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Table 5: Peak VO
2
Treatment Effect Across Studies
Study
Population
Bayesian
VO
2
Estimate
Bayesian
Posterior
Probability
Primary Analysis with
Borrowing
FIX-HF-5C & FIX-HF-5
Imputation (Death = 0)
0.836
0.989
Imputation (Death = lowest peak VO
2
)
0.693
0.988
Completed Cases (No Imputation)
0.603
0.978
Pooled
FIX-HF-5C & FIX-HF-5
Completed Cases (No Imputation)
0.749
0.999
FIX-HF-5C Alone
Imputation (Death = 0)
0.799
0.960
Imputation (Death = lowest peak VO
2
)
0.611
0.957
Completed Cases (No Imputation)
0.480
0.916
FIX-HF-5 Alone
Imputation (Death = 0)
1.074
1.00
Completed Case (No Imputation)
1.080
1.00
Secondary Effectiveness Endpoints
MLWHFQ results at 24 weeks are presented in Table 3 and show that the CCM
group was statistically significantly superior over the control group (p < 0.001) in each
study.
Table 6: Change in MLWHFQ at 24 Weeks by Study
Difference (95% CI) in
MLWHFQ Total Score Between
Groups
p-value
(1-sided)
Pooled data
-10.9 (-14.6, -7.2)
< 0.001
FIX-HF-5C
-11.7 (-17.6, -5.9)
< 0.001
FIX-HF-5
Subgroup
-10.8 (-15.6, -6.1)
< 0.001
The percentage of patients improving by 1 or more NYHA class by study was
statistically significantly superior in the CCM group compared to the control group
(p < 0.001 in each study; Table 7).
Table 7: Patients Achieving ≥ 1 Class Improvement in NYHA at 24 Weeks by Study
Change in ≥ 1 Class
in NYHA Class
CCM
Control
p-value
(1-sided)
Pooled data
104/173 (60.1%)
59/169 (34.9%)
< 0.001
FIX-HF-5C
57/70 (81.4%)
32/75 (42.7%)
< 0.001
FIX-HF-5 Subgroup
47/103 (45.6%)
27/94 (28.7%)
< 0.001
In the FIX-HF-5C study, the p-value for the comparison of mean peak VO
2
at 24
weeks for CCM compared to control among observations with RER > 1.05 was
0.1100. Therefore, this secondary effectiveness endpoint was not met with FIX-HF-
5C data alone. When data were pooled from the FIX-HF-5 and FIX-HF-5C studies,
the treatment effect was estimated as 0.62 mL/kg/min with a p-value of 0.009. In
addition, the endpoint was met in the FIX-HF-5 subgroup (Table 8).