Angioedema
Conditions
Keywords
MeSH: angioneurotic edema, complement 1 inactivators, Hereditary Angioedema Type I or II, Acquired Angioedema
Brief summary
A multicentre study to investigate the pharmacokinetics, clinical efficacy and safety of nanofiltered Cetor® (called C1-esteraseremmer-N during the development phase) for the treatment of hereditary angioedema (HAE) will be performed. This study (KB2003.01) consists of three parts: Part A - pharmacokinetics (phase II); Part B - treatment of attacks of angioedema (phase III); and Part C - prophylactic use of C1 inhibitor (phase III). Parts B + C will provide data on the efficacy of C1-esteraseremmer-N. The changes within the manufacturing process of C1-esteraseremmer-N, compared to Cetor® nanofiltration and omission of hepatitis B immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide more safety regarding viruses. In part C of the study, a number of HAE patients will be treated prophylactically with open-label C1-esteraseremmer-N for a period of 16 weeks. The number of attacks occurring will be compared with historical data. If possible, also some patients treated prophylactically with C1 inhibitor for acquired angioedema will be included.
Detailed description
A multicentre study to investigate the pharmacokinetics, clinical efficacy and safety of nanofiltered Cetor® (called C1-esteraseremmer-N during the development phase) for the treatment of hereditary angioedema (HAE) will be performed. This study (KB2003.01) consists of three parts: Part A - pharmacokinetics (phase II); Part B - treatment of attacks of angioedema (phase III); and Part C - prophylactic use of C1 inhibitor (phase III). Parts B + C will provide data on the efficacy of C1-esteraseremmer-N. The changes within the manufacturing process of C1-esteraseremmer-N, compared to Cetor® nanofiltration and omission of hepatitis B immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide more safety regarding viruses. In part C of the study, a number of HAE patients will be treated prophylactically with open-label C1-esteraseremmer-N for a period of 16 weeks. The number of attacks occurring will be compared with historical data. If possible, also some patients treated prophylactically with C1 inhibitor for acquired angioedema will be included.
Interventions
every 5-7 days
Sponsors
Prothya Biosolutions
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Inclusion criteria for hereditary angioedema patients: * Established diagnosis of hereditary angioedema type I or II: markedly decreased C1 inhibitor activity; decreased (type I), normal or elevated (type II) level of C1 inhibitor antigen; decreased level of C4. * Patients already using C1 inhibitor concentrate for prophylaxis of angioedema attacks. * Age ≥ 16 years * Signed informed consent by patient and patient's legal representative if under 18 years old Inclusion criteria for acquired angioedema patients: * Established diagnosis of acquired angioedema: recurrent attacks of angioedema without urticaria; no family history; decreased functional C1 inhibitor; decreased level of C4. * Autoantibodies to C1 inhibitor or decreased C1q or onset after the third decade of life. * Age ≥ 16 years * Patient already using C1 inhibitor concentrate for prophylaxis of angioedema attacks * Signed informed consent by patient and patient's legal representative if under 18 years old
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective in this clinical study is to evaluate the prevention of angioedema attacks in patients prophylactically treated with C1-esteraseremmer-N. | 16 weeks |
Secondary
| Measure | Time frame |
|---|---|
| The secondary objective of this study is to evaluate the safety and clinical activity of C1-esteraseremmer-N. | 16 weeks |
Countries
Netherlands
Outcome results
None listed