Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05929807 | A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia | PHASE2 | ENROLLING BY_INVITATION | 140 | — | — | Jun 21, 2023 | Mar 1, 2039 | Oct 27, 2025 | 17 | United States, Australia +8 |
| NCT05598320 | A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia | PHASE2 | COMPLETED | 84 | — | — | Mar 3, 2023 | Aug 13, 2025 | Jan 15, 2026 | 10 | United States, Australia +5 |
| NCT04085523 | A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia | PHASE2 | COMPLETED | 57 | — | — | Jun 24, 2020 | Oct 1, 2024 | May 22, 2025 | 15 | United States, Australia +6 |
Incidence of Treatment-Emergent Adverse Events
Number of standard deviations
cm per year
The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set.
| Arm | Type | Description |
|---|---|---|
| TransCon CNP 100 mcg | EXPERIMENTAL | TransCon CNP 100 mcg delivered once weekly by subcutaneous injection |
| TransCon CNP | EXPERIMENTAL | Once weekly double-blinded treatment with SC injection of 100 µg/kg of TransCon CNP for 52 weeks |
| Placebo for TransCon CNP | PLACEBO_COMPARATOR | Once weekly double-blinded treatment with SC injection of 100 µg/kg of Placebo for TransCon CNP for 52 weeks |
| Open-Label Extension Period: TransCon CNP | EXPERIMENTAL | Participants who completed the 52-week blinded treatment period continued into the open-label extension period and received treatment with TransCon CNP 100 µg/kg delivered once weekly by subcutaneous injection. |
| TransCon CNP 6 mcg | EXPERIMENTAL | TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection. |
| TransCon CNP 20 mcg | EXPERIMENTAL | TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection. |
| TransCon CNP 50 mcg | EXPERIMENTAL | TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection. |
| Placebo | PLACEBO_COMPARATOR | Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection. |
| Name | Type | Description |
|---|---|---|
| TransCon CNP | DRUG | 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. |
| Placebo for TransCon CNP | DRUG | Once-weekly subcutaneous injection of 100 µg/kg placebo for TransCon CNP |
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 writte...