NCT04394286

A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

An Open-Label, Multinational, Phase 1/2 Study of the Safety and Dose Escalation of SHP648, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua in Hemophilia B Subjects

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Baxalta now part of Shire · Industry
• Sex: Male
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and dose escalation of SHP648 an adeno-associated viral vector for gene transfer in hemophilia B participants.

Detailed description

This study will consists of 3 dose cohorts with 2-7 participants in each of the three ascending dose cohorts. Initially 2 participants will be dosed in Cohort 1, followed by dosing of up to 5 additional participants if the cohort is expanded. Participants in cohort 2 and 3 will receive 2-fold or 3-fold dose escalation to their respective preceding cohort doses if required.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	SHP648	Participants will receive a single IV infusion of SHP648 in Cohort 1, 2, 3 on Day 0.

Timeline

• Start date: 2020-05-13
• Primary completion: 2021-05-03
• Completion: 2021-05-03
• First posted: 2020-05-19
• Last updated: 2022-05-19
• Results posted: 2022-05-19

Locations

2 sites across 2 countries: Spain, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study