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NCT05127226 (ION582 in Angelman Syndrome): BVCT ex-ante prediction vs. reported outcome

On 3 Jan 2024, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. The 22 Jul 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially insufficient.

Clinical Trial

NCTID
NCT05127226
Acronym
HALOS
Drug Name
ION582
Drug MoA
intrathecal anti-UBE3A-AS ASO
Drug Modality
small interfering RNA (siRNA) antisense oligonucleotide
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Angelman Syndrome
Human Patients
51
Sponsor
Ionis Pharmaceuticals
Phase
Phase 1 Phase 2

Prediction of , index 1404

Prospective Prediction
Prediction Index
1404
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient clinical benefit non-superior non-inferior to GTX102)
Technical Prediction
SUCCESS (statistically significant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
201 days in advance
Readout Data Interpretation
97% patients have improvements in SAS-CGI-C endpoint and consistent improvements in multiple functions; advance to pivotal trial alone by Ionis.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially insufficient
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
71
Lead sponsor
Ionis Pharmaceuticals, Inc.
Conditions
Angelman Syndrome
Interventions
DRUG: ION582
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2021-12-22
Primary completion
2029-03
Completion
2029-03
Primary outcomes
  • To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters). (Part 1: Up to Week 45; Part 2: Up to Week 81)

NCT05127226 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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