Trial Outcomes & Findings for A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema (NCT NCT04030598)
NCT ID: NCT04030598
Last Updated: 2023-04-03
Results Overview
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 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).
COMPLETED
PHASE2
23 participants
Week 1 to Week 17
2023-04-03
Participant Flow
Participants took part in the study at 7 investigative sites from 7 January 2020 to 1 March 2021.
Participants with hereditary angioedema were concurrently enrolled in Part A and B, respectively. In Part A, 20 participants with hereditary angioedema type I/type II (HAE-1/HAE-2) were randomized in 2:1 ratio to receive donidalorsen or placebo for 13 weeks. In Part B, 3 participants with hereditary angioedema with normal C1-inhibitor (HAE-nC1-INH) received donidalorsen for 13 weeks after Part A.
Participant milestones
| Measure |
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.
|
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.
|
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.
|
|---|---|---|---|
|
Overall Study
STARTED
|
6
|
14
|
3
|
|
Overall Study
COMPLETED
|
6
|
13
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
0
|
Reasons for withdrawal
| Measure |
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.
|
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.
|
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.
|
|---|---|---|---|
|
Overall Study
Voluntary Withdrawal
|
0
|
1
|
0
|
Baseline Characteristics
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
Baseline characteristics by cohort
| Measure |
Part A: Placebo
n=6 Participants
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.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
Total
n=23 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
40.0 years
n=5 Participants
|
37.8 years
n=7 Participants
|
34.0 years
n=5 Participants
|
37.26 years
n=4 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
16 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
22 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
22 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Week 1 to Week 17Population: The intent-to-treat (ITT) population included all enrolled or randomized participants.
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 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).
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Time-normalized Number of HAE Attacks (Per Month) From Week 1 to Week 17
|
2.21 HAE attacks per month
Standard Deviation 1.558
|
0.23 HAE attacks per month
Standard Deviation 0.268
|
1.52 HAE attacks per month
Standard Deviation 2.221
|
SECONDARY outcome
Timeframe: Week 5 to Week 17Population: ITT population included all enrolled or randomized participants.
The Week 5 to end of on-treatment period HAE attack rate was calculated for each participant as number of HAE attacks occurring from Week 5 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 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).
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Time-normalized Number of Investigator-confirmed HAE Attacks (Per Month) From Week 5 to Week 17
|
2.06 HAE attacks per month
Standard Deviation 1.574
|
0.07 HAE attacks per month
Standard Deviation 0.267
|
1.78 HAE attacks per month
Standard Deviation 2.795
|
SECONDARY outcome
Timeframe: Week 5 to Week 17Population: ITT population included all enrolled or randomized participants.
The Week 5 to end of on-treatment period HAE attack rate was calculated for each participant as number of moderate or severe HAE attacks occurring from Week 5 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 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 attack severity: Mild: transient or mild discomfort, Moderate: mild to moderate limitation in activity, some assistance needed, and Severe: marked limitation in activity, assistance required.
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Time-normalized Number of Moderate or Severe Investigator-confirmed HAE Attacks (Per Month) From Week 5 to Week 17
|
1.25 HAE attacks per month
Standard Deviation 1.208
|
0.05 HAE attacks per month
Standard Deviation 0.178
|
0.89 HAE attacks per month
Standard Deviation 1.540
|
SECONDARY outcome
Timeframe: Week 5 to Week 17Population: ITT population included all enrolled or randomized participants. Overall number of participants analyzed is the number of participants with data available for analyses.
Clinical response was defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction from Baseline in HAE attack rate from Week 5 to Week 17. The HAE attack rate was calculated as number of investigator-confirmed HAE attacks occurring from Week 5 to 28 days after last dose administration, 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).
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=13 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Number of Participants With Clinical Response by Week 17
≥ 50% Reduction
|
2 Participants
|
13 Participants
|
2 Participants
|
|
Number of Participants With Clinical Response by Week 17
≥ 70% Reduction
|
1 Participants
|
12 Participants
|
2 Participants
|
|
Number of Participants With Clinical Response by Week 17
≥ 90% Reduction
|
0 Participants
|
12 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Week 5 to Week 17Population: ITT population included all enrolled or randomized participants.
The Week 5 to end of on-treatment period HAE attack rate was calculated for each participant as number of HAE attacks requiring acute therapy occurring from Week 5 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 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 medical intervention or hospitalization marked on the case report form (CRFs).
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Number of Investigator-confirmed HAE Attacks Requiring Acute Therapy From Week 5 to Week 17
|
1.25 HAE attacks per month
Standard Deviation 1.208
|
0.05 HAE attacks per month
Standard Deviation 0.178
|
0.89 HAE attacks per month
Standard Deviation 1.540
|
SECONDARY outcome
Timeframe: Weeks 9 and 17Population: ITT population included all enrolled or randomized participants. Number analyzed is number of participants with data available for analysis at specified time point.
High-molecular-weight kininogen (HMWK) is an abundant protein found in plasma and it has a critical role in acute attacks of HAE. During HAE attack plasma kallikrein cleaves HMWK producing cleaved HMWK (cHMWK) and bradykinin, the major biologic peptide that promotes the edema, one of the characteristic traits of HAE. Percentage of cHMWK levels were assessed to evaluate pharmacodynamics of donidalorsen.
