Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)

NCT ID: NCT02865720

Last Updated: 2021-06-02

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-09-08
• Study Completion Date: 2017-06-23

Brief Summary

The purpose of this study is to determine if an investigational treatment is safe and well tolerated when administered by intravenous (IV) infusion in Japanese subjects with HAE.

Conditions

Hereditary Angioedema (HAE)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Subjects 2 to 5 years of age

500 U of CINRYZE will be administered by IV infusion twice weekly for 12 weeks

Group Type: EXPERIMENTAL

CINRYZE 500 U

Intervention Type: DRUG

IV infusion administered twice weekly

Subjects 6 years of age and older

1000 U of CINRYZE will be administered by IV infusion twice weekly for 12 weeks.

Group Type: EXPERIMENTAL

CINRYZE 1000 U

Intervention Type: DRUG

IV infusion administered twice weekly

Interventions

CINRYZE 500 U

IV infusion administered twice weekly

Intervention Type: DRUG

CINRYZE 1000 U

IV infusion administered twice weekly

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents.

2. Be ≥2 years of age.

3. Meet the following minimum body weight criteria:

• Subjects 2 to 5 years of age must weigh at least 12.5 kg; and
• Subjects 6 years of age and above must weigh at least 25 kg.

4. Have a confirmed diagnosis of Type I or Type II HAE. NOTE: Diagnosis may be based on historical data including family history, clinical symptoms (characteristic attacks), or documentation of low level of C1 INH protein and/or C1 INH activity.

5. Have a history of at least one angioedema attack per month (on average) during the 3 consecutive months immediately before enrollment.

6. Agree to adhere to the protocol-defined schedule of assessments and procedures.

7. Agree to avoid his/her known angioedema attack triggers during the study to the best of his/her ability.

8. If a female of reproductive age, be postmenopausal (≥12 months following cessation of menstruation), surgically sterile, or following an acceptable method of birth control (and agree to continue its use through 1 month after the last dose of study drug):

• Non-hormonal methods (eg, abstinence, barrier control) for at least 1 complete menstrual cycle before the Screening Visit.
• Stable doses of estrogen and/or progestin containing products for at least 2 months before the Screening Visit.

9. If a male of reproductive age, be surgically sterile or agree to follow an acceptable method of birth control (eg, abstinence, barrier control) from the Screening Visit through 2 months after the last dose of study drug.

10. 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 or minor (<20 years of age), have a parent/legal guardian who is informed of the nature of the study provide written informed consent (ie, permission) for the child to participate in the study before any study-specific procedures are performed. Assent will be obtained from children ≥14 years of age.

Exclusion Criteria

1. Have a history of hypercoagulability (abnormal blood clotting).

2. Have a diagnosis of acquired angioedema or be known to have C1 INH antibodies.

3. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE) or other blood products.

4. Have received C1 INH therapy or any blood products within 3 days before the first dose of study drug.

5. Have had signs or symptoms of an angioedema attack within 2 days before the first dose of study drug.

6. Have any change (start, stop, or change in dose) in androgen therapy (eg, danazol, oxandrolone, stanozolol, testosterone), tranexamic acid, epsilon-aminocaproic acid (EACA), or other antifibrinolytics within 14 days before the first dose of study drug.

7. If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (eg, estrogen/progestin containing products) within 2 months before the first dose of study drug.

8. Be pregnant or breastfeeding.

9. Have received an investigational drug other than those required for prevention or treatment of angioedema attacks within 30 days before the first dose of study drug.

10. 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.

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Toyohashi Municipal Hospital

Toyohashi, Aiti, Japan

Asahi General Hospital

Asahi, Chiba, Japan

Gunma University Hospital

Maebashi, Gunma, Japan

Kobe University Hospital

Kobe, Hyōgo, Japan

Heart Life Hospital

Nakagusuku, Nakagami, Japan

Naha City Hospital

Naha, Okinawa, Japan

Shiman University Hospital

Izumo, Shimane, Japan

Adachi kyosai Hospital

Adachi City, Tokyo, Japan

Hiroshima University Hospital

Hiroshima, , Japan

Tomakomai City Hospital

Tomakomai, , Japan

Countries

Japan

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

0624-209

Identifier Type: OTHER

Identifier Source: secondary_id

SHP616-209

Identifier Type: -

Identifier Source: org_study_id