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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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

No Placebo-Only Group

Summary

This trial uses TransCon CNP to help children and adolescents with achondroplasia grow their bones properly. It is for those who have already been part of a previous trial. The treatment continues for an extended period.

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.

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.

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

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

Primary study objectives

Height Z-scores

Safety and Tolerability

Secondary study objectives

Annualized Growth Velocity

Side effects data

From 2024 Phase 2 trial • 57 Patients • NCT04085523

50%

Upper respiratory tract infection

50%

Nasopharyngitis

40%

Cough

30%

Arthralgia

30%

Headache

30%

Gastroenteritis

30%

Respiratory tract infection

30%

Pain in extremity

20%

Vomiting

20%

Nasal congestion

20%

Oropharyngeal pain

20%

Rhinorrhoea

20%

Diarrhoea

20%

Otitis media

20%

Pyrexia

10%

Viral infection

10%

Injection Site Reaction

10%

COVID-19

10%

Snoring

10%

Epistaxis

10%

Febrile Convulsion

10%

Ear Pain

10%

Abdominal Pain Upper

100%

80%

60%

40%

20%

Study treatment Arm

TransCon CNP 50 mcg

Placebo

TransCon CNP 100 mcg

TransCon CNP 6 mcg

TransCon CNP 20 mcg

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: TransCon CNP 100 mcgExperimental Treatment1 Intervention

TransCon CNP 100 mcg 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

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.