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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06297486
Other study ID # SPK-8011-302
Secondary ID 2023-504537-46
Status Recruiting
Phase Phase 3
First received
Last updated
Start date March 13, 2024
Est. completion date September 4, 2035

Study information

Verified date May 2024
Source Spark Therapeutics, Inc.
Contact Clinical Trial Director
Phone Spark Therapeutics, Inc.
Email clinicaltrials@sparktx.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis.


Recruitment information / eligibility

Status Recruiting
Enrollment 85
Est. completion date September 4, 2035
Est. primary completion date May 2027
Accepts healthy volunteers No
Gender Male
Age group 18 Years and older
Eligibility Inclusion Criteria: - Have a negative anti-AAV-Spark200 neutralizing antibody (NAb) test result. - Are adult males with severe or moderately severe hemophilia A, defined as endogenous FVIII activity =3%, as documented by a certified laboratory (historically or during the Screening Period) and where the FVIII:C level is measured more than 96 hours after the prior dose of an extended-half-life FVIII - Have =150 documented exposure days to an FVIII protein product such as recombinant, plasma-derived, or extended half-life FVIII product - Have no prior history of hypersensitivity or anaphylaxis associated with the administration of any FVIII product. - Have screening hepatic ultrasound without evidence of cirrhosis and no laboratory or clinical evidence per the Investigator's judgment of advanced liver disease or cirrhosis. - Have a negative test for inhibitor against FVIII (ie, <0.6 Bethesda units [BU]) during screening. - Have no documented FVIII inhibitor (ie, <0.6 BU), FVIII half-life <6 hours, or FVIII recovery <66% in the 5 years prior to screening. - Candidates who completed successful immune tolerance induction (ITI) at least 5 years before screening are eligible, provided they have had no evidence of inhibitor recurrence (permanent or temporary) within 5 years prior to screening as may be indicated by detection of an inhibitor, FVIII half-life <6 hours, or FVIII recovery <66% since completing ITI. - If human immunodeficiency virus (HIV)-positive at screening, have an adequate cluster of differentiation 4 (CD4) count (>200/mm3) and undetectable viral load (<50 genome copies [gc]/mL), are on an antiretroviral drug regimen, and have completed at least 12 weeks of this treatment regimen prior to screening. - Meet the following inclusion criteria by cohort: - Cohort A: have documented history of prior treatment with FVIII prophylaxis (defined as receiving a prescribed dose and frequency of FVIII infusions with the intent to treat continuously for 52 weeks per year) for a minimum of 6 months prior to screening; and are willing to continue their FVIII prophylaxis during the Lead-In Period of this study (minimum of 24 weeks). - Cohort B: have documented history of prior treatment with FVIII on demand for a minimum of 6 months that shows =5 treated bleeds in the last 6 months prior to screening. - Cohort C: have documented history of prior treatment with emicizumab prophylaxis for a minimum of 6 months prior to screening. Exclusion Criteria: - Have an inherited or acquired bleeding disorder other than hemophilia A - Have inherited or acquired thrombophilia, have signs of thromboembolic disease in the Investigator's judgment, or are on current treatment for thromboembolic disease. A history of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing is not considered an exclusion criterion. - Have concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study or that would, in the opinion of the Investigator or Sponsor, preclude the candidate's safe participation in and completion of the study, or the interpretation of the study results.

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
SPK-8011
Participants will receive a single dose of SPK-8011, administered by intravenous (IV) infusion, on Day 1.

