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A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia (AttaCH)

Primary Purpose

Achondroplasia

Status
Enrolling by invitation
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
TransCon CNP
Sponsored by
Ascendis Pharma Growth Disorders 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

3 Years - 15 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Written, signed informed consent of the parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC). For participants who are below the age of consent, a written assent will be obtained in accordance with applicable requirements as required by IRB/HREC/IEC. Upon reaching the legal age of consent, depending on applicable requirements, these participants will be asked to give their own written consent. Participants with achondroplasia who have completed a clinical trial with TransCon CNP. Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of TransCon CNP and to follow the protocol. Considered eligible based on the safety evaluations performed for evaluating stopping/holding rule criteria during the prior TransCon CNP clinical trial. Exclusion Criteria: Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris[hydroxymethyl]aminomethane, succinate, and methoxy polyethylene glycol [mPEG]). Have received any dose of prescription medications, investigational medicinal product (other than TransCon CNP). Sexually active female participants and female partners of male participants of childbearing potential not using a highly effective form of contraceptive (including oral, injectable, or implantable contraception, or intrauterine device (IUD)) for the entire trial period and for 90 days post end of the trial. Participants with serum 25-hydroxy-vitamin D (25OHD) levels of <50 nmol/L (<20 ng/mL) at Visit 1 must be on treatment regimen of Vitamin D supplementation. Any disease or condition that, in the opinion of the investigator, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, complications or manifestations, or medications that might impact safety or be considered confounding.

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

TransCon CNP 100 mcg

Arm Description

TransCon CNP 100 mcg delivered once weekly by subcutaneous injection

Outcomes

Primary Outcome Measures

Safety and Tolerability
Incidence of Treatment-Emergent Adverse Events
Height Z-scores
Number of standard deviations

Secondary Outcome Measures

Annualized Growth Velocity
cm per year

Full Information

First Posted
June 6, 2023
Last Updated
August 17, 2023
Sponsor
Ascendis Pharma Growth Disorders A/S
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1. Study Identification

Unique Protocol Identification Number
NCT05929807
Brief Title
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
Acronym
AttaCH
Official Title
A Phase 2, Multicenter, Long-Term, Open Label Extension Trial Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children and Adolescents With Achondroplasia
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
June 21, 2023 (Actual)
Primary Completion Date
January 2039 (Anticipated)
Study Completion Date
March 2039 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ascendis Pharma Growth Disorders 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
TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial. Participants who complete a prior TransCon CNP trial and meet all eligibility criteria will be invited to continue into the long-term open label extension trial to receive 100 µg CNP/kg/week of TransCon CNP. Trial treatment will be completed when the participant reaches 16 years of age for females and 18 years of age for males and have femur and tibial epiphyseal closure. TransCon CNP treatment will continue if femur and tibial epiphyseal closure is not confirmed at the age of 16 years for females, and 18 years for males. Treatment with TransCon CNP will be completed once femur and tibial epiphyseal closure is confirmed by radiographic imaging. The trial duration is individual for each trial participant. Visits will occur every 12-14 weeks throughout the trial.

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, Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Patients will roll over from previous TransCON CNP clinical trials
Masking
None (Open Label)
Allocation
N/A
Enrollment
140 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
TransCon CNP 100 mcg
Arm Type
Experimental
Arm Description
TransCon CNP 100 mcg delivered once weekly by subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
TransCon CNP
Intervention Description
TransCon CNP drug product is a lyophilized powder in a single-use vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.
Primary Outcome Measure Information:
Title
Safety and Tolerability
Description
Incidence of Treatment-Emergent Adverse Events
Time Frame
Through trial completion, an average of 10 years
Title
Height Z-scores
Description
Number of standard deviations
Time Frame
Through trial completion, an average of 10 years
Secondary Outcome Measure Information:
Title
Annualized Growth Velocity
Description
cm per year
Time Frame
Through trial completion, an average of 10 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Written, signed informed consent of the parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC). For participants who are below the age of consent, a written assent will be obtained in accordance with applicable requirements as required by IRB/HREC/IEC. Upon reaching the legal age of consent, depending on applicable requirements, these participants will be asked to give their own written consent. Participants with achondroplasia who have completed a clinical trial with TransCon CNP. Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of TransCon CNP and to follow the protocol. Considered eligible based on the safety evaluations performed for evaluating stopping/holding rule criteria during the prior TransCon CNP clinical trial. Exclusion Criteria: Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris[hydroxymethyl]aminomethane, succinate, and methoxy polyethylene glycol [mPEG]). Have received any dose of prescription medications, investigational medicinal product (other than TransCon CNP). Sexually active female participants and female partners of male participants of childbearing potential not using a highly effective form of contraceptive (including oral, injectable, or implantable contraception, or intrauterine device (IUD)) for the entire trial period and for 90 days post end of the trial. Participants with serum 25-hydroxy-vitamin D (25OHD) levels of <50 nmol/L (<20 ng/mL) at Visit 1 must be on treatment regimen of Vitamin D supplementation. Any disease or condition that, in the opinion of the investigator, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, complications or manifestations, or medications that might impact safety or be considered confounding.
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
72202
Country
United States
Facility Name
Ascendis Pharma Investigational Site
City
Columbia Falls
State/Province
Montana
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
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
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Ascendis Pharma Investigational Site
City
Dublin
ZIP/Postal Code
D01 YC76
Country
Ireland

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

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