21 Participants Needed

BAX 888 for Hemophilia A

Recruiting at 32 trial locations
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

What is the purpose of this trial?

The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults.Participants will receive one infusion with BAX 888 at the hemophilia treatment center. During the study, participants will visit their study clinic multiple times.

Research Team

SD

Study Director

Principal Investigator

Shire

Eligibility Criteria

This trial is for male adults aged 18-75 with severe hemophilia A, who have had more than 150 exposure days to FVIII treatments and at least three bleeding episodes in the past year. Participants must be willing to use barrier contraception and comply with study requirements like providing semen samples and keeping a diary of bleeding episodes.

Inclusion Criteria

Signed informed consent
I am a man between 18 and 75 years old.
Participant is willing and able to comply with the requirements of the protocol, including provision of semen samples, maintenance of a diary of bleeding episodes and FVIII protein use
See 3 more

Exclusion Criteria

Received an investigational intervention or participated in another clinical trial within 4 weeks prior to enrollment or within 5 half-lives of the investigational drug administration, whichever is longer
My body does not strongly fight off the AAV8 virus used in treatment.
I have a known immune disorder such as myeloma or lymphoma.
See 24 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous infusion of BAX 888 at varying doses depending on cohort assignment

1 day
1 visit (in-person)

Dose Escalation and Expansion

Participants are monitored for FVIII activity levels to determine dose escalation or cohort expansion

14 weeks

Follow-up

Participants are monitored for safety, effectiveness, and immune response after treatment

Up to 5 years

Treatment Details

Interventions

  • BAX 888
Trial Overview The trial is testing BAX 888's safety and determining its proper dose for treating severe hemophilia A. Participants will receive one infusion of BAX 888 at a treatment center and make multiple visits to their study clinic for monitoring.
Participant Groups
3Treatment groups
Experimental Treatment
Group I: Cohort 3Experimental Treatment1 Intervention
Cohort 3 participants will receive a single peripheral IV infusion of BAX 888 at a dose of 1.2\*10\^13 cp/kg on the day of dosing (Day 0).
Group II: Cohort 2Experimental Treatment1 Intervention
Cohort 2 participants will receive a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
Group III: Cohort 1Experimental Treatment1 Intervention
Cohort 1 participants will receive a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).

Find a Clinic Near You

Who Is Running the Clinical Trial?

Baxalta now part of Shire

Lead Sponsor

Trials
111
Recruited
9,100+

Flemming Ornskov

Baxalta now part of Shire

Chief Executive Officer since 2013

MD, MPH

Ludwig N. Hantson

Baxalta now part of Shire

Chief Medical Officer since 2015

PhD

Baxalta Innovations GmbH, now part of Shire

Industry Sponsor

Trials
19
Recruited
8,000+

Takeda Development Center Americas, Inc.

Industry Sponsor

Trials
58
Recruited
10,800+
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