Trial · NCT05139810
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
NCT05139810, Phase 3, Completed, Ionis Pharmaceuticals, Inc.; results first posted 2025-03-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE). Trials, record NCT05139810. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05139810
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- 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 number
- NCT05139810
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 91 participants, actual
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Conditions
- Hereditary Angioedema
- Interventions
- Donidalorsen, Placebo
- First posted
- 2021-12-01
- Last updated
- 2025-03-06
- Other registries' IDs
- eudract:2021-002571-19
- Results first posted
- 2025-03-06
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time-Normalized Investigator-Confirmed (IC) HAE Attack Rate (Per Month) From Week 1 to Week 25 | Week 1 to Week 25 | The time-adjusted HAE attack rate was calculated as number of IC HAE attacks occurring from Week 1 to Week 25, 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 region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). |
| Secondary | Time-Normalized IC HAE Attack Rate (Per Month) From Week 5 to Week 25 | Week 5 to Week 25 | The time-adjusted HAE attack rate was calculated as number of IC HAE attacks occurring from Week 5 to Week 25, 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 region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). |
| Secondary | Percentage of IC HAE Attack-Free Participants From Week 5 to Week 25 | Week 5 to Week 25 | 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 region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Percentages are rounded off to the nearest decimal. |
| Secondary | Time-Normalized Moderate or Severe IC HAE Attack Rate (Per Month) From Week 5 to Week 25 | Week 5 to Week 25 | The time-adjusted HAE attack rate was calculated as number of investigator-confirmed moderate or severe HAE attacks occurring from Week 5 to Week 25, 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 region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). |
| Secondary | Number of Participants With a Clinical Response From Week 5 to Week 25 | Week 5 to Week 25 | Clinical response was defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction from Baseline in HAE attack rate from Week 5 to Week 25. The HAE attack rate between Week 5 and Week 25 for each participant is calculated as number of HAE attacks occurring from Week 5 to week 25 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 region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Baseline= Run-in period which is the period from screening to the last day prior to Study Day 1. |
| Secondary | IC HAE Attack Rate Requiring Acute HAE Therapy From Week 5 to Week 25 | Week 5 to Week 25 | Time-adjusted HAE attack rate is calculated as number of IC HAE attacks requiring acute therapy occurring from Week 5 to Week 25, divided by number of days the participant contributed to 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 region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). HAE attacks requiring acute therapy included those attacks with following concomitant medications c1 esterase inhibitors (human and recombinant), plasma kallikrein inhibitor (human), and bradykinin antagonist. |
| Secondary | Percentage of Participants Who Are Well Controlled on the Angioedema Control Test (AECT) at Week 25 | Week 25 | The AECT is a validated participant-reported outcome instrument to assess disease activity in participants with recurrent angioedema. The questionnaire consists of 4 questions asking about the frequency and severity of angioedema experienced in last 4 weeks. Each question has 5 response choices with total score ranging from 0 to 16. The AECT can be used to identify participants with poorly controlled disease by working with a cutoff value of greater than or equal to 10 points. Participants who score less than 10 points (0-9) in the AECT have poorly controlled disease whereas participants with well-controlled disease score 10-16 points. Percentages are rounded off to the nearest decimal. |
