What side effects are associated with this type of intervention?

The most common treatments for achondroplasia, particularly those involving C-type Natriuretic Peptide (CNP) like TransCon CNP, are generally well-tolerated but can have some side effects. These may include injection site reactions, such as redness, swelling, or pain. Other potential side effects could involve gastrointestinal symptoms like nausea or vomiting, and in some cases, there may be concerns about the long-term impact on cardiovascular health due to the systemic effects of CNP. Regular monitoring and follow-up with healthcare providers are essential to manage and mitigate these side effects.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for achondroplasia that utilize C-type Natriuretic Peptide (CNP) to promote bone growth by inhibiting the FGFR3 pathway. The clinical trial for TransCon CNP is investigating this novel approach. Other treatments for achondroplasia have focused on managing symptoms and complications rather than directly addressing the underlying genetic cause. The development of therapies like TransCon CNP represents a promising area of research aimed at modifying the disease process itself.

What other types of research exist?

Promising novel alternatives to TransCon CNP for Achondroplasia patients include vosoritide, a C-type natriuretic peptide analog that has shown efficacy in promoting bone growth by inhibiting the FGFR3 pathway. Another potential treatment is infigratinib, an FGFR1-3 inhibitor, which is being investigated for its ability to promote bone growth in Achondroplasia. Both therapies are in advanced stages of clinical development and may offer effective treatment options by 2024.

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Other

TransCon CNP for Achondroplasia (AttaCH 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 through trial completion, an average of 10 years

Awards & highlights

Summary

This trial will study the effects of TransCon CNP on people aged 2-18 with achondroplasia. Treatment will continue until femur and tibial epiphyseal closure is confirmed, with visits every 12-14 weeks.

Who is the study for?

This trial is for children and adolescents with achondroplasia who have already been part of a previous TransCon CNP study. They must be able to receive weekly injections, have parental consent, and meet safety criteria from the prior trial. Those unable to complete the study or at risk due to other conditions are excluded.Check my eligibility

What is being tested?

The long-term safety, tolerability, and effectiveness of TransCon CNP given once a week are being tested in young patients with achondroplasia until they reach skeletal maturity (16 years for females, 18 years for males), confirmed by radiographic imaging.See study design

What are the potential side effects?

Specific side effects aren't listed here but may include reactions related to subcutaneous injection or hypersensitivity to ingredients like trehalose, tris[hydroxymethyl]aminomethane, succinate, and methoxy polyethylene glycol.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through trial completion, an average of 10 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through trial completion, an average of 10 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and through trial completion, an average of 10 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Height Z-scores

Safety and Tolerability

Secondary outcome measures

Annualized Growth Velocity

Trial Design

1Treatment groups

Experimental Treatment

Group I: TransCon CNP 100 mcgExperimental Treatment1 Intervention

TransCon CNP 100 mcg delivered once weekly by subcutaneous injection

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

The most common treatments for Achondroplasia focus on promoting bone growth by targeting specific molecular pathways. TransCon CNP, a C-type Natriuretic Peptide, works by inhibiting the FGFR3 pathway, which is overactive in Achondroplasia due to mutations in the FGFR3 gene. This inhibition helps normalize bone growth, addressing the root cause of the condition. Other treatments, such as growth hormone therapy, stimulate overall growth but do not specifically target the FGFR3 pathway. Understanding these mechanisms is crucial for patients and healthcare providers to select the most effective treatment that directly addresses the genetic abnormalities in Achondroplasia.

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Who is running the clinical trial?

Ascendis Pharma Growth Disorders A/SLead Sponsor

3 Previous Clinical Trials

174 Total Patients Enrolled

3 Trials studying Achondroplasia

174 Patients Enrolled for Achondroplasia

Silas Gylvin, MDStudy DirectorAscendis Pharma

1 Previous Clinical Trials

57 Total Patients Enrolled

1 Trials studying Achondroplasia

57 Patients Enrolled for Achondroplasia

Adebola Giwa, MDStudy DirectorAscendis Pharma

2 Previous Clinical Trials

317 Total Patients Enrolled

2 Trials studying Achondroplasia

317 Patients Enrolled for Achondroplasia

Media Library

TransCon CNP (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05929807 — Phase 2 & 3

Achondroplasia Research Study Groups: TransCon CNP 100 mcg

Achondroplasia Clinical Trial 2023: TransCon CNP Highlights & Side Effects. Trial Name: NCT05929807 — Phase 2 & 3

TransCon CNP (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05929807 — Phase 2 & 3

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