Locations

Country Name City State
United States Boston Children's Hospital Boston Massachusetts
United States University of North Carolina - Chapel Hill Chapel Hill North Carolina
United States Cincinnati Children's Hospital Cincinnati Ohio
United States University Hospitals Cleveland Cleveland Ohio
United States Nationwide Children's Hospital Columbus Ohio
United States Cook Children's Hospital Fort Worth Texas
United States University of Florida Gainesville Florida
United States Penn State Hershey Medical Center Hershey Pennsylvania
United States The University of Texas Health Science Center at Houston-Gulf States Hemophilia & Thrombosis Center Houston Texas
United States Loma Linda University Health Loma Linda California
United States Orthopaedic Institute for Children/Orthopaedic Hemophilia Treatment Center Los Angeles California
United States University of Minnesota Minneapolis Minnesota
United States Weill Cornell Medical Hospital New York New York
United States Newark Beth Israel Newark New Jersey
United States Kaiser Permanente-Oakland Medical Center Oakland California
United States University of Pennsylvania Philadelphia Pennsylvania
United States Phoenix Children's Hospital Phoenix Arizona
United States Oregon Health & Sciences University Portland Oregon
United States Kaiser Permanente-Roseville Medical Center Roseville California
United States Kaiser Permanente -Sacramento Medical Center Sacramento California
United States University of Utah Salt Lake City Utah
United States Kaiser Permanente -San Francisco Medical Center San Francisco California
United States University of California - San Francisco San Francisco California
United States Kaiser Permanente- Santa Clara Medical Center Santa Clara California
United States Bloodworks NW Seattle Washington
United States Kaiser Permanente-Vallejo Medical Center Vallejo California
United States Kaiser Permanente -Walnut Creek Medical Center Walnut Creek California

Sponsors (1)

Lead Sponsor Collaborator
Spark Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Annualized Bleed Rate (ABR) for All Bleeds [Cohort A] Up to 66 weeks post-SPK-8011 infusion
Secondary Median FVIII: C levels [Cohort A] Up to approximately 10 years
Secondary ABR for treated bleeds [Cohort A] Up to approximately 10 years
Secondary Percentage of Participants with ABR=0 for all bleeds; treated bleeds; treated spontaneous bleeds; and treated joint and target joint bleeds [Cohort A] Up to approximately 10 years
Secondary ABR for treated spontaneous, joint, and target joint bleeds [Cohort A] Up to approximately 10 years
Secondary Annualized FVIII dosage [Cohort A] Up to approximately 10 years
Secondary Proportion of Resolved Target Joints [Cohort A] Up to approximately 10 years
Secondary Mean Change of Total Hemophilia Joint Health Score [Cohort A] Baseline, up to 66 weeks post-SPK-8011 infusion
Secondary FVIII:C levels over time [Cohort A] Up to approximately 10 years
Secondary Mean ABR for all bleeds and treated bleeds [Cohort C] Up to approximately 10 years
Secondary FVIII: C levels over time from Week 26 [Cohort C] Up to approximately 10 years
Secondary Proportion of Participants Who Receive IV Methylprednisolone (IVMP) Prior to Week 8 (Early IVMP) [Cohorts A, B, C] Up to approximately 10 years
Secondary Proportion of Participants Who Receive Secondary Oral Immunomodulation [Cohorts A, B, C] Up to approximately 10 years
Secondary Median Time to First Immunomodulation-related Corticosteroid Dose [Cohorts A, B, C] Up to approximately 10 years
Secondary Median Duration of Secondary Oral Immunomodulation [Cohorts A, B, C] Up to approximately 10 years
Secondary Proportion of Participants with FVIII Inhibitor Development [Cohorts A, B, C] Up to approximately 10 years
Secondary Proportion of participants with Treatment-related AEs of ALT Elevation [Cohorts A, B, C] Up to approximately 10 years
Secondary Proportion of participants with treatment-related AEs of Infusion Reaction [Cohorts A, B, C] Up to approximately 10 years
Secondary Number of participants with abnormal physical exam findings, abnormal vital signs, and abnormal selected clinical laboratory test results [Cohorts A, B, C] Up to approximately 10 years
Secondary Proportion of participants with AEs and SAEs [Cohorts A, B, C] Up to approximately 10 years
Secondary Proportion of participants with AESIs [Cohorts A, B, C] Up to approximately 10 years
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