NCT04085523|CompletedPhase 2ResultsDMCFDA Drug

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

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

Ascendis Pharma A/S ClinicalTrials.gov

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1 other identifier

TCC-201

Study Type

interventional

Target

Locations

7 countries

Sites

Timeline

RegisteredSep 2019

StartedJun 2020

CompletionOct 2024

Brief 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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P25-P50 for phase_2

Timeline

Completed

Started Jun 2020

Typical duration for phase_2

Geographic Reach

7 countries

14 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

4.3 years study duration

First Submitted

Initial submission to the registry

September 9, 2019

Completed

2 days until next milestone

First Posted

Study publicly available on registry

September 11, 2019

Completed

10 months until next milestone

Study Start

First participant enrolled

June 24, 2020

Completed

2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 27, 2022

Completed

1.4 years until next milestone

Results Posted

Study results publicly available

March 7, 2024

Completed

7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2024

Completed

Last Updated

May 22, 2025

Status Verified

May 1, 2025

Enrollment Period

2.3 years

First QC Date

September 9, 2019

Results QC Date

December 27, 2023

Last Update Submit

May 7, 2025

Conditions

Achondroplasia

Keywords

AchondroplasiaDwarfism

Outcome Measures

Primary Outcomes (1)

Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment
The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set.
52 weeks

Study Arms (6)

TransCon CNP 6 mcg

EXPERIMENTAL

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

Drug: TransCon CNP

TransCon CNP 20 mcg

EXPERIMENTAL

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

Drug: TransCon CNP

TransCon CNP 50 mcg

EXPERIMENTAL

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

Drug: TransCon CNP

TransCon CNP 100 mcg

EXPERIMENTAL

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

Drug: TransCon CNP

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo for TransCon CNP

Open-Label Extension Period: TransCon CNP

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.

Drug: TransCon CNP

Interventions

TransCon CNPDRUG

TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

Open-Label Extension Period: TransCon CNPTransCon CNP 100 mcgTransCon CNP 20 mcgTransCon CNP 50 mcgTransCon CNP 6 mcg

Placebo for TransCon CNPDRUG

Weekly subcutaneously injection of placebo.

Placebo

Eligibility Criteria

Age2 Years - 10 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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

You may not qualify if:

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
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

Contact the study team to confirm eligibility.