| Secondary | Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire Total Score at Week 25 | Week 25 | The AE-QoL questionnaire is a validated tool to assess symptom-specific health-related QOL impairment in participants suffering from recurrent angioedema. It is a self-administered questionnaire comprising 17 questions across 4 domains: functioning, fatigue/mood, fears/shame, and food. The responses are scored from 0 to 4 where, 0 = never, 1 = rarely, 2 = occasionally, 3 = often, 4 = very often. The AE-QoL domain scores and total score were calculated by using the following formula: (Sum score of all completed items) / (maximum sum score of all possible items) × 100. Total scores ranges from 0 to 100, with higher scores indicating greater impairment. Negative change from baseline indicates improvement. The calculated domain and total scores were not raw scores but linear transformations to a 0 to 100 scale. Baseline is defined as the score on Study Day 1. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Time-Normalized Investigator-Confirmed (IC) HAE Attack Rate (Per Month) From Week 1 to Week 25 | Week 1 to Week 25 | The FAS included all randomized participants who received at least 1 dose of the study drug (donidalorsen or placebo). As pre-specified in the protocol and statistical analysis plan, for purposes of analysis, data for placebo participants from Cohort A and Cohort B was pooled for comparison to donidalorsen treated participants. | HAE attacks per month | LEAST_SQUARES_MEAN |
| SECONDARY | Time-Normalized IC HAE Attack Rate (Per Month) From Week 5 to Week 25 | Week 5 to Week 25 | The FAS included all randomized participants who received at least 1 dose of the study drug (donidalorsen or placebo). As pre-specified in the protocol and statistical analysis plan, for purposes of analysis, data for placebo participants from Cohort A and Cohort B was pooled for comparison to donidalorsen treated participants. | HAE attacks per month | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of IC HAE Attack-Free Participants From Week 5 to Week 25 | Week 5 to Week 25 | The FAS included all randomized participants who received at least 1 dose of the study drug (donidalorsen or placebo). As pre-specified in the protocol and statistical analysis plan, for purposes of analysis, data for placebo participants from Cohort A and Cohort B was pooled for comparison to donidalorsen treated participants. | Percentage of participants | NUMBER |
| SECONDARY | Time-Normalized Moderate or Severe IC HAE Attack Rate (Per Month) From Week 5 to Week 25 | Week 5 to Week 25 | The FAS included all randomized participants who received at least 1 dose of the study drug (donidalorsen or placebo). As pre-specified in the protocol and statistical analysis plan, for purposes of analysis, data for placebo participants from Cohort A and Cohort B was pooled for comparison to donidalorsen treated participants. | HAE attacks per month | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With a Clinical Response From Week 5 to Week 25 | Week 5 to Week 25 | The FAS included all randomized participants who received at least 1 dose of the study drug (donidalorsen or placebo). As pre-specified in the protocol and statistical analysis plan, for purposes of analysis, data for placebo participants from Cohort A and Cohort B was pooled for comparison to donidalorsen treated participants. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | IC HAE Attack Rate Requiring Acute HAE Therapy From Week 5 to Week 25 | Week 5 to Week 25 | The FAS included all randomized participants who received at least 1 dose of the study drug (donidalorsen or placebo). As pre-specified in the protocol and statistical analysis plan, for purposes of analysis, data for placebo participants from Cohort A and Cohort B was pooled for comparison to donidalorsen treated participants. | HAE attacks per month | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants Who Are Well Controlled on the Angioedema Control Test (AECT) at Week 25 | Week 25 | The FAS included all randomized participants who received at least 1 dose of the study drug (donidalorsen or placebo). Overall number analyzed is the number of participants with data available for analysis at the specified timepoint. As pre-specified in the protocol and statistical analysis plan, for purposes of analysis, data for placebo participants from Cohort A and Cohort B was pooled for comparison to donidalorsen treated participants. | Percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire Total Score at Week 25 | Week 25 | The FAS included all randomized participants who received at least 1 dose of the study drug (donidalorsen or placebo). Overall number of participants analyzed is the number of participants with data available for analysis at the specified time point. As pre-specified in the protocol and statistical analysis plan, for purposes of analysis, data for placebo participants from Cohort A and Cohort B was pooled for comparison to donidalorsen treated participants. | Score on a scale | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group30
