A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension Period

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 60

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
6. Written, signed informed consent of the parent(s) or legal guardian(s) of the participant and written assent of the participant as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC)
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 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 ACH, that affects growth potential of long bones
6.History of any bone-related surgery that affects growth potential of long bones, such as orthopedic reconstructive urgery and osteotomy (Limb-lengthening with full recovery is allowed with a minimum of 12 months of bone healing. Foramen magnum decompression and laminectomy with full recovery are allowed with minimum of 6 months of bone healing. History of 8 plate epiphysiodesis is allowed, but the plates must have been removed prior to Screening with minimum 4 weeks of healing).
7.Have a form of skeletal dysplasia other than ACH or known medical conditions that result in short stature or abnormal growth.
8.History or presence of malignant disease, other than basal cell epithelioma/carcinoma or completely resected squamous skin cancer with no recurrence for 12 months per medical records
9.History or presence of the following:
•Chronic anemia (resolved iron deficiency anemia is allowed)
•Significant cardiovascular disease per the judgement of the investigator, such as congenital heart disease (uncomplicated patent ductus arteriosus and atrial or ventricular septal defect with repair are allowed), aortic insufficiency, clinically significant arrhythmias, congestive heart failure with NYHA class II and above, or other conditions that impair regulation of blood pressure or heart rate
•Condition that impacts hemodynamic stability (such as autonomic dysfunction, orthostatic intolerance)
•History of chronic renal insufficiency
•Chronic or recurrent illness that can affect hydration or volume status. This may include conditions associated with decreased nutritional intake
or increased volume loss
•Bone fracture within 6 months prior to Screening Visit (within 2 months for fracture of digits)
•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 presents undue risk from receiving study drug
10.Child has significant electrocardiogram abnormalities, including evidence of a previous myocardial infarction, left ventricular hypertrophy, flat T waves (particularly in the inferior leads) or more
than minor non-specific ST-T wave changes or:
•QRS>90 milliseconds (msec)
•QT interval corrected using Fridericia's formula (QTcF)>440 msec
•PR interval>170 msec
•Complete right or left bundle branch block
11.Requires, or anticipated to require, chronic (>4 weeks) or repeated (more than twice per year) treatment with oral corticosteroids during participation in the trial (low and mid-dose inhaled corticosteroids are allowed. High-dose inhaled corticosteroids are

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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