Mintun · The New England journal of medicine 2021 · phase 2 randomized controlled trial · n=257

Donanemab in Early Alzheimer's Disease.

Cited 1756 times in the scientific literature.

Level 2 - randomized trial

Individual randomized controlled trial

PubMed 33720637 · doi:10.1056/NEJMoa2100708 · record verified 2026-08-29

What was done

A multicenter phase 2 randomized controlled trial evaluated donanemab (an antibody targeting deposited amyloid-beta) versus placebo in 257 patients with early symptomatic Alzheimer's disease confirmed by tau and amyloid PET deposition. Participants were randomized 1:1 to receive intravenous donanemab (700 mg for 3 doses, then 1400 mg every 4 weeks; n = 131) or placebo (n = 126) for up to 72 weeks. The primary outcome was the change from baseline to 76 weeks on the Integrated Alzheimer's Disease Rating Scale (iADRS; range 0 to 144). Secondary clinical outcomes included CDR-SB, ADAS-Cog13, ADCS-iADL, and MMSE, along with PET amyloid and tau burden.

What was found

Baseline iADRS was 106 in both arms. At 76 weeks, iADRS declined by 6.86 points with donanemab versus 10.06 points with placebo (difference 3.20; 95% CI, 0.12 to 6.27; P = 0.04). Most secondary clinical outcomes showed no substantial difference between groups. Amyloid plaque reduction was 85.06 centiloids greater and global tau load reduction was 0.01 greater with donanemab than placebo at 76 weeks. Amyloid-related cerebral edema or effusions (mostly asymptomatic) occurred in the donanemab group.

Why it matters

This study shows that antibody-mediated clearance of deposited amyloid-beta can produce modest slowing of composite cognitive-functional decline over 76 weeks in early Alzheimer's disease.

Limits

The sample size was modest (n = 257) and follow-up was limited to 76 weeks. Most secondary clinical outcomes did not show statistically significant differences, the primary endpoint confidence interval was wide with a lower bound near zero, and treatment was associated with amyloid-related cerebral edema or effusions.

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