Phase 4
Completed N=20
A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE
Source: ClinicalTrials.gov NCT00914966 ↗Enrolled (actual)
20
Serious AEs
7.7%
Results posted
Jul 2013
Primary outcomePrimary: Number of Subjects With Adverse Events, Hospitalizations, Thrombotic Events, Treatment-emergent C1 INH Antibodies, Post-baseline Toxicity Grade Increases in Clinical Laboratory Parameters, and Post-dose Vital Signs Changes of Potential Clinical Importance — 15; 11; 11; 0 participants
Summary
The objectives of the study were:
1. To assess the safety and tolerability of escalating doses of CINRYZE.
2. To assess the effect of an escalating dose algorithm for CINRYZE on hereditary angioedema (HAE) attack rates.
3. To assess the immunogenicity of CINRYZE.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects With Adverse Events, Hospitalizations, Thrombotic Events, Treatment-emergent C1 INH Antibodies, Post-baseline Toxicity Grade Increases in Clinical Laboratory Parameters, and Post-dose Vital Signs Changes of Potential Clinical Importance |
15; 11; 11; 0; 1; 0 | — |
| SECONDARY Treatment Effect of Escalating Doses of CINRYZE on HAE Attack Rates |
4; 0; 5; 1; 0; 1 | — |
Eligibility Criteria
Inclusion Criteria
To be eligible for this protocol, subjects must:
- Be ≥6 years of age and ≥25 kg body weight.
- Have a confirmed diagnosis of HAE with a documented history of swelling of the face, extremities, gastrointestinal tract, genitalia, or larynx and a history of at least one of the following:
- C1 INH gene mutation
- C4 level below the lower limit of the reference range
- C1 INH antigen level below the lower limit of the reference range
- Functional C1 INH level below the lower limit of the reference range
- Family history of HAE (i.e., grandparent, parent, sibling)
- Have a history of >1.0 HAE attack per month (average) of any severity during the 3 consecutive months prior to screening while receiving the recommended CINRYZE dosing of 1000 Units every 3 to 4 days via intravenous injection.
- If an adult, be informed of the nature of the study and provide written informed consent before any study-specific procedures are performed.
OR
- If a child, have a parent/legal guardian who is willing and able to provide written informed consent for the child to participate in the study (with assent from the child when appropriate).
Exclusion Criteria
To be eligible for this protocol, subjects must not:
- Have, as determined by the investigator and/or the sponsor's medical monitor, any surgical or medical condition that could interfere with the administration of study drug or interpretation of study results.
- Have a history of abnormal blood clotting or other coagulopathy.
- Be taking prescription anticoagulant medication.
- Have a history of allergic reaction to CINRYZE or other blood products.
- Have participated in any other investigational drug study within the past 30 days (other than CINRYZE protocols).
- Have received any blood products (other than CINRYZE) within 60 days prior to screening.
- Have any of the following laboratory values at screening:
- Hemoglobin 20 x 10^9/L
- Platelet count 400 x 10^9/L
- Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >2.0 x the upper limit of normal
- Blood urea nitrogen and/or creatinine >2.0 x the upper limit of normal
- Be pregnant or breastfeeding.
Data sourced from ClinicalTrials.gov (NCT00914966). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.