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

Achondroplasia Clinical Trial

— 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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05929807
• Other study ID #: ASND0039
• Status: Enrolling by invitation
• Phase: Phase 2/Phase 3
• Start date: June 21, 2023
• Est. completion date: March 2039

Study information

• Verified date: April 2024
• Source: Ascendis Pharma A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 140
• Est. completion date: March 2039
• Est. primary completion date: January 2039
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 15 Years

Eligibility

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.

Related Conditions & MeSH terms

• Achondroplasia

Intervention

Drug:
• TransCon CNP
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.

Locations

Country	Name	City	State
Australia	Ascendis Pharma Investigational Site	Parkville	Victoria
Austria	Ascendis Investigational Site	Linz
Denmark	Ascendis Pharma Investigational Site	Copenhagen
Germany	Ascendis Investigational Site	Berlin
Ireland	Ascendis Pharma Investigational Site	Dublin
New Zealand	Ascendis Investigational Site	Auckland
United States	Ascendis Investigational Site	Aurora	Colorado
United States	Ascendis Pharma Investigational Site	Buffalo	New York
United States	Ascendis Pharma Investigational Site	Columbia Falls	Montana
United States	Ascendis Investigational Site	Houston	Texas
United States	Ascendis Pharma Investigational Site	Little Rock	Arkansas
United States	Ascendis Pharma Investigational Site	Madison	Wisconsin
United States	Ascendis Investigational Site	Saint Paul	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma Growth Disorders A/S

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Denmark,  Germany,  Ireland,  New Zealand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability	Incidence of Treatment-Emergent Adverse Events	Through trial completion, an average of 10 years
Primary	Height Z-scores	Number of standard deviations	Through trial completion, an average of 10 years
Secondary	Annualized Growth Velocity	cm per year	Through trial completion, an average of 10 years