A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

Ascendis Pharma

Status and phase: Completed; Phase 2

Conditions: Achondroplasia

Treatments: Drug: Placebo for TransCon CNP; Drug: TransCon CNP

Study type: Interventional

Funder types: Industry

Identifiers

NCT04085523

TCC-201

Details and patient eligibility

About

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.

Enrollment: 57 patients

Sex: All

Ages: 2 to 10 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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57 participants in 6 patient groups, including a placebo group

TransCon CNP 6 mcg

Experimental group

Description:

TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection.

Treatment:

Drug: TransCon CNP

TransCon CNP 20 mcg

Experimental group

Description:

TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection.

Treatment:

Drug: TransCon CNP

TransCon CNP 50 mcg

Experimental group

Description:

TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection.

Treatment:

Drug: TransCon CNP

TransCon CNP 100 mcg

Experimental group

Description:

TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection.

Treatment:

Drug: TransCon CNP

Placebo

Placebo Comparator group

Description:

Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection.

Treatment:

Drug: Placebo for TransCon CNP

Open-Label Extension Period: TransCon CNP

Experimental group

Description:

Participants who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection.

Treatment:

Drug: TransCon CNP