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Trial record 1 of 1 for:    NCT04085523
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A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

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ClinicalTrials.gov Identifier: NCT04085523
Recruitment Status : Enrolling by invitation
First Posted : September 11, 2019
Last Update Posted : November 3, 2020
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

Tracking Information
First Submitted Date  ICMJE September 9, 2019
First Posted Date  ICMJE September 11, 2019
Last Update Posted Date November 3, 2020
Actual Study Start Date  ICMJE June 24, 2020
Estimated Primary Completion Date June 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 9, 2019)
  • Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [ Time Frame: 52 weeks ]
    Safety and tolerability of once weekly TransCon CNP treatment or placebo
  • Annualized height velocity (centimeters/year) at 52 weeks [ Time Frame: 52 weeks ]
    Annualized height velocity measured in centimeters over 52 weeks for TransCon CNP or placebo
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
Official Title  ICMJE 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 12 Months in Prepubertal Children With Achondroplasia
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
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
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Achondroplasia
Intervention  ICMJE
  • 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.
  • Drug: Placebo for TransCon CNP
    Weekly subcutaneously injection of placebo.
Study Arms  ICMJE
  • TransCon CNP 6 mcg
    TransCon CNP 6 mcg CNP/kg or placebo mimicking TransCon CNP 6 mcg delivered once weekly by subcutaneous injection
    Interventions:
    • Drug: TransCon CNP
    • Drug: Placebo for TransCon CNP
  • TransCon CNP 20 mcg
    TransCon CNP 20 mcg CNP/kg or placebo mimicking TransCon CNP 20 mcg delivered once weekly by subcutaneous injection
    Interventions:
    • Drug: TransCon CNP
    • Drug: Placebo for TransCon CNP
  • TransCon CNP 50 mcg
    TransCon CNP 50 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection
    Interventions:
    • Drug: TransCon CNP
    • Drug: Placebo for TransCon CNP
  • TransCon CNP 100 mcg
    TransCon CNP 100 mcg CNP/kg or placebo mimicking TransCon CNP 100 mcg delivered once weekly by subcutaneous injection
    Interventions:
    • Drug: TransCon CNP
    • Drug: Placebo for TransCon CNP
  • TransCon CNP >100 mcg
    TransCon CNP >100 mcg CNP/kg delivered once weekly by subcutaneous injection (to be determined after completion of 100 mcg cohort)
    Interventions:
    • Drug: TransCon CNP
    • Drug: Placebo for TransCon CNP
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: September 9, 2019)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2026
Estimated Primary Completion Date June 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 10 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04085523
Other Study ID Numbers  ICMJE TCC-201
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ascendis Pharma A/S
Study Sponsor  ICMJE Ascendis Pharma A/S
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Will Charlton, MD Ascendis Pharma A/S
PRS Account Ascendis Pharma A/S
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP