BAX 855 Continuation

NCT ID: NCT01945593

Last Updated: 2021-05-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 218 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-10-15
• Study Completion Date: 2018-03-02

Brief Summary

To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.

Conditions

Hemophilia A

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Fixed BAX855 prophylaxis

45-80 IU/kg twice weekly to once per week.

Group Type: EXPERIMENTAL

BAX855

Intervention Type: BIOLOGICAL

Antihemophilic Factor (Recombinant), PEGylated

Pharmacokinetic (PK)-tailored BAX 855 prophylaxis

PK-tailored prophylactic BAX855 regimen based on participant's individual PK profile to maintain a Factor VIII (FVIII) trough level

Group Type: EXPERIMENTAL

BAX855

Intervention Type: BIOLOGICAL

Antihemophilic Factor (Recombinant), PEGylated

Interventions

BAX855

Antihemophilic Factor (Recombinant), PEGylated

Intervention Type: BIOLOGICAL

Other Intervention Names

ADYNOVATE; BAX 855; PEGylated Recombinant Factor VIII

Eligibility Criteria

Inclusion Criteria

Participants Transitioning from Other BAX 855 Studies:

Participants transitioning from other BAX 855 studies can be provided with the continuation study informed consent form (ICF) prior to the end of study visit to review and consider participation in this continuation study. These participants will complete any additional screening assessments within 2 weeks of the previous study's end of study visit and will return to the study site within 6 (± 1) weeks of the previous study end of study visit to confirm eligibility for this continuation study.

• Participants transitioning from other BAX 855 studies who meet ALL of the following criteria are eligible for this study:

1. Participant has completed a previous BAX 855 study and is willing to immediately transition into this continuation study.

2. Participant is ≤75 years of age at screening of the previous BAX 855 study.

3. Participant continues to have a Karnofsky (for participants aged ≥ 16 years) or Lansky (for participants aged < 16 years) performance score of ≥ 60.

4. Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening.

5. Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.

6. If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.

7. Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.

• BAX 855 Naïve Participants:

BAX 855 naïve participants who are ≥ 12 years of age can only be enrolled in this continuation study after enrollment in the phase 2/3 pivotal study is closed. BAX 855 naïve participants who are < 12 years of age can only be enrolled in this continuation study after enrollment in the pediatric previously treated patient (PTP) study is closed.

• Enrolment of BAX 855 naïve participants will only start once the sponsor has notified the study sites accordingly.

BAX 855 naïve participants who meet ALL of the following criteria are eligible for this study:

1. Participant is ≤75 years of age at screening.

2. Participant is naïve to BAX 855.

3. Participant has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory at screening after at least a 72-hour washout period.

4. Participant aged ≥ 6 years has documented previous treatment with plasma-derived FVIII or rFVIII for ≥ 150 exposure days (EDs).

5. Participant aged < 6 years has documented previous treatment with plasma-derived FVIII concentrates or rFVIII for ≥ 50 EDs.

6. Participant is currently receiving prophylaxis or on-demand therapy with FVIII.

7. Participant has a Karnofsky (for participants aged ≥ 16 years) or Lansky (for participants aged < 16 years) performance score of ≥ 60.

8. Participant is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening.

9. Participant is HCV- by antibody or PCR testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.

10. If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.

11. Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.

EXCLUSION CRITERA

• Participants Transitioning from Other BAX 855 Studies:

Participants transitioning from other BAX 855 studies who meet ANY of the following criteria are not eligible for this study:

1. Participant had detectable factor VIII (FVIII) inhibitory antibodies (≥ 0.6 Bethesda unit (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.

2. Participant has developed FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay as determined at central laboratory in a previous BAX 855 study).

3. Participant has acquired a hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease) in a previous BAX 855 study.

4. Participant has severe chronic hepatic dysfunction (eg, ≥ 5 times upper limit of normal alanine aminotransferase [ALT], as confirmed by central laboratory at screening).

5. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.

6. Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.

7. Participant is scheduled to use other PEGylated drugs during study participation.

8. Participant is planning to take part in any other clinical study during the course of the continuation study, with the exception of any other parallel BAX 855 study.

9. Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.

10. Participant is a family member or employee of the investigator.

• BAX 855 Naïve Participants:

BAX 855 naïve participants who meet ANY of the following criteria are not eligible for this study:

1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.

2. Participant has history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening.

3. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).

4. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG), or Tween 80.

5. Participant has severe chronic hepatic dysfunction eg, ≥ 5 times upper limit of normal ALT, as confirmed by central laboratory at screening).

6. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.

7. Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.

8. Participant has current or recent (< 30 days) use of other PEGylated drugs prior to study participation or scheduled use of such drugs during study participation.

9. Participant has participated in another clinical study involving an IP other than BAX 855 or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product (IP) or investigational device during the course of this study.

10. Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.

11. Participant is a family member or employee of the investigator.

• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Baxalta Innovations GmbH, now part of Shire

INDUSTRY

Sponsor Role: collaborator

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Phoenix Childrens Hospital

Phoenix, Arizona, United States

University of Colorado

Aurora, Colorado, United States

University of Florida College of Medicine

Gainesville, Florida, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States

University of Kentucky Medical Center

Lexington, Kentucky, United States

University of Louisville

Louisville, Kentucky, United States

Tulane University School of Medicine

New Orleans, Louisiana, United States

Children's Mercy Hospitals & Clinics

Kansas City, Missouri, United States

North Shore-Long Island Jewish Health System

New Hyde Park, New York, United States

New York - Presbyterian/Weill Cornell Medical Center

New York, New York, United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Duke University Medical Center

