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NCT05676034 (AMX0035 in Wolfram Syndrome): BVCT ex-ante prediction vs. reported outcome

On 13 Dec 2023, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. The 17 Oct 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically probably significant and commercially probably sufficient.

Clinical Trial

NCTID
NCT05676034
Acronym
HELIOS
Drug Name
AMX0035
Drug MoA
sodium phenylbutyrate + taurursodiol
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases / Genetic Diseases
Indication
Wolfram Syndrome
Human Patients
12
Sponsor
Amylyx Pharmaceuticals
Phase
Phase 2

Prediction of , index 1374

Prospective Prediction
Prediction Index
1374
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient clinical benefit)
Technical Prediction
SUCCESS (statistically significant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
309 days in advance
Readout Data Interpretation
AMX0035 achieved +36.7 min*ng/mL C-Peptide Response as the primary efficacy endpoint; and phase 2a safe and 100% participants meeting prespecified responding criteria (24 week -0.26% HbA1c; +7.6% target glucose range; +0.05 vision acuity; 62.5% CGIC and 75% PGIC).
Press Release
Additional Readout Data
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically probably significant and commercially probably sufficient
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Phase
Phase 2
Status
Active not recruiting
Enrollment
12
Lead sponsor
Amylyx Pharmaceuticals Inc.
Conditions
Wolfram Syndrome
Interventions
DRUG: AMX0035
Design
Single group · None
Primary purpose
Treatment
Start
2023-03-03
Primary completion
2024-07-23
Completion
2028-05-31
Primary outcomes
  • To evaluate the effect of AMX0035 on residual beta-cell function by monitoring C-peptide levels during a 0-240 minutes mixed-meal tolerance test (MMTT) (24 weeks)
  • To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome (208 weeks)

NCT05676034 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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