Expect These Alzheimer Phase 3 Trial Results in 2016
While you’re waiting for other stuff to happen to Martin Shkreli, anticipate these results from interventional phase 3 trials in Alzheimer disease in 2016.*
February 2016
- Safety and Efficacy Study Evaluating TRx0237 in Subjects With Mild to Moderate Alzheimer’s Disease: Tau Therapeutics will complete its international, 15-month, phase 3 study of 2 doses of the anti-tau TRx0237 in 800+ people with mild-moderate AD. Primary efficacy measures are the ADAS-cog11 and the ADCS-CGIC. Secondary efficacy measures include the ADCS-ADL23 and the MMSE. Biomarkers to support any disease-modifying effects are temporal-lobe glucose uptake via FDG-PET and whole-brain volume via MRI. In this phase 3 study and another (see below), TRx0237 (75 or 125 mg bid) will be delivered in a new “stable reduced form” (LMTX®), to address any delivery issues in the phase 2 trial.
- The Study of Nasal Insulin in the Fight Against Forgetfulness (SNIFF**): Sponsored by the University of Southern California with collaborators Wake Forest and the National Institute on Aging, this placebo-controlled, 18-month, phase 2/3 study is assessing the potential cognitive benefits of intranasal insulin (Humulin® R U-100) in 240 people with amnestic MCI or mild AD. Primary outcome measures include the ADAS-cog12 and daily-functional outcomes. Maybe there is a market for Afrezza® after all?
May 2016
- Safety and Efficacy Study Evaluating TRx0237 in Subjects With Mild Alzheimer’s Disease: This is Tau’s companion phase 3 of TRx0237 (aka LMTX®). However, an alternative dosage of 100 mg bid is being given. Expected enrollment is 700 people with mild AD, and trial duration is 18 months. As in the 2-dose study, primary efficacy measures include the ADAS-cog11 and the ADCS-CGIC, along with the biomarker assessment of FDG-PET.
October 2016
- Study of Lu AE58054 in Patients With Mild-Moderate Alzheimer’s Disease Treated With Donepezil (STARSHINE): Lundbeck and Otsuka will complete an international, 24-week, phase 3 trial of their 5-HT6-receptor antagonist (Lu AE58054, idalopirdine) in 930 people with mild-moderate AD. All enrollees are taking 10 mg of donepezil. The primary outcome measure is the ADAS-cog. Lu AE58054 is generally considered a symptomatic therapy (like donepezil), but anticipate Lundbeck/Otsuka to make claims (outright or veiled) about disease modification–providing that cognition data are positive.
December 2016
- A Study to Evaluate Albumin and Immunoglobulin in Alzheimer’s Disease (AMBAR): Despite Baxter’s failure with IV Ig in AD, Grifols Biologicals, Inc., plowed ahead with an international, phase 2/3 trial of “short-term” plasma exchange with 20% albumin ± IV Ig in 350 people with mild-moderate AD. Sham plasma exchange has been incorporated into the trial design. The primary outcome measure at 14 months is the ADAS-cog. Secondary outcome measures include CSF biomarkers and structural changes via MRI.
AD = Alzheimer disease; ADAS-cog = Alzheimer’s Disease Assessment Scale-cognition; ADCS-ADL23= Alzheimer’s Disease Cooperative Study – Activities of Daily Living; ADCS-CGIC = Alzheimer’s Disease Cooperative Study – Clinical Global Impression of Change; CSF = cerebrospinal fluid; FDG-PET = 18F-fluorodeoxyglucose positron emission tomography; MCI = mild cognitive impairment; MMSE = Mini-Mental State Examination; MRI = magnetic resonance imaging.
* Based on my guided search of clinicaltrials.gov. So warning: the list may not be complete.
** Ugh.
A native East Tennessean, Barbara Martin is a formerly practicing, board-certified neurologist who received her BS (psychology, summa cum laude) and MD from Duke University before completing her postgraduate training (internship, residency, fellowship) at the Hospital of the University of Pennsylvania in Philadelphia. She has worked in academia, private practice, medical publishing, drug market research, and continuing medical education (CME). For the last 3 years, she has worked in a freelance capacity as a medical writer, analyst, and consultant. Follow Dr. Barbara Martin on Google + and Twitter.
