A Gene Transfer Study for Hemophilia A

Description

This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.

Study Start Date

December, 01 2016

Estimated Completion Date

December 2019

Interventions

  • Genetic: SPK-8011

Study ID

Spark Therapeutics -- SPK-8011-101

Status

Recruiting

Trial ID

NCT03003533

Study Type

Interventional

Trial Phase

Phase 1/Phase 2

Enrollment Quota

30

Sponsor

Spark Therapeutics

Inclusion Criteria

  • Males age18 years or older
  • Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma FVIII activity levels ? 2% of normal
  • Have received >150 exposure days (EDs) to FVIII concentrates or cryoprecipitate
  • Have experienced >10 bleeding events over the previous 12 months only if receiving on-demand therapy and having FVIII baseline level 1-2% of normal
  • Have no prior history of allergic reaction to any FVIII product
  • Have no measurable inhibitor against factor VIII inhibitor as assessed by the central laboratory and have no prior history of inhibitors to FVIII protein
  • Agree to use reliable barrier contraception

Exclusion Criteria

  • Evidence of active hepatitis B or C
  • Currently on antiviral therapy for hepatitis B or C
  • Have significant underlying liver disease
  • Have serological evidence* of HIV-1 or HIV-2 with CD4 counts ?200/mm3 (* participants who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll)
  • Have detectable antibodies reactive with AAV-Spark200 capsid
  • Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks

