Trial record 2 of 2 for:
IONIS HTTRx | Huntington Disease
Study in Huntington's Disease Patients Who Participated in Prior Investigational Studies of ISIS 443139
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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. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03342053 |
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Recruitment Status :
Active, not recruiting
First Posted : November 14, 2017
Last Update Posted : November 16, 2018
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Sponsor:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
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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) |
| Actual 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 |
| Actual Study Start Date : | November 27, 2017 |
| Estimated Primary Completion Date : | December 2019 |
| Estimated Study Completion Date : | December 2019 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Huntington disease
MedlinePlus related topics:
Huntington's Disease
| Arm | Intervention/treatment |
|---|---|
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Experimental: IONIS HTTRx Cohort A
IONIS HTTRx is administered intrathecally for 14 months at dose level A.
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Drug: IONIS HTTRx
Intrathecal injection
Other Name: ISIS 443139 |
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Experimental: IONIS HTTRx Cohort B
IONIS HTTRx is administered intrathecally for 14 months at dose level B.
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Drug: IONIS HTTRx
Intrathecal injection
Other Name: ISIS 443139 |
Primary Outcome Measures :
- 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:
- CSF huntingtin protein concentration (fM) [ Time Frame: 74 weeks ]
- Ventricular volume (mL) [ Time Frame: 74 weeks ]
- 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, Older Adult) |
| 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
Locations
| Canada, British Columbia | |
| University of British Columbia | |
| Vancouver, British Columbia, Canada, V6T 2B5 | |
| Germany | |
| Charite University Berlin | |
| Berlin, Germany, 10117 | |
| Ruhr-University of Bochum | |
| Bochum, Germany, 44791 | |
| Ulm University Hospital | |
| Ulm, Germany, 89081 | |
| United Kingdom | |
| University Hospitals Birmingham | |
| Birmingham, United Kingdom, B15 2TH | |
| Cambridge University Hospital | |
| Cambridge, United Kingdom, CB2 0PY | |
| University Hospital of Wales | |
| Cardiff, United Kingdom, CF14 4XN | |
| University College London | |
| London, United Kingdom, WC1N 3BG | |
| University of Manchester, St. Mary's Hospital | |
| 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 16, 2018 |
| Last Verified: | November 2018 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes | |
| Studies a U.S. FDA-regulated Device Product: | No | |
| Product Manufactured in and Exported from the U.S.: | Yes | |
Keywords provided by Ionis Pharmaceuticals, Inc.:
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Huntington's Disease HTTRx |
Additional relevant MeSH terms:
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Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases |
Genetic Diseases, Inborn Cognition Disorders Neurocognitive Disorders Mental Disorders Dementia Chorea Dyskinesias |