Trial · NCT04085523

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

NCT04085523, Phase 2, Completed, Ascendis Pharma A/S; results first posted 2024-03-07

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia. Trials, record NCT04085523. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04085523

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
NCT number: NCT04085523
Status: Completed
Phases: Phase 2
Enrollment: 57 participants, actual
Lead sponsor: Ascendis Pharma A/S
Conditions: Achondroplasia
Interventions: TransCon CNP, Placebo for TransCon CNP
First posted: 2019-09-11
Last updated: 2026-07-02
Results first posted: 2024-03-07

Source ClinicalTrials.gov

Registered outcomes1

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	52 weeks	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.

Source ClinicalTrials.gov

Outcomes1

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	52 weeks		cm/year	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group5

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	TransCon CNP 6 mcg	4.09	95% Confidence Interval 3.34 to 4.84	cm/year
Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	TransCon CNP 20 mcg	4.52	95% Confidence Interval 3.82 to 5.22	cm/year
Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	TransCon CNP 50 mcg	5.16	95% Confidence Interval 4.43 to 5.90	cm/year
Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	TransCon CNP 100 mcg	5.42	95% Confidence Interval 4.74 to 6.11	cm/year
Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	Placebo	4.35	95% Confidence Interval 3.75 to 4.94	cm/year

Source ClinicalTrials.gov

Participant flow36

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Double-Blind Period (Weeks 0 to 52)	STARTED	TransCon CNP 6 mcg	10
Double-Blind Period (Weeks 0 to 52)	STARTED	TransCon CNP 20 mcg	11
Double-Blind Period (Weeks 0 to 52)	STARTED	TransCon CNP 50 mcg	10
Double-Blind Period (Weeks 0 to 52)	STARTED	TransCon CNP 100 mcg	11
Double-Blind Period (Weeks 0 to 52)	STARTED	Placebo	15
Double-Blind Period (Weeks 0 to 52)	STARTED	Open-Label Extension Period: TransCon CNP	0
Double-Blind Period (Weeks 0 to 52)	COMPLETED	TransCon CNP 6 mcg	10
Double-Blind Period (Weeks 0 to 52)	COMPLETED	TransCon CNP 20 mcg	11
Double-Blind Period (Weeks 0 to 52)	COMPLETED	TransCon CNP 50 mcg	10
Double-Blind Period (Weeks 0 to 52)	COMPLETED	TransCon CNP 100 mcg	11

1 to 10 of 36Source ClinicalTrials.gov

See all 36 participant flow

Adverse events342

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Headache	Nervous system disorders	TransCon CNP 6 mcg	0	10	0
other	Headache	Nervous system disorders	TransCon CNP 20 mcg	1	11	1
other	Headache	Nervous system disorders	TransCon CNP 50 mcg	3	10	4
other	Headache	Nervous system disorders	TransCon CNP 100 mcg	2	11	2
other	Headache	Nervous system disorders	Placebo	2	15	8
other	Headache	Nervous system disorders	Open-Label Extension Period: TransCon CNP	17	57	40
other	Pyrexia	General disorders	TransCon CNP 6 mcg	1	10	2
other	Pyrexia	General disorders	TransCon CNP 20 mcg	4	11	7
other	Pyrexia	General disorders	TransCon CNP 50 mcg	2	10	2
other	Pyrexia	General disorders	TransCon CNP 100 mcg	2	11	4

1 to 10 of 342Source ClinicalTrials.gov

See all 342 adverse events

Adverse event totals18

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	TransCon CNP 6 mcg	0	10
Total, all-cause mortality	TransCon CNP 20 mcg	0	11
Total, all-cause mortality	TransCon CNP 50 mcg	0	10
Total, all-cause mortality	TransCon CNP 100 mcg	0	11
Total, all-cause mortality	Placebo	0	15
Total, all-cause mortality	Open-Label Extension Period: TransCon CNP	0	57
Total, other adverse events	TransCon CNP 6 mcg	9	10
Total, other adverse events	TransCon CNP 20 mcg	11	11
Total, other adverse events	TransCon CNP 50 mcg	10	10
Total, other adverse events	TransCon CNP 100 mcg	10	11

1 to 10 of 18Source ClinicalTrials.gov

See all 18 adverse event totals

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
37823031	RESULT	PubMed
42319555	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04085523

GET https://conductscience.org/api/trials/v1/NCT04085523/flow?page=2

API reference For AI assistants