ClinicalTrials.gov
ClinicalTrials.gov Menu

Study in Huntington's Disease Patients Who Participated in Prior Investigational Studies of ISIS 443139

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.
ClinicalTrials.gov Identifier: NCT03342053
Recruitment Status : Not yet recruiting
First Posted : November 14, 2017
Last Update Posted : November 27, 2017
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Brief Summary:
This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of IONIS-HTTRx administered intrathecally to adult patients with Huntington's Disease.

Condition or disease Intervention/treatment Phase
Huntington's Disease Drug: IONIS HTTRx Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 46 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Huntington's Disease Patients Who Participated in Prior Investigational Studies of ISIS 443139
Estimated Study Start Date : November 2017
Estimated Primary Completion Date : September 30, 2019
Estimated Study Completion Date : September 30, 2019

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: IONIS HTTRx Cohort A
IONIS HTTRx is administered intrathecally for 14 months at dose level A.
Drug: IONIS HTTRx
Intrathecal injection
Other Name: ISIS 443139
Experimental: IONIS HTTRx Cohort B
IONIS HTTRx is administered intrathecally for 14 months at dose level B.
Drug: IONIS HTTRx
Intrathecal injection
Other Name: ISIS 443139



Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events [safety and tolerability] [ Time Frame: Participants will be followed for the duration of the study; an expected 74 weeks ]
    The number of participants with adverse events


Other Outcome Measures:
  1. CSF huntingtin protein concentration (fM) [ Time Frame: 74 weeks ]
  2. Ventricular volume (mL) [ Time Frame: 74 weeks ]
  3. Huntington's disease cognitive assessment battery composite score (unitless) [ Time Frame: 74 weeks ]


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:   25 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Must have completed dosing in ISIS 443139-CS1

Key Exclusion Criteria:

  • Any new condition or worsening of existing condition that could make the patient unsuitable for participation or interfere with the patient participating in and/or completing the study

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): NCT03342053


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

Locations
Canada, British Columbia
University of British Columbia Completed
Vancouver, British Columbia, Canada, V6T 2B5
Germany
Charite University Berlin Completed
Berlin, Germany, 10117
Ruhr-University of Bochum Completed
Bochum, Germany, 44791
Ulm University Hospital Completed
Ulm, Germany, 89081
United Kingdom
University Hospitals Birmingham Completed
Birmingham, United Kingdom, B15 2TH
Cambridge University Hospital Completed
Cambridge, United Kingdom, CB2 0PY
University Hospital of Wales Completed
Cardiff, United Kingdom, CF14 4XN
University College London Completed
London, United Kingdom, WC1N 3BG
University of Manchester, St. Mary's Hospital Completed
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.

Additional Information:
Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03342053     History of Changes
Other Study ID Numbers: ISIS 443139-CS2
First Posted: November 14, 2017    Key Record Dates
Last Update Posted: November 27, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Keywords provided by Ionis Pharmaceuticals, Inc.:
Huntington's Disease
HTTRx

Additional relevant MeSH terms:
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders