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Hemophilia APhase 2/3NCT01736475

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

A Phase 2/3 study testing Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method and PEGylated Recombinant Factor VIII in people with Hemophilia A, run by Baxalta now part of Shire at 72 sites in 20 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 12 to 65 years, men only
Conditions the study is for
Hemophilia A
What is being tested?
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method and PEGylated Recombinant Factor VIII
Could I receive a placebo?
No, everyone receives an active treatment
How many people
159 participants
Where
72 sitesin 20 countries
Study dates
31 Jan 2013to 17 Jul 2014
Phase
Phase 2/3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 65 years
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Main 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

Main 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).

Trial locations and contacts

72 sites in 20 countries.

72 sites in 20 countries

United States17 sites

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322

  • University of Colorado

    Aurora, Colorado, 80045

  • University of North Carolina Chapel Hill

    Chapel Hill, North Carolina, 27599

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • Palmetto Health

    Columbia, South Carolina, 29203

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • University of Florida, College of Medicine

    Gainesville, Florida, 32610

  • Penn State Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • Children's Mercy Hospitals & Clinics

    Kansas City, Missouri, 66211

  • University of Kentucky Medical Center

    Lexington, Kentucky, 40504

  • University of Louisville Hospital

    Louisville, Kentucky, 40202

  • Tulane University Medical School

    New Orleans, Louisiana, 70124

  • Weill Cornell Medical College-New York Presbyterian Hospital

    New York, New York, 10065

  • Bleeding and Clotting Disorders Institute

    Peoria, Illinois, 61614

  • University of Utah Health Sciences Center

    Salt Lake City, Utah, 84132

  • Puget Sound Blood Group

    Seattle, Washington, 98104

Other trials in United States

Japan8 sites

  • Hiroshima University Hospital

    Hiroshima, Hiroshima, 734-8551

  • Nara Medical University Hospital

    Kashihara-shi, Nara, 634-8522

  • St. Marianna University School of Medicine Hospital

    Kawasaki-shi, Kanagawa, 216-8511

  • University of Occupational and Environmental Health Hospital

    Kitakyushu-shi, Fukuoka, 807-8556

  • Nagoya University Hospital

    Nagoya, Aichi-ken, 466-8560

  • Hyogo College of Medicine Hospital

    Nishinomiya-shi, Hyōgo, 663-8501

  • Tokyo Medical University Hospital

    Shinjuku-ku, Tokyo-To, 160-0023

  • Ogikubo Hospital

    Suginami-ku, Tokyo-To, 167-8515

Other trials in Japan

United Kingdom7 sites

  • Bristol Royal Hospital for Children

    Bristol, Avon, BS2 8BJ

  • Leicester Royal Infirmary

    Leicester, Leicestershire, LE1 5WW

  • Royal Free Hospital

    London, Greater London, NW3 2QG

  • St Thomas' Hospital

    London, Greater London, SE1 7EH

  • Manchester Royal Infirmary

    Manchester, Greater Manchester, M13 9WL

  • Royal Manchester Children's Hospital

    Manchester, Greater Manchester, M13 9WL

  • Churchill Hospital

    Oxford, Oxfordshire, OX3 7LJ

Other trials in United Kingdom

South Korea5 sites

  • Pusan National University Hospital

    Busan, 602-739

  • Eulji University Hospital

    Daejeon, 302-120

  • Chonnam National University Hwasun Hospital

    Hwasun, Jeollanam-do, 519-763

  • Kyung hee University Hospital at Gangdong

    Seoul, 134-727

  • Ulsan University Hospital

    Ulsan, 682-714

Other trials in South Korea

Australia4 sites

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000

  • The Alfred Hospital

    Clayton, Victoria, 3168

  • Fremantle Hospital

    Fremantle, Western Australia, 6160

  • Hollywood Specialist Centre

    Nedlands, Western Australia, 6009

Other trials in Australia

Germany4 sites

  • Vivantes Klinikum im Friedrichshain - Landsberger Allee

    Berlin, 10249

  • Gerinnungszentrum Rhein-Ruhr

    Duisburg, North Rhine-Westphalia, 47051

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246

  • Werlhof-Institut MVZ

    Hanover, 30159

Other trials in Germany

Spain4 sites

  • Complejo Hospitalario Universitario A Coruña

    A Coruña, La Coruña, 15006

  • Hospital Regional Universitario de Malaga

    Málaga, Málaga, 29010

  • Hospital Universitari Son Espases

    Palma de Mallorca, Balearic Islands, 07010

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026

