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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04085523
Recruitment Status : Active, not recruiting
First Posted : September 11, 2019
Last Update Posted : December 22, 2022
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

Study Description
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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.

Condition or disease Intervention/treatment Phase
Achondroplasia Drug: TransCon CNP Drug: Placebo for TransCon CNP Drug: sWfI Placebo for TransCon CNP Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 57 participants
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
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
Actual Study Start Date : June 24, 2020
Actual Primary Completion Date : September 27, 2022
Estimated Study Completion Date : September 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
TransCon CNP 6 mcg
TransCon CNP 6 mcg CNP/kg or placebo mimicking TransCon CNP 6 mcg delivered once weekly by subcutaneous injection
 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.

Drug: sWfI Placebo for TransCon CNP
Weekly subcutaneously injection of placebo.
TransCon CNP 20 mcg
TransCon CNP 20 mcg CNP/kg or placebo mimicking TransCon CNP 20 mcg delivered once weekly by subcutaneous injection
 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.
TransCon CNP 50 mcg
TransCon CNP 50 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection
 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.
TransCon CNP 100 mcg
TransCon CNP 100 mcg CNP/kg or placebo mimicking TransCon CNP 100 mcg delivered once weekly by subcutaneous injection
 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.
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)
 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.


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [ Time Frame: 52 weeks ]
    Safety and tolerability of once weekly TransCon CNP treatment or placebo

  2. 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


Eligibility Criteria
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Ages Eligible for Study:   2 Years to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04085523


Locations
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United States, Arkansas
Ascendis Pharma Investigational Site
Little Rock, Arkansas, United States, 72211
United States, Colorado
Ascendis Pharma Investigational Site
Aurora, Colorado, United States, 80045
United States, Minnesota
Ascendis Pharma Investigational Site
Saint Paul, Minnesota, United States, 55102
United States, Missouri
Ascendis Pharma Investigational Site
Columbia, Missouri, United States, 65212
United States, New York
Ascendis Pharma Investigational Site
Buffalo, New York, United States, 14203
United States, Texas
Ascendis Pharma Investigational Site
Houston, Texas, United States, 77030
United States, Washington
Ascendis Pharma Investigational Site
Seattle, Washington, United States, 98105
United States, Wisconsin
Ascendis Pharma Investigational Site
Madison, Wisconsin, United States, 53705
Australia, Victoria
Ascendis Pharma Investigational Site
Parkville, Victoria, Australia, 3052
Austria
Ascendis Pharma Investigational Site
Linz, Austria, 4020
Denmark
Ascendis Pharma Investigational Site
Copenhagen, Denmark, 2100
Germany
Ascendis Pharma Investigational Site
Berlin, Germany, 13353
Ireland
Ascendis Pharma Investigational Site
Dublin, Ireland, D01 YC76
New Zealand
Ascendis Pharma Investigational Site
Auckland, New Zealand, 1023
Portugal
Ascendis Pharma Investigational Site
Coimbra, Portugal, 3000-602
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
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Study Director: Adebola Giwa, MD Ascendis Pharma
More Information
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Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT04085523    
Other Study ID Numbers: TCC-201
First Posted: September 11, 2019    Key Record Dates
Last Update Posted: December 22, 2022
Last Verified: December 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ascendis Pharma A/S:
Achondroplasia
Dwarfism
Additional relevant MeSH terms:
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Achondroplasia
Dwarfism
Bone Diseases, Developmental
Bone Diseases
 Musculoskeletal Diseases
Osteochondrodysplasias
Genetic Diseases, Inborn