Not recruitingRare disease

Study on the Effects of TransCon CNP in Children Aged 2-11 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 a condition called Achondroplasia, which is a type of growth disorder in children. The trial is testing a new treatment called TransCon CNP, which is a solution for injection. This treatment involves a special substance known as 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 evaluate how well TransCon CNP can help improve growth in children with 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 during this time. After the initial 52 weeks, there will be an open-label extension period where all participants may receive the actual treatment. The study aims to observe changes in growth over the course of the trial.

The trial is specifically for children aged 2 to 11 years who have been diagnosed with Achondroplasia. The main goal is to see if the treatment can increase the rate of growth in these children. The study will also look at changes in height compared to the baseline measurements taken at the start of the trial. This research is important for understanding how TransCon CNP might help children with this growth disorder in the future.

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 and 11 years old and able to stand without assistance.
The parent or legal guardian must provide written, signed consent for the participant to join the trial.
Step 2
Initial assessment
An initial assessment will be conducted, including a review of medical history, a physical examination, and tests such as vital signs, ECG, and laboratory tests to confirm eligibility.
Step 3
Treatment phase
The participant will receive a subcutaneous injection of either TransCon CNP or a placebo once a week for 52 weeks. The injection is a solution administered under the skin.
The parent or legal guardian must be willing and able to administer these weekly injections.
Step 4
Monitoring and evaluation
Throughout the 52-week period, the participant's growth will be monitored to evaluate the efficacy of TransCon CNP. The primary measure is the annualized growth velocity at week 52.
Secondary evaluations include changes in height Z-score from the baseline at week 52.
Step 5
Open label extension
After the initial 52-week period, participants may enter an open label extension phase, where all participants receive TransCon CNP to further assess long-term effects.

Who can join the trial?

7 criteria

The parent(s) or legal guardian(s) must provide a written and signed agreement to allow the child to participate in the study.
The child must be a boy or girl between the ages of 2 and 11 at the time of the initial screening.
The child must have a clinical diagnosis of **Achondroplasia (ACH)**, which is a condition affecting bone growth, and there must be genetic proof of this diagnosis.
The child must be able to stand without help.
The parent(s) or legal guardian(s) must be willing and able to give weekly **subcutaneous (SC)** injections, which means injections under the skin, and follow the study's guidelines.
The child must have at least six months of growth and disease history from a previous study called **ACHieve (TCC-NHS-01)** or similar information available from medical records, which will be confirmed by a medical professional.
The child must be considered eligible based on their medical history, a physical check-up, and the results of vital signs, heart tests (**ECG**), and lab tests done during the screening period.

Who cannot join the trial?

8 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 are not eligible.
Participants who are not able to follow the study procedures or instructions will be excluded.
Those with other medical conditions that might interfere with the study's results cannot join.
Participants who are taking medications that could affect the study's outcomes are not allowed.
Individuals who have participated in another clinical trial recently may be excluded.
Anyone who is pregnant or planning to become pregnant during the study cannot participate.
Participants who have allergies or reactions to the study medication or similar drugs will be excluded.

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Verified sites

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

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

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Investigated drugs

TransCon CNP is a medication being studied for its potential to help children with achondroplasia grow. Achondroplasia is a condition that affects bone growth, leading to shorter stature. TransCon CNP is given as an injection under the skin once a week. The goal of this medication is to improve growth in children with this condition over a period of 52 weeks.

C-Type Natriuretic Peptide Conjugated To A Multi-Arm Polyethylene Glycol Carrier Molecule Through A Cleavable Linker

What is already known about the treatment

TransCon CNP – This medication is administered through a subcutaneous injection, 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, TransCon CNP works by releasing a C-type natriuretic peptide, which helps regulate bone growth and development. It is classified pharmacologically as a growth modulator.

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 development and may require assistance with certain physical activities. The condition is present from birth and remains throughout life, with growth patterns differing from those of average stature individuals.