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Hereditary Angioedema| donidalorsen: Cohort A

.1058.

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

This is a Phase 3, multi-center, double-blind, randomized, placebo-controlled study of donidalorsenin up to 84 participants. Participants will be randomized in a 2:1 ratio to Cohort A (donidalorsenor placebo every 4 weeks) or Cohort B (donidalorsenor placebo every 8 weeks), respectively. Within each Cohort, participants will be randomized in a 3:1 ratio to receive donidalorsenor 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.

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donidalorsen: Cohort A

Purpose of Study

The purpose of this study is to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsenon the quality and pattern of HAE attacks and their impact on quality of life (QoL).

Eligibility

Age:
12 +
Sex:
Female Icon + Male Icon

Key Inclusion / Exclusion Criteria

Inclusion Criteria

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

Participants must:

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

Exclusion Criteria

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)
Any clinically-significant abnormalities in screening laboratory values that would render a participant unsuitable for inclusion in the study
Treatment with another investigational drug or biological agent within 1 month or 5 half-lives, whichever is longer, of Screening
Participated in a prior ISIS 721744 study

Exposure to any of the following medications:

Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption within 4 weeks prior to Screening
Chronic prophylaxis with Takhzyro, Haegarda, Cinryze or Orladeyo within 5 half-lives prior to Screening
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

Conditions:

Hereditary Angioedema

Status:

Recruiting

Phase:

Phase 3

Clinical Trials ID

NCT05139810

Read Detailed Summary:

Start Date / End Date:

12 2021 - 10 2023

Intervention:

donidalorsen: Cohort A

Enrollment:

84

Full Details:

https://clinicaltrials.gov/ct2/show/NCT05139810