The purpose of this study was to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS-MAPTRx in patients with Mild Alzheimer's Disease.
This was a randomized, double-blind, placebo-controlled study in 46 participants, followed by an Open-Label Extension. This study consisted of two parts: Part 1: a randomized, double-blind, placebo-controlled multiple ascending dose period in participants with Mild Alzheimer's Disease, followed by Part 2: the open-label, long-term extension period.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
OTHER
Masking
DOUBLE
Enrollment
46
IONIS MAPTRx injections.
Artificial CSF injections.
Montreal Neurological Hospital
Montreal, Canada
Clinical Research Services Turku CRST
Turku, Finland
St Josef Hospital
Bochum, Germany
Part 1: Number of Participants With Adverse Events That Are Related to Treatment With ISIS 814907
An adverse event (AE) was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of medicinal (investigational) product, whether or not the AE was considered related to the medicinal (investigational) product. A TEAE was defined as any AE that starts or worsens on or after the date of first dose of study treatment. TEAEs were categorised as mild, moderate, and severe to aid in severity assessment.
Time frame: From first dose of study drug up to Week 37 in Part 1
Part 2: Number of Participants With Adverse Events That Are Related to Treatment With ISIS 814907
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of medicinal (investigational) product, whether or not the AE was considered related to the medicinal (investigational) product. A TEAE was defined as any AE that starts or worsens on or after the date of first dose of study treatment. TEAEs were categorised as mild, moderate, and severe to aid in severity assessment.
Time frame: From first dose of study drug up to Week 64 in Part 2
CSF Trough Concentration of ISIS 814907
Time frame: Pre dose on Day 85 in Part 1 and Day 337 in Part 2
Maximum Observed Drug Concentration (Cmax) of ISIS 814907 in Plasma
Time frame: Pre-dose, 0.5, 1, 2, 3, 4, and 5 hours post-intrathecal (IT) bolus injection on Day 85 in Part 1 and on Day 337 in Part 2
Time Taken to Reach Maximal Concentration (Tmax) of ISIS 814907 in Plasma
Time frame: Pre-dose, 0.5, 1, 2, 3, 4, and 5 hours post-IT bolus injection on Day 85 in Part 1 and on Day 337 in Part 2
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Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE)
Bonn, Germany
MVZ Mittweida Gbr
Mittweida, Germany
Universittsklinikum Ulm
Ulm, Germany
VU University Medical Center
Amsterdam, Netherlands
QPS Netherlands BV
Groningen, Netherlands
Minnesmottagningen
Mölndal, Sweden
Karolinska University Hospital Huddinge
Stockholm, Sweden
...and 3 more locations
Terminal Elimination Half-life (t1/2λz) of ISIS 814907 in Plasma
Time frame: Pre-dose, 0.5, 1, 2, 3, 4, and 5 hours post-IT bolus injection on Day 85 in Part 1 and on Day 337 in Part 2
Areas Under the Plasma Concentration-time Curve From Zero Time (Predose) to 24 Hours After the IT Administration (AUC0-24h) of ISIS 814907
Time frame: Pre-dose, 0.5, 1, 2, 3, 4, and 5 hours post-IT bolus injection on Day 85 in Part 1 and on Day 337 in Part 2