TransCon CNP for Achondroplasia
(ApproaCH Trial)

Recruiting in Palo Alto (17 mi)

+9 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Ascendis Pharma Growth Disorders A/S

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

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.

Inclusion Criteria

I can stand up on my own without help.

My guardian can give me weekly shots and follow the study rules.

My recent health checks and tests show I am fit for the trial.

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Exclusion Criteria

I need urgent surgery due to severe neck spine compression.

Have a clinically significant finding or arrhythmia as determined by the investigator in consultation with the medical monitor that indicates abnormal cardiac function or conduction

I have had surgery on my bones that could affect their growth.

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Treatment Details

Interventions

Placebo for TransCon CNP (Placebo)
TransCon CNP (Peptide)

Trial OverviewThe 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.

Participant Groups

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