A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-01-07

Study Completion Date

2021-03-01

Brief Summary

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The purpose of this study was to evaluate the clinical efficacy, safety, and tolerability of donidalorsen in participants with hereditary angioedema (HAE) type 1 (HAE-1), HAE type 2 (HAE-2), or HAE with normal C1-inhibitor (C1-INH) and to evaluate the effect of donidalorsen on plasma prekallikrein (PKK) and other relevant biomarkers.

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Detailed Description

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This was a randomized, double-blind, placebo-controlled study in 23 participants conducted concurrently in 2 parts (Part A and Part B); participants were allocated into Part A or Part B according to type of HAE (i.e., either HAE-1/HAE-2 in Part A or HAE-nC1-INH in Part B). Part A was randomized, double-blind, and placebo-controlled; and Part B was open-label. The length of participation in the study was approximately 8 months, which included an up to 8-week screening period, a 12-week treatment period, and a 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

TRIPLE

Participants Investigators Outcome Assessors

Part A was randomized, double-blind; Part B was open-label.

Study Groups

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Part A: Placebo

Participants with hereditary angioedema type I/type II (HAE-1/HAE-2) received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo matching solution administered SC

Part A: Donidalorsen 80 mg

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

Group Type EXPERIMENTAL

Donidalorsen

Intervention Type DRUG

Donidalorsen administered SC

Part B: Donidalorsen 80 mg

Participants with hereditary angioedema with normal C1-inhibitor (HAE-nC1-INH) received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.

Group Type EXPERIMENTAL

Donidalorsen

Intervention Type DRUG

Donidalorsen administered SC

Interventions

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Donidalorsen

Donidalorsen administered SC

Intervention Type DRUG

Placebo

Placebo matching solution administered SC

Intervention Type DRUG

Other Intervention Names

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ISIS 721744 IONIS-PKK-LRx

Eligibility Criteria

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Inclusion Criteria

* Documented diagnosis of HAE-1/HAE-2 (for inclusion in Part A) or HAE-nC1-INH (for inclusion in Part B)
* Participants must have experienced a minimum of 2 HAE attacks (assessed by the Angioedema Activity Score \[AAS\] and confirmed by the investigator) during the screening period
* Access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks

Exclusion Criteria

* Anticipated use of short-term prophylaxis for angioedema attacks for a pre-planned procedure during the screening or study periods
* Concurrent diagnosis of any other type of recurrent angioedema, including acquired or idiopathic angioedema
* Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C, or chronic hepatitis B
* Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
* Treatment with another investigational drug or biological agent within 1 month or 5 half-lives, whichever is longer, of screening
* Exposure to any of the following medications:

* Angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptive or hormonal replacement therapy) within 4 weeks prior to screening
* Chronic prophylaxis with Lanadelumab within 10 weeks prior to screening
* Oligonucleotides (including small interfering ribonucleic acid \[RNA\]) within 4 months of screening (if single dose received) or within 12 months of screening (if multiple doses received)

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No