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Percentage of Cleaved High Molecular Weight Kininogen (cHMWK) Levels at Weeks 9 and 17
Week 9
|
5.62 percentage of cHMWK levels
Standard Deviation 3.255
|
2.07 percentage of cHMWK levels
Standard Deviation 1.241
|
1.03 percentage of cHMWK levels
Standard Deviation 0.115
|
|
Percentage of Cleaved High Molecular Weight Kininogen (cHMWK) Levels at Weeks 9 and 17
Week 17
|
7.00 percentage of cHMWK levels
Standard Deviation 4.338
|
2.35 percentage of cHMWK levels
Standard Deviation 1.353
|
2.13 percentage of cHMWK levels
Standard Deviation 0.929
|
SECONDARY outcome
Timeframe: Weeks 9 and 17Population: ITT population included all enrolled or randomized participants. Number analyzed is number of participants with data available for analysis at specified timepoint.
Prekallikrein (PKK) has a critical role in acute attacks of HAE. During HAE attack PKK is activated to form plasma kallikrein. Plasma kallikrein cleaves HMWK producing cleaved HMWK (cHMWK) and bradykinin, the major biologic peptide that promotes the edema, one of the characteristic traits of HAE. Prekallikrein levels were measured to assess pharmacodynamics of donidalorsen.
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Prekallikrein (PKK) Activity Levels at Weeks 9 and 17
Week 9
|
95.467 milligram per liter (mg/L)
Standard Deviation 23.7193
|
37.676 milligram per liter (mg/L)
Standard Deviation 14.5639
|
25.630 milligram per liter (mg/L)
Standard Deviation 19.2671
|
|
Prekallikrein (PKK) Activity Levels at Weeks 9 and 17
Week 17
|
98.600 milligram per liter (mg/L)
Standard Deviation 34.5944
|
37.795 milligram per liter (mg/L)
Standard Deviation 38.6618
|
28.615 milligram per liter (mg/L)
Standard Deviation 22.8042
|
SECONDARY outcome
Timeframe: Weeks 9 and 17Population: ITT population included all enrolled or randomized participants.
Treatment options for HAE included on-demand treatment of attacks and prophylaxis. On-demand medication options included supplementation of C1-INH (either plasma-derived or recombinant C1-INH concentrate) and inhibition of BK2 receptor activation (BK2-receptor antagonist). The number of participants who used on-demand medication at Week 9 (Day 57) and at Week 17 (end of the on-treatment period) were reported.
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Number of Participants Who Consumed On-demand Medication at Weeks 9 and 17
Week 17
|
6 Participants
|
11 Participants
|
3 Participants
|
|
Number of Participants Who Consumed On-demand Medication at Weeks 9 and 17
Week 9
|
6 Participants
|
12 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Baseline, Weeks 9 and 17Population: ITT population included all enrolled or randomized participants. Number analyzed is the number of participants with data available at specified timepoint.
The AE-QoL was developed to measure health-related quality of life (HRQoL) impairment in participants with recurrent angioedema. The AE-QoL is a self-administered questionnaire that can be completed in less than 5 minutes. It comprises 17 items across 4 domains: functioning, fatigue/mood, fears/shame, and food. Responses use a 5-point Likert scale ranging from '0 = never' to '4 = very often.' Per-participant scores for each domain were computed using the appropriate scoring algorithm applied to the question response scores for each domain. Per-participant total scores (including all 4 domains) were similarly computed using the question response scores for all 17 questions. The outputs from the scoring algorithm were normalized on a scale ranging from 0 (less adverse impact) to 100 (most adverse impact). Global total score ranges from 0 to 100, with higher scores indicating greater impairment. Mixed model for repeated measures (MMRM) was used for analyses.
Outcome measures
| Measure |
Part A: Placebo
n=6 Participants
Participants with HAE-1/HAE-2 received placebo subcutaneously (SC) every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part A: Donidalorsen 80 mg
n=14 Participants
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 Participants
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.
|
|---|---|---|---|
|
Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire Total Score at Weeks 9 and 17
Change from Baseline at Week 9
|
-1.50 score on a scale
Standard Error 4.413
|
-27.42 score on a scale
Standard Error 2.854
|
25.49 score on a scale
Standard Error 17.421
|
|
Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire Total Score at Weeks 9 and 17
Change from Baseline at Week 17
|
-6.15 score on a scale
Standard Error 4.671
|
-26.85 score on a scale
Standard Error 3.133
|
26.96 score on a scale
Standard Error 20.918
|
Adverse Events
Part A: Placebo
Part A: Donidalorsen 80 mg
Part B: Donidalorsen 80 mg
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Part A: Placebo
n=6 participants at risk
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.
|
Part A: Donidalorsen 80 mg
n=14 participants at risk
Participants with HAE-1/HAE-2 received donidalorsen, 80 mg, SC, every 4 weeks at Weeks 1, 5, 9, and 13.
|
Part B: Donidalorsen 80 mg
n=3 participants at risk
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.
|
|---|---|---|---|
|
Gastrointestinal disorders
Nausea
|
16.7%
1/6 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
7.1%
1/14 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
0.00%
0/3 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/6 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
0.00%
0/14 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
33.3%
1/3 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
|
Nervous system disorders
Headache
|
33.3%
2/6 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
14.3%
2/14 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
0.00%
0/3 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
|
General disorders
Application site rash
|
0.00%
0/6 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
0.00%
0/14 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
33.3%
1/3 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
|
General disorders
Feeling hot
|
0.00%
0/6 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
0.00%
0/14 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
33.3%
1/3 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
|
General disorders
Hyperhidrosis
|
0.00%
0/6 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
0.00%
0/14 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
33.3%
1/3 • From first dose of study drug up to end of the study (Up to Week 26)
The safety population included all enrolled participants who received at least 1 dose of study drug (donidalorsen or placebo).
|
Additional Information
Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place