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COMPLETEDPHASE1/PHASE2

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NCT03734588•Spark Therapeutics, Inc.

Summary

SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.

Eligibility

Age

18 - No limit years

Sex

MALE

Healthy Volunteers

Inclusion Criteria

•1. Be male and ≥18 years of age;
•2. Have clinically severe hemophilia A, defined as:
•1. \<1% (\<1 IU/dL) endogenous FVIII activity levels as historically documented by a certified laboratory or screening data results; OR
•2. 1-2% (1-2 IU/dL) endogenous FVIII activity levels and \> 10 bleeding events per year (in the last 52 weeks prior to screening); OR
•3. 1-2% (1-2 IU/dL) endogenous FVIII activity levels and on prophylaxis;
•3. Have had \>150 exposure days (EDs) to any recombinant and/or plasma-derived FVIII concentrates or cryoprecipitates
•4. Have no prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration
•5. Have no measurable inhibitor against FVIII as assessed by central laboratory, have no confirmed history of clinically significant FVIII inhibitor, and no clinical signs or symptoms of decreased response to FVIII administration (Note: family history of inhibitors will not exclude study participation)
•6. Agree to use reliable barrier contraception after the administration of SPK-8016 until notified by the Investigator.

Exclusion Criteria

•1. Have active hepatitis B or C
•2. Have significant underlying liver disease.
•3. Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm3. Participants who are HIV-positive and stable, with an adequate CD4 count (\>200/mm3) and undetectable viral load, and are on an antiretroviral drug regimen are eligible to enroll
•4. Have detectable antibodies reactive with AAV-Spark capsid
•5. Have history of chronic infection or other chronic disease
•6. Have been dosed in a previous gene therapy research trial within the last 52 weeks or with an investigational drug within the last 12 weeks
•7. Any concurrent clinically significant major disease (such as liver abnormalities or type I diabetes) or other condition that, in the opinion of the Investigator and/or Sponsor, makes the subject unsuitable for participation in the study;
•8. Unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol.

Locations (11)

Orthopaedic Institute for Children

Los Angeles, California, United States

Illinois Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States

University of Michigan

Ann Arbor, Michigan, United States

Mississippi Center for Advanced Medicine

Madison, Mississippi, United States

Weill Cornell Medicine

New York, New York, United States

Oregon Health & Science University

Portland, Oregon, United States

Penn State Health

Hershey, Pennsylvania, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Jefferson University Hospitals

Philadelphia, Pennsylvania, United States

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

Key Dates

Start Date

January 30, 2019

Primary Completion Date

October 14, 2020

Completion Date

January 19, 2023

Last Updated

February 23, 2024

Enrollment

ACTUAL participants

Conditions

Adeno-Associated Virus (AAV)Blood Coagulation DisorderBlood Coagulation Disorders, InheritedCoagulation Protein DisordersFactor VIII (FVIII)Factor VIII (FVIII) DeficiencyFactor VIII (FVIII) GeneFactor VIII (FVIII) ProteinGenetic Diseases, InbornGenetic Diseases, X-LinkedGene TherapyGene TransferHematologic DiseasesHemorrhagic DisordersRecombinantVectorInhibitors

Interventions

SPK-8016

GENETIC

Transfusion Surveillance in Anaesthesia

NCT06403163

Transfusion ReactionTransfusion Related Complication+6 more

View study

RECRUITING

Evaluation of Hemostasis in Bleeding and Thrombotic Disorders

NCT00178594

Blood Coagulation Disorders, InheritedThrombotic Disorder

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 23, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Inclusion Criteria

Exclusion Criteria

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