Other trials in Spain

Czechia3 sites

  • Fakultni nemocnice Brno

    Brno, 61300

  • Fakultni nemocnice Olomouc

    Olomouc, 775 20

  • Fakultni nemocnice v Motole

    Prague, 150 06

Other trials in Czechia

Malaysia3 sites

  • Hospital Pulau Pinang

    George Town, Pulau Pinang, 10990

  • Hospital Tengku Ampuan Rahimah

    Klang, Selangor, 41200

  • Pusat Darah Negara

    Kuala Lumpur, 50400

Other trials in Malaysia

Austria2 sites

  • Landes-Frauen-und Kinderklinik Linz

    Linz, 4020

  • AKH - Medizinische Universität Wien

    Vienna, 1090

Other trials in Austria

Israel2 sites

  • Rambam Health Care Campus

    Haifa, 3109601

  • Chaim Sheba Medical Center

    Tel Aviv, 64239

Other trials in Israel

Lithuania2 sites

  • Vilnius University Hospital Santariskiu Clinics, Public Institution

    Vilnius, 08661

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

    Vilnius, LT-08406

Other trials in Lithuania

Poland2 sites

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-952

  • Wojewodzki Szpital Specjalistyczny im.M.Kopernika w Lodzi

    Lodz, 93-510

Other trials in Poland

Sweden2 sites

  • Skånes Universitetssjukhus, Malmö

    Malmö, 20502

  • Karolinska Universitetssjukhuset, Solna

    Stockholm, 17176

Other trials in Sweden

Ukraine2 sites

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

    Donetsk, 83045

  • SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

    Lviv, 79044

Other trials in Ukraine

Bulgaria1 site

  • SHAT of Oncohaematology Diseases

    Sofia, 1527

Other trials in Bulgaria

Netherlands1 site

  • Academisch Medisch Centrum

    Amsterdam, 1105 AZ

Other trials in Netherlands

Romania1 site

  • Sanador SRL

    Bucharest, 011026

Other trials in Romania

Switzerland1 site

  • Universitatsspital Zurich

    Zurich, 8091

Other trials in Switzerland

Taiwan1 site

  • Tri-Service General Hospital

    Taipei, 11490

Other trials in Taiwan

About this study

Official title: A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients With Severe Hemophilia A

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.

Conditions
Hemophilia A
Being tested
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII
Medical subject headings
Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Antihemophilic Factor (Recombinant) - Plasma/Albumin Free MethodBiological

    Also called ADVATE

    Pharmacokinetic (PK) evaluation of ADVATE

  • PEGylated Recombinant Factor VIIIBiological

    Also called BAX 855

    Pharmacokinetic (PK) evaluation of BAX 855

  • PEGylated Recombinant Factor VIIIBiological

    Also called BAX 855

    Prophylaxis treatment

  • PEGylated Recombinant Factor VIIIBiological

    Also called BAX 855

    On-demand treatment

Study groups in NCT01736475
Study groupRoleWhat they receive
ProphylaxisExperimentalNot described
Receives: Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method; Biological: PEGylated Recombinant Factor VIII
On-demandExperimentalNot described
Receives: Biological: PEGylated Recombinant Factor VIII

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Annualized Bleeding Rate (ABR)

    Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy.

    Measured: 9 months

Secondary outcomes (30)

  1. Rate of Success of BAX 855 for Treatment of Bleeding Episodes

    Success in the control of bleeding was defined as a rating of excellent or good using the Efficacy Rating Scale for Treatment of Bleeding Episodes measured 24 hours after initiation of treatment for the bleeding episode. EXCELLENT: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring. GOOD: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. FAIR: Probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion. Required more than 1 infusion for complete resolution. NONE: No improvement or condition worsens.

    Measured: At least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm.

  2. Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes

    Measured: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].

  3. Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes

    Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)*(12/365.2425)

    Measured: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].

  4. Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion

    Measured: Prophylactic Infusion: ≥50 exposure days or 6 months (±2 weeks), whichever occurs last. PK Infusion: PK #1 Pre-infusion within 30 minutes; Post-infusion 10 min, and 0.5, 1, 3, 6, 24, 32, 48, 56 hours (h). PK #2 also at Post-infusion 96h

  5. Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis

    Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis.

