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NCT05598320 (TransCon CNP in Achondroplasia): BVCT ex-ante prediction vs. reported outcome

On 29 Aug 2024, BioinvestGPT's BVCT predicted technical success and commercial partial success for this trial. The 16 Sep 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially maybe sufficient (weaker efficacy yet better adherence with once-weekly administration).

Clinical Trial

NCTID
NCT05598320
Acronym
ApproaCH
Drug Name
TransCon CNP
Drug MoA
long-lasting c-type natriuretic peptide
Drug Modality
peptide prodrug
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Achondroplasia
Human Patients
84
Sponsor
Ascendis Pharma
Phase
Phase 2 Phase 3

Prediction of , index 660

Prospective Prediction
Prediction Index
660
Prediction Date
Commercial Prediction
partial SUCCESS (commercially maybe sufficient yet moderate clinical benefit in AGV inferior to vosoritide due to suboptimal dosage)
Technical Prediction
SUCCESS (statistically significant clinical benefit in AGV against placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
18 days in advance
Readout Data Interpretation
once-weekly TransCon CNP achieved an AGV with LS mean treatment difference 1.49 cm/year; which is lower than 1.57cm/year achieved by the once-daily SoC vosoritide.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially maybe sufficient (weaker efficacy yet better adherence with once-weekly administration)
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

Official title
A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children With Achondroplasia Followed by an Open Label Extension Period
Phase
Phase 2, Phase 3
Status
Completed
Enrollment
84
Lead sponsor
Ascendis Pharma Growth Disorders A/S
Conditions
Achondroplasia
Interventions
DRUG: Navepegritide (TransCon CNP); DRUG: Placebo for Navepegritide (TransCon CNP)
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2023-03-03
Primary completion
2024-08-09
Completion
2025-08-13
Results first posted
2026-07-14
Primary outcomes
  • Annualized Growth Velocity (AGV) at Week 52 (At Week 52)

NCT05598320 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials