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NCT04030598 · ClinicalTrials.gov registry record · Phase 2
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
A Phase 2 study, sponsored by Ionis Pharmaceuticals.
- Completed
- Registry status
- Phase 2
- Development phase
- 23
- Enrollment target
NCT04030598: Completed Phase 2 study, sponsored by Ionis Pharmaceuticals.
NCT04030598 is a Phase 2 study that has completed, run by Ionis Pharmaceuticals. The registered enrollment target is 23 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (83% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04030598, a Phase 2 study, has completed, sponsored by Ionis Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 23 participants
- Enrollment target
Study Summary
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.
Primary Outcome
The Week 1 to end of on-treatment period HAE attack rate was calculated for each participant as number of HAE attacks occurring from Week 1 to 28 days after the last dose date divided by the number of days the participant contributed to the period multiplied by 28 days. An HAE attack was defined as an event with signs or symptoms consistent with an attack in at least 1 of the locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary
Interventions
- DRUG Placebo
- DRUG Donidalorsen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 23 participants |
| Start Date | 2020-01-07 |
| Est. Completion | 2021-03-01 |
| Phase | Phase 2 |
What the finished NCT04030598 record still lists
NCT04030598 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 23 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (83% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT04030598 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04030598 about?
NCT04030598 is a clinical study titled "A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema". 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 (...
What is the current status of trial NCT04030598?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 23 participants. The study started on 2020-01-07. Estimated completion is 2021-03-01.
What interventions are being tested in trial NCT04030598?
The interventions under investigation include: Placebo (DRUG), Donidalorsen (DRUG).
Who is sponsoring clinical trial NCT04030598?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04030598's enrollment target sits among peer trials
23 1784th of 2000 higher than 211 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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