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Study on the Effects of Navepegritide and Lonapegsomatropin in Children with Achondroplasia

Verified siteInvestigationalNo 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 Achondroplasia, a condition that affects bone growth in children, leading to shorter stature. The study will use two treatments: Navepegritide and Lonapegsomatropin. These treatments are given as a solution for injection under the skin. The purpose of the study is to evaluate how these treatments affect growth in children with Achondroplasia and to assess their safety.

Participants in the study will receive the treatments once a week over a period of 156 weeks, which is about three years. The study will monitor the children's growth and overall health during this time. The researchers will compare the effects of using both treatments together to using Navepegritide alone. This will help determine if the combination of treatments is more effective in promoting growth.

Throughout the study, the safety of the treatments will be closely observed. This includes checking for any side effects and monitoring the children's health through regular medical assessments. The study aims to provide valuable information on the potential benefits and safety of these treatments for children with Achondroplasia.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the trial
Upon joining the trial, the participant must have a clinical diagnosis of achondroplasia confirmed by genetic testing. The participant should be between 2 to 11 years old and able to stand without assistance.
The parent or legal guardian must provide written consent for the participant to join the trial.
Step 2
Initial assessment
An initial assessment will be conducted to gather baseline data. This includes measurements of height, body composition, and other relevant health indicators.
The assessment will also include a review of the participant's medical history and any previous growth data.
Step 3
Medication administration
The participant will receive a combination of two medications: navepegritide and lonapegsomatropin. Both medications are administered as a subcutaneous injection once a week.
The medication TransCon hGH contains lonapegsomatropin, and TransCon CNP contains navepegritide. These are solutions for injection.
Step 4
Ongoing assessments
Throughout the trial, regular assessments will be conducted to monitor growth and health. These assessments occur at weeks 26, 52, 104, and 156.
The assessments will include measurements of height, body composition, and other health indicators. Safety assessments will also be conducted, including vital signs, laboratory tests, and physical examinations.
Step 5
End of trial
The trial is expected to last for 156 weeks, concluding in July 2027.
At the end of the trial, a final assessment will be conducted to evaluate the overall effects of the treatment on growth and health.

Who can join the trial?

6 criteria

The child's parent(s) or legal representative(s) must provide a written, signed agreement to participate in the study. This is called an informed consent.
The child must be a boy or girl between the ages of 2 and 11 years old at the time of the first visit.
The child must have a clinical diagnosis of Achondroplasia (ACH), which is a condition affecting bone growth, confirmed by a genetic test showing a specific type of genetic pattern called a heterozygote genotype. Previous test results can be used to prove this diagnosis.
The child must be able to stand without help.
The child's parent(s), caregiver(s), or legal guardian(s) must be willing and able to give weekly injections under the skin, known as subcutaneous (SC) injections, and follow all study requirements.
There must be at least 6 months of information about the child's growth and disease history available from previous studies or medical records.

Who cannot join the trial?

7 criteria

Children who do not have Achondroplasia, a condition that affects bone growth, cannot participate.
Children who are not within the specified age range for the study cannot participate.
Children who have other medical conditions that might interfere with the study cannot participate.
Children who are taking medications that could affect the study results cannot participate.
Children who have had previous treatments that might influence the study outcomes cannot participate.
Children who are unable to follow the study procedures cannot participate.
Children whose parents or guardians do not give permission for them to participate cannot be included.

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Verified sites

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

Children's Health Ireland
Verified
Dublin, Ireland
Rigshospitalet
Verified
Copenhagen, Denmark

Trial locations

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Investigated drugs

Navepegritide
is a medication being studied for its potential to help children with achondroplasia grow taller. Achondroplasia is a condition that affects bone growth, leading to shorter stature. This medication is being tested to see if it can improve the growth rate in these children.
Lonapegsomatropin
is another medication included in the study, used in combination with navepegritide. It is being evaluated to see if it can further enhance the growth effects when used together with navepegritide. The study aims to understand how these two medications work together to promote better growth outcomes in children with achondroplasia.

Lonapegsomatropin

What is already known about the treatment

Navepegritide
Navepegritide is administered as an injection, typically given once a week. It is currently being studied in clinical trials for its potential to aid growth in children with achondroplasia, a condition characterized by short stature. The medication works by targeting specific pathways that influence bone growth, aiming to enhance linear growth. It is classified as a growth-promoting agent.
Lonapegsomatropin
Lonapegsomatropin is also administered via injection on a weekly basis. It is under investigation in clinical trials for its use in children with achondroplasia, focusing on improving growth outcomes. This medication functions by mimicking natural growth hormone, stimulating growth in bones and tissues. It falls under the category of growth hormone therapies.

Investigated diseases

Achondroplasia – Achondroplasia is a genetic disorder that affects bone growth, leading to dwarfism. It is characterized by short stature with disproportionately short arms and legs, a large head, and distinctive facial features. 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 challenges with balance and coordination. Over time, individuals may also develop spinal stenosis, which can lead to back pain and other complications. Despite these challenges, individuals with achondroplasia typically have normal intelligence and can lead full, productive lives.