SL1009-02

8 evidence topics · 3 sources

Objective

Survival comparison

“SL1009-02 compared survival of subjects who received DCA in SL1009-01 to an external control derived from a published 2012 natural history study of PDCD by DeBrosse et al.” (opens the source at this quote in a new tab)

Location and study date

Noncontemporaneous cohorts

“temporal separation (average 18 years)” (opens the source at this quote in a new tab)

Study design

Eligibility criteria

No evidence found.

Treatment

Study outcomes

Rationale for using survival as the primary endpoint

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

No evidence found.

Description of analysis sets

FDA assessment of imputation in the external-control analysis

“imputation protocol was inconsistently applied introducing bias against the external control group.” (opens the source at this quote in a new tab)

Additional analyses described in the subsequent resubmission announcement

“additional survival analyses to support the application” (opens the source at this quote in a new tab)
“Saol conducted further analyses of existing data” (opens the source at this quote in a new tab)

Results

Participant disposition

No evidence found.

Baseline characteristics

FDA assessment of severity imbalance

“structural brain abnormalities most commonly associated with severe disease favored the DCA-treated group.” (opens the source at this quote in a new tab)

Efficacy results

Sponsor-reported survival result

“DCA treatment improved survival (p=0.027) compared to appropriately matched controls.” (opens the source at this quote in a new tab)

FDA assessment of the proposed comparison

“cannot be used to assess the treatment effect of DCA on survival” (opens the source at this quote in a new tab)

Quantitative results of the additional resubmission analyses

No evidence found.

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

No evidence found.

Study limitations

Summary

This comparison reused the SL1009-01 treatment cohort and was not an independent randomized trial. The FDA identified inconsistent imputation, disease-severity imbalance, and an average temporal separation of 18 years between cohorts in its 2025 assessment. These limitations prevent interpreting the sponsor-reported p=0.027 as an established causal survival benefit. The subsequent resubmission announcement describes further analyses, including FDA-requested survival analyses, but this report contains no quantitative results from those additional analyses.