CompletedPhase 2

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

United States57 participantsStarted 2020-06-24

Plain-language summary

The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.

Who can participate

Age range2 Years – 10 Years

SexALL

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Inclusion criteria

✓. Clinical diagnosis of ACH with genetic confirmation
✓. Age between 2 to 10 years old (inclusive) at Screening Visit
✓. Prepubertal (Stage 1 breasts for girls or testicular volume \< 4ml for boys) at Screening Visit
✓. Able to stand without assistance
✓. Caregiver willing and able to administer subcutaneous injections of study drug

Exclusion criteria

✕. Clinically significant findings at Screening that:
✕. Have received treatment (\>3 months) of human growth hormone (hGH) or other medications known 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
✕. Have received any study drug or device intended to affect stature or body proportionality at any time
✕. History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones

What they're measuring

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

Timeframe: 52 weeks

Trial details

NCT IDNCT04085523

SponsorAscendis Pharma A/S

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-09-27

Results submitted2023-12-27

View on ClinicalTrials.gov More Achondroplasia trials