A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

Part of paid clinical trials in Saint Paul, Minnesota.

Sponsor
Ascendis Pharma A/S
Study ID
NCT06079398
Phase
PHASE2
Status
Recruiting
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Conditions

  • Achondroplasia

Eligibility Criteria

Sex
ALL
Age
0 Years - 2 Years
Healthy Volunteers
Not accepted

Interventions

  • Navepegritide — DRUG
    Once-weekly subcutaneous injection of 100 µg/kg Navepegritide
  • Placebo for Navepegritide — DRUG
    Once-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide

Study Details

This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to \< 2 years at the time of randomization.

Key Dates

Start date
Jan 23, 2024
Status verified
Apr 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2028

Study Design

Enrollment
72 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Navepegritide
    Once weekly double-blinded treatment with SC injection of 100 µg/kg of Navepegritide for 52 weeks
  • Placebo Comparator: Placebo for Navepegritide
    Once weekly double-blinded treatment with SC injection of 100 µg/kg of Placebo for Navepegritide for 52 weeks

Primary Outcome Measure

To evaluate the safety and tolerability of Navepegritide [ Time Frame: 52 weeks ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Ascendis Investigational SiteSaint PaulMinnesota55102-
Ascendis Investigational SiteHoustonTexas77030-
Ascendis Investigational SiteMadisonWisconsin53705-

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Ascendis Investigational Site· Saint Paul, MNAscendis Investigational Site· Houston, TXAscendis Investigational Site· Madison, WI

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