Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
|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: NCT03734588|
Recruitment Status : Active, not recruiting
First Posted : November 8, 2018
Last Update Posted : November 2, 2020
|Condition or disease||Intervention/treatment||Phase|
|Adeno-Associated Virus (AAV) Blood Coagulation Disorder Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Factor VIII (FVIII) Factor VIII (FVIII) Deficiency Factor VIII (FVIII) Gene Factor VIII (FVIII) Protein Genetic Diseases, Inborn Genetic Diseases, X-Linked Gene Therapy Gene Transfer Hematologic Diseases Hemorrhagic Disorders Recombinant Vector Inhibitors||Genetic: SPK-8016||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors|
|Actual Study Start Date :||January 30, 2019|
|Estimated Primary Completion Date :||November 2020|
|Estimated Study Completion Date :||November 2021|
All participants who meet the eligibility criteria will receive an outpatient single intravenous (i.v.) administration of SPK-8016.
adeno-associated viral vector
- Number of study-related adverse events, including clinically significant abnormal laboratory values. [ Time Frame: 52 weeks ]Adverse events.
- Occurrence of hepatic transaminase elevation requiring immunosuppression. [ Time Frame: 52 weeks ]Number of incidences of hepatic transaminase elevation where immunosuppression is required.
- Number of bleeding events (spontaneous and traumatic) after vector administration. [ Time Frame: 52 weeks ]Bleeding events.
- Number of FVIII infusions after vector administration. [ Time Frame: 52 weeks ]FVIII infusions.
- Peak and steady-state FVIII activity levels. [ Time Frame: 52 weeks ]Peak and steady-state FVIII activity levels assessed by coagulation clotting assays.
- Vector shedding of SPK-8016 in bodily fluids. [ Time Frame: 52 weeks ]Vector shedding.
- Incidence of immune response to AAV capsid protein and transgene product. [ Time Frame: 52 weeks ]
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): NCT03734588
|United States, Illinois|
|Illinois Bleeding and Clotting Disorders Institute|
|Peoria, Illinois, United States, 61615|
|United States, Mississippi|
|Mississippi Center for Advanced Medicine|
|Madison, Mississippi, United States, 39110|
|United States, New York|
|Weill Cornell Medicine|
|New York, New York, United States, 10065|
|United States, Oregon|
|Oregon Health & Science University|
|Portland, Oregon, United States, 97239|
|United States, Pennsylvania|
|Penn State Health|
|Hershey, Pennsylvania, United States, 17033|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Jefferson University Hospitals|
|Philadelphia, Pennsylvania, United States, 19107|
|United States, Virginia|
|Virginia Commonwealth University School of Medicine|
|Richmond, Virginia, United States, 23219|
|Principal Investigator:||Lindsey George, MD||Children's Hospital of Philadelphia|