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

Primary Purpose

Hereditary Angioedema

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Donidalorsen
Sponsored by
Ionis Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Angioedema focused on measuring Hereditary Angioedema, HAE

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 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

Sites / Locations

  • Ionis Investigative Site
  • Ionis Investigative Site
  • Ionis Investigative Site
  • Ionis Investigative Site
  • Ionis Investigative Site
  • Ionis Investigative Site
  • Ionis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Donidalorsen

Arm Description

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.

Outcomes

Primary Outcome Measures

Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity

Secondary Outcome Measures

The Time-normalized HAE Attacks (per Month) by Treatment
Plasma Prekallikrein (PKK) Levels
Consumption of On-demand Medications
Angioedema Quality of Life (AE-QoL) Questionnaire Score
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

Full Information

First Posted
March 11, 2020
Last Updated
March 19, 2023
Sponsor
Ionis Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04307381
Brief Title
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
Official Title
An Open-Label Extension Study of ISIS 721744 in Patients With Hereditary Angioedema
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
April 1, 2020 (Actual)
Primary Completion Date
April 2025 (Anticipated)
Study Completion Date
April 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ionis Pharmaceuticals, Inc.

4. Oversight

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
Data Monitoring Committee
No

5. Study Description

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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema
Keywords
Hereditary Angioedema, HAE

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Donidalorsen
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Donidalorsen
Other Intervention Name(s)
ISIS 721744, IONIS-PKK-LRx
Intervention Description
Donidalorsen administered SC
Primary Outcome Measure Information:
Title
Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity
Time Frame
Up to Week 221
Secondary Outcome Measure Information:
Title
The Time-normalized HAE Attacks (per Month) by Treatment
Time Frame
Up to Week 221
Title
Plasma Prekallikrein (PKK) Levels
Time Frame
Up to Week 221
Title
Consumption of On-demand Medications
Time Frame
Up to Week 221
Title
Angioedema Quality of Life (AE-QoL) Questionnaire Score
Description
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
Time Frame
Up to Week 221

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Satisfactory completion of ISIS 721744-CS2 (index study) through Week 17 with an acceptable safety and tolerability profile, per Sponsor and Investigator judgement Able and willing to participate in a 64-week study Females must be non-pregnant, non-lactating and either surgically sterile or post-menopausal 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 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
Facility Information:
Facility Name
Ionis Investigative Site
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85251
Country
United States
Facility Name
Ionis Investigative Site
City
Santa Monica
State/Province
California
ZIP/Postal Code
90404
Country
United States
Facility Name
Ionis Investigative Site
City
Plymouth
State/Province
Minnesota
ZIP/Postal Code
55446
Country
United States
Facility Name
Ionis Investigative Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45231
Country
United States
Facility Name
Ionis Investigative Site
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Ionis Investigative Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
Ionis Investigative Site
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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