NCT04687137

Expanded Access Program With Lanadelumab for Japanese People With Hereditary Angioedema

Open-arm, Japan Expanded Access Program With Lanadelumab (TAK-743) for Japanese Patients With Hereditary Angioedema

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Takeda · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
DRUG	TAK-743 300 mg	TAK-743 300 mg, subcutaneous injection every 2 or 4 weeks

Timeline

• Start date: 2021-02-10
• Primary completion: 2022-06-18
• Completion: 2022-06-18
• First posted: 2020-12-29
• Last updated: 2023-06-26
• Results posted: 2023-06-26

Locations

9 sites across 1 country: Japan

Regulatory

• FDA-regulated drug study