A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Ascendis Pharma Growth Disorders A/S
Study ID
NCT05929807
Phase
PHASE2/PHASE3
Status
Enrolling By Invitation

Conditions

  • Achondroplasia

Eligibility Criteria

Sex
ALL
Age
3 Years - 15 Years
Healthy Volunteers
Not accepted

Interventions

  • TransCon CNP — DRUG
    TransCon CNP drug product is a lyophilized powder in a single-use vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

Study Details

TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial. Participants who complete a prior TransCon CNP trial and meet all eligibility criteria will be invited to continue into the long-term open label extension trial to receive 100 µg CNP/kg/week of TransCon CNP. Trial treatment will be completed when the participant reaches 16 years of age for females and 18 years of age for males and have femur and tibial epiphyseal closure. TransCon CNP treatment will continue if femur and tibial epiphyseal closure is not confirmed at the age of 16 years for females, and 18 years for males. Treatment with TransCon CNP will be completed once femur and tibial epiphyseal closure is confirmed by radiographic imaging. The trial duration is individual for each trial participant. Visits will occur every 12-14 weeks throughout the trial.

Key Dates

Start date
Jun 21, 2023
Status verified
Oct 2025
Primary completion
Jan 31, 2039
Completion
Mar 31, 2039

Study Design

Enrollment
140 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: TransCon CNP 100 mcg
    TransCon CNP 100 mcg delivered once weekly by subcutaneous injection

Primary Outcome Measure

Safety and Tolerability [ Time Frame: Through trial completion, an average of 10 years ]

Locations (8)

FacilityCityStateZIPSite coordinators
Ascendis Pharma Investigational SiteLittle RockArkansas72202-
Ascendis Investigational SiteAuroraColorado80045-
Ascendis Investigational SiteWilmingtonDelaware19803-
Ascendis Investigational SiteSaint PaulMinnesota55102-
Ascendis Pharma Investigational SiteColumbia FallsMontana65212-
Ascendis Pharma Investigational SiteBuffaloNew York14203-
Ascendis Investigational SiteHoustonTexas77030-
Ascendis Pharma Investigational SiteMadisonWisconsin53705-

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