A Study of BAX 888 in Male Adults With Severe Hemophilia A

Completed · Phase 1/Phase 2

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT03370172

Run by

Baxalta now part of Shire

People needed

4

Starts

2018-02-27

Expected to finish

2024-07-09

Last updated by the study team

2025-09-11

Who can join

Age: 18 and older, up to 75. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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 , and documented evidence of >=3 hemorrhages over the previous 12 months requiring treatment with exogenous FVIII or use of FVIII prophylaxis because of history of frequent bleeding episodes.
• History of greater than (>) 150 exposure days to exogenously administered FVIII concentrates or cryoprecipitate.
• Sexually active men must agree to use barrier contraception (combination of a condom and spermicide) or limit sexual intercourse to post-menopausal, surgically sterilized, or contraception-practicing partners for a minimum of 6 months after administration of BAX 888, or until BAX 888 genomes are no longer detected in the semen, whichever is sooner.
• 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.
• Signed informed consent.

You may not qualify if…

• Bleeding disorder(s) other than hemophilia A.
• Personal laboratory evidence of having developed inhibitors to FVIII protein at any time (>=0.6 Bethesda units [BU] on any single test).
• Documented prior allergic reaction to any FVIII product.
• Anti-Adeno-associated virus, serotype 8 (AAV8) neutralizing antibody titer >=1:5. Participants whose laboratory assessments are less than or equal to (<=) 1:10 may be re-tested within the same screening window and, if eligibility criterion is met on retest, may be enrolled after confirmation by the Sponsor Medical Monitor.
• Known hypersensitivity to prednisolone or prednisone, or to any of the excipients.
• Having a disease in which treatment with prednisolone or prednisone is not tolerated (including but not limited to osteoporosis with vertebral fractures, difficult to control hypertension, and difficult to control diabetes).
• Evidence of markers of potential underlying risk for autoimmune mediated hepatic disease:
• Anti-smooth muscle antibody assay results >=40 (Inova QUANTA LiteTM Actin IgG enzyme-linked immunosorbent assay [ELISA]); values of 31 to 39 will be flagged as possibly abnormal and the Investigator and Medical Monitor will evaluate the participant for eligibility.
• Elevated anti-liver-kidney microsomal antibody type 1 (LKM1) titers.
• Total immunoglobulin G (IgG) >1.5*upper limit of normal (ULN).
• Antinuclear antibody (ANA) titer >1:320; OR ANA titer >1:80 if demonstrated concurrently with alanine aminotransferase (ALT) that is >ULN.
• Active Hepatitis virus (Hepatitis C): As indicated by detectable hepatitis C virus (HCV) ribonucleic acid (RNA) by polymerase chain reaction (PCR).
• Hepatitis B: If surface antigen is positive.
• Seropositive for Human Immunodeficiency Virus (HIV).
• Receiving systemic antiviral and/or interferon therapy within 4 weeks prior to enrollment.
• Clinically significant infections (e.g. systemic fungal infections) requiring systemic treatment.
• Known immune disorder (including myeloma and lymphoma).
• Concurrent chemotherapy or biological therapy for treatment of neoplastic disease or other disorders.
• An absolute neutrophil count <1000 cells per cubic millimeter (cells/mm\^3).
• Markers of hepatic inflammation or cirrhosis as evidenced by 1 or more of the following:
• Platelet count of <150,000/microliter (mcL).
• Serum albumin level is below the central laboratory's lower limit of normal and FibroSURE is >=0.48 (i.e., Metavir staging of F2 or greater). Of note, in participants with a known history of Gilbert's syndrome, a Fibrotest cannot be used for fibrosis testing.
• Total bilirubin >1.5*ULN and direct bilirubin >=0.5 milligram per deciliter (mg/dL).
• ALT or aspartate aminotransferase (AST) >1.0*ULN.
• Alkaline phosphatase (AP) >2.0*ULN.

Where it is running

• Phoenix Childrens Hospital — Phoenix, Arizona, United States
• Orthopaedic Hospital DBA Orthopaedic Hemophilia Treatment Center — Los Angeles, California, United States
• UC Davis Medical Center — Sacramento, California, United States
• University of Colorado Hemophilia & Thrombosis Center — Aurora, Colorado, United States
• Mount Sinai Medical Center — New York, New York, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Medical University of South Carolina (MUSC) — Charleston, South Carolina, United States
• Gulf States Hemophilia and Thrombophilia Center — Houston, Texas, United States
• AKH - Medizinische Universität Wien — Vienna, Austria
• Hôpital de la Timone — Marseille, Bouches-du-Rhône, France
• Hôpital Morvan — Brest, Finistere, France
• CHU Rennes - Hopital Pontchaillou — Rennes, Ille Et Vilaine, France
• CHU Tours - Hôpital Trousseau — Tours, Indre Et Loire, France
• CHU de Nantes Site Hotel Dieu — Nantes, Loire Atlantique, France
• Hopital Jeanne de Flandre - CHU Lille — Lille, Nord, France
• Groupement Hospitalier Est- Hôpital Louis Pradel — Bron, Rhone, France
• Hôpital Bicêtre — Le Kremlin-Bicêtre, Val De Marne, France
• Klinikum der Johann Wolfgang Goethe-Universitaet — Frankfurt am Main, Hesse, Germany
• Universitaetsklinikum Carl Gustav Carus TU Dresden — Dresden, Saxony, Germany
• Vivantes Klinikum im Friedrichshain — Berlin, Germany
• Semmelweis Egyetem — Budapest, Hungary
• Hospital Universitari Vall d'Hebron — Barcelona, Spain
• Hospital Universitario La Paz — Madrid, Spain
• Hospital Regional Universitario de Malaga — Málaga, Spain
• Hospital Clinico Universitario de Salamanca — Salamanca, Spain

Full record on ClinicalTrials.gov

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