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

NCT05246033 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2023-11-02

No results posted yet for this study

Summary

Purpose of the study:

The main purpose of this study is to determine the safety and evaluate the effect of a once weekly dose of TransCon CNP in prepubertal children with achondroplasia in China.

Study Treatments:

TransCon CNP is an investigational (new) drug, which means that it is currently being tested, and therefore is considered experimental. TransCon CNP is designed to provide a sustained exposure of active CNP by subcutaneous (under the skin) injection once weekly.

The Randomized Period of this study is a double-blinded and placebo-controlled. "Placebo-controlled" means that some participants will receive injections that don't contain any TransCon CNP (placebo injection - no active ingredient). "Double-blinded" means that neither the participant nor the study doctor will know which treatment the participant will be receiving, except in an emergency.

After completion of the Randomized Period the trial participant may be invited to take part of the Open-Label Period of this study.

"Open-label" means that all participants will receive injections that contain TransCon CNP; regardless of which treatment (TransCon CNP or placebo) was assigned during the 52 weeks (1 year) Randomized Period/blinded treatment period. It also means that both the participant and the study doctor will know which treatment, and which dose the participant receives.

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

  • Vibeke Breinholt · Ascendis Pharma

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
2022-01-05
Primary Completion
2023-03-15
Completion
2024-03-31
FDA Drug
Yes

Countries

  • China

Study Locations

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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 NCT05246033 on ClinicalTrials.gov