DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE

Information source: Shire
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Hereditary Angioedema

Intervention: C1 inhibitor (human) [C1 INH] (Biological)

Phase: Phase 4

Status: Completed

Sponsored by: Shire

Official(s) and/or principal investigator(s):
Jennifer Schranz, MD, FRCP(C), Study Director, Affiliation: ViroPharma

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.

Clinical Details

Official title: A Phase 4 Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE® (C1 Inhibitor [Human]) as Prophylactic Therapy in Subjects With Inadequately Controlled Hereditary Angioedema Attacks

Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Primary outcome: 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

Secondary outcome:

Treatment Effect of Escalating Doses of CINRYZE on HAE Attack Rates

Use of Rescue Therapy and/or Other Therapy for Treatment of HAE Symptoms

Detailed description: Qualifying subjects entered a 3-step dose escalation algorithm:

- Step 1: 1500 Units twice per week (starting dosing regimen for all subjects in the

study)

- Step 2: 2000 Units twice per week

- Step 3: 2500 Units twice per week

Each step consisted of 12 weeks of safety monitoring, followed by calculation of average monthly angioedema attack rate based on subject reports of angioedema symptoms (regardless of intensity) and actual duration of therapy for that step. If a subject was deemed a "success" at a given step and the investigator and medical monitor determined that it was safe for the subject to continue on that dose, the subject entered a 3 month follow-up period at that dose level with continued safety monitoring. The subject could not re-enter the study for purposes of dose escalation during the follow-up period. If a subject was not deemed a "success," the subject initiated the next highest step of the dose escalation algorithm provided that the investigator and medical monitor agreed that dose escalation was appropriate. If at the end of Step 3 (2500 Units), a subject was not deemed a "success," then the Week 12 visit represented study completion and the subject was referred to the physician who manages their HAE care.

Eligibility

Minimum age: 6 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria: To be eligible for this protocol, subjects must: 1. Be ≥6 years of age and ≥25 kg body weight. 2. 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)

3. 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. 4. If an adult, be informed of the nature of the study and provide written informed consent before any study-specific procedures are performed. OR 5. 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: 1. 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. 2. Have a history of abnormal blood clotting or other coagulopathy. 3. Be taking prescription anticoagulant medication. 4. Have a history of allergic reaction to CINRYZE or other blood products. 5. Have participated in any other investigational drug study within the past 30 days (other than CINRYZE protocols). 6. Have received any blood products (other than CINRYZE) within 60 days prior to screening. 7. Have any of the following laboratory values at screening:

- Hemoglobin <8 g/dL

- White blood cell count <2 x 10^9/L or >20 x 10^9/L

- Platelet count <50 x 10^9/L or >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

8. Be pregnant or breastfeeding.

Locations and Contacts

Allergy, Asthma and Immunology Associates, Scottsdale, Arizona 85251, United States

Family Allergy and Asthma Center, Atlanta, Georgia 30342, United States

Institute for Asthma and Allergy, Wheaton, Maryland 20902, United States

Winthrop University Hospital, Mineola, New York 11501, United States

University of Cincinnati Medical Center, Cincinnati, Ohio 45267, United States

Allergy and Asthma Research Group, Eugene, Oregon 97401, United States

Baker Allergy, Asthma and Dermatology Research Center, Lake Oswego, Oregon 97035, United States

East Tennessee Center for Clinical Research, Knoxville, Tennessee 37909, United States

Bryan, Texas 77802, United States

AARA Research Center, Dallas, Texas 75231, United States

Marycliff Allergy Specialist, Spokane, Washington 99204, United States

Additional Information

Starting date: July 2009
Last updated: March 19, 2014

Page last updated: August 23, 2015

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017