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Statistical Review

Statistical Review. Boris Zaslavsky, Ph.D., Dr. Sc. FDA/CBER/OBE/DB. Study Design . Cross-over with Two Periods and Two Sequences. Subjects were randomized to either C1INH-nf or Placebo in Period 1. Treatment was then switched in the second period.

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Statistical Review

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  1. Statistical Review Boris Zaslavsky, Ph.D., Dr. Sc. FDA/CBER/OBE/DB

  2. Study Design • Cross-over with Two Periods and Two Sequences. • Subjects were randomized to either C1INH-nf or Placebo in Period 1. Treatment was then switched in the second period. • This enabled use of each subject as his/her own control, thereby controlling the variability.

  3. Primary Efficacy Endpoint The number of attacks of angioedema during each treatment phase (normalized for the number of days the subject participated in this phase)

  4. Primary Endpoint Analysis: The crossoveranalysis evaluating the number of attacks was based on a Poisson distribution and used the GEE method as implemented in the SAS statistical procedure PROC GENMOD. Statistical analysis for crossoverstudy design was performed with effects for treatment, period, and subject within treatment

  5. Assumptions for the Analysisof Crossover Design • Responses from different subjects are assumed to be independent • Responses within subjects are assumed to be correlated

  6. Variables in the Analysis • Response Variable: The total number of attacks in each period divided by the number of days the subject was in that period. • Independent Variables: Treatment, Sequence (Placebo/C1INH-nf vs. C1INH-nf/Placebo), and Period.

  7. Treatment by Period: No of Attacks/Days

  8. Treatment by Period as Analyzed: No of Attacks/Days

  9. Results • The difference in the number of attacks during treatment with Cinryze and treatment with Placebo - statistically significant (p < 0.001) • Sequence (Placebo/C1INH-nf vs. C1INH-nf/Placebo) – non-significant • Period – non-significant

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