Gene Therapy Study of SPK-8011QQ for Hemophilia A
This study is testing a gene therapy called SPK-8011QQ in adult men with severe or moderately severe hemophilia A (a bleeding disorder where blood doesn't clot properly). The main goal is to see how safe SPK-8011QQ is and how well people tolerate it. Researchers will be looking for any side effects and their severity over approximately five years. To join, you must be at least 18 years old, male, and have a confirmed diagnosis of severe or moderately severe hemophilia A, meaning your FVIII:C activity levels are 3% or less. The study plans to enroll 5 participants, but its current status is unclear.
- Study design
- This is an interventional study, meaning participants will receive the study drug SPK-8011QQ. It plans to enroll 5 adult male participants.
- What's involved
- Participants will receive SPK-8011QQ intravenously (into a vein). The study will monitor for side effects for up to approximately 5 years.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for adverse events for up to approximately 5 years after receiving the intervention.
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NCT07226206
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
Recruiting
PHASE1Ages 18+InterventionalTreatmentHoffmann-La Roche
~5 participants
Updated 2026-08-04 on ClinicalTrials.gov
What's tested:SPK-8011QQ
At a glance
Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of participants with adverse events (AEs)
Measured over Up to approximately 5 years
+5 more outcomes measured
Conditions
Where it's being run
3 sites across 1 statesCalifornia3
Study leadership
- Clinical Trials · STUDY_DIRECTOR · Hoffmann-La Roche
Who to contact
Reference Study ID Number: XO46084 https://forpatients.roche.com/ No email attachments.
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Eligibility criteria
Inclusion
Signed Informed Consent Form (ICF)
≥18 years of age at the time of signing the ICF
Male sex assigned at birth
Severe or moderately severe hemophilia A, defined as endogenous FVIII:C activity levels ≤3%, as documented (historically or during the Screening Period) by a certified laboratory and where the FVIII:C level is measured more than 96 hours after the prior dose of an extended half-life FVIII replacement product or more than 72 hours after the prior dose of a standard half-life FVIII replacement product
Have documented treatment for a minimum of 6 months prior to screening with either of the following: plasma coagulation factor VIII (FVIII) prophylaxis, defined as receiving a prescribed dose and frequency of FVIII infusions with the intent to treat continuously for 52 weeks per year; or FVIII on demand, with a history of ≥ 5 breakthrough bleeds in the 6 months prior to screening
No prior history of hypersensitivity or anaphylaxis associated with the administration of any FVIII product
Have ≥150 exposure days to a FVIII protein product such as recombinant, plasma-derived, or extended half-life FVIII product
Negative screening test for inhibitor against FVIII (i.e., \<0.6 BU)
Candidates with prior FVIII inhibitors who are tolerized having completed successful 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
Confirmed negative anti-Spark200 antibodies as documented through central laboratory testing of a serum sample
Acceptable hepatobiliary function according to all of the following criteria: ALT, AST, and ALP ≤2×ULN and INR \<1.4 at the time of screening; No evidence of cirrhosis or advanced liver disease on screening liver ultrasound; Otherwise no laboratory or clinical evidence of liver disease or cirrhosis, per the Investigator's judgement
Adequate renal function, defined as creatinine clearance ≥30 mL/min/1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration formula; patients on dialysis are not eligible for the study
Platelet count ≥50,0000 cells/µL
Negative HIV test at screening, with the following exception: Individuals with a positive HIV test at screening are eligible provided they are stable on an antiretroviral treatment regimen, have a cluster of differentiation (CD4) count \>200/mm3, and undetectable viral load (\<50 gc/mL)
Negative hepatitis B surface antigen (HBsAg) at screening
Positive hepatitis surface antibody (HBsAb) at screening, or a negative HBsAb at screening accompanied by either of the following: Negative hepatitis B core antibody (HBcAb); Positive HBcAb and negative hepatitis B virus (HBV) DNA test
Negative hepatitis C virus (HCV) antibody test at screening, or positive HCV antibody test at screening accompanied by negative HCV RNA test
Otherwise appropriate medical history and physical and laboratory evaluation that are acceptable for inclusion in this clinical trial
Are able and willing to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the completion of applicable patient-reported outcome questionnaires
Agreement to adhere to the contraception requirements described in the protocol
Exclusion
Are currently undergoing antiviral therapy for chronic hepatitis B or chronic hepatitis C
Have an inherited or acquired bleeding disorder other than hemophilia A
Have known inherited or acquired thrombophilia, have signs of thromboembolic disease in the Investigator's judgement, 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 had prior treatment with a vector or gene transfer agent. Nucleic acid-based vaccines, such as the vaccine for coronavirus disease 2019 (COVID-19), are not considered gene transfer agents
Are receiving an investigational drug concurrently or have received an investigational drug within 30 days or 5 half-lives of the last investigational drug administration, whichever is longer
Have a major surgical procedure planned in the 15-month period following SPK-8011QQ infusion
Are unable (or unwilling) to receive blood or blood products (or any standard-of-care treatment for a life-threatening condition)
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 preclude the candidate's safe participation in and completion of the study, or the interpretation of the study results
History of malignancy within 5 years prior to screening and up to investigational study drug administration (Day 1) with the following exceptions: Participants with curatively treated basal or squamous cell carcinoma of the skin at any time prior to investigational study drug administration (Day 1) are eligible
What this trial measures
- Incidence of participants with adverse events (AEs)Up to approximately 5 years
- Severity of AEs as determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0 grading scaleUp to approximately 5 years
- Incidence of adverse events of special interest (AESIs)Up to approximately 5 years
- Incidence of serious adverse events (SAEs)Up to approximately 5 years
- Incidence of treatment-related AEsUp to approximately 5 years
- Number of participants with abnormal laboratory valuesUp to approximately 5 years