What is the mechanism of action of this treatment?

TransCon CNP is a sustained release formulation of C-type Natriuretic Peptide (CNP), which promotes bone growth by inhibiting the signaling pathway of fibroblast growth factor receptor 3 (FGFR3). FGFR3 is overactive in Achondroplasia, leading to impaired bone growth and short stature. By blocking this pathway, CNP helps to normalize bone growth and improve height outcomes in patients. This mechanism is crucial for Achondroplasia patients as it directly targets the underlying cause of their growth issues, offering a potential therapeutic approach to enhance their quality of life.

What side effects are associated with this type of intervention?

Common treatments for Achondroplasia, particularly those involving C-type Natriuretic Peptide (CNP) like TransCon CNP, aim to promote bone growth. Known side effects of CNP treatments can include injection site reactions, hypotension (low blood pressure), and headaches. Other potential side effects may involve gastrointestinal issues such as nausea and vomiting. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate any adverse effects effectively.

What prior FDA approvals exist?

As of now, the FDA has not approved any drugs specifically for the treatment of Achondroplasia that utilize C-type Natriuretic Peptide (CNP) or similar mechanisms to promote bone growth. The current trial involving TransCon CNP is exploring a novel approach by using a sustained release form of CNP to address the underlying growth issues in children with Achondroplasia. Other treatments for Achondroplasia have primarily focused on surgical interventions and growth hormone therapy, but these do not involve CNP or related pathways.

What other types of research exist?

Promising alternatives to TransCon CNP for Achondroplasia treatment include vosoritide and meclozine. Vosoritide, a C-type natriuretic peptide analog, has shown positive results in increasing growth velocity in children with Achondroplasia. Meclozine, an antihistamine, has demonstrated potential in preclinical studies to promote bone growth by inhibiting FGFR3 signaling, which is implicated in Achondroplasia.

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Peptide Hormone Analog

TransCon CNP for Achondroplasia

Phase 2

Waitlist Available

Research Sponsored by Ascendis Pharma A/S

Eligibility Criteria Checklist

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

Must have

Able to stand without assistance

Age between 2 to 10 years old (inclusive) at Screening Visit

Must not have

Have received any study drug or device intended to affect stature or body proportionality at any time

Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Summary

This trial tests a shot called TransCon CNP in children aged 2 to 10 with Achondroplasia. The shot helps bones grow by delivering a special protein. The goal is to see if it can improve growth in these children. TransCon CNP has shown promising results in increasing growth in children with achondroplasia.

Who is the study for?

This trial is for prepubertal children aged 2-10 with Achondroplasia, able to stand without assistance and have a caregiver willing to give injections. Kids who've had growth treatments or need surgery that could interfere with the study can't join.

What is being tested?

The trial tests TransCon CNP, given weekly via injection, against a placebo in kids with Achondroplasia. It's randomized and double-blind, meaning neither the researchers nor participants know who gets the real treatment.

What are the potential side effects?

While specific side effects are not listed here, they may include reactions at the injection site, general discomfort or other issues related to growth treatments which will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can stand up on my own without help.

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I am between 2 and 10 years old.

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I am prepubertal according to the screening visit.

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I have been diagnosed with ACH and it's genetically confirmed.

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My caregiver can give me shots under my skin as part of the study.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have never used drugs or devices to alter my height or body shape.

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I have taken medication to alter my height or body shape in the last 6 months.

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I have a condition affecting my bone growth, but it's not Achondroplasia.

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 Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment

Trial Design

6Treatment groups

Experimental Treatment

Placebo Group

Group I: TransCon CNP 6 mcgExperimental Treatment2 Interventions

TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection.

Group II: TransCon CNP 50 mcgExperimental Treatment2 Interventions

TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection.

Group III: TransCon CNP 20 mcgExperimental Treatment2 Interventions

TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection.

Group IV: TransCon CNP 100 mcgExperimental Treatment2 Interventions

TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection.

Group V: Open-Label Extension PeriodExperimental Treatment1 Intervention

Subjects who complete the 52-week blinded treatment period can continue into the 104-week open-label extension period and will receive treatment with TransCon CNP.

Group VI: PlaceboPlacebo Group1 Intervention

Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

TransCon CNP

2020

Completed Phase 2

~60

Placebo for TransCon CNP

2020

Completed Phase 2

~60

0 / 8Eligibility criteria met

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

TransCon CNP is a sustained release formulation of C-type Natriuretic Peptide (CNP), which promotes bone growth by inhibiting the signaling pathway of fibroblast growth factor receptor 3 (FGFR3). FGFR3 is overactive in Achondroplasia, leading to impaired bone growth and short stature. By blocking this pathway, CNP helps to normalize bone growth and improve height outcomes in patients. This mechanism is crucial for Achondroplasia patients as it directly targets the underlying cause of their growth issues, offering a potential therapeutic approach to enhance their quality of life.