OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
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ClinicalTrials.gov Identifier: NCT05139810 |
Recruitment Status :
Completed
First Posted : December 1, 2021
Last Update Posted : January 3, 2024
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Sponsor:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
Tracking Information | |||||
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First Submitted Date ICMJE | November 18, 2021 | ||||
First Posted Date ICMJE | December 1, 2021 | ||||
Last Update Posted Date | January 3, 2024 | ||||
Actual Study Start Date ICMJE | December 3, 2021 | ||||
Actual Primary Completion Date | November 6, 2023 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Time-Normalized Number of Investigator-Confirmed HAE Attacks (per Month) From Week 1 to Week 25 [ Time Frame: Week 1 to Week 25 ] | ||||
Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE) | ||||
Official Title ICMJE | A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE) | ||||
Brief Summary | The purpose of this study is to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL). | ||||
Detailed Description | This is a Phase 3, multi-center, double-blind, randomized, placebo-controlled study of donidalorsen in up to 84 participants. Participants will be randomized in a 2:1 ratio to Cohort A (donidalorsen or placebo every 4 weeks) or Cohort B (donidalorsen or placebo every 8 weeks), respectively. Within each Cohort, participants will be randomized in a 3:1 ratio to receive donidalorsen or matching-placebo. The length of participation in the study is approximately 11 months, which includes an up to 8-week Screening Period, a 25-week Treatment Period, and an up to 13-week Post-treatment Period. | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 3 | ||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE | Hereditary Angioedema | ||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
91 | ||||
Original Estimated Enrollment ICMJE |
84 | ||||
Actual Study Completion Date ICMJE | November 9, 2023 | ||||
Actual Primary Completion Date | November 6, 2023 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria
Exclusion Criteria
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Sex/Gender ICMJE |
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Ages ICMJE | 12 Years and older (Child, Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Belgium, Bulgaria, Canada, Denmark, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Turkey, United Kingdom, United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT05139810 | ||||
Other Study ID Numbers ICMJE | ISIS 721744-CS5 2021-002571-19 ( EudraCT Number ) |
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Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Ionis Pharmaceuticals, Inc. | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Ionis Pharmaceuticals, Inc. | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE | Not Provided | ||||
PRS Account | Ionis Pharmaceuticals, Inc. | ||||
Verification Date | December 2023 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |