Not recruitingRare disease

Study on the Long-Term Safety and Effectiveness of Vosoritide for Children with Achondroplasia

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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 condition known as Achondroplasia, which is a genetic disorder affecting bone growth, leading to shorter stature. The study is evaluating a treatment called BMN 111, also known as Voxzogo, which is a medication designed to help improve growth in children with this condition. Voxzogo is administered as a solution for injection under the skin.

The purpose of this study is to assess the long-term safety and effectiveness of BMN 111 in promoting growth in children with achondroplasia. Participants in the study will receive regular injections of the medication and will be monitored over an extended period. The study will track growth rates and any potential side effects to ensure the treatment is both safe and beneficial for the children involved.

Throughout the study, various health checks will be conducted, including physical exams and imaging tests like X-rays, to observe changes in bone growth and overall health. The study aims to provide valuable information on how BMN 111 can help children with achondroplasia achieve better growth outcomes and improve their quality of life.

The research process

The trial runs in 7 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 a previous study, identified as Study 111-301.
Consent is required from a parent or guardian, and from the participant if they are of legal age or reach it during the study.
Step 2
Baseline visit
A baseline visit includes a pregnancy test for females aged 10 years or older or those who have begun menstruation.
Participants must agree to use contraception if sexually active.
Step 3
Medication administration
The medication used is Voxzogo, which contains the active substance vosoritide.
It is administered as a solution for injection under the skin (subcutaneous use).
Dosages available are 0.4 mg, 0.56 mg, and 1.2 mg, with the specific dosage determined by the study protocol.
Step 4
Regular monitoring
Height growth velocity is measured annually to assess efficacy.
Safety is monitored through the incidence of adverse events, changes in vital signs, physical examinations, and various tests including ECG and echocardiograms.
Step 5
Additional assessments
Imaging and hip monitoring are conducted to evaluate bone morphology and quality.
Blood tests are performed to monitor hormone levels such as FSH and LH for participants over 8 years old.
Step 6
Quality of life evaluation
Quality of life is assessed using questionnaires like QoLISSY and PedsQL.
Functional independence is evaluated with the Wee-FIM assessment.
Step 7
Study duration
The study is expected to continue until December 31, 2030.
Participants are required to perform all study procedures as outlined in the protocol.

Who can join the trial?

5 criteria

The child must have completed a previous study called Study 111-301.
The child's parent(s) or guardian(s) must be willing and able to sign a document called informed consent, which means they understand the study and agree to let their child participate. If the child is old enough, they may also need to agree by signing a similar document called assent.
If the child is a girl who is 10 years old or older, or if she has started having her period, she must have a negative pregnancy test at the start of the study and be willing to take more pregnancy tests during the study.
If the child is sexually active, they must agree to use birth control as described in the study's rules.
The child must be willing and able to do all the activities and tests required by the study.

Who cannot join the trial?

7 criteria

Children who have any other medical condition that might interfere with the study.
Children who are taking medications that could affect their growth.
Children who have had surgery that could impact their growth.
Children who have participated in another clinical trial recently.
Children who have allergies to the study medication or its ingredients.
Children who have a history of certain serious health issues.
Children who are not able to follow the study instructions or attend study visits.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Hospital Sant Joan De Deu Barcelona
Esplugues De Llobregat, Spain
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Hospital Universitario Virgen De La Victoria
Malaga, Spain
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Otto Von Guericke Universitaet Magdeburg
Magdeburg, Germany

Trial locations

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Investigated drugs

BMN 111 is a medication being studied for its effects on children with achondroplasia, a condition that affects bone growth. The trial aims to evaluate how safe and well-tolerated this medication is over a long period, as well as its effectiveness in promoting growth in these children.

Vosoritide

What is already known about the treatment

BMN 111 – This medication is administered as an injection and is currently being studied in clinical trials for its safety and effectiveness in treating achondroplasia, a condition characterized by short stature. BMN 111 is in the investigational stage, with ongoing research to determine its long-term impact on growth in children with this condition. It works by targeting specific pathways involved in bone growth, aiming to promote normal growth patterns. BMN 111 is classified as a therapeutic agent for growth disorders.

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 enlarged 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 individuals with achondroplasia grow, they may experience a range of physical challenges, including limited range of motion at the elbows, bowed legs, and a sway of the lower back. Despite these challenges, individuals with achondroplasia typically have normal intelligence and can lead full, productive lives. The progression of the condition is generally stable after childhood, with most growth occurring in the first few years of life.