Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease
NCT ID: NCT02519036
Last Updated: 2019-05-31
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
46 participants
INTERVENTIONAL
2015-08-06
2017-11-08
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ISIS 443139 10 mg
Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 10 mg
ISIS 443139, 10 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 30 mg
Participants received ISIS 443139, 30 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 30 mg
ISIS 443139, 30 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 60 mg
Participants received ISIS 443139, 60 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 60 mg
ISIS 443139, 60 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 90 mg
Participants received ISIS 443139, 90 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 90 mg
ISIS 443139, 90 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 120 mg
Participants received ISIS 443139, 120 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 120 mg
ISIS 443139, 120 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
Placebo
Participants received placebo, by intrathecal injection, on Study Days 1, 29, 57, and 85.
Placebo
Placebo was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
Interventions
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ISIS 443139 10 mg
ISIS 443139, 10 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 30 mg
ISIS 443139, 30 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 60 mg
ISIS 443139, 60 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 90 mg
ISIS 443139, 90 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
ISIS 443139 120 mg
ISIS 443139, 120 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
Placebo
Placebo was administered by intrathecal injection, on Study Days 1, 29, 57, and 85.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Male or female, aged 25 to 65 years, inclusive, at the time of informed consent
* Able and willing to meet all study requirements, including 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 participant to select trial visits and to be available to the Study Center by phone if needed
* Able to tolerate MRI scans, blood draws and lumbar punctures
* Reside within 4 hours travel of the Study Center
Exclusion Criteria
* Recent treatment with another investigational drug, biological agent, or device
* Prior treatment with an antisense oligonucleotide \[including small interfering ribonucleic acid (siRNA)\]
* Any history of gene therapy or cell transplantation or any other experimental brain surgery
* Presence of an implanted shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter
* History of post-lumbar-puncture headache of moderate or severe intensity and/or blood patch
* Malignancy within 5 years of Screening, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
* Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study
25 Years
65 Years
ALL
No
Sponsors
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Ionis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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University of British Columbia
Vancouver, British Columbia, Canada
Charite University Berlin
Berlin, , Germany
Ruhr-University of Bochum
Bochum, , Germany
Ulm University Hospital
Ulm, , Germany
University Hospitals Birmingham
Birmingham, , United Kingdom
Cambridge University Hospital
Cambridge, , United Kingdom
University Hospital of Wales
Cardiff, , United Kingdom
University College London
London, , United Kingdom
University of Manchester, St. Mary's Hospital
Manchester, , United Kingdom
Countries
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References
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Rodrigues FB, Ferreira JJ, Wild EJ. Huntington's Disease Clinical Trials Corner: June 2019. J Huntingtons Dis. 2019;8(3):363-371. doi: 10.3233/JHD-199003.
Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM; Phase 1-2a IONIS-HTTRx Study Site Teams. Targeting Huntingtin Expression in Patients with Huntington's Disease. N Engl J Med. 2019 Jun 13;380(24):2307-2316. doi: 10.1056/NEJMoa1900907. Epub 2019 May 6.
Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: February 2018. J Huntingtons Dis. 2018;7(1):89-98. doi: 10.3233/JHD-189001.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Related Links
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Ionis Pharmaceuticals, Inc. home page
Other Identifiers
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2015-000381-66
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ISIS 443139-CS1
Identifier Type: -
Identifier Source: org_study_id
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