RecruitingRare disease

Study on the Effectiveness and Safety of Navepegritide for Adolescents 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 achondroplasia, a condition that affects bone growth, leading to shorter stature. The study will evaluate a treatment called TransCon CNP, which is a solution for injection. This treatment involves a special protein called c-type natriuretic peptide that is linked to a carrier molecule to help it work effectively in the body. The purpose of the study is to assess how well this treatment works in promoting growth in adolescents aged 12 to under 18 years who have achondroplasia.

Participants in the study will receive weekly injections of either TransCon CNP or a placebo for a period of 52 weeks. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure that the results are unbiased. The main goal is to see if the treatment can improve growth over the course of the study.

Throughout the study, participants will be monitored for any changes in their height and overall health. The study aims to provide valuable information on the safety and effectiveness of TransCon CNP in treating achondroplasia, potentially offering a new option for managing this condition in adolescents.

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 study
Upon joining the study, informed consent or assent is required. This involves signing a document that explains the study and confirms understanding and agreement to participate. For those under the legal age of consent, a parent or legal guardian must also provide consent.
Participants must be between 12 and less than 18 years old, with a clinical diagnosis of achondroplasia confirmed by genetic testing.
Step 2
Randomization and initial assessment
Participants are randomly assigned to receive either the study medication, TransCon CNP, or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.
An initial assessment is conducted, including a review of medical history and a physical examination. A historical height measurement from medical records, taken between 6 to 15 months prior to screening, is required.
Step 3
Treatment phase
Participants receive a subcutaneous injection of the assigned treatment once a week for 52 weeks. The injection is administered under the skin.
Parents or legal guardians are responsible for administering the weekly injections and following the study protocol.
Step 4
Monitoring and follow-up
Throughout the study, regular monitoring is conducted to assess the participant's growth and overall health. This includes measuring height and evaluating any changes from the baseline height Z-score.
The primary goal is to evaluate the average growth velocity (AGV) at week 52.
Step 5
Completion of the study
At the end of the 52-week treatment period, a final assessment is conducted to evaluate the efficacy and safety of the treatment.
The study is estimated to conclude by January 1, 2027.

Who can join the trial?

5 criteria

The participant, or their parent(s) or legal guardian(s), must provide a written, signed agreement to join the study. This is called informed consent. If the participant is too young to give consent, they will need to give a written agreement called assent. When they reach the legal age, they will need to provide their own written consent.
The participant must be a boy or girl aged between 12 and less than 18 years old at the time they are randomly chosen to join the study.
The participant must have a clinical diagnosis of achondroplasia, which is a condition affecting bone growth, confirmed by a genetic test. Previous test results can be used as proof.
The participant's parent(s) or legal guardian(s) must be willing and able to give weekly injections under the skin, known as subcutaneous (SC) injections, and follow the study's rules.
There must be at least one past measurement of the participant's height while standing, available from medical records. This measurement should have been taken between 6 to 15 months before the screening for the study.

Who cannot join the trial?

7 criteria

Children and adolescents who do not have Achondroplasia, a condition affecting bone growth, cannot participate.
Individuals outside the specified age range for the study cannot participate.
Participants who are not available for the entire duration of the study cannot participate.
Those with other medical conditions that might interfere with the study cannot participate.
Individuals who are currently participating in another clinical trial cannot participate.
Participants who have had recent surgery or plan to have surgery during the study cannot participate.
Those who are unable to comply with the study procedures cannot participate.

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Verified sites

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

Children's Health Ireland
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Dublin, Ireland
Hopital Necker Enfants Malades
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Paris, France
Rigshospitalet
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Copenhagen, Denmark

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Investigated drugs

Navepegritide is a medication being studied for its potential to help with growth in adolescents with achondroplasia. Achondroplasia is a condition that affects bone growth, leading to shorter stature. This medication is given as an injection under the skin once a week. The trial aims to see if it can safely and effectively improve growth over a period of 52 weeks.

What is already known about the treatment

Navepegritide – This medication is administered through subcutaneous injections, typically given once a week. It is currently being studied in clinical trials for its effectiveness and safety in treating achondroplasia in children and adolescents. The main therapeutic indication is to promote growth in individuals with this condition. At the molecular level, navepegritide works by targeting specific pathways that influence bone growth and development. It is classified pharmacologically as a growth-promoting agent.

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. Children with achondroplasia typically have normal intelligence and life expectancy. As they grow, they may experience delayed motor skills and may require assistance with certain physical activities. The condition is present from birth and remains throughout life.