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BAX 888

Phase 1

Hemophilia A | Small molecule | Hematology |Takeda Pharmaceutical Company Limited|Last Updated: Sep 11, 2025

Success Probability

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment4

FDA Designations

No designations recorded

Clinical trial landscape

BAX 888 · 1 trial · 1 indication

Phase 1 1
NCT03370172A Study of BAX 888 in Male Adults With Severe Hemophilia AHemophilia A
COMPLETED4 Analytics
PHASE1COMPLETED
A Study of BAX 888 in Male Adults With Severe Hemophilia A
Hemophilia AUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With BAX 888-Related Adverse Events (AEs)
From first dose up to end of the study (approximately 6 years)

An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A Serious adverse event (SAE) is an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. AEs include both serious and non-serious adverse events including development of FVIII inhibitory antibodies, clinically significant changes in standard laboratory parameters, physical exam, and vital signs.

Secondary Endpoints

Change From Baseline in Circulating Plasma FVIII Activity Level
Baseline, up to Month 60
Number of Participants With Clinically Significant Change From Baseline in Circulating Plasma FVIII Antigen Level
Baseline, up to Month 60
Annualized Bleed Rate (ABR)
Up to approximately 6 years 4 months
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposePREVENTION

Treatment Arms

ArmTypeDescription
Cohort 1: BAX 888 2.0*10^12 cp/kgEXPERIMENTALCohort 1 participants received 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).
Cohort 2: BAX 888 6.0*10^12 cp/kgEXPERIMENTALCohort 2 participants received 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).

Interventions

NameTypeDescription
BAX 888DRUGParticipants will receive a single peripheral IV infusion of BAX 888 in Cohort 1 and 2 Day 0.
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Eligibility Criteria

Age Range18 Years to 75 Years
SexMALE
Healthy VolunteersNo
Study Sites26

Inclusion Criteria: * Male, aged 18 to 75 years at the time of screening. * Established severe hemophilia A (FVIII:C \<1%, measured following \>=5 days without FVIII treatment) and/or documented intron 1 inversion or intron 22 inversion mutation in the F8 gene, consistent with severe hemophilia A ,...

Countries:United StatesAustriaFranceGermanyHungarySpain
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Frequently asked questions about BAX 888

What is BAX 888 used for?

BAX 888 is an investigational small molecule being developed for the treatment of hemophilia A, a bleeding disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes BAX 888?

BAX 888 is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker symbol TAK. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with hemophilia A.

What phase is BAX 888 in?

BAX 888 is in Phase 1 clinical development. It is an investigational drug and has not yet been approved for commercial use. The Phase 1 trial has been completed, and the drug remains under investigation for hemophilia A.

What clinical trials is BAX 888 in?

BAX 888 has one completed Phase 1 clinical trial registered as NCT03370172, titled 'A Study of BAX 888 in Male Adults With Severe Hemophilia A.' The trial enrolled 4 participants and was conducted in the United States, Austria, France, Germany, Hungary, and Spain.

Is BAX 888 the same as any other drug?

No alternative names for BAX 888 have been disclosed. The drug is identified solely by its code name BAX 888 in clinical trial registrations and development documentation.