Trial Outcomes & Findings for Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients (NCT NCT01359969)
NCT ID: NCT01359969
Last Updated: 2024-03-29
Results Overview
Time to beginning of relief of symptoms that showed the response to treatment based on the overall VAS score decrease of ≥ 20 mm from baseline. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the oro-pharyngeal-laryngeal (OPL) VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination. Time to beginning of relief will also be calculated based on the Investigator Score (IS) and Treatment Effect Questionnaire (TEQ).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
57 participants
Primary outcome timeframe
The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion VAS score decrease of ≥ 20 mm from baseline.
Results posted on
2024-03-29
Participant Flow
Male and female patients, from 2 up to and including 13 years of age with a clinically suspected and/or confirmed diagnosis of HAE will be recruited for this study. Patients will be identified and invited to participate by the investigators at the respective study centers.
Screening details: Pts with a med hist of HAE (age 2-13 yrs) were invited for a screening visit. At screening the diagnosis HAE was confirmed via Central lab testing. If the diagnosis was confirmed, pts were eligible for treatment if an acute attack would occur and they presented to study center within 5 hrs of onset of attack. In total 57 pts signed ICF and 20 pts had one or more HAE attacks treated.
Participant milestones
| Measure |
Recombinant Human C1 Inhibitor
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
rhC1INH: Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
|
|---|---|
|
Overall Study
STARTED
|
57
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
37
|
Reasons for withdrawal
| Measure |
Recombinant Human C1 Inhibitor
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
rhC1INH: Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
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|---|---|
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Overall Study
Study continued until at least 20 patients had been treated for at least one treatment.
|
37
|
Baseline Characteristics
Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients
Baseline characteristics by cohort
| Measure |
Recombinant Human C1 Inhibitor
n=20 Participants
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
rhC1INH: Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
|
|---|---|
|
Age, Categorical
<=18 years
|
20 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
9.55 years
STANDARD_DEVIATION 3.304 • n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
19 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Romania
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Hungary
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Czechia
|
2 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
2 participants
n=5 Participants
|
|
Region of Enrollment
North Macedonia
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
9 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
1 participants
n=5 Participants
|
|
Height at screening
|
125.6 centimeters
STANDARD_DEVIATION 25.78 • n=5 Participants
|
|
Weight at presentation
|
34.81 kilograms
STANDARD_DEVIATION 20.554 • n=5 Participants
|
PRIMARY outcome
Timeframe: The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion VAS score decrease of ≥ 20 mm from baseline.Population: No statistical analyses have been specified for this primary end point. As this was an open-label single arm study, no statistical analyses could be provided.
Time to beginning of relief of symptoms that showed the response to treatment based on the overall VAS score decrease of ≥ 20 mm from baseline. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the oro-pharyngeal-laryngeal (OPL) VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination. Time to beginning of relief will also be calculated based on the Investigator Score (IS) and Treatment Effect Questionnaire (TEQ).
Outcome measures
| Measure |
Recombinant Human C1 Inhibitor
n=20 Participants
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
rhC1INH: Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
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|---|---|
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Time to Beginning of Relief Based on Visual Analogue Scale (VAS) Was Defined as the Time, in Minutes, From Time of Infusion to the Beginning of Relief.
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60 minutes
Interval 60.0 to 65.0
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SECONDARY outcome
Timeframe: The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion all locations where VAS Scores were recorded.Population: No statistical analyses for this end point
Time to minimal symptoms was defined as the time at which the Overall VAS score fell below 20 mm for all locations where VAS Scores were recorded. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines provided to answer each question related to the severity of symptoms and the patient's condition. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the OPL VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination.
Outcome measures
| Measure |
Recombinant Human C1 Inhibitor
n=20 Participants
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
rhC1INH: Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
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|---|---|
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Time to Minimal Symptoms Based on Patient's VAS Scores; Time From the Start of the Infusion of Study Medication to the First Assessment Time at Which the Overall Severity VAS Reaches a Value of Less Than 20 mm for All Locations
|
122.5 minutes
Interval 120.0 to 126.0
|
Adverse Events
Recombinant Human C1 Inhibitor
Serious events: 3 serious events
Other events: 11 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Recombinant Human C1 Inhibitor
n=20 participants at risk
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
rhC1INH: Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal oedema
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Gastrointestinal disorders
Vomiting
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Bronchitis
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Pneumonia
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Tonsillitis
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Viral infection
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Eye disorders
Eye swelling
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
Other adverse events
| Measure |
Recombinant Human C1 Inhibitor
n=20 participants at risk
Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
rhC1INH: Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
|
|---|---|
|
Vascular disorders
Lymphocyte morphology abnormal
|
10.0%
2/20 • Number of events 2 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Vascular disorders
Pallor
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Vascular disorders
Body temperature increased
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Respiratory, thoracic and mediastinal disorders
Catarrh
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal oedema
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Nervous system disorders
Headache
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Eye disorders
Eye swelling
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Ear and labyrinth disorders
Middle ear effusion
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Nasopharyngitis
|
15.0%
3/20 • Number of events 3 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Viral infection
|
10.0%
2/20 • Number of events 2 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Bronchitis
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Pharyngitis streptococcal
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Pharyngotonsillitis
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Pneumonia
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Tonsillitis
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Infections and infestations
Upper respiratory tract infection
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Gastrointestinal disorders
Vomiting
|
15.0%
3/20 • Number of events 3 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Gastrointestinal disorders
Abdominal pain
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Gastrointestinal disorders
Dysphagia
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
General disorders
Feeling cold
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
General disorders
Peripheral swelling
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
5.0%
1/20 • Number of events 1 • Investigators are required to document all AEs occurring during the clinical study, commencing with the signing of the ICF through the last follow-up visit (Day 90 after the last treatment).
The reported values are based on the treatment-emergent (serious) adverse events (TE(S)AE), which are the (S)AEs with an onset at any time between the start of treatment and 97 days after treatment for any treated attack. The AEs are only analyzed for the 20 treated patients. The TLFs are based on these 20 treated and analyzed patients. A total of 11 patients experienced 30 TEAEs after treatment with rhC1INH. A total of 3 patients experienced 9 TESAEs after treatment with rhC1INH.
|
Additional Information
Anurag Relan, MD, VP Clinical Research & Medical Affairs
Pharming Technologies BV
Phone: +31 715247400
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place