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Trial record 3 of 23 for:    achondroplasia

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04085523
Recruitment Status : Not yet recruiting
First Posted : September 11, 2019
Last Update Posted : September 11, 2019
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

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 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 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 12 Months in Prepubertal Children With Achondroplasia
Estimated Study Start Date : November 2019
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021


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
The placebo for TransCon CNP contains the same excipients as TransCon CNP drug product but does not contain TransCon CNP. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

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
The placebo for TransCon CNP contains the same excipients as TransCon CNP drug product but does not contain TransCon CNP. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

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
The placebo for TransCon CNP contains the same excipients as TransCon CNP drug product but does not contain TransCon CNP. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

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
The placebo for TransCon CNP contains the same excipients as TransCon CNP drug product but does not contain TransCon CNP. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

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
The placebo for TransCon CNP contains the same excipients as TransCon CNP drug product but does not contain TransCon CNP. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.




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



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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

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


Contacts
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Contact: Medical Affairs Director +1 855-795-2467 ext 4420 accomplishtrial@ascendispharma.com

Locations
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United States, Arkansas
Ascendis Pharma Investigational Site
Little Rock, Arkansas, United States, 72211
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, Washington
Ascendis Pharma Investigational Site
Seattle, Washington, United States, 98105
Australia, Victoria
Ascendis Pharma Investigational Site
Parkville, Victoria, Australia, 3052
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
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Study Director: Will Charlton, MD Ascendis Pharma A/S
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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: September 11, 2019
Last Verified: September 2019

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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