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OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

NCT ID: NCT05139810

Last Updated: 2025-03-06

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

91 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-03

Study Completion Date

2023-11-09

Brief Summary

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The purpose of this study was 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).

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Detailed Description

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This was a Phase 3, multi-center, double-blind, randomized, placebo-controlled study of donidalorsen in 91 participants. Participants were randomly assigned 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 were randomized in a 3:1 ratio to receive donidalorsen or matching-placebo. The study included an up to 8-week Screening Period, a 24-week Treatment Period, and an up to 13-week Post-treatment Period.

Conditions

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Hereditary Angioedema

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Pooled Placebo

Participants with hereditary angioedema type I/type II (HAE-1/HAE-2) received placebo subcutaneously (SC) either every 4 weeks (Weeks 1, 5, 9, 13, 17, and 21) or 8 weeks (Weeks 1, 9, and 17)

Group Type PLACEBO_COMPARATOR

Donidalorsen

Intervention Type DRUG

Donidalorsen was administered by SC injection.

Cohort A: Donidalorsen 80 mg

Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, 13, 17, and 21.

Group Type EXPERIMENTAL

Donidalorsen

Intervention Type DRUG

Donidalorsen was administered by SC injection.

Cohort B: Donidalorsen 80 mg

Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 8 weeks at Weeks 1, 9, and 17.

Group Type EXPERIMENTAL

Placebo

Intervention Type DRUG

Donidalorsen-matching placebo was administered by SC injection.

Interventions

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Donidalorsen

Donidalorsen was administered by SC injection.

Intervention Type DRUG

Placebo

Donidalorsen-matching placebo was administered by SC injection.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Participants, or their legally appointed and authorized representatives, must provide written and signed informed consent form (ICF)/assent
2. Participants must be aged ≥ 12 years at the time of informed consent and, as applicable, assent
3. Participants must have a documented diagnosis of hereditary angioedema type 1 (HAE-1)/hereditary angioedema type 2 (HAE-2)
4. Participants must:

1. Experience a minimum of 2 HAE attacks (confirmed by the Investigator) during the Screening Period
2. Be willing to complete the participant reported outcomes (PRO) assessments throughout the study
5. Participants must have access to, and the ability to use acute medication(s) to treat angioedema attacks

Exclusion Criteria

1. Concurrent diagnosis of any other type of recurrent angioedema, including acquired, idiopathic angioedema or HAE with normal C1-INH (also known as HAE Type III)
2. Any clinically-significant abnormalities in screening laboratory values that would render a participant unsuitable for inclusion in the study
3. Treatment with another investigational drug or biological agent within 1 month or 5 half-lives, whichever is longer, of Screening
4. Participated in a prior ISIS 721744 study
5. Exposure to any of the following medications:

1. Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption within 4 weeks prior to Screening
2. Chronic prophylaxis with Takhzyro, Haegarda, Cinryze and Ruconest or Orladeyo within 5 half-lives prior to Screening
3. Oligonucleotides (including small interfering ribonucleic acid \[siRNA\]) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received. This exclusion does not apply to vaccines
6. Recent history (3 years) of, or current drug or alcohol abuse
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Ionis Investigative Site

Scottsdale, Arizona, United States

Site Status

Ionis Investigative Site

San Diego, California, United States

Site Status

Ionis Investigative Site

Santa Monica, California, United States

Site Status

Ionis Investigative Site

Walnut Creek, California, United States

Site Status

Ionis Investigative Site

Colorado Springs, Colorado, United States

Site Status

Ionis Investigative Site

Tampa, Florida, United States

Site Status

Ionis Investigative Site

Kansas City, Kansas, United States

Site Status

Ionis Investigative Site

Boston, Massachusetts, United States

Site Status

Ionis Investigative Site

Ann Arbor, Michigan, United States

Site Status

Ionis Investigative Site

St Louis, Missouri, United States

Site Status

Ionis Investigative Site

Columbus, Ohio, United States

Site Status

Ionis Investigative Site

Toledo, Ohio, United States

Site Status

Ionis Investigative Site

Hershey, Pennsylvania, United States

Site Status

Ionis Investigative Site

Dallas, Texas, United States

Site Status

Ionis Investigative Site

Murray, Utah, United States

Site Status

Ionis Investigative Site

Brussels, , Belgium

Site Status

Ionis Investigative Site

Brussels, , Belgium

Site Status

Ionis Investigative Site

Edegem, , Belgium

Site Status

Ionis Investigative Site

Sofia, , Bulgaria

Site Status

Ionis Investigative Site

Sofia, , Bulgaria

Site Status

Ionis Investigative Site

Ottawa, Ontario, Canada

Site Status

Ionis Investigative Site

Edmonton, , Canada

Site Status

Ionis Investigative Site

Odense, , Denmark

Site Status

Ionis Investigative Site

La Tronche, , France

Site Status

Ionis Investigative Site

Marseille, , France

Site Status

Ionis Investigative Site

Paris, , France

Site Status

Ionis Investigative Site

Berlin, , Germany

Site Status

Ionis Investigative Site

Frankfurt, , Germany

Site Status

Ionis Investigative Site

Munich, , Germany

Site Status

Ionis Investigative Site

Ashkelon, , Israel

Site Status

Ionis Investigative Site

Haifa, , Israel

Site Status

Ionis Investigative Site

Tel Aviv, , Israel

Site Status

Ionis Investigative Site

Catania, , Italy

Site Status

Ionis Investigative Site

Milan, , Italy

Site Status

Ionis Investigative Site

Napoli, , Italy

Site Status

Ionis Investigative Site

Padua, , Italy

Site Status

Ionis Investigative Site

Palermo, , Italy

Site Status

Ionis Investigative Site

Amsterdam, , Netherlands

Site Status

Ionis Investigative Site

Groningen, , Netherlands

Site Status

Ionis Investigative Site

Krakow, , Poland

Site Status

Ionis Investigative Site

Barcelona, , Spain

Site Status

Ionis Investigative Site

Barcelona, , Spain

Site Status

Ionis Investigative Site

Madrid, , Spain

Site Status

Ionis Investigative Site

Seville, , Spain

Site Status

Ionis Investigative Site

Valencia, , Spain

Site Status

Ionis Investigative Site

Ankara, , Turkey (Türkiye)

Site Status

Ionis Investigative Site

Istanbul, , Turkey (Türkiye)

Site Status

Ionis Investigative Site

Izmir, , Turkey (Türkiye)

Site Status

Ionis Investigative Site

Birmingham, , United Kingdom

Site Status

Ionis Investigative Site

Bristol, , United Kingdom

Site Status

Ionis Investigative Site

London, , United Kingdom

Site Status

Countries

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United States Belgium Bulgaria Canada Denmark France Germany Israel Italy Netherlands Poland Spain Turkey (Türkiye) United Kingdom

References

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Riedl MA, Tachdjian R, Lumry WR, Craig T, Karakaya G, Gelincik A, Stobiecki M, Jacobs JS, Gokmen NM, Reshef A, Gompels MM, Manning ME, Bordone L, Newman KB, Treadwell S, Wang S, Yarlas A, Cohn DM; OASIS-HAE Team. Efficacy and Safety of Donidalorsen for Hereditary Angioedema. N Engl J Med. 2024 Jul 4;391(1):21-31. doi: 10.1056/NEJMoa2402478. Epub 2024 May 31.

Reference Type RESULT
PMID: 38819395 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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2021-002571-19

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ISIS 721744-CS5

Identifier Type: -

Identifier Source: org_study_id

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