Clinical trial NCT04085523: A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
Official title: ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Achondroplasia
Keywords: Achondroplasia
Dwarfism
Lead sponsor: Ascendis Pharma A/S
Sponsor class: INDUSTRY
First posted: 2019-09-11
Enrollment: 57
Sex: ALL
Minimum age (years): 2
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: There are 5 cohorts enrolling approximately 60 subjects who will be randomized to receive either TransCon CNP or Placebo in a 3:1 ratio
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

1. Clinical diagnosis of ACH with genetic confirmation
2. Age between 2 to 10 years old (inclusive) at Screening Visit
3. Prepubertal (Stage 1 breasts for girls or testicular volume \< 4ml for boys) at Screening Visit
4. Able to stand without assistance
5. Caregiver willing and able to administer subcutaneous injections of study drug

Exclusion Criteria:

1. Clinically significant findings at Screening that:

   * are expected to require surgical intervention during participation in the trial or
   * are musculoskeletal in nature, such as Salter-Harris fractures and severe hip pain or
   * otherwise are considered by investigator or Medical Monitor/Medical Expert to make a participant unfit to receive study drug or undergo trial related procedures
2. Have received treatment (\>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time
3. Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
4. Have received any study drug or device intended to affect stature or body proportionality at any time
5. History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones

Primary outcomes:
measure: Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment
timeFrame: 52 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Ascendis Pharma Investigational Site
state: Arkansas
city: Aurora
country: United States
facility: Ascendis Pharma Investigational Site
state: Colorado
city: Saint Paul
country: United States
facility: Ascendis Pharma Investigational Site
state: Minnesota
city: Columbia
country: United States
facility: Ascendis Pharma Investigational Site
state: Missouri
city: Buffalo
country: United States
facility: Ascendis Pharma Investigational Site
state: New York
city: Houston
country: United States
facility: Ascendis Pharma Investigational Site
state: Texas
city: Seattle
country: United States
facility: Ascendis Pharma Investigational Site
state: Washington
city: Madison
country: United States
facility: Ascendis Pharma Investigational Site
state: Wisconsin
city: Parkville
country: Australia
facility: Ascendis Pharma Investigational Site
state: Victoria
city: Linz
country: Austria
facility: Ascendis Pharma Investigational Site
city: Copenhagen
country: Denmark
facility: Ascendis Pharma Investigational Site
city: Berlin
country: Germany
facility: Ascendis Pharma Investigational Site
city: Dublin
country: Ireland
facility: Ascendis Pharma Investigational Site
city: Auckland
country: New Zealand
facility: Ascendis Pharma Investigational Site
city: Coimbra
country: Portugal
facility: Ascendis Pharma Investigational Site
Study officials:
affiliation: Ascendis Pharma, Inc.
name: Aimee D. Shu, MD
role: STUDY_DIRECTOR
References:
citation: Savarirayan R, Hoernschemeyer DG, Ljungberg M, Zarate YA, Bacino CA, Bober MB, Legare JM, Hogler W, Quattrin T, Abuzzahab MJ, Hofman PL, White KK, Ma NS, Schnabel D, Sousa SB, Mao M, Smith A, Chakraborty M, Giwa A, Winding B, Volck B, Shu AD, McDonnell C. Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial. EClinicalMedicine. 2023 Oct 2;65:102258. doi: 10.1016/j.eclinm.2023.102258. eCollection 2023 Nov.