BAX 826 Dose-Escalation Safety Study

NCT ID: NCT02716194

Last Updated: 2021-05-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-03-03
• Study Completion Date: 2017-01-17

Brief Summary

1. To assess tolerability and safety of BAX 826 after a single infusion in previously treated patients (PTPs) with severe hemophilia A

2. To determine the pharmacokinetic (PK) parameters of BAX 826 compared to ADVATE

3. To evaluate immunogenicity of polysialic acid linked to Factor VIII (FVIII)

Conditions

Hemophilia A

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort 1 - Low dose

The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level.

Group Type: EXPERIMENTAL

BAX 826

Intervention Type: BIOLOGICAL

Octocog alfa

Intervention Type: BIOLOGICAL

Cohort 2 - Medium dose

The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level.

Group Type: EXPERIMENTAL

BAX 826

Intervention Type: BIOLOGICAL

Octocog alfa

Intervention Type: BIOLOGICAL

Cohort 3 - High dose

The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level.

Group Type: EXPERIMENTAL

BAX 826

Intervention Type: BIOLOGICAL

Octocog alfa

Intervention Type: BIOLOGICAL

Interventions

BAX 826

Intervention Type: BIOLOGICAL

Octocog alfa

Intervention Type: BIOLOGICAL

Other Intervention Names

BAX826; ADVATE (Antihemophilic Factor [Recombinant]); ADVATE

Eligibility Criteria

Inclusion Criteria

1. Previously treated male participants aged 18 to 65 years (inclusive) at the time of screening

2. Diagnosis of severe hemophilia A (Factor VIII level <1%)

3. Previously treated with FVIII concentrates for ≥150 documented Exposure Days (EDs)

4. Karnofsky performance score of ≥60

5. Human immunodeficiency virus negative (HIV-); or HIV+ with stable disease

6. Hepatitis C virus negative (HCV-); or HCV+ with chronic stable hepatitis as assessed by the investigator

7. Able to understand and have provided written informed consent including signature on an informed consent form (ICF) approved by an ethics committee (EC)

8. Have provided written authorization for use and disclosure of protected health information

9. Agree to abide by the study schedule and to return for the required assessments

10. Willing and able to comply with the requirements of the protocol

Exclusion Criteria

1. Detectable FVIII inhibitor at screening, with a titer ≥0.6 Bethesda Unit (BU)

2. Documented history of FVIII inhibitors with a titer ≥0.4 BU at any time prior to screening

3. Known clinical hypersensitivity towards mouse or hamster proteins or to polysialic acid (PSA)

4. Scheduled elective surgery during study participation

5. Severe chronic hepatic dysfunction

6. Severe renal impairment

7. Currently receiving, or has recently received (less than 3 months prior to study participation), or is scheduled to receive during the course of the study, other PSA-ylated drugs

8. Have received another investigational drug within 30 days prior to study entry and/or is scheduled to receive additional investigational drug during the course of the study in the context of another investigational drug study

9. Diagnosis of an inherited or acquired hemostatic defect other than hemophilia A

10. Currently receiving, or scheduled to receive during the course of the study, an immune-modulating drug other than antiretroviral chemotherapy

11. Has a clinically significant medical, psychiatric or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect the safety or compliance of the participant during the study

12. Is a family member or employee of the investigator

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

UMHAT "Sv. Georgi", EAD

Plovdiv, , Bulgaria

Werlhof-Institut

Hanover, Lower Saxony, Germany

Medizinische Hochschule Hannover

Hanover, Lower Saxony, Germany

Vivantes Klinikum im Friedrichshain - Landsberger Allee

Berlin, , Germany

Universitaetsklinikum des Saarlandes

Homburg, , Germany

Universitaetsklinikum Gießen

Marburg, , Germany

Semmelweis Egyetem AOK I.sz. Belgyogyaszati Klinika

Budapest, , Hungary

Presidio Ospedaliero di Castelfranco Veneto

Castelfranco Veneto, Treviso, Italy

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Policlinico Umberto I di Roma-Università di Roma La Sapienza

Roma, , Italy

Radboud University Nijmegen Medical Centre

Nijmegen, , Netherlands

Erasmus Medisch Centrum

Rotterdam, , Netherlands

Instytut Hematologii i Transfuzjologii

Warsaw, , Poland

FSBI "Kirov SR Institute of Hematology and Blood Transfusion FMBA"

Kirov, , Russia

FSBI "Hematological Research Center" MoH of RF

Moscow, , Russia

SBEI HPE "Samara State Medical University" of the MoH of the RF

Samara, , Russia

Complejo Hospitalario Universitario A Coruña

A Coruña, La Coruña, Spain

Hospital General Universitario de Alicante

Alicante, , Spain

Hospital Universitari Vall d'Hebron

Barcelona, , Spain

Hospital Universitario La Paz

Madrid, , Spain

Hospital Regional Universitario de Malaga

Málaga, , Spain

Hospital Universitario Son Espases

Palma de Mallorca, , Spain

Royal Cornwall Hospital

Truro, Cornwall, United Kingdom

Royal London Hospital

London, Greater London, United Kingdom

Royal Free Hospital

London, Greater London, United Kingdom

St Thomas' Hospital Centre for Haemostasis & Thrombosis

London, Greater London, United Kingdom

Manchester Royal Infirmary

Manchester, Greater Manchester, United Kingdom

University Hospital of Wales

Cardiff, West Glamorgan, United Kingdom

Countries

Bulgaria; Germany; Hungary; Italy; Netherlands; Poland; Russia; Spain; United Kingdom

References

Tiede A, Allen G, Bauer A, Chowdary P, Collins P, Goldstein B, Jiang HJ, Kӧck K, Takacs I, Timofeeva M, Wolfsegger M, Srivastava S. SHP656, a polysialylated recombinant factor VIII (PSA-rFVIII): First-in-human study evaluating safety, tolerability and pharmacokinetics in patients with severe haemophilia A. Haemophilia. 2020 Jan;26(1):47-55. doi: 10.1111/hae.13878. Epub 2019 Nov 28.

Reference Type: DERIVED

PMID: 31778283 (View on PubMed)

Other Identifiers

2015-004079-60

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

291501

Identifier Type: -

Identifier Source: org_study_id