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Time-Normalized Investigator-Confirmed (IC) HAE Attack Rate (Per Month) From Week 1 to Week 25 | Pooled Placebo | 2.26 | 95% Confidence Interval 1.657 to 3.085 | HAE attacks per month | |
| Time-Normalized Investigator-Confirmed (IC) HAE Attack Rate (Per Month) From Week 1 to Week 25 | Cohort A: Donidalorsen 80 mg | 0.44 | 95% Confidence Interval 0.265 to 0.727 | HAE attacks per month | |
| Time-Normalized Investigator-Confirmed (IC) HAE Attack Rate (Per Month) From Week 1 to Week 25 | Cohort B: Donidalorsen 80 mg | 1.02 | 95% Confidence Interval 0.651 to 1.594 | HAE attacks per month | |
| Time-Normalized IC HAE Attack Rate (Per Month) From Week 5 to Week 25 | Pooled Placebo | 2.25 | 95% Confidence Interval 1.594 to 3.183 | HAE attacks per month | |
| Time-Normalized IC HAE Attack Rate (Per Month) From Week 5 to Week 25 | Cohort A: Donidalorsen 80 mg | 0.30 | 95% Confidence Interval 0.151 to 0.581 | HAE attacks per month | |
| Time-Normalized IC HAE Attack Rate (Per Month) From Week 5 to Week 25 | Cohort B: Donidalorsen 80 mg | 0.90 | 95% Confidence Interval 0.529 to 1.520 | HAE attacks per month | |
| Percentage of IC HAE Attack-Free Participants From Week 5 to Week 25 | Pooled Placebo | 9.1 | Percentage of participants | ||
| Percentage of IC HAE Attack-Free Participants From Week 5 to Week 25 | Cohort A: Donidalorsen 80 mg | 53.3 | Percentage of participants | ||
| Percentage of IC HAE Attack-Free Participants From Week 5 to Week 25 | Cohort B: Donidalorsen 80 mg | 34.8 | Percentage of participants | ||
| Time-Normalized Moderate or Severe IC HAE Attack Rate (Per Month) From Week 5 to Week 25 | Pooled Placebo | 1.15 | 95% Confidence Interval 0.718 to 1.831 | HAE attacks per month |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pooled Placebo | 22 | |
| Overall Study | STARTED | Cohort A: Donidalorsen 80 mg | 46 | |
| Overall Study | STARTED | Cohort B: Donidalorsen 80 mg | 23 | |
| Overall Study | Full Analysis Set (FAS) | Pooled Placebo | 22 | |
| Overall Study | Full Analysis Set (FAS) | Cohort A: Donidalorsen 80 mg | 45 | |
| Overall Study | Full Analysis Set (FAS) | Cohort B: Donidalorsen 80 mg | 23 | |
| Overall Study | COMPLETED | Pooled Placebo | 0 | |
| Overall Study | COMPLETED | Cohort A: Donidalorsen 80 mg | 1 | |
| Overall Study | COMPLETED | Cohort B: Donidalorsen 80 mg | 3 | |
| Overall Study | NOT COMPLETED | Pooled Placebo | 22 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events63
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Headache | Nervous system disorders | Pooled Placebo | 4 | 22 | |
| other | Headache | Nervous system disorders | Cohort A: Donidalorsen 80 mg | 7 | 45 | |
| other | Headache | Nervous system disorders | Cohort B: Donidalorsen 80 mg | 2 | 23 | |
| other | Injection Site Erythema | General disorders | Pooled Placebo | 0 | 22 | |
| other | Injection Site Erythema | General disorders | Cohort A: Donidalorsen 80 mg | 6 | 45 | |
| other | Injection Site Erythema | General disorders | Cohort B: Donidalorsen 80 mg | 1 | 23 | |
| other | Nasopharyngitis | Infections and infestations | Pooled Placebo | 5 | 22 | |
| other | Nasopharyngitis | Infections and infestations | Cohort A: Donidalorsen 80 mg | 6 | 45 | |
| other | Nasopharyngitis | Infections and infestations | Cohort B: Donidalorsen 80 mg | 3 | 23 | |
| other | Influenza | Infections and infestations | Pooled Placebo | 2 | 22 |
1 to 10 of 63Source ClinicalTrials.gov
See all 63 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pooled Placebo | 0 | 22 |
| Total, all-cause mortality | Cohort A: Donidalorsen 80 mg | 0 | 45 |
| Total, all-cause mortality | Cohort B: Donidalorsen 80 mg | 0 | 23 |
| Total, other adverse events | Pooled Placebo | 15 | 22 |
| Total, other adverse events | Cohort A: Donidalorsen 80 mg | 22 | 45 |
| Total, other adverse events | Cohort B: Donidalorsen 80 mg | 13 | 23 |
| Total, serious adverse events | Pooled Placebo | 1 | 22 |
| Total, serious adverse events | Cohort A: Donidalorsen 80 mg | 0 | 45 |
| Total, serious adverse events | Cohort B: Donidalorsen 80 mg | 0 | 23 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05139810
GET https://conductscience.org/api/trials/v1/NCT05139810/results?page=2