Active not recruitingPHASE2INTERVENTIONAL

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

This research study, called a Phase 2 trial, is testing a medicine named BMN 111 for children who have achondroplasia. Achondroplasia is a lifelong condition that causes shorter arms and legs. This particular study is an 'extension study,' meaning it follows on from an earlier trial. Researchers want to find out if BMN 111 is safe to use for a long time and if it continues to help children after they've already received it for two years in a previous study. Children in this study will get daily injections of BMN 111. The main goal is to check how well children tolerate the medicine and if there are any side effects over a longer period. It's open to children who have already been treated with BMN 111 in an earlier study.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

BioMarin Pharmaceutical

Enrolment target

Start

26 Jan 2016

Estimated completion

01 Feb 2028

Achondroplasia

What is this study about?

This study is looking at a medicine called BMN 111 for children who have achondroplasia. Achondroplasia is a genetic condition that affects how bones grow, usually leading to short stature and shorter arms and legs. While we've learned a lot about this condition, researchers are always working to find new ways to support children and improve their quality of life. This particular study builds on earlier research into BMN 111.

BMN 111 is designed to help address some of the growth differences seen in achondroplasia. This study is called a "Phase 2 Open-Label Extension" study. "Phase 2" means researchers are still carefully studying the medicine to understand its effects, safety, and correct dose. "Open-Label" means that everyone involved, including the children, their families, and the study team, will know that the child is receiving BMN 111.

The main purpose of this study is to check on the long-term safety and how well children tolerate BMN 111. It's specifically for children who have already taken BMN 111 for two years in a previous study. By continuing to follow these children, researchers hope to gather more information about possible side effects over time and to see if the medicine continues to be helpful. This information is really important for understanding if BMN 111 could be a good long-term treatment option.

Key takeaways

This study is for children with achondroplasia who have already used BMN 111 for two years.
It aims to see if BMN 111 is safe and works well over a longer period.
Children will receive daily injections of BMN 111 at home.
Regular clinic visits will be needed to monitor health and progress.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, children must have already completed two years of treatment with BMN 111 in a previous study (called 111-202). Their parents or guardians must be willing to give permission for them to take part. If a child is old enough to understand, they will also be asked if they want to participate. If they reach adulthood during the study, they will then give their own permission.

There are also some other requirements. For example, if a participant is sexually active, they must agree to use effective contraception during the study. Girls aged 10 and over, or who have started their periods, will need to have pregnancy tests. Parents or caregivers must be able to give daily injections and be willing to learn how to do this.

Children cannot join if they are already taking other experimental medicines, if the study doctor thinks they might not be able to follow the study rules, or if they have other medical conditions that could make taking part unsafe. Also, if they stopped taking BMN 111 during the previous study, or if they are pregnant or planning to become pregnant, they won't be able 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.

Has your child already completed two years of BMN 111 treatment in the 111-202 study?
Are you, as a parent or guardian, willing to give permission for your child to join?
Are you able to give daily injections at home after training?
Does your child not have any other serious medical conditions that the study doctor thinks would interfere with participation or safety?
Is your child not currently pregnant or planning to become pregnant if they are of childbearing age?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will receive daily injections of BMN 111. These injections will be given at home by a parent or caregiver after they've received training from the study team. You'll have regular visits to the study clinic where your child will have check-ups, measurements taken, and potentially blood tests to monitor their health and the medicine's effects. The study team will explain all the specific appointments and what each one involves.

Throughout the study, you'll be asked to keep records and communicate regularly with the study team about your child's health and any effects of the medicine. The total duration of participation in this long-term extension study will be explained by the study team, as it continues from an earlier trial. You are free to stop your child's participation at any time, for any reason.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. For your child, a potential benefit of this study is continued access to BMN 111, which may help with aspects of their growth and development related to achondroplasia. This study also helps researchers learn more, which could benefit others with achondroplasia in the future. As with any medicine, there are potential risks, including side effects from BMN 111 or discomfort from injections or study procedures like blood tests. The study team will explain all known or potential risks in detail before you decide to participate. Remember, your child's participation is completely voluntary, and you have the right to withdraw them from the study at any time without giving a reason, and it will not affect their regular medical care.

Locations (9)

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

Children's Hospital & Research Center Oakland
Verified postcode
Oakland, United States
Harbor - UCLA Medical Center
Verified postcode
Torrance, United States
Ann and Robert H. Lurie Childrens Hospital of Chicago
Verified postcode
Chicago, United States
Johns Hopkins McKusick - Institute of Genetic Medicine
Verified postcode
Baltimore, United States
Vanderbilt University
Verified postcode
Nashville, United States
Baylor College of Medicine
Verified postcode
Houston, United States
Murdoch Children's Research Institute
Verified postcode
Parkville, Australia
Institut Necker
Verified postcode
Paris, France
Guys & St. Thomas NHS Foundation Trust Evelina Hospital
Verified postcode
London, United Kingdom

Common questions

What is achondroplasia?

Achondroplasia is a condition that affects how bones grow, leading to shorter arms and legs and shorter height.

What is BMN 111?

BMN 111 is a medicine being studied to help with bone growth in children with achondroplasia.

How is BMN 111 given?

BMN 111 is given as a daily injection, usually administered by a parent or caregiver at home.

Can my child join if they haven't taken BMN 111 before?

No, this study is only for children who have already completed two years of BMN 111 treatment in a previous study.

What does 'long-term safety' mean?

It means the study will check for any side effects or health changes that might happen over a longer period of using the medicine.