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Table 2 Summary of efficacy results for primary analysis, sensitivity analyses, and secondary endpoints (responder analyses) for the masitinib 4.5 mg/kg/day and titrated masitinib 6.0 mg/kg/day parallel groups (FAS population)

From: Masitinib for mild-to-moderate Alzheimer’s disease: results from a randomized, placebo-controlled, phase 3, clinical trial

M4.5 parallel group

 

PBO (N=176)

M4.5 (N=182)

Between group difference

P value

 

Primary analysis

Change from baseline at week 24 (±SE)

LSM (97.5% CI)

 
 

ADAS-Cog (primary endpoint)

0.69 (±0.54)

−1.46 (±0.51)

−2.15 [−3.48, −0.81]

<0.001

 

ADCS-ADL (primary endpoint)

−0.81 (±0.79)

1.01 (±0.76)

1.82 [−0.15, 3.79]

0.038

 

Sensitivity analysis

Change from baseline at week 24 (±SE)

LSM (95% CI)

 
 

ADAS-Cog (JTR analysis)

0.85 (±0.54)

−1.04 (±0.52)

−1.89 [−3.06, −0.72]

0.002

 

ADCS-ADL (JTR analysis)

−0.90 (±0.79)

0.81 (±0.76)

1.71 [−0.01, 3.43]

0.051

 

ADAS-Cog (MI analysis)

0.88 (±0.63)

−1.16 (±0.65)

−2.04 [−3.41, −0.67]

0.004

 

ADCS-ADL (MI analysis)

−0.95 (±0.93)

0.77 (±0.90)

1.72 [−0.26, 3.70]

0.089

 

ADAS-Cog (ITT analysis) a

0.67 (±0.53)

−1.41 (±0.50)

−2.08 [−3.22, −0.94]

<0.001

 

ADCS-ADL (ITT analysis) a

−0.06 (±0.83)

1.14 (±0.80)

1.20 [−0.60, 3.00]

0.192

 

Responder analyses (secondary endpoints)

Response rate, n (%)

Odds Ratio (95% CI)

 
 

Clinical response rate

23 (13.1%)

41 (22.5%)

1.96 [1.11, 3.46]

0.020

 

CIBIC-plus improvement

36 (20.5%)

47 (25.8%)

1.71 [1.02, 2.85]

0.040

 

CIBIC-plus worsening

37 (21.0%)

23 (12.6%)

0.64 [0.36, 1.14]

0.127

tM6.0 parallel group

 

tPBO (N=91)

tM6.0 (N=186)

Between group difference

P value

 

Primary analysis

Change from baseline at week 24 (±SE)

LSM (97.5% CI)

 
 

ADAS-Cog (primary endpoint)

0.25 (±0.60)

−0.18 (±0.47)

−0.43 [−1.81, 0.95]

0.483

 

ADCS-ADL (primary endpoint)

0.37 (±0.81)

0.57 (±0.62)

0.20 [−1.64, 2.04]

0.807

  1. Unless otherwise stated, this table summarizes data from patients in the masitinib 4.5 mg/kg/day parallel group full analysis dataset (PBO n=176; M4.5 n=182). All assessments were prespecified in the protocol or Statistical Analysis Plan prior to unblinding. M4.5 masitinib treatment-arm from the masitinib 4.5 mg/kg/day parallel group, PBO placebo treatment-arm from the masitinib 4.5 mg/kg/day parallel group, tM6.0 masitinib treatment-arm from the titrated 6.0 mg/kg/day parallel group, tPBO placebo treatment-arm from the titrated 6.0 mg/kg/day parallel group, LSM least-squares mean, JTR jump-to-reference, MI multiple imputation, ITT intention-to-treat population, ADAS-cog Alzheimer’s Disease Assessment Scale. For ADAS-cog (scores range from 0 to 70) a positive change from baseline indicates worsening dementia and a negative between group difference (masitinib minus placebo) favors masitinib. ADCS-ADL Alzheimer’s Disease Cooperative Study Activities of Daily Living Inventory scale. For ADCS-ADL (scores range from 0 to 78) a negative change from baseline indicates worsening function and a positive between group difference (masitinib minus placebo) favors masitinib. CIBIC-plus: Clinician’s Interview-Based Impression of Change plus Caregiver Input (scores of 1-3 correspond with improvement, scores of 5-7 correspond with worsening compared with baseline). Clinical response defined as decrease from baseline at week 24 in ADAS-cog of ≥4, without deterioration in ADCS-ADL or worsening in the CIBIC-plus scale. SE: Standard error. CI: Confidence Interval at 97.5% for primary endpoint and at 95% for sensitivity analyses. Sensitivity and secondary endpoint analyses on the titrated masitinib 6.0 mg/kg/day dataset were not performed because, in accordance to protocol, the primary endpoint was not met for this parallel group. a Sensitivity analysis performed on the ITT population (PBO n=185; M4.5 n=186)
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Alzheimer's Research & Therapy

ISSN: 1758-9193