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BAY81-8973 Pediatric Safety and Efficacy Trial

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01311648
Recruitment Status : Active, not recruiting
First Posted : March 9, 2011
Last Update Posted : July 22, 2020
Sponsor:
Information provided by (Responsible Party):
Bayer

Brief Summary:

The primary objective is to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.

The secondary objectives are

  • To assess the safety and efficacy of BAY81-8973 during surgeries.
  • To assess incremental recovery of BAY 81-8973.
  • To assess pharmacokinetic parameters in a subset of children. (PTPs and PUPs/MTPs -participation in pharmacokinetic [PK] sampling is voluntary and requires consent).

Condition or disease Intervention/treatment Phase
Haemophilia A Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973) Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 94 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy
Actual Study Start Date : June 9, 2011
Actual Primary Completion Date : September 9, 2019
Estimated Study Completion Date : October 30, 2020


Arm Intervention/treatment
Experimental: Arm 1
Part A will investigate a total of 50 PTPs up to 12 years of age. Part B will include at least 25 PUPs, plus up to additional 25 PUPs/MTPs
Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973)
PTPs prophylaxis treatment: 25-50 IU/kg at least 2x/week. PUPs/MTPs: 15 -50 IU/kg at least 1x/week.




Primary Outcome Measures :
  1. Annualized number of bleeds within 48 hours (h) after a prophylaxis injection [ Time Frame: Up to 9 months ]

Secondary Outcome Measures :
  1. Incidence of inhibitory antibody [ Time Frame: Up to 9 months ]
  2. Total annualized consumption of FVIII per subject [ Time Frame: Up to 9 months ]
  3. Number of infusions for the treatment of a bleed [ Time Frame: Up to 9 months ]
  4. Recovery of FVIII after infusion [ Time Frame: Up to 9 months ]
  5. Half-life (t1/2) [ Time Frame: Up to 24 hours after end of infusion ]
    Participation in the PK evaluations is optional.



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Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male, aged </= 12 years
  • Severe hemophilia A defined as < 1% FVIII:C
  • PTPs (previously treated patients) > 50 Exposure days (ED) with any FVIII products
  • PUPs (previously untreated patients) no prior exposure to any FVIII products
  • MTPs (minimally treated patients) not previously having received more than 3 Exposure Days (ED) with any FVIII products
  • No current evidence or history of inhibitory antibody

Exclusion Criteria:

  • History of FVIII inhibitor formation
  • Diagnosed with other bleeding disorder
  • Platelet count < 100000 cells/μL
  • Kidney or liver dysfunction
  • Known hypersensitivity to any FVIII

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01311648


Locations
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United States, Louisiana
New Orleans, Louisiana, United States, 70112
United States, Ohio
Cincinnati, Ohio, United States, 45229
Cleveland, Ohio, United States, 44106-2602
Columbus, Ohio, United States, 43205-2696
Argentina
Bahía Blanca, Buenos Aires, Argentina, B8001HXM
Bulgaria
Plovdiv, Bulgaria, 4002
Varna, Bulgaria, 9010
Canada, Alberta
Edmonton, Alberta, Canada, T6G 2C8
Canada, Ontario
Hamilton, Ontario, Canada, L8N 3Z5
Toronto, Ontario, Canada, M5G 1X8
Denmark
Århus N, Denmark, 8200
Hungary
Budapest, Hungary, 1089
Debrecen, Hungary, 4032
Mohacs, Hungary, 7700
Ireland
Crumlin, Dublin, Ireland, 12
Israel
Ramat Gan, Israel, 5262000
Italy
Roma, Lazio, Italy, 00165
Milano, Lombardia, Italy, 20122
Bari, Puglia, Italy, 70126
Catania, Sicilia, Italy, 95123
Padova, Veneto, Italy, 35128
Latvia
Riga, Latvia, LV-1004
Lithuania
Vilnius, Lithuania, 08406
Mexico
Guadalajara, Jalisco, Mexico, 44280
San Juan del Río, Querétaro, Mexico, 76800
Oaxaca, Mexico, 68000
Norway
Oslo, Norway
Poland
Lodz, Poland, 91-738
Warszawa, Poland, 00-576
Wroclaw, Poland, 50-368
Romania
Bucharest, Romania, 011026
Bucharest, Romania, 022328
Cluj-Napoca, Romania, 400370
Timisoara, Romania, 300011
Russian Federation
Kazan, Russian Federation, 139445
Kirov, Russian Federation, 610027
Volgograd, Russian Federation, 400138
Spain
Esplugues de LLobregat, Barcelona, Spain, 08950
A Coruña, Spain, 15006
Alicante, Spain, 03010
Cáceres, Spain, 10003
Madrid, Spain, 28046
Valencia, Spain, 46026
Sponsors and Collaborators
Bayer
Investigators
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Study Director: Bayer Study Director Bayer
Additional Information:
Publications of Results:
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Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT01311648    
Other Study ID Numbers: 13400
2010-021781-29 ( EudraCT Number )
First Posted: March 9, 2011    Key Record Dates
Last Update Posted: July 22, 2020
Last Verified: July 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Bayer:
Recombinant factor VIII
Pediatric use
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants