What side effects are associated with this type of intervention?

The clinical trial for TransCon CNP in children with Achondroplasia aims to evaluate its efficacy and safety. Common side effects for treatments promoting endochondral ossification and bone growth, such as CNP analogs, may include injection site reactions, headaches, and potential impacts on blood pressure. As with any treatment, monitoring for adverse effects is crucial to ensure safety and efficacy.

What prior FDA approvals exist?

As of now, there are no FDA-approved drugs specifically for the treatment of Achondroplasia that work through the mechanism of promoting endochondral ossification and bone growth similar to CNP (C-type Natriuretic Peptide). The clinical trial mentioned is investigating the efficacy and safety of TransCon CNP, which is designed to promote bone growth in children with Achondroplasia. This represents a novel approach in the treatment landscape for this condition.

What other types of research exist?

Promising novel alternatives to CNP for Achondroplasia patients include vosoritide, a C-type natriuretic peptide analog, which has shown positive results in increasing growth velocity in clinical trials. Additionally, gene therapy approaches and other growth-promoting agents like FGFR3 inhibitors are being explored as potential treatments.

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TransCon CNP for Achondroplasia (ApproaCH Trial)

Phase 2 & 3

Waitlist Available

Research Sponsored by Ascendis Pharma Growth Disorders A/S

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Summary

This trial tests if regular injections can help children with Achondroplasia grow taller by making their bones grow faster. Vosoritide has shown promising results in increasing growth in children with achondroplasia.

Who is the study for?

This trial is for children aged 2 to 11 with confirmed Achondroplasia who can stand without assistance. They must have a history of growth and disease from previous trials or medical records, and their parents must consent and be able to administer weekly injections. Children with chronic anemia, renal insufficiency, recent participation in other clinical trials, closed epiphysis, hypersensitivity to the drug components, other growth disorders or conditions affecting stature are excluded.

What is being tested?

The trial tests TransCon CNP's effectiveness on improving annual growth velocity compared to placebo over one year in children with Achondroplasia. It's a double-blind study where neither participants nor researchers know who receives the real treatment until after the results are collected. Afterward, there's an open-label extension for another year.

What are the potential side effects?

While specific side effects aren't listed here, common concerns may include reactions at injection sites such as pain or swelling, potential allergic reactions if sensitive to ingredients in TransCon CNP or placebo components. Other risks could involve how the body reacts overall to new treatments.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Annualized Growth Velocity

Secondary study objectives

Height Z-score

Side effects data

From 2024 Phase 2 trial • 57 Patients • NCT04085523

36%

Pyrexia

27%

Cough

27%

Nasal congestion

18%

Gastroenteritis

18%

Rhinorrhoea

18%

Vomiting

18%

Epistaxis

18%

Otitis media

18%

Fatigue

Vitamin D deficiency

Upper respiratory tract infection

COVID-19

Snoring

Pain in extremity

Viral infection

Injection Site Reaction

Ear Pain

Headache

Abdominal Pain Upper

100%

80%

60%

40%

20%

Study treatment Arm

TransCon CNP 20 mcg

TransCon CNP 6 mcg

Placebo

TransCon CNP 50 mcg

TransCon CNP 100 mcg

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: TransCon CNPExperimental Treatment1 Intervention

Once weekly double-blinded treatment with SC injection of 100 µg/kg of TransCon CNP for 52 weeks

Group II: Placebo for TransCon CNPPlacebo Group1 Intervention

Once weekly double-blinded treatment with SC injection of 100 µg/kg of Placebo for TransCon CNP for 52 weeks

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

TransCon CNP

2020

Completed Phase 2

~60

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

C-type Natriuretic Peptide (CNP) is a promising treatment for Achondroplasia as it promotes endochondral ossification and bone growth, processes that are impaired in this condition. By enhancing these processes, CNP can improve growth velocity and overall bone development in children with Achondroplasia, addressing the core growth deficiencies caused by the genetic mutation. This mechanism of action is crucial for improving the quality of life and physical development in affected patients.