Trial Outcomes & Findings for A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (NCT NCT00851409)
NCT ID: NCT00851409
Last Updated: 2018-06-26
Results Overview
Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).
COMPLETED
PHASE2
25 participants
8 weeks
2018-06-26
Participant Flow
Patients with a history of frequent attacks, defined as "HAE" attacks occurring at least every two weeks were included in the study.
Participant milestones
| Measure |
Recombinant Human C1 Inhibitor
Weekly administration of 50 IU/kg recombinant human C1 inhibitor
Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
|
|---|---|
|
Overall Study
STARTED
|
25
|
|
Overall Study
COMPLETED
|
25
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration
Baseline characteristics by cohort
| Measure |
Recombinant Human C1 Inhibitor
n=25 Participants
Weekly administration of 50 IU/kg recombinant human C1 inhibitor
Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
25 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
20 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 8 weeksPrior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).
Outcome measures
| Measure |
Recombinant Human C1 Inhibitor
n=25 Participants
Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
|
|---|---|
|
HAE Attacks/Week
Historical
|
0.9 attacks/week
Interval 0.51 to 1.3
|
|
HAE Attacks/Week
Prophylaxis
|
0.4 attacks/week
Interval 0.27 to 0.56
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SECONDARY outcome
Timeframe: 8 weeks"PK/PD" parameters will be based on concentration time curves after the 1st and 8th "rhC1INH" administration.(ratio visit 8/ visit 1, based on the area under the curve from baseline up to 4 hours after administration (AUC 0-4)
Outcome measures
| Measure |
Recombinant Human C1 Inhibitor
n=25 Participants
Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
|
|---|---|
|
The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.
Functional C1 Inhibitor level
|
1.06 ratio
Interval 0.98 to 1.14
|
|
The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.
Antigenic C1 Inhibitor level
|
1.02 ratio
Interval 0.91 to 1.15
|
|
The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.
C4 level
|
1.00 ratio
Interval 0.74 to 1.33
|
Adverse Events
Recombinant Human C1 Inhibitor
Serious adverse events
| Measure |
Recombinant Human C1 Inhibitor
n=25 participants at risk
Weekly administration of 50 IU/kg recombinant human C1 inhibitor
Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
|
|---|---|
|
Infections and infestations
Appendicitis
|
4.0%
1/25 • Number of events 1 • Treatment emergent adverse events ("TEAEs") were defined as events with start from onset of study drug administration. All Serious Adverse Events("SAEs")are listed.
All Adverse Events ("AEs")occurring within 7 days of onset after study drug administration have been listed.
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal edema
|
4.0%
1/25 • Number of events 1 • Treatment emergent adverse events ("TEAEs") were defined as events with start from onset of study drug administration. All Serious Adverse Events("SAEs")are listed.
All Adverse Events ("AEs")occurring within 7 days of onset after study drug administration have been listed.
|
Other adverse events
| Measure |
Recombinant Human C1 Inhibitor
n=25 participants at risk
Weekly administration of 50 IU/kg recombinant human C1 inhibitor
Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia-mild
|
4.0%
1/25 • Number of events 1 • Treatment emergent adverse events ("TEAEs") were defined as events with start from onset of study drug administration. All Serious Adverse Events("SAEs")are listed.
All Adverse Events ("AEs")occurring within 7 days of onset after study drug administration have been listed.
|
|
Blood and lymphatic system disorders
Anaemia-moderate
|
4.0%
1/25 • Number of events 1 • Treatment emergent adverse events ("TEAEs") were defined as events with start from onset of study drug administration. All Serious Adverse Events("SAEs")are listed.
All Adverse Events ("AEs")occurring within 7 days of onset after study drug administration have been listed.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator must provide a copy of any results communication to the sponsor for review at least 30 days prior to its submission or disclosure. The sponsor may request to delete information identified by sponsor as confidential information prior to submitting such manuscript and/or abstract for publication. For a multi-center study, the investigator must wait (i) at least 24 months after the study is completed at all sites or (ii) until after the multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER