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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease

This study is currently recruiting participants.
Verified June 2017 by Ionis Pharmaceuticals, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03186989
First Posted: June 14, 2017
Last Update Posted: October 2, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
  Purpose
The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS-MAPTRx in patients with Mild Alzheimer's Disease

Condition Intervention Phase
Mild Alzheimer's Disease Drug: IONIS MAPTRx Other: Placebo Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Other
Official Title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ISIS 814907 in Patients With Mild Alzheimer's Disease

Resource links provided by NLM:


Further study details as provided by Ionis Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Incidence and severity of adverse events that are related to treatment with IONIS MAPTRx [ Time Frame: Up to 253 Days ]
    The safety and tolerability of ascending dose-levels of multiple IT bolus administrations of IONIS MAPTRx will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with IONIS MAPTRx


Secondary Outcome Measures:
  • Pharmacokinetics (PK) after ascending dose-levels of multiple IT bolus administrations of IONIS MAPTRx (trough concentration) [ Time Frame: Up to 253 days ]
    The cerebrospinal fluid (CSF) trough concentrations of IONIS MAPTRx will be assessed after ascending dose-levels of multiple IT bolus administrations of IONIS MAPTRx

  • Pharmacokinetics (PK) after ascending dose-levels of multiple IT bolus administrations of IONIS MAPTRx (maximum observed drug concentration or Cmax) [ Time Frame: Up to 253 days ]
    The Plasma pharmacokinetics (maximum observed drug concentration or Cmax)) of IONIS MAPTRx will be assessed after ascending dose-levels of multiple IT bolus administrations of IONIS MAPTRx

  • Pharmacokinetics after ascending dose-levels of multiple IT bolus administrations on IONIS MAPTRx (time taken to reach maximal concentration or Tmax) [ Time Frame: Up to 253 days ]
    The plasma pharmacokinetics (time taken to reach maximal concentration or Tmax) of IONIS MAPTRx will be assessed following single and multiple dose IT administration

  • Pharmacokinetics after ascending dose-levels of multiple IT bolus administrations on IONIS MAPTRx (Plasma terminal elimination half-life (t1/2λz) [ Time Frame: Up to 253 days ]
    The plasma pharmacokinetics (Plasma terminal elimination half-life (t1/2λz) of IONIS-MAPTRx will be assessed following single and multiple dose IT administration

  • Pharmacokinetics after ascending dose-levels of multiple IT bolus administrations on IONIS MAPTRx (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the IT administration (AUCt) [ Time Frame: Up to 253 days ]
    The plasma pharmacokinetics (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the IT administration (AUCt) of IONIS MAPTRx will be assessed following single and multiple dose IT administration


Estimated Enrollment: 44
Actual Study Start Date: June 5, 2017
Estimated Study Completion Date: February 29, 2020
Estimated Primary Completion Date: January 31, 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: IONIS-MAPTRx
IONIS MAPTRx (Study Drug)
Drug: IONIS MAPTRx
IONIS MAPTRx is administered intrathecally at 4 week intervals over the course of a 13 week treatment period for dose levels A, B, C, and D
Other Name: ISIS 814907
Placebo Comparator: Placebo
Artificial CSF
Other: Placebo
A placebo is administered intrathecally at 4 week intervals over the course of 13 weeks

Detailed Description:
Allocation: RandomizedEndpoint Classification: Safety Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator) Primary Purpose: Treatment
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   50 Years to 74 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males or females aged 50-74 years, inclusive, at the time of informed consent
  • Diagnosed with mild Alzheimers disease, including CSF biomarkers consistent with this diagnosis
  • Body Mass Index BMI ≥ 18 and ≤ 35 kg/m2 and total body weight > 50 kg (110 lbs)
  • Able and willing to meet all study requirements, including toleration for MRI scans, blood draws and lumbar punctures, travel to Study Center and participation in all procedures and measurements at study visits
  • Have a trial partner who is reliable, competent and at least 18 years of age, is willing to accompany the patient to select trial visits and to be available to the Study Center by phone if needed
  • Reside within 4 hours travel of the Study Center

Exclusion Criteria:

  • Treatment with another Study Drug, biological agent, or device within one-month of Screening or 5 half-lives of investigational agent, whichever is longer
  • Clinically significant medical condition which would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study
  • Use of a disallowed CNS-active or antipsychotic medication within 4 weeks prior to Screening punctures
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03186989


Contacts
Contact: Ionis Pharmaceuticals 800-679-4747 patients@ionisph.com

Locations
Canada
Montreal Neurological Hospital Recruiting
Montréal, Canada
Finland
Clinical Research Services Turku CRST Recruiting
Turku, Finland
Germany
Charite - Universitaetsmedizin Berlin Campus Mitte Not yet recruiting
Berlin, Germany
St Josef Hospital Recruiting
Bochum, Germany
MVZ Mittweida Gbr Recruiting
Mittweida, Germany
Universittsklinikum Ulm Recruiting
Ulm, Germany
Netherlands
Faculty Medical Sciences UMCG Neurology Recruiting
Groningen, Netherlands
Sweden
Goteborgs Universitet- Sahlgrenska Akademin Recruiting
Göteborg, Sweden
Karolinska University Hospital Huddinge Recruiting
Stockholm, Sweden
United Kingdom
Royal Liverpool University Hospital Recruiting
Liverpool, United Kingdom
University College London Hospitals NHS Foundation Trust Recruiting
London, United Kingdom
Sheffield Institute for Translational Neuroscience (SITraN) Recruiting
Sheffield, United Kingdom
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
  More Information

Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03186989     History of Changes
Other Study ID Numbers: ISIS 814907-CS1
First Submitted: June 6, 2017
First Posted: June 14, 2017
Last Update Posted: October 2, 2017
Last Verified: June 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Ionis Pharmaceuticals, Inc.:
Mild Alzheimer's Disease
ISIS 814907

Additional relevant MeSH terms:
Alzheimer Disease
Dementia
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Tauopathies
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders