Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

NCT ID: NCT01736475

Last Updated: 2021-05-20

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

159 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-01-31

Study Completion Date

2014-07-17

Brief Summary

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To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.

Detailed Description

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Conditions

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Hemophilia A

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

Study Groups

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Prophylaxis

Group Type EXPERIMENTAL

Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method

Intervention Type BIOLOGICAL

Pharmacokinetic (PK) evaluation of ADVATE

PEGylated Recombinant Factor VIII

Intervention Type BIOLOGICAL

Pharmacokinetic (PK) evaluation of BAX 855

PEGylated Recombinant Factor VIII

Intervention Type BIOLOGICAL

Prophylaxis treatment

On-demand

Group Type EXPERIMENTAL

PEGylated Recombinant Factor VIII

Intervention Type BIOLOGICAL

On-demand treatment

Interventions

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Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method

Pharmacokinetic (PK) evaluation of ADVATE

Intervention Type BIOLOGICAL

PEGylated Recombinant Factor VIII

Pharmacokinetic (PK) evaluation of BAX 855

Intervention Type BIOLOGICAL

PEGylated Recombinant Factor VIII

Prophylaxis treatment

Intervention Type BIOLOGICAL

PEGylated Recombinant Factor VIII

On-demand treatment

Intervention Type BIOLOGICAL

Other Intervention Names

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ADVATE BAX 855 BAX 855 BAX 855

Eligibility Criteria

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Inclusion Criteria

* Participant and/or legal representative has/have voluntarily provided signed informed consent
* Participant is 12 to 65 years old at the time of screening
* Participant is male with severe hemophilia A (Factor VIII (FVIII) clotting activity \< 1%) as confirmed by central laboratory at screening after the appropriate washout period or a documented FVIII clotting activity \<1%
* Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥150 documented exposure days (EDs)
* Participant is currently receiving prophylaxis or on-demand therapy with FVIII
* Participant is willing and able to comply with the requirements of the protocol

Exclusion Criteria

* Participant has detectable FVIII inhibitory antibodies (≥ 0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
* Participant has history of FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time prior to screening
* Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
Minimum Eligible Age

12 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Baxalta now part of Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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University of Colorado

Aurora, Colorado, United States

Site Status

University of Florida, College of Medicine

Gainesville, Florida, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States

Site Status

University of Kentucky Medical Center

Lexington, Kentucky, United States

Site Status

University of Louisville Hospital

Louisville, Kentucky, United States

Site Status

Tulane University Medical School

New Orleans, Louisiana, United States

Site Status

Children's Mercy Hospitals & Clinics

Kansas City, Missouri, United States

Site Status

Weill Cornell Medical College-New York Presbyterian Hospital

New York, New York, United States

Site Status

University of North Carolina Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

The Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Palmetto Health

Columbia, South Carolina, United States

Site Status

University of Utah Health Sciences Center

Salt Lake City, Utah, United States

Site Status

Puget Sound Blood Group

Seattle, Washington, United States

Site Status

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Site Status

The Alfred Hospital

Clayton, Victoria, Australia

Site Status

Fremantle Hospital

Fremantle, Western Australia, Australia

Site Status

Hollywood Specialist Centre

Nedlands, Western Australia, Australia

Site Status

Landes-Frauen-und Kinderklinik Linz

Linz, , Austria

Site Status

AKH - Medizinische Universität Wien

Vienna, , Austria

Site Status

SHAT of Oncohaematology Diseases

Sofia, , Bulgaria

Site Status

Fakultni nemocnice Brno

Brno, , Czechia

Site Status

Fakultni nemocnice Olomouc

Olomouc, , Czechia

Site Status

Fakultni nemocnice v Motole

Prague, , Czechia

Site Status

Gerinnungszentrum Rhein-Ruhr

Duisburg, North Rhine-Westphalia, Germany

Site Status

Vivantes Klinikum im Friedrichshain - Landsberger Allee

Berlin, , Germany

Site Status

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Site Status

Werlhof-Institut MVZ

Hanover, , Germany

Site Status

Rambam Health Care Campus

Haifa, , Israel

Site Status

Chaim Sheba Medical Center

Tel Aviv, , Israel

Site Status

Nagoya University Hospital

Nagoya, Aichi-ken, Japan

Site Status

University of Occupational and Environmental Health Hospital

Kitakyushu-shi, Fukuoka, Japan

Site Status

Hiroshima University Hospital

Hiroshima, Hiroshima, Japan

Site Status

Hyogo College of Medicine Hospital

Nishinomiya-shi, Hyōgo, Japan

Site Status

St. Marianna University School of Medicine Hospital

Kawasaki-shi, Kanagawa, Japan

Site Status

Nara Medical University Hospital

Kashihara-shi, Nara, Japan

Site Status

Tokyo Medical University Hospital

Shinjuku-ku, Tokyo-To, Japan

Site Status

Ogikubo Hospital

Suginami-ku, Tokyo-To, Japan

Site Status

Vilnius University Hospital Santariskiu Clinics, Public Institution

Vilnius, , Lithuania

Site Status

Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos

Vilnius, , Lithuania

Site Status

Hospital Pulau Pinang

George Town, Pulau Pinang, Malaysia

Site Status

Hospital Tengku Ampuan Rahimah

Klang, Selangor, Malaysia

Site Status

Pusat Darah Negara

Kuala Lumpur, , Malaysia

Site Status

Academisch Medisch Centrum

Amsterdam, , Netherlands

Site Status

Uniwersyteckie Centrum Kliniczne

Gdansk, , Poland

Site Status

Wojewodzki Szpital Specjalistyczny im.M.Kopernika w Lodzi

Lodz, , Poland

Site Status

Sanador SRL

Bucharest, , Romania

Site Status

Chonnam National University Hwasun Hospital

Hwasun, Jeollanam-do, South Korea

Site Status

Pusan National University Hospital

Busan, , South Korea

Site Status

Eulji University Hospital

Daejeon, , South Korea

Site Status

Kyung hee University Hospital at Gangdong

Seoul, , South Korea

Site Status

Ulsan University Hospital

Ulsan, , South Korea

Site Status

Hospital Universitari Son Espases

Palma de Mallorca, Balearic Islands, Spain

Site Status

Complejo Hospitalario Universitario A Coruña

A Coruña, La Coruña, Spain

Site Status

Hospital Regional Universitario de Malaga

Málaga, Málaga, Spain

Site Status

Hospital Universitari i Politecnic La Fe

Valencia, , Spain

Site Status

Skånes Universitetssjukhus, Malmö

Malmo, , Sweden

Site Status

Karolinska Universitetssjukhuset, Solna

Stockholm, , Sweden

Site Status

Universitatsspital Zurich

Zurich, , Switzerland

Site Status

Tri-Service General Hospital

Taipei, , Taiwan

Site Status

SI V.K.Gusak Emergency and Reconstructive Surgery Institute of NAMSU Center of IT

Donetsk, , Ukraine

Site Status

SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

Lviv, , Ukraine

Site Status

Bristol Royal Hospital for Children

Bristol, Avon, United Kingdom

Site Status

Royal Free Hospital

London, Greater London, United Kingdom

Site Status

St Thomas' Hospital

London, Greater London, United Kingdom

Site Status

Manchester Royal Infirmary

Manchester, Greater Manchester, United Kingdom

Site Status

Royal Manchester Children's Hospital

Manchester, Greater Manchester, United Kingdom

Site Status

Leicester Royal Infirmary

Leicester, Leicestershire, United Kingdom

Site Status

Churchill Hospital

Oxford, Oxfordshire, United Kingdom

Site Status

Countries

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United States Australia Austria Bulgaria Czechia Germany Israel Japan Lithuania Malaysia Netherlands Poland Romania South Korea Spain Sweden Switzerland Taiwan Ukraine United Kingdom

References

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Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, Engl W, Dyck-Jones J, Fuerlinger M, Patrone L, Ewenstein B, Abbuehl B. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015 Aug 27;126(9):1078-85. doi: 10.1182/blood-2015-03-630897. Epub 2015 Jul 8.

Reference Type DERIVED
PMID: 26157075 (View on PubMed)

Other Identifiers

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2012-003599-38

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

261201

Identifier Type: -

Identifier Source: org_study_id

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