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NCT05228145 (NGN101 in CLN5 Batten Disease): 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 18 Nov 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically probably insignificant and commercially probably insufficient.

Clinical Trial

NCTID
NCT05228145
Acronym
CLN5-200
Drug Name
NGN101
Drug MoA
AAV9-delivered CLN5 trans gene
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
CLN5 Batten Disease
Human Patients
6
Sponsor
Neurogene
Phase
Phase 1 Phase 2

Prediction of , index 1709

Prospective Prediction
Prediction Index
1709
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in UBDRS or visual acuity)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak clinical benefit in UBDRS or visual acuity)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
110 days in advance
Readout Data Interpretation
NGN101’s further development has been discontinued
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically probably insignificant 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 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
6
Lead sponsor
Neurogene Inc.
Conditions
Neuronal Ceroid Lipofuscinosis CLN5
Interventions
GENETIC: NGN-101
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2022-01-31
Primary completion
2028-11
Completion
2028-11
Primary outcomes
  • Incidence of Treatment Emergent Adverse Events (TEAEs) (5 years (multiple visits))
  • Incidence of Serious Adverse Events (SAEs) (5 years (multiple visits))
  • Incidence of clinical laboratory abnormalities (5 years (multiple visits))
  • Incidence of new nerve conduction study (NCS) abnormalities (5 years (multiple visits))
  • Incidence of new physical and neurologic exam abnormalities (5 years (multiple visits))

NCT05228145 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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