The expanded access program allows people to gain access to an unlicensed treatment on compassionate grounds. Lanadelumab, also known as TAK-743, is a medicine to help prevent hereditary angioedema attacks. Lanadelumab is not yet licensed for use in Japan. The main aim of this study is to allow Japanese teenagers and adults with type I or type II hereditary angioedema to be treated with lanadelumab, through the expanded access program in Japan. Participants can either have taken part in the previous study SHP643-302 or can be new participants. Participants just completing study SHP643-302 who reach the criteria can automatically take part in this study. However, for new participants, the study doctor will check who can take part at the first study visit. For those who can take part, new participants will receive injections of lanadelumab just under the skin. Eventually, after training, some of these will be able to inject themselves with lanadelumab in the same way. Participants who injected themselves with lanadelumab in study SHP643-302 can continue to do so during this study. The study doctors will decide if each participant will be treated with lanadelumab every 2 weeks or every 4 weeks. Treatment with lanadelumab will continue until lanadelumab is commercially available in Japan or the sponsor (Takeda) stops the study. Participants can visit the clinic during treatment if needed. If treatment continues after 6 months, participants will visit the clinic every 12 weeks for a check-up. This will include noting any hereditary angioedema attacks and side effects from the treatment. After 7 months of treatment, the study staff will check-up with each participant every 2 weeks by telephone. After treatment has finished, participants will visit the clinic for a final-check-up 4 weeks later.
This study is Japan Expanded Access Program with TAK-743. The study drug in this study is called TAK-743. TAK-743 will be administered to people who have Type I or II hereditary angioedema (HAE). Two types of participants will be enrolled into this study: * Participants who rollover from Study SHP643-302 (NCT04180163). * Participants who are non-rollovers (ie, were not participants in Study SHP643-302). Participants who discontinue from Study SHP643-302 after providing informed consent are not eligible to enroll in this study. All participants will be asked to administer TAK-743 300 mg with subcutaneous injection every 2 weeks throughout this study. Participants who is stable with over 6-month administration of 300mg every 2 weeks can be switched to 300mg every 4 weeks. This multi-center trial will be conducted in Japan (approximately 15 sites). The overall time to participate in this study is over 182 days. Participants will make multiple visits to the clinic basically every 2 weeks until Day 182, and will be contacted by telephone every 2 weeks after Day 182 plus multiple visit every 12 weeks after Day 182 for a follow-up assessment until study completion.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
TAK-743 300 mg, subcutaneous injection every 2 or 4 weeks
Toyohashi Municipal Hospital
Toyohashi, Aichi-ken, Japan
Asahi General Hospital
Asahi, Chiba, Japan
Tomakomai City Hospital
Tomakomai, Hokkaido, Japan
Kobe University Hospital
Kobe, Hyōgo, Japan
Tokai University Hospital
Isehara, Kanagawa, Japan
Yokohama City University Hospital
Yokohama, Kanagawa, Japan
Osaka University Hospital
Suita, Osaka, Japan
Saiyu Soka Hospital
Sōka, Saitama, Japan
Hiroshima University Hospital
Hiroshima, Japan
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Serious TEAEs and Adverse Events of Special Interest (AESI)
TEAEs were defined as adverse events (AEs) with onset at the time of or following the first exposure to lanadelumab in this study, or medical conditions present prior to the start of treatment but increasing in severity or relationship at the time of or following the start of treatment. Serious TEAEs were defined as any untoward clinical manifestation of signs, symptoms, outcomes (related to IP or not) that at any dose: resulted in death, was life-threatening, required inpatient/prolongation of hospitalization, resulted in persistent/significant disability/incapacity, congenital abnormality/birth defect, important medical event. AESI were defined as investigator-reported hypersensitivity reactions, events of disordered coagulation as bleeding/hypercoagulable. Relatedness to study drug was based on Investigator's discretion. TEAEs were classified and reported as Hereditary Angioedema (HAE) attack and non-HAE attack AEs in this outcome measure.
Time frame: From start of study drug administration (in current study) up to end of study (EOS) (Day 294)
Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Tests
Clinical laboratory testing included clinical serum chemistry, hematology, coagulation and urinalysis. Any change in clinical laboratory abnormalities which were deemed clinically significant by the investigator were recorded as TEAEs.
Time frame: From start of study drug administration (in current study) up to EOS (Day 294)
Number of Participants With Clinically Significant Abnormalities in Vital Signs
Vital signs parameters included blood pressure, heart rate, body temperature and respiratory rate. Any change in vital sign abnormalities which were deemed clinically significant by the investigator were recorded as TEAEs.
Time frame: From start of study drug administration (in current study) up to EOS (Day 294)
Time to First HAE Attack After Day 0 for the Efficacy Evaluation Period for Non-Rollover Participants
A HAE attack was defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). The time to the first HAE attack (days) was calculated from the date and time of the first dose of lanadelumab for the efficacy evaluation period (Day 0 through Day 182) to the date and time of the first in HAE attack after the first open-label dose for the efficacy evaluation period of Day 0 through Day 182. The time to the first Investigator-confirmed HAE attack (days) was summarized using Kaplan-Meier (KM) methods.
Time frame: Day 0 (in current study) through Day 182
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