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Gene Therapy Trial Report

Summary

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


NCTID NCT03734588 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Hemophilia A
Disease Ontology Term DOID:12134
Compound Name SPK-8016
Sponsor Spark Therapeutics, Inc.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 4 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant F8
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 5E11 vg/kg (n=4)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2018-11-06
Completion Date 2023-01-19
Last Update 2024-02-23

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 11
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Roche (maker of Hemlibra) acquired Spark in 2019, announced they would discontinue development of this program in Q2 2023 update

Resources/Links