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NCT06049082 (Nebulization in Alpha 1-Antitrypsin Deficiency): BVCT ex-ante prediction vs. reported outcome

On 31 Jul 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 12 Dec 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant (AAT concentration) and commercially TBD (wait for FEV1 data).

Clinical Trial

NCTID
NCT06049082
Acronym
Serpentine-1
Drug Name
Nebulization (KB408)
Drug MoA
inhaled replication-defective HSV1-delivered SERPINA1
Drug Modality
inhaled formulation
Drug Class
First-In-Class
Therapeutic Area
Cardiovascular Diseases and Nutritional and Metabolic Diseases
Indication
Alpha 1-Antitrypsin Deficiency (AATD)
Human Patients
12
Sponsor
Krystal Biotech
Phase
Phase 1

Prediction of , index 1706

Prospective Prediction
Prediction Index
1706
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in FEV1)
Technical Prediction
partial SUCCESS (statistically significant clinical benefit in lung AAT concentration) partial FAILURE (statistically insignificant clinical benefit in lung NE level)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
134 days in advance
Readout Data Interpretation
KB408 achieved 5.3-9.7 micromolar mean total AAT protein; inferior to the 10.8 micromolar mean total AAT protein achieved by WVE006
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant (AAT concentration) and commercially TBD (wait for FEV1 data)
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase 1 Study of Inhaled KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
Phase
Phase 1
Status
Recruiting
Enrollment
15
Lead sponsor
Krystal Biotech, Inc.
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
DRUG: KB408 (Nebulization)
Design
Non randomized · Single group · None
Primary purpose
Treatment
Start
2024-02-15
Primary completion
2025-12
Completion
2026-12
Primary outcomes
  • To evaluate safety and tolerability of KB408 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in vital signs, ECG, spirometry, and clinical laboratory test results (2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b))

NCT06049082 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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