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NCT05579691 (CTI-1601 in Friedreich Ataxia): BVCT ex-ante prediction vs. reported outcome

On 7 Jan 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. 2 readouts followed: 12 Feb 2024, True Negative (TN); 16 Dec 2024, True Negative (TN).

Clinical Trial

NCTID
NCT05579691
Drug Name
CTI-1601
Drug MoA
TAT-delivered FXN recombinant fusion protein
Drug Modality
fusion protein biologic
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Friedreich Ataxia
Human Patients
28
Sponsor
Larimar Therapeutics
Phase
Phase 2

Prediction of , index 1409

Prospective Prediction
Prediction Index
1409
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit inferior to omaveloxolone)
Technical Prediction
partial SUCCESS (statistically significant yet weak clinical benefit due to TAT delivery)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
36 days in advance
Readout Data Interpretation
insignificant FXN levels
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant yet weak and commercially insufficient
Prediction Classification
True Negative (TN)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
344 days in advance
Readout Data Interpretation
nomlabofusp achieved early trends yet very weak improvements in mFARS
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically maybe significant yet weak (mFARS) and commercially probably insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 in Adult Subjects With Friedreich's Ataxia
Phase
Phase 2
Status
Completed
Enrollment
28
Lead sponsor
Larimar Therapeutics, Inc.
Conditions
Friedreich Ataxia
Interventions
BIOLOGICAL: CTI-1601; OTHER: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2022-09-21
Primary completion
2023-12-04
Completion
2023-12-04
Primary outcomes
  • Number of Participants with Treatment Emergent Adverse Events (Through study completion, an average of 93 days)

NCT05579691 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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