A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase

NCT01426763 | Completed Phase 2 Results

• 2 other identifiers: 0624-204SPD616-204
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 4

Brief Summary

The objectives of the study are to:

1. 1.Evaluate the safety and tolerability of subcutaneously administered CINRYZE with recombinant human hyaluronidase (rHuPH20) in subjects with hereditary angioedema (HAE) who previously participated in CINRYZE Study 0624-200 (NCT01095497)
2. 2.Characterize the pharmacokinetics and pharmacodynamics of subcutaneously administered CINRYZE with rHuPH20
3. 3.Assess the immunogenicity of CINRYZE following subcutaneous (SC) administration of CINRYZE with rHuPH20

Conditions

Hereditary Angioedema

Keywords

HAE; C1 esterase inhibitor; C1 INH; Subcutaneous; hyaluronidase; rHuPH20

Outcome Measures

Primary Outcomes (1)

• Incidence and Severity of Adverse Events, Number of Participants With Local Injection Site Reactions, and Number of Participants Who Discontinue Study Drug or Withdraw From the Study

  18 days

Secondary Outcomes (3)

• Mean Change C1 Inhibitor (C1INH)

  18 days

• Mean Change C4 Compliment

  18 days

• Number of Subjects With C1 INH Antibodies

  Day 1 (pre-dose), Day 18 (168 h post Dose 4), and 30 (±2) days after the last dose of study drug (Dose 4)

Study Arms (2)

SC CINRYZE with rHuPH20 Dose Level 1

EXPERIMENTAL

Subcutaneous injection of 1000 Units of CINRYZE with 20,000 Units of rHuPH20 twice weekly for two weeks

Biological: CINRYZE with rHuPH20

SC CINRYZE with rHuPH20 Dose Level 2

EXPERIMENTAL

Subcutaneous injection of 2000 Units of CINRYZE with 40,000 Units of rHuPH20 twice weekly for two weeks

Biological: CINRYZE with rHuPH20

Interventions

CINRYZE with rHuPH20 BIOLOGICAL

Also known as: C1 esterase inhibitor (human), Recombinant human hyaluronidase

SC CINRYZE with rHuPH20 Dose Level 1; SC CINRYZE with rHuPH20 Dose Level 2

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• To be eligible for this protocol, a subject must:
• Provide informed consent/assent, as appropriate.
• Have previously participated in CINRYZE Study 0624-200 and completed the subcutaneous therapy period in that study.
• During the 3 consecutive months prior to screening, have a history of less than 1 HAE attack per month (average) that required treatment with C1 INH therapy or other blood products.
• Agree to avoid his/her known HAE triggers during the study to the best of his/her ability.

You may not qualify if:

• To be eligible for this protocol, a subject must not:
• Have received C1 INH therapy or any blood products for treatment or prevention of an HAE attack within 14 days prior to the first dose of study drug.
• Have received any ecallantide (Kalbitor), icatibant (Firazyr), or antifibrinolytics (e.g., tranexamic acid) within 14 days prior to the first dose of study drug.
• Have any change (start, stop, or change in dose) in androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 14 days prior to the first dose of study drug.
• If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progestin containing products) within 3 months prior to the first dose of study drug.
• Have a history of abnormal blood clotting.
• Have a history of allergic reaction to products containing C1 INH or other blood products.
• Have a known allergy to hyaluronidase or any other ingredient in rHuPH20.
• Be pregnant or breastfeeding.
• Have received an investigational study drug within 30 days prior to the first dose of study drug.

Sponsors & Collaborators

• Shire: lead
• Halozyme Therapeutics: collaborator

Study Sites (4)

ViroPharma Investigational Site

Scottsdale, Arizona, 85251, United States

Location

ViroPharma Investigational Site

Walnut Creek, California, 94598, United States

Location

ViroPharma Investigational Site

Atlanta, Georgia, 30342, United States

Location

ViroPharma Investigational Site

Dallas, Texas, 75231, United States

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

SERPING1 protein, human; Complement C1 Inhibitor Protein

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Complement C1 Inactivator Proteins; Serpins; Peptides; Amino Acids, Peptides, and Proteins; Complement Inactivator Proteins; Complement System Proteins; Immunoproteins; Blood Proteins; Proteins

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 25, 2011
• First Posted: August 31, 2011
• Study Start: September 12, 2011
• Primary Completion: November 28, 2011
• Study Completion: November 28, 2011
• Last Updated: July 19, 2021
• Results First Posted: January 4, 2013

Record last verified: 2021-06

Locations

US (4)