Completed

ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period

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What is being tested

TransCon CNP

+ Placebo for TransCon CNP

Drug
Who is being recruted

Achondroplasia+6

+ Bone Diseases

+ Bone Diseases, Developmental

From 2 to 10 Years
See all eligibility criteria
How is the trial designed

Treatment Study

Placebo-ControlledPhase 2
Interventional
Study Start: June 2020
See protocol details

Summary

Principal SponsorAscendis Pharma A/S
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: June 24, 2020

Actual date on which the first participant was enrolled.

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.

Official TitleACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period
Principal SponsorAscendis Pharma A/S
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

57 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.



Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 2 to 10 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

AchondroplasiaBone DiseasesBone Diseases, DevelopmentalDwarfismEndocrine System DiseasesMusculoskeletal DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesOsteochondrodysplasiasGenetic Diseases, Inborn

Criteria

Inclusion Criteria: 1. Clinical diagnosis of ACH with genetic confirmation 2. Age between 2 to 10 years old (inclusive) at Screening Visit 3. Prepubertal (Stage 1 breasts for girls or testicular volume \< 4ml for boys) at Screening Visit 4. Able to stand without assistance 5. Caregiver willing and able to administer subcutaneous injections of study drug Exclusion Criteria: 1. Clinically significant findings at Screening that: * are expected to require surgical intervention during participation in the trial or * are musculoskeletal in nature, such as Salter-Harris fractures and severe hip pain or * otherwise are considered by investigator or Medical Monitor/Medical Expert to make a participant unfit to receive study drug or undergo trial related procedures 2. Have received treatment (\>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time 3. Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit 4. Have received any study drug or device intended to affect stature or body proportionality at any time 5. History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

6 intervention groups are designated in this study

16.667% chance of being blinded to the placebo group

Treatment Groups

Group I

Experimental
Participants who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection.

Group II

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

Group III

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

Group IV

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

Group 5

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

Group 6

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

Study Objectives

Primary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 15 locations

Suspended

Ascendis Pharma Investigational Site

Little Rock, United StatesOpen Ascendis Pharma Investigational Site in Google Maps
Suspended

Ascendis Pharma Investigational Site

Aurora, United States
Suspended

Ascendis Pharma Investigational Site

Saint Paul, United States
Suspended

Ascendis Pharma Investigational Site

Columbia, United States
Completed15 Study Centers