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

TransCon CNP, a novel drug developed by Ascendis Pharma, is being studied in several clinical trials for the treatment of achondroplasia in children and adolescents. Achondroplasia is the most common form of dwarfism, characterized by short stature and disproportionate body features. These trials aim to evaluate the safety, efficacy, and long-term effects of TransCon CNP, which is administered as a weekly subcutaneous injection. The studies focus on different age groups, from infants to adolescents, and assess various aspects of growth, body proportions, and quality of life improvements.

Table of Contents

What is TransCon CNP?

TransCon CNP, also known as navepegritide, is an investigational medication being developed to treat achondroplasia in children and adolescents[1]. It is a synthetically manufactured peptide that consists of C-type natriuretic peptide (CNP) conjugated to a multi-arm polyethylene glycol carrier molecule through a cleavable linker[2].

How Does TransCon CNP Work?

TransCon CNP is designed to provide sustained release of CNP, a naturally occurring molecule that plays a role in bone growth. By delivering CNP in a controlled manner, TransCon CNP aims to promote bone growth and improve height in individuals with achondroplasia[3].

What Condition Does TransCon CNP Treat?

TransCon CNP is being developed to treat achondroplasia, which is the most common form of dwarfism. Achondroplasia is a genetic condition that affects bone growth, resulting in short stature and other skeletal abnormalities[4]. People with achondroplasia have an average adult height of about 4 feet for women and 4 feet 4 inches for men.

Ongoing Clinical Trials

Several clinical trials are currently underway to evaluate the safety and effectiveness of TransCon CNP in different age groups:

  • A Phase 2 trial (AttaCH) for children and adolescents with achondroplasia[1]
  • A Phase 2 trial for infants (0 to <2 years of age) with achondroplasia[2]
  • A Phase 2b trial (COACH) combining TransCon CNP with lonapegsomatropin in children aged 2-11 years[3]
  • A Phase 2b trial (teACH) for adolescents aged 12-17 years[4]
  • A Phase 2b trial (ApproaCH) for children aged 2-11 years[5]

Potential Benefits

The main goals of TransCon CNP treatment are:

  • Improving linear growth and height in children and adolescents with achondroplasia[1]
  • Enhancing overall growth velocity (how fast a child grows)[5]
  • Potentially improving other aspects of achondroplasia, such as body proportions and quality of life[3]

Safety and Side Effects

The ongoing clinical trials are closely monitoring the safety of TransCon CNP. Some areas of focus include:

  • Monitoring for any adverse events or side effects[1]
  • Assessing cardiac function and heart-related issues[4]
  • Evaluating bone health and growth plate status[5]
  • Checking for any allergic reactions to the medication[2]

It’s important to note that as an investigational drug, the full safety profile of TransCon CNP is still being established through these clinical trials.

Administration

TransCon CNP is administered as a subcutaneous injection (an injection under the skin) once weekly[1]. The medication is provided as a solution for injection, and parents or caregivers will be trained on how to administer the injections at home[2].

Conclusion

TransCon CNP represents a promising potential treatment for children and adolescents with achondroplasia. By targeting the underlying mechanisms of bone growth, this medication aims to improve height and potentially other aspects of this condition. As clinical trials progress, more information will become available about its effectiveness and safety profile. If you have a child with achondroplasia, you may want to discuss with your doctor whether participating in a clinical trial or considering this treatment in the future might be appropriate.

Trial Name Age Group Duration Primary Endpoints Key Features
AttaCH Children and Adolescents 156 weeks Safety, tolerability, and efficacy on growth Long-term extension trial
Infant Study 0 to <2 years 52 weeks + OLE Safety, tolerability, and effect on growth Includes placebo control
COACH 2 to 11 years 156 weeks AGV at Week 52 Combines TransCon CNP with lonapegsomatropin
teACH 12 to <18 years 52 weeks Efficacy on growth Focuses on adolescents
ApproaCH 2 to 11 years 52 weeks + OLE AGV at Week 52 Placebo-controlled with open-label extension

FAQ

  • What is TransCon CNP and how does it work? TransCon CNP is a drug that contains C-type natriuretic peptide (CNP) conjugated to a multi-arm polyethylene glycol carrier molecule through a cleavable linker. It is designed to promote bone growth in individuals with achondroplasia by regulating the overactive FGFR3 signaling that causes the condition.
  • Who is eligible to participate in these clinical trials? The trials are designed for children and adolescents with achondroplasia, with specific age ranges varying by study. Generally, participants must have a confirmed genetic diagnosis of achondroplasia and meet certain health criteria. Some trials focus on ages 2-11, while others include adolescents up to 18 years old.
  • How is TransCon CNP administered in these trials? TransCon CNP is administered as a weekly subcutaneous injection. Parents or caregivers are trained to give these injections at home, making it convenient for long-term treatment.
  • What are the main outcomes being measured in these trials? The primary outcomes include annualized growth velocity (AGV), changes in height Z-scores, and safety assessments. Secondary outcomes often include body proportions, quality of life measures, and developmental milestones.
  • How long do these clinical trials last? The duration of the trials varies, but many include a 52-week initial treatment period followed by longer-term extension phases. Some trials last up to 156 weeks (3 years) to assess long-term safety and efficacy.

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

  • Study on the Effects of TransCon CNP in Children Aged 2-11 with Achondroplasia

    Not recruiting

    1
    Investigated drugs:
    Denmark Ireland Spain

Glossary

  • Achondroplasia: The most common form of dwarfism, characterized by short stature and disproportionate body features due to a genetic mutation affecting bone growth.
  • TransCon CNP: A drug containing C-type natriuretic peptide conjugated to a multi-arm polyethylene glycol carrier molecule, designed to promote bone growth in individuals with achondroplasia.
  • Annualized Growth Velocity (AGV): The rate of growth in height over a year, typically measured in centimeters per year (cm/year).
  • Height Z-score: A measure of how a child's height compares to the average height of children of the same age and sex in the general population.
  • Subcutaneous Injection: An injection given into the fatty tissue just beneath the skin.
  • Open Label Extension (OLE): A phase of a clinical trial where all participants receive the active treatment, often following a placebo-controlled phase.
  • QTcF: The QT interval on an electrocardiogram corrected for heart rate using Fridericia's formula, used to assess cardiac safety.
  • Cervicomedullary Decompression: A surgical procedure to relieve pressure on the brainstem and upper spinal cord, sometimes necessary in individuals with achondroplasia.
  • FGFR3: Fibroblast Growth Factor Receptor 3, the gene mutated in achondroplasia that leads to overactive signaling and impaired bone growth.
  • Polyethylene Glycol (PEG): A compound used in drug delivery to improve the pharmacokinetics and stability of therapeutic proteins.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-transcon-cnp-for-children-and-adolescents-with-achondroplasia/
  2. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-transcon-cnp-for-infants-with-achondroplasia/
  3. http://clinicaltrials.eu/trial/study-on-the-effects-of-navepegritide-and-lonapegsomatropin-in-children-with-achondroplasia/
  4. http://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-navepegritide-for-adolescents-with-achondroplasia/
  5. http://clinicaltrials.eu/trial/study-on-the-effects-of-transcon-cnp-in-children-aged-2-11-with-achondroplasia/