    Measured: Treatment of Bleeding Episode (BE): Minor/Moderate BE every 12 to 24 hours until bleeding is resolved; Major BE every 8 to 12 hours until bleeding is resolved. Per BE for Maintenance of Hemostasis: within 48 hours after bleeding episode resolution.

  6. Percentage of Participants With Adverse Events

    Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Measured: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].

  7. Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination

    Number of participants who received BAX855, with immunogenicity data from study completion/termination visit. FVIII = factor VIII; PEG-VIII = polyethylene glycol-factor VIII; Anti-CHO = Anti-Chinese hamster ovary

    Measured: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].

  8. Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study

    The HAEMO-SYM has two subscales: pain and bleeds. HAEMO-SYM subscale scores are calculated by taking the mean of the items in each subscale and transforming them to a 0 (none or absent) to 100 (very severe) scale. Given that higher scores indicate more severe symptoms on the Haemo-SYM and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates an improvement (reduction in symptoms). Conversely, a positive change score indicates worsening symptoms.

    Measured: Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm].

  9. Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study

    Change from Baseline to End of Study for SF-36 Questionnaire is provided. Scores for individual SF-36 categories range from 0 to 100 with higher scores representing better health. Given that higher scores indicate better health-related quality of life (HRQoL) and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates a worsening of HRQoL.

    Measured: Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm]

  10. Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)

    Terminal half-life calculated as log_e2/λz where λz is the terminal elimination rate constant. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

    Measured: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

  11. Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)

    The mean residence time (MRT) w as calculated as total area under the moment curve divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

    Measured: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

  12. Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)

    Clearance in dL/(kg.h) will be calculated as the dose in IU/kg divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

    Measured: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

  13. Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)

    Incremental recovery (IR) in (IU/dL)/ (IU/kg) calculated as: IR = (Cmax- (C pre-infusion)) / (Dose/kg), where C =concentration. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

    Measured: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

  14. Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)

    Calculated by WinNonlin NCA (Model 201, calculation method: Linear Trapezoidal Linear/Log Interpolation). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

    Measured: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

  15. Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)

    The apparent volume of distribution at steady state (Vss) will be calculated as: Vss = Clearance * Mean Residence Time. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

    Measured: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

  16. Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)

    Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

    Measured: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

  17. Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)

    Tmax in hours will be defined as the time to reach Cmax. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

    Measured: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

  18. Change in Vital Signs From Screening - Temperature

    Measured: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination

  19. Change in Vital Signs From Screening - Pulse Rate

    Measured: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination

  20. Change in Vital Signs From Screening - Respiratory Rate

    Measured: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination

  21. Changes in Vital Signs From Screening - Blood Pressure

    Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP)

    Measured: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination

  22. Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein

    Measured: Screening, week 2, week 4, month 3, study completion/termination

  23. Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase

    Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino)

    Measured: Screening, week 2, week 4, month 3, study completion/termination

  24. Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)

    Measured: Screening, week 2, week 4, month 3, study completion/termination

  25. Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin

    Measured: Screening, week 2, week 4, month 3, study completion/termination

  26. Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes

    Measured: Screening, week 2, week 4, month 3, study completion/termination

  27. Changes in Hematology Laboratory Assessments From Screening - Hematocrit

    Measured: Screening, week 2, week 4, month 3, study completion/termination

  28. Changes in Hematology Laboratory Assessments From Screening - Hemoglobin

    Measured: Screening, week 2, week 4, month 3, study completion/termination

  29. Changes in Hematology Laboratory Assessments From Screening - Erythrocytes

    Measured: Screening, week 2, week 4, month 3, study completion/termination

  30. Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)

    Measured: Screening, week 2, week 4, month 3, study completion/termination

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Nov 2012 (estimated)
  2. Study start31 Jan 2013
  3. Primary completion17 Jul 2014
  4. Study completion17 Jul 2014

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Prevention
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
159 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01736475
Sponsor's protocol ID
261201
Other IDs
2012-003599-38
Status last verified
Apr 2021
First submitted
21 Nov 2012
Last updated
20 May 2021
Data monitoring committee
Yes
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Publications

  • 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.

    PubMed 26157075

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method and PEGylated Recombinant Factor VIII in people with Hemophilia A, run by Baxalta now part of Shire at 72 sites in 20 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 72 sites in United States, Japan, United Kingdom, South Korea, Australia and 15 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Sep 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Annualized Bleeding Rate (ABR) (time frame: 9 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01736475, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2021.

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