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

Achondroplasia Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04085523
• Other study ID #: TCC-201
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: June 24, 2020
• Est. completion date: September 2024

Study information

• Verified date: February 2024
• Source: Ascendis Pharma A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 57
• Est. completion date: September 2024
• Est. primary completion date: September 27, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 10 Years

Eligibility

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

Related Conditions & MeSH terms

• Achondroplasia

Intervention

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.
• Placebo for TransCon CNP
Weekly subcutaneously injection of placebo.

Locations

Country	Name	City	State
Australia	Ascendis Pharma Investigational Site	Parkville	Victoria
Austria	Ascendis Pharma Investigational Site	Linz
Denmark	Ascendis Pharma Investigational Site	Copenhagen
Germany	Ascendis Pharma Investigational Site	Berlin
Ireland	Ascendis Pharma Investigational Site	Dublin
New Zealand	Ascendis Pharma Investigational Site	Auckland
Portugal	Ascendis Pharma Investigational Site	Coimbra
United States	Ascendis Pharma Investigational Site	Aurora	Colorado
United States	Ascendis Pharma Investigational Site	Buffalo	New York
United States	Ascendis Pharma Investigational Site	Columbia	Missouri
United States	Ascendis Pharma Investigational Site	Houston	Texas
United States	Ascendis Pharma Investigational Site	Little Rock	Arkansas
United States	Ascendis Pharma Investigational Site	Madison	Wisconsin
United States	Ascendis Pharma Investigational Site	Saint Paul	Minnesota
United States	Ascendis Pharma Investigational Site	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma A/S

Countries where clinical trial is conducted

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

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set.	52 weeks