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

University of Oklahoma

Oklahoma City, Oklahoma, United States

Penn State Hershey Cancer Center

Hershey, Pennsylvania, United States

Palmetto Health Richland

Columbia, South Carolina, United States

University of Utah

Salt Lake City, Utah, United States

Puget Sound Blood Group

Seattle, Washington, United States

Royal Adelaide Hospital

Adelaide, South Australia, Australia

The Alfred Hospital

Melbourne, Victoria, Australia

Fremantle Hospital

Fremantle, Western Australia, Australia

Landes-Frauen-und Kinderklinik Linz

Linz, , Austria

Universitatsklinik fur Innere Medizin I

Vienna, , Austria

UMHAT "Sv. Georgi", EAD

Plovdiv, , Bulgaria

SHAT of Oncohaematology Diseases

Sofia, , Bulgaria

MHAT 'Sv. Marina', EAD

Varna, , Bulgaria

Fakultni nemocnice Brno

Brno, , Czechia

Fakultni nemocnice Olomouc

Olomouc, , Czechia

Fakultni nemocnice v Motole

Prague, , Czechia

Werlhof-Institut GmbH

Hanover, Lower Saxony, Germany

Gerinnungszentrum Rhein-Ruhr

Duisburg, North Rhine-Westphalia, Germany

Vivantes Klinikum im Friedrichshain - Landsberger Allee

Berlin, , Germany

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Prince of Wales Hospital

Shatin, , Hong Kong

Rambam Health Care Campus

Haifa, , Israel

Chaim Sheba Medical Center

Ramat Gan, , Israel

Nagoya University Hospital

Nagoya, Aichi-ken, Japan

University of Occupational and Environmental Health Hospital

Kitakyushu-shi, Fukuoka, Japan

Hiroshima University Hospital

Hiroshima, Hiroshima, Japan

St. Marianna University School of Medicine Hospital

Kawasaki-shi, Kanagawa, Japan

Nara Medical University Hospital

Kashihara-shi, Nara, Japan

Ogikubo Hospital

Suginami City, Tokyo, Japan

Tokyo Medical University Hospital

Shinjuku-ku, Tokyo-To, Japan

Vilnius University Hospital Santariskiu Clinics, Public Institution

Vilnius, , Lithuania

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

Vilnius, , Lithuania

Penang General Hospital

George Town, Pulau Pinang, Malaysia

Hospital Umum Sarawak

Kuching, Sarawak, Malaysia

Hospital Sibu

Sibu, Sarawak, Malaysia

Hospital Ampang

Ampang, Selangor, Malaysia

Pusat Darah Negara

Kuala Lumpur, , Malaysia

Hospital Pulau Pinang

Pulau Pinang, , Malaysia

Academisch Medisch Centrum

Amsterdam, , Netherlands

Uniwersyteckie Centrum Kliniczne

Gdansk, , Poland

Wojewodzki Szpital Specjalistyczny im.M.Kopernika w Lodzi

Lodz, , Poland

Sanador SRL

Bucharest, , Romania

LLC "Alba Dent"

Kirov, , Russia

Regional Clinical Hospital

Krasnoyarsk, , Russia

Chonnam National University Hwasun Hospital

Hwasun-gun, Jeollanam-do, South Korea

Pusan National University Hospital

Busan, , South Korea

Eulji University Hospital

Daejeon, , South Korea

Kyung Hee University Hospital at Gangdong

Seoul, , South Korea

Ulsan University Hospital

Ulsan, , South Korea

Hospital Universitari Son Espases

Palma de Mallorca, Balearic Islands, Spain

Complejo Hospitalario Universitario A Coruña

A Coruña, La Coruña, Spain

Hospital Regional Universitario de Malaga

Málaga, Málaga, Spain

Hospital Universitario La Paz

Madrid, , Spain

Hospital Universitari i Politecnic La Fe

Valencia, , Spain

Sjukhusapoteket Malmo

Malmo, , Sweden

Karolinska

Stockholm, , Sweden

Universitaetsspital Zuerich

Zurich, , Switzerland

Taichung Veterans General Hospital

Taichung, , Taiwan

Tri-Service General Hospital

Taipei, , Taiwan

Ankara University Medical Faculty

Ankara, , Turkey (Türkiye)

Akdeniz University Faculty of Medicine

Antalya, , Turkey (Türkiye)

Istanbul University Cerrahpasa Medical Faculty

Istanbul, , Turkey (Türkiye)

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

Donetsk, , Ukraine

SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

Lviv, , Ukraine

Bristol Royal Hospital for Children

Bristol, Avon, United Kingdom

St Thomas' Hospital

London, Greater London, United Kingdom

Royal Free Hospital

London, Greater London, United Kingdom

Great Ormond Street Hospital for Children

London, Greater London, United Kingdom

Royal Manchester Children's Hospital

Manchester, Greater Manchester, United Kingdom

Southampton General Hospital

Southampton, Hampshire, United Kingdom

Leicester Royal Infirmary

Leicester, Leicestershire, United Kingdom

Birmingham Children's Hospital

Birmingham, West Midlands, United Kingdom

The Churchill Hospital

Oxford, , United Kingdom

Countries

United States; Australia; Austria; Bulgaria; Czechia; Germany; Hong Kong; Israel; Japan; Lithuania; Malaysia; Netherlands; Poland; Romania; Russia; South Korea; Spain; Sweden; Switzerland; Taiwan; Turkey (Türkiye); Ukraine; United Kingdom

Provided Documents

Document Type: Study Protocol: Protocol

View Document

Document Type: Study Protocol: Protocol Amendment 1

View Document

Document Type: Study Protocol: Protocol Amendment 4

View Document

Document Type: Study Protocol: Protocol Amendment 7

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2013-002236-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

261302

Identifier Type: -

Identifier Source: org_study_id