Gender

Male

Ages

18 Years and older

Accepts Healthy Volunteers

No

Study Locations and Contact Information (54)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Stacey Croteau MD None Stacy.Croteau@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Stacey Croteau MD None Stacy.Croteau@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Stacey Croteau MD None Stacy.Croteau@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Stacey Croteau MD None Stacy.Croteau@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Stacey Croteau MD None Stacy.Croteau@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Stacey Croteau MD None Stacy.Croteau@childrens.harvard.edu
Pennslyvania State University Milton S Hershey Medical Center - Hershey, Pennsylvania 174.2 miles Elaine Eyster MD None meyster@hmc.psu.edu
Pennslyvania State University Milton S Hershey Medical Center - Hershey, Pennsylvania 174.2 miles Elaine Eyster MD None meyster@hmc.psu.edu
Pennslyvania State University Milton S Hershey Medical Center - Hershey, Pennsylvania 174.2 miles Elaine Eyster MD None meyster@hmc.psu.edu
Pennslyvania State University Milton S Hershey Medical Center - Hershey, Pennsylvania 174.2 miles Elaine Eyster MD None meyster@hmc.psu.edu
Pennslyvania State University Milton S Hershey Medical Center - Hershey, Pennsylvania 174.2 miles Elaine Eyster MD None meyster@hmc.psu.edu
Pennslyvania State University Milton S Hershey Medical Center - Hershey, Pennsylvania 174.2 miles Elaine Eyster MD None meyster@hmc.psu.edu
Weill Cornell MedicineComprehensive Center for Hemophilia and Coagulation Disorders - New York, New York 187.1 miles Catherine McGuinn MD 212-746-3978 cam9061@med.cornell.edu
Weill Cornell MedicineComprehensive Center for Hemophilia and Coagulation Disorders - New York, New York 187.1 miles Catherine McGuinn MD 212-746-3978 cam9061@med.cornell.edu
Weill Cornell MedicineComprehensive Center for Hemophilia and Coagulation Disorders - New York, New York 187.1 miles Catherine McGuinn MD 212-746-3978 cam9061@med.cornell.edu
Weill Cornell MedicineComprehensive Center for Hemophilia and Coagulation Disorders - New York, New York 187.1 miles Catherine McGuinn MD 212-746-3978 cam9061@med.cornell.edu
Weill Cornell MedicineComprehensive Center for Hemophilia and Coagulation Disorders - New York, New York 187.1 miles Catherine McGuinn MD 212-746-3978 cam9061@med.cornell.edu
Weill Cornell MedicineComprehensive Center for Hemophilia and Coagulation Disorders - New York, New York 187.1 miles Catherine McGuinn MD 212-746-3978 cam9061@med.cornell.edu
The Childrens Hospital of Philadelphia - Philadelphia, Pennsylvania 270.8 miles Lindsey George MD None GeorgeL@email.chop.edu
The Childrens Hospital of Philadelphia - Philadelphia, Pennsylvania 270.8 miles Lindsey George MD None GeorgeL@email.chop.edu
The Childrens Hospital of Philadelphia - Philadelphia, Pennsylvania 270.8 miles Lindsey George MD None GeorgeL@email.chop.edu
The Childrens Hospital of Philadelphia - Philadelphia, Pennsylvania 270.8 miles Lindsey George MD None GeorgeL@email.chop.edu
The Childrens Hospital of Philadelphia - Philadelphia, Pennsylvania 270.8 miles Lindsey George MD None GeorgeL@email.chop.edu
The Childrens Hospital of Philadelphia - Philadelphia, Pennsylvania 270.8 miles Lindsey George MD None GeorgeL@email.chop.edu
Virginia Commonwealth University School of Medicine - Richmond, Virginia 473.7 miles J Christian Barrett MD 804-827-3306 jcbarrett@vcu.edu
Virginia Commonwealth University School of Medicine - Richmond, Virginia 473.7 miles J Christian Barrett MD 804-827-3306 jcbarrett@vcu.edu
Virginia Commonwealth University School of Medicine - Richmond, Virginia 473.7 miles J Christian Barrett MD 804-827-3306 jcbarrett@vcu.edu
Virginia Commonwealth University School of Medicine - Richmond, Virginia 473.7 miles J Christian Barrett MD 804-827-3306 jcbarrett@vcu.edu
Virginia Commonwealth University School of Medicine - Richmond, Virginia 473.7 miles J Christian Barrett MD 804-827-3306 jcbarrett@vcu.edu
Virginia Commonwealth University School of Medicine - Richmond, Virginia 473.7 miles J Christian Barrett MD 804-827-3306 jcbarrett@vcu.edu
Hemophilia Center of Western Pennsylvania - Pittsburgh, Pennsylvania 478.5 miles Judith Kadosh RN BSN 412-209-7263 JKadosh@itxm.org
Hemophilia Center of Western Pennsylvania - Pittsburgh, Pennsylvania 478.5 miles Judith Kadosh RN BSN 412-209-7263 JKadosh@itxm.org
Hemophilia Center of Western Pennsylvania - Pittsburgh, Pennsylvania 478.5 miles Judith Kadosh RN BSN 412-209-7263 JKadosh@itxm.org
Hemophilia Center of Western Pennsylvania - Pittsburgh, Pennsylvania 478.5 miles Judith Kadosh RN BSN 412-209-7263 JKadosh@itxm.org
Hemophilia Center of Western Pennsylvania - Pittsburgh, Pennsylvania 478.5 miles Judith Kadosh RN BSN 412-209-7263 JKadosh@itxm.org
Hemophilia Center of Western Pennsylvania - Pittsburgh, Pennsylvania 478.5 miles Judith Kadosh RN BSN 412-209-7263 JKadosh@itxm.org
East Carolina University - Greenville, North Carolina 577.1 miles Darla Liles MD 252-744-1888 lilsd@ecu.edu
East Carolina University - Greenville, North Carolina 577.1 miles Darla Liles MD 252-744-1888 lilsd@ecu.edu
East Carolina University - Greenville, North Carolina 577.1 miles Darla Liles MD 252-744-1888 lilsd@ecu.edu
East Carolina University - Greenville, North Carolina 577.1 miles Darla Liles MD 252-744-1888 lilsd@ecu.edu
East Carolina University - Greenville, North Carolina 577.1 miles Darla Liles MD 252-744-1888 lilsd@ecu.edu
East Carolina University - Greenville, North Carolina 577.1 miles Darla Liles MD 252-744-1888 lilsd@ecu.edu
Mississippi Center for Advanced Medicine - Madison, Mississippi 1,246.5 miles Spencer K Sullivan MD 601-499-0935 SKSullivan@msadvancedmedicine.com
Mississippi Center for Advanced Medicine - Madison, Mississippi 1,246.5 miles Spencer K Sullivan MD 601-499-0935 SKSullivan@msadvancedmedicine.com
Mississippi Center for Advanced Medicine - Madison, Mississippi 1,246.5 miles Spencer K Sullivan MD 601-499-0935 SKSullivan@msadvancedmedicine.com
Mississippi Center for Advanced Medicine - Madison, Mississippi 1,246.5 miles Spencer K Sullivan MD 601-499-0935 SKSullivan@msadvancedmedicine.com
Mississippi Center for Advanced Medicine - Madison, Mississippi 1,246.5 miles Spencer K Sullivan MD 601-499-0935 SKSullivan@msadvancedmedicine.com
Mississippi Center for Advanced Medicine - Madison, Mississippi 1,246.5 miles Spencer K Sullivan MD 601-499-0935 SKSullivan@msadvancedmedicine.com
University of California Davis Hemostasis and Thrombosis Center - Sacramento, California 2,689.7 miles Adam Giermasz MD PhD 916-734-1293 giermasz@ucdavis.edu
University of California Davis Hemostasis and Thrombosis Center - Sacramento, California 2,689.7 miles Adam Giermasz MD PhD 916-734-1293 giermasz@ucdavis.edu
University of California Davis Hemostasis and Thrombosis Center - Sacramento, California 2,689.7 miles Adam Giermasz MD PhD 916-734-1293 giermasz@ucdavis.edu
University of California Davis Hemostasis and Thrombosis Center - Sacramento, California 2,689.7 miles Adam Giermasz MD PhD 916-734-1293 giermasz@ucdavis.edu
University of California Davis Hemostasis and Thrombosis Center - Sacramento, California 2,689.7 miles Adam Giermasz MD PhD 916-734-1293 giermasz@ucdavis.edu
University of California Davis Hemostasis and Thrombosis Center - Sacramento, California 2,689.7 miles Adam Giermasz MD PhD 916-734-1293 giermasz@ucdavis.edu

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