NCT05598320|CompletedPhase 2ResultsDMCFDA Drug

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia

ApproaCH

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

Ascendis Pharma Growth Disorders A/S ClinicalTrials.gov

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1 other identifier

ASND0036

Study Type

interventional

Target

Locations

7 countries

Sites

Timeline

RegisteredOct 2022

StartedMar 2023

CompletionAug 2025

Brief Summary

The purpose of this clinical trial was to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of 100 µg TransCon CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period in children aged 2 to 11 years with genetically confirmed Achondroplasia. The double-blind, placebo-controlled treatment period was followed by an Open Label Extension (OLE) period of a 52-week duration.

Trial Health

On Track

Trial Health Score

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

Enrollment

participants targeted

Target at P50-P75 for phase_2

Timeline

Completed

Started Mar 2023

Geographic Reach

7 countries

10 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

2.4 years study duration

First Submitted

Initial submission to the registry

October 25, 2022

Completed

3 days until next milestone

First Posted

Study publicly available on registry

October 28, 2022

Completed

4 months until next milestone

Study Start

First participant enrolled

March 3, 2023

Completed

1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 9, 2024

Completed

1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

August 13, 2025

Completed

11 months until next milestone

Results Posted

Study results publicly available

July 14, 2026

Completed

Last Updated

July 14, 2026

Status Verified

June 1, 2026

Enrollment Period

1.4 years

First QC Date

October 25, 2022

Results QC Date

June 16, 2026

Last Update Submit

June 16, 2026

Conditions

Achondroplasia

Outcome Measures

Primary Outcomes (1)

Annualized Growth Velocity (AGV) at Week 52
Annualized growth velocity is defined as (height - baseline height)/(date of height assessment - date of baseline) \* 365.25. Annualized growth velocity reported in terms of centimeters (cm) per year. Missing values at Week 52 were imputed by a multiple imputation method.
At Week 52

Secondary Outcomes (2)

Change From Baseline in Height Z-score (ACH-specific) at Week 52
Baseline, Week 52
Change From Baseline in Height Z-score (CDC-Based) at Week 52
Baseline, Week 52

Study Arms (3)

Navepegritide

EXPERIMENTAL

Participants received Navepegritide (TransCon CNP) 100 micrograms per kilogram (µg/kg) delivered once weekly by subcutaneous injection for a treatment period of up to 52 weeks.

Drug: Navepegritide (TransCon CNP)

Placebo for Navepegritide

PLACEBO COMPARATOR

Participants received placebo matched for Navepegritide (TransCon CNP) 100 µg/kg delivered once weekly by subcutaneous injection for a treatment period of up to 52 weeks.

Drug: Placebo for Navepegritide (TransCon CNP)

Open-Label Extension Period: Navepegritide

EXPERIMENTAL

Participants who completed the 52-week treatment period continued into the open-label extension period and received treatment with navepegritide (TransCon CNP) doses up to Week 104. All participants received navepegritide at a dose of 100 μg/kg/week.

Drug: Navepegritide (TransCon CNP)

Interventions

Navepegritide (TransCon CNP)DRUG

Once-weekly subcutaneous injection of 100 µg/kg Navepegritide (TransCon CNP)

NavepegritideOpen-Label Extension Period: Navepegritide

Placebo for Navepegritide (TransCon CNP)DRUG

Once-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide (TransCon CNP)

Placebo for Navepegritide

Eligibility Criteria

Age2 Years - 11 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

Written, signed informed consent of the parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC).
Male or female, between 2 and 11 years of age (inclusive) at the time of Screening.
Clinical diagnosis of Achondroplasia (ACH) with documented genetic confirmation available.
Able to stand without assistance.
Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of Investigational Medicinal Product (IMP) and to follow the protocol.
At least six months of growth and disease history from ACHieve (TCC-NHS-01) trial or comparable growth and disease history available from medical records (pending confirmation by Medical Monitor).
Considered eligible based on the medical history, physical examination, and the results of vital signs, ECG and clinical laboratory tests performed during the Screening period

You may not qualify if:

Participation (i.e., signed informed consent) in any interventional clinical trial before within 3 months prior to screening.
Closed epiphysis.
Known or suspected hypersensitivity to the IMP or related products (trehalose, tris\[hydroxymethyl\]aminomethane, succinate, and mPEG).
Had a growth disorder or medical condition other than ACH that results in short stature or abnormal growth such as severe ACH with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, celiac disease, hypothyroidism, hyperthyroidism, pre-diabetes, or diabetes mellitus.
Have received any dose of prescription medications and IMP or surgical intervention intended to affect stature, growth, or body proportionality at any time.
Required, or anticipated to require, chronic (\> 4 weeks) or repeated treatment (more than twice/year and \>3 weeks/year) with systemic corticosteroids during participation in the trial. Chronic use of high-dose inhaled corticosteroids was not allowed.
Known history of presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.
Known history of any bone-related surgery affecting growth potential of long bones, such as:
Cervicomedullary decompression surgery without anticipated need for repeat decompression during the time of the trial are allowed with minimum of 6 months of bone healing.
Ventriculoperitoneal (VP) shunt and laminectomy with full recovery are allowed with minimum of 6 months of bone healing.
Bone fracture within 6 months prior to screening (within 2 months for fracture of digits and buckle fractures).
Clinically significant findings at Screening, such as:
Expected to require surgical intervention during participation in the trial. Common surgeries, such as insertion of grommets, adenoidectomy, tonsillectomy, or myringotomy tube placement, are permitted.
Severe untreated sleep apnea or newly initiated sleep apnea treatment (e.g., Continuous Positive Airway Pressure \[CPAP\] in the previous 2 months prior to Screening).
Musculoskeletal disease, such as Salter-Harris fractures or clinical and/or radiographic evidence of severe hip pathology, or
+15 more criteria

Contact the study team to confirm eligibility.