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An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

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: NCT04307381
Recruitment Status : Active, not recruiting
First Posted : March 13, 2020
Last Update Posted : February 7, 2024
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety and efficacy of extended dosing of donidalorsen administered subcutaneously (SC), with alternative dosing and/or dose frequency with donidalorsen in participants with hereditary angioedema (HAE).

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Drug: Donidalorsen Phase 2

Detailed Description:

This is an open-label extension study of donidalorsen in up to 24 participants with HAE. The length of participation in the study is approximately 68 weeks, which includes an up to 4-week qualification period, a 52-week treatment period, and a 12-week post-treatment period. Following the Week 53 treatment period visit, participants will receive donidalorsen in an extended treatment period for up to an additional 156 weeks. Participants taking part in the extended treatment period will enter the 12-week post-treatment period after completion of, or early termination from, the extended treatment period.

This study was extended to allow participants to receive donidalorsen for an additional 156 weeks following the initial 53-week treatment period.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study of ISIS 721744 in Patients With Hereditary Angioedema
Actual Study Start Date : April 1, 2020
Estimated Primary Completion Date : April 2025
Estimated Study Completion Date : April 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Donidalorsen
Participants will be administered donidalorsen SC for up to 53 weeks. Participants will also be administered donidalorsen in the extended treatment period for an additional 156 weeks, up to week 209.
 Drug: Donidalorsen
Donidalorsen administered SC
Other Names:
  • ISIS 721744
  • IONIS-PKK-LRx


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity [ Time Frame: Up to Week 221 ]

Secondary Outcome Measures :
  1. The Time-normalized HAE Attacks (per Month) by Treatment [ Time Frame: Up to Week 221 ]
  2. Plasma Prekallikrein (PKK) Levels [ Time Frame: Up to Week 221 ]
  3. Consumption of On-demand Medications [ Time Frame: Up to Week 221 ]
  4. Angioedema Quality of Life (AE-QoL) Questionnaire Score [ Time Frame: Up to Week 221 ]
    The AE-QoL was developed to measure health-related quality of life (HRQoL) impairment in participants with recurrent angioedema. The AE-QoL is a self-administered questionnaire that can be completed in less than 5 minutes. It comprises 17 items across 4 domains: functioning, fatigue/mood, fears/shame, and food. Responses use a 5-point Likert scale ranging from 'never' to 'very often.' Global and domain scores range from 0 to 100, with higher scores indicating greater impairment


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Satisfactory completion of ISIS 721744-CS2 (index study) through Week 17 with an acceptable safety and tolerability profile, per Sponsor and Investigator judgement
  2. Able and willing to participate in a 64-week study
  3. Females must be non-pregnant, non-lactating and either surgically sterile or post-menopausal
  4. Males must be surgically sterile or abstinent* or if engaged in sexual relations with a female of child-bearing potential, participant is utilizing an acceptable contraceptive method
  5. Participants must have access to, and the ability to use, ≥ 1 acute medication(s) (e.g., plasma-derived or recombinant C1- inhibitor (C1-INH) concentrate or a bradykinin-2 [BK-2] antagonist) to treat angioedema attacks

Exclusion Criteria:

1. Have any new condition or worsening of an existing condition or change or anticipated change in medication, which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study

Contacts and Locations
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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): NCT04307381


Locations
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United States, Arizona
Ionis Investigative Site
Scottsdale, Arizona, United States, 85251
United States, California
Ionis Investigative Site
Santa Monica, California, United States, 90404
United States, Minnesota
Ionis Investigative Site
Plymouth, Minnesota, United States, 55446
United States, Ohio
Ionis Investigative Site
Cincinnati, Ohio, United States, 45231
United States, Pennsylvania
Ionis Investigative Site
Hershey, Pennsylvania, United States, 17033
United States, Texas
Ionis Investigative Site
Dallas, Texas, United States, 75231
Netherlands
Ionis Investigative Site
Amsterdam, Netherlands, 1105 AZ
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
More Information
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04307381    
Other Study ID Numbers: ISIS 721744-CS3
2020-000197-14 ( EudraCT Number )
First Posted: March 13, 2020    Key Record Dates
Last Update Posted: February 7, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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.:
Hereditary Angioedema
HAE
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
 Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes