Not recruitingRare disease

Study on Long-Term Safety and Effects of Vosoritide in Children with Achondroplasia

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the long-term effects of a treatment for Achondroplasia, a condition that affects bone growth and results in short stature. The treatment being tested is called BMN 111, also known as Voxzogo, which is given as a daily injection under the skin. The main goal of the study is to evaluate the safety and how well children with achondroplasia tolerate this treatment over an extended period.

Participants in the study will receive daily injections of BMN 111 and will be monitored for any side effects or changes in their health. The study will also look at how the treatment affects growth, including height and body proportions, to see if it helps improve these aspects in children with achondroplasia. The study is designed to gather information over a long period to ensure the treatment is safe and effective for long-term use.

Throughout the study, various health checks will be conducted, such as physical exams and imaging tests, to ensure the participants' well-being. The study aims to provide valuable insights into the potential benefits and risks of using BMN 111 for treating achondroplasia in children, contributing to better management of the condition in the future.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Participation begins after completing 24 months of previous treatment with BMN 111 in a prior study.
Written consent from a parent or guardian is required, and if under 18, written assent from the participant may also be needed.
Step 2
Baseline visit
A baseline visit includes a negative pregnancy test for females aged 10 or older or those who have started menstruating.
Participants must be willing to perform all study procedures and caregivers must be trained to administer daily injections.
Step 3
Daily medication administration
The medication used is Voxzogo, which contains the active substance vosoritide.
It is administered as a subcutaneous injection, meaning it is injected under the skin.
The medication is available in two dosages: 0.56 mg and 1.2 mg, and is given daily.
Step 4
Safety monitoring
Safety is monitored through the occurrence of any adverse events, serious adverse events, and results from laboratory tests such as urinalysis, blood chemistry, and hematology.
Regular checks of vital signs, physical examinations, echocardiograms, X-rays, and clinical hip assessments are conducted.
Step 5
Efficacy assessment
Efficacy is evaluated by measuring changes in height growth velocity, height, final adult height, and body proportions.
Measurements include height, weight, head circumference, and lengths of arms and legs, among others.
Step 6
Study duration
The study is expected to continue until December 31, 2024.
Participants are required to use effective contraception if sexually active during the study.

Who can join the trial?

6 criteria

Must have completed 24 months (plus or minus 14 days) of treatment with BMN 111 in a previous study.
Parent(s) or guardian(s) must be willing and able to provide written, signed consent after understanding the study details. If the participant is under 18, they must also provide written agreement if required.
If sexually active, must be willing to use a highly effective method of birth control while participating in the study.
Females aged 10 years or older, or those who have started their menstrual cycle, must have a negative pregnancy test at the start of the study and be willing to have more tests during the study.
Must be willing and able to perform all study procedures as physically possible.
Caregivers must be willing to give daily injections to the participants and complete the necessary training. This does not apply in France.

Who cannot join the trial?

7 criteria

Children who do not have Achondroplasia cannot participate. Achondroplasia is a condition that affects bone growth, leading to shorter stature.
Children who are not within the specified age range for the study cannot participate.
Children who have any other medical conditions that might interfere with the study cannot participate.
Children who are unable to receive daily injections cannot participate.
Children who have participated in other clinical trials recently may not be eligible.
Children who have allergies or reactions to the study medication cannot participate.
Children who are not able to follow the study procedures or attend regular visits cannot participate.

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Verified sites

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Hopital Necker Enfants Malades
Verified
Paris, France

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Investigated drugs

BMN 111 is a medication being studied for its long-term safety and effectiveness in children with achondroplasia, a condition that affects bone growth. The trial involves giving daily injections of BMN 111 to see how well children tolerate it over time and to monitor any potential side effects. The goal is to determine if BMN 111 can be a safe and effective treatment option for managing achondroplasia in children.

Vosoritide

What is already known about the treatment

BMN 111 – This medication is administered through daily subcutaneous injections. It is currently being studied in clinical trials, specifically for its long-term safety and effectiveness in children with achondroplasia, a condition characterized by abnormal bone growth. BMN 111 works by targeting specific pathways involved in bone development, aiming to promote normal growth patterns. It is classified as a therapeutic agent for skeletal dysplasias, focusing on improving bone growth and development.

Investigated diseases

Achondroplasia – Achondroplasia is a genetic disorder that affects bone growth, leading to dwarfism. It is characterized by an average-sized trunk, short arms and legs, and a slightly larger head with a prominent forehead. The condition is caused by a mutation in the FGFR3 gene, which affects the conversion of cartilage to bone, particularly in the long bones. As children with achondroplasia grow, they may experience delayed motor development and may have difficulty with certain physical activities due to their shorter stature. The condition is present from birth and remains throughout life, with growth patterns differing from those of average height individuals. Despite these differences, individuals with achondroplasia typically have normal intelligence and can lead full, productive lives.