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

Sponsor

Ascendis Pharma Growth Disorders A/S (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05929807

Collaborator

(none)

140

Enrollment

Location

Arm

188.3

Anticipated Duration (Months)

0.7

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Achondroplasia	Drug: TransCon CNP	Phase 2/Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

140 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

Patients will roll over from previous TransCON CNP clinical trialsPatients will roll over from previous TransCON CNP clinical trials

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Jun 21, 2023

Anticipated Primary Completion Date :

Jan 1, 2039

Anticipated Study Completion Date :

Mar 1, 2039

Arms and Interventions

Arm	Intervention/Treatment
Experimental: TransCon CNP 100 mcg 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. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

Arm

Intervention/Treatment

Experimental: TransCon CNP 100 mcg

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. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

Outcome Measures

Primary Outcome Measures

Safety and Tolerability [Through trial completion, an average of 10 years]
Incidence of Treatment-Emergent Adverse Events
Height Z-scores [Through trial completion, an average of 10 years]
Number of standard deviations

Secondary Outcome Measures

Annualized Growth Velocity [Through trial completion, an average of 10 years]
cm per year

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 15 Years

Sexes Eligible for Study:

All

Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Ascendis Pharma Investigational Site	Parkville	Victoria	Australia	3052

Sponsors and Collaborators

Ascendis Pharma Growth Disorders A/S

Investigators

Study Director: Adebola Giwa, MD, Ascendis Pharma

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Ascendis Pharma Growth Disorders A/S

ClinicalTrials.gov Identifier:

NCT05929807

Other Study ID Numbers:

ASND0039

First Posted:

Jul 3, 2023

Last Update Posted:

Jul 3, 2023

Last Verified:

Jun 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Ascendis Pharma Growth Disorders A/S

Achondroplasia

Dwarfism

Additional relevant MeSH terms:

Achondroplasia

Study Results

No Results Posted as of Jul 3, 2023