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NCT06405633 (WVE-006 in alpha-1 antitrypsin deficiency): BVCT ex-ante prediction vs. reported outcome

On 10 Aug 2024, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. The 16 Oct 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially TBD (pending FEV1 data).

Clinical Trial

NCTID
NCT06405633
Acronym
RestorAATion-2
Drug Name
WVE-006
Drug MoA
GalNAc-conjugated ADAR-dependent mRNA single-base modifier
Drug Modality
base editing
Drug Class
First-In-Class
Therapeutic Area
Cardiovascular Diseases and Nutritional and Metabolic Diseases
Indication
alpha-1 antitrypsin deficiency (AATD)
Human Patients
24
Sponsor
Wave Life Sciences
Phase
Phase 1 Phase 2

Prediction of , index 1742

Prospective Prediction
Prediction Index
1742
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient additive clinical benefit in FEV1 superior to both fazirsiran and KB408)
Technical Prediction
SUCCESS (statistically significant additive clinical benefit in lung AAT/NE concentration)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
67 days in advance
Readout Data Interpretation
WVE-006 achieved 10.8 micromolar mean total AAT protein at day 15 (meeting the level that has been the basis for regulatory approval)
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially TBD (pending FEV1 data)
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants With AATD Pi*ZZ on WVE-006 (RestorAATion-2)
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
24
Lead sponsor
Wave Life Sciences USA, Inc.
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
DRUG: WVE-006
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2024-07-19
Primary completion
2026-09
Completion
2026-09
Primary outcomes
  • The proportion of participants with adverse events (Up to 36 Weeks)

NCT06405633 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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