-term follow-up for children who previously participated in a clinical trial for Ornithine Transcarbamylase Deficiency (OTCD), a rare genetic liver disorder. Its main purpose is to see how safe the investigational treatment was and how well it worked over time for those who received it. The study will also gather information on the natural progression of OTCD in children who were part of the previous trial but did not receive the specific study treatment. No new medication is given in this follow-up study; participants will continue with their usual medical care as prescribed by their doctors. This research helps us understand the disease and the effects of potential new treatments over many years.

At a glance

Status

Recruiting

Sponsor

iECURE, Inc.

Enrolment target

Start

23 Dec 2024

Estimated completion

01 Jul 2041

Ornithine Transcarbamylase Deficiency Ornithine Transcarbamylase Deficiency Disease Urea Cycle Disorders, Inborn

What is this study about?

This study is a special check-up for children who have a rare genetic liver condition called Ornithine Transcarbamylase Deficiency (OTCD). This condition means the body can't properly process certain waste products, which can lead to serious health problems. These children previously took part in an earlier study where some might have received a new, unapproved treatment (an investigational product) and others did not.

The main goal of this current study is to carefully monitor the health of these children over a long period. For those who received the investigational treatment, doctors want to see if it was safe in the long run and if its benefits lasted. For those who were in the earlier study but didn't receive the new treatment, this study will help us understand how OTCD naturally progresses and affects children's lives over many years. Gathering this 'natural history' information is very important for comparing new treatments in the future.

It's important to know that in this follow-up study, no one will be given any new medications or treatments. All participants will continue to receive the standard medical care and prescriptions from their own doctors, just as they normally would. This study is purely about observing and collecting information over time to learn more about OTCD and the effects of previous study treatments.

Key takeaways

This is a long-term follow-up study for children with OTCD.
It checks on health and progress for up to 14.5 years.
No new study medication will be given; usual care continues.
Participation requires being part of a previous iECURE study.
Helps understand long-term effects of previous treatments and the disease's natural course.
Your child's ongoing medical care will not be affected.

Who may be eligible?

This study is for very specific children. To be considered, a child must have already taken part in a previous clinical trial sponsored by iECURE for their Ornithine Transcarbamylase Deficiency (OTCD). They might have either finished that previous study or stopped it early. It's not for children who haven't been in an iECURE study before.

Parents or legal guardians of the child must be willing and able to follow all the study's instructions and attend any necessary appointments. When the child first joined the earlier study, their parents or guardians would have given permission (consent), and the child, if old enough, would have also agreed (assent) for their information to be used.

A child cannot join this follow-up study if they decide to participate in another clinical trial that involves a different unapproved drug or gene therapy. This is to make sure that the information collected in this study isn't influenced by other new treatments.

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.

Has your child already taken part in an iECURE clinical trial for OTCD?
Were you or your guardian willing to follow the rules of that previous study?
Did you (as parent/guardian) give permission for your child to be in the earlier study (and if old enough, did your child agree)?
Is your child currently NOT taking part in another new drug or gene therapy trial (outside of this follow-up)?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, it will involve being monitored for a long time, specifically up to 14 and a half years. This means regular check-ups and assessments by doctors. The study aims to gather information about your child's health over time, including how their body is working and how their OTCD condition is progressing. You will not be given any new study medication; your child will continue to receive all their usual and prescribed medical care from their own doctors, as they do now. The types of assessments might include blood tests, physical examinations, and possibly other tests that doctors usually use to monitor OTCD. The exact schedule of visits and tests will be explained in detail by the study team. You always have the right to withdraw your child from the study at any time.

Potential risks and benefits

The main benefit of participating is contributing valuable long-term information that helps doctors and researchers better understand Ornithine Transcarbamylase Deficiency (OTCD) and the lasting effects of potential new treatments, which can ultimately improve care for others in the future. Since no new medications are given, the risks involved are generally those associated with routine medical monitoring for OTCD, such as blood tests which might cause minor discomfort or bruising. There are no direct personal benefits for your child in terms of receiving a new treatment, as this is purely an observational study. You are free to withdraw your child from the study at any time, for any reason, without affecting their standard medical care.

Locations (1)

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

Great Ormond Street Hospital
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is Ornithine Transcarbamylase Deficiency (OTCD)?

OTCD is a rare genetic condition where the body can't properly get rid of harmful waste products, which can build up and cause serious health issues, especially affecting the brain.

Will my child receive any new medication in this study?

No, your child will not receive any new study medication in this follow-up study. They will continue with their usual care as prescribed by their doctors.

How long will this study last?

This study is a long-term follow-up that could last for up to 14 and a half years.

Why is this study important if no new treatment is given?

It's important because it helps doctors understand the long-term safety and effects of treatments given in previous studies, and how OTCD naturally progresses over many years, which aids future research.

Will participating affect my child's regular medical care?

No, participating in this study will not affect your child's regular medical care. They will continue to be looked after by their own doctors as normal.

How to find out more

George Diaz, M.D., Ph.D.

medinfo@iecure.com 1-877-694-3558 Official trial record

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