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

Primary Purpose

Achondroplasia

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
TransCon CNP
Placebo for TransCon CNP
sWfI Placebo for TransCon CNP
Sponsored by
Ascendis Pharma A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Achondroplasia focused on measuring Achondroplasia, Dwarfism

Eligibility Criteria

2 Years - 10 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site
  • Ascendis Pharma Investigational Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Other

Other

Other

Other

Other

Arm Label

TransCon CNP 6 mcg

TransCon CNP 20 mcg

TransCon CNP 50 mcg

TransCon CNP 100 mcg

TransCon CNP >100 mcg

Arm Description

TransCon CNP 6 mcg CNP/kg or placebo mimicking TransCon CNP 6 mcg delivered once weekly by subcutaneous injection

TransCon CNP 20 mcg CNP/kg or placebo mimicking TransCon CNP 20 mcg delivered once weekly by subcutaneous injection

TransCon CNP 50 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection

TransCon CNP 100 mcg CNP/kg or placebo mimicking TransCon CNP 100 mcg delivered once weekly by subcutaneous injection

TransCon CNP >100 mcg CNP/kg delivered once weekly by subcutaneous injection (to be determined after completion of 100 mcg cohort)

Outcomes

Primary Outcome Measures

Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Safety and tolerability of once weekly TransCon CNP treatment or placebo
Annualized height velocity (centimeters/year) at 52 weeks
Annualized height velocity measured in centimeters over 52 weeks for TransCon CNP or placebo

Secondary Outcome Measures

Full Information

First Posted
September 9, 2019
Last Updated
September 11, 2023
Sponsor
Ascendis Pharma A/S
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1. Study Identification

Unique Protocol Identification Number
NCT04085523
Brief Title
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

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 24, 2020 (Actual)
Primary Completion Date
September 27, 2022 (Actual)
Study Completion Date
September 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ascendis Pharma A/S

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Achondroplasia
Keywords
Achondroplasia, Dwarfism

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
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
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
57 (Actual)

8. Arms, Groups, and Interventions

Arm Title
TransCon CNP 6 mcg
Arm Type
Other
Arm Description
TransCon CNP 6 mcg CNP/kg or placebo mimicking TransCon CNP 6 mcg delivered once weekly by subcutaneous injection
Arm Title
TransCon CNP 20 mcg
Arm Type
Other
Arm Description
TransCon CNP 20 mcg CNP/kg or placebo mimicking TransCon CNP 20 mcg delivered once weekly by subcutaneous injection
Arm Title
TransCon CNP 50 mcg
Arm Type
Other
Arm Description
TransCon CNP 50 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection
Arm Title
TransCon CNP 100 mcg
Arm Type
Other
Arm Description
TransCon CNP 100 mcg CNP/kg or placebo mimicking TransCon CNP 100 mcg delivered once weekly by subcutaneous injection
Arm Title
TransCon CNP >100 mcg
Arm Type
Other
Arm Description
TransCon CNP >100 mcg CNP/kg delivered once weekly by subcutaneous injection (to be determined after completion of 100 mcg cohort)
Intervention Type
Drug
Intervention Name(s)
TransCon CNP
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Placebo for TransCon CNP
Intervention Description
Weekly subcutaneously injection of placebo.
Intervention Type
Drug
Intervention Name(s)
sWfI Placebo for TransCon CNP
Intervention Description
Weekly subcutaneously injection of placebo.
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Description
Safety and tolerability of once weekly TransCon CNP treatment or placebo
Time Frame
52 weeks
Title
Annualized height velocity (centimeters/year) at 52 weeks
Description
Annualized height velocity measured in centimeters over 52 weeks for TransCon CNP or placebo
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Clinical diagnosis of ACH with genetic confirmation Age between 2 to 10 years old (inclusive) at Screening Visit Prepubertal (Stage 1 breasts for girls or testicular volume < 4ml for boys) at Screening Visit Able to stand without assistance Caregiver willing and able to administer subcutaneous injections of study drug Exclusion Criteria: 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 Have received treatment (>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit Have received any study drug or device intended to affect stature or body proportionality at any time History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Silas Gylvin, MD
Organizational Affiliation
Ascendis Pharma
Official's Role
Study Director
Facility Information:
Facility Name
Ascendis Pharma Investigational Site
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72211
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55102
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Columbia
State/Province
Missouri
ZIP/Postal Code
65212
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Buffalo
State/Province
New York
ZIP/Postal Code
14203
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53705
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Ascendis Pharma Investigational Site
City
Linz
ZIP/Postal Code
4020
Country
Austria
Facility Name
Ascendis Pharma Investigational Site
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Ascendis Pharma Investigational Site
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Ascendis Pharma Investigational Site
City
Dublin
ZIP/Postal Code
D01 YC76
Country
Ireland
Facility Name
Ascendis Pharma Investigational Site
City
Auckland
ZIP/Postal Code
1023
Country
New Zealand
Facility Name
Ascendis Pharma Investigational Site
City
Coimbra
ZIP/Postal Code
3000-602
Country
Portugal

12. IPD Sharing Statement

Learn more about this trial

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

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