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

NCT04085523 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 57

Last updated 2025-05-22

Study results available
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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.

Conditions

Interventions

DRUG

TransCon CNP

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.

DRUG

Placebo for TransCon CNP

Weekly subcutaneously injection of placebo.

Sponsors & Collaborators

  • Ascendis Pharma A/S

    lead INDUSTRY

Principal Investigators

  • Aimee D. Shu, MD · Ascendis Pharma, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-06-24
Primary Completion
2022-09-27
Completion
2024-10-01
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Austria
  • Denmark
  • Germany
  • Ireland
  • New Zealand
  • Portugal

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04085523 on ClinicalTrials.gov