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

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What is this study about?

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.

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.

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.

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.

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.

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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Rigshospitalet	Copenhagen	Denmark
Children’s Health Ireland	Dublin	Ireland

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	01.05.2024
Ireland	Not recruiting	01.05.2024

Trial locations

Investigated drugs:

Lonapegsomatropin

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.

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.

Trial ID:

2023-508341-40-00

Protocol code:

ASND0042

Trial Phase:

Therapeutic exploratory (Phase II)

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?