Actively Recruiting
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
Led by Hoffmann-La Roche · Updated on 2026-04-27
5
Participants Needed
2
Research Sites
271 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
CONDITIONS
Official Title
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed Informed Consent Form (ICF)
- 18 years of age or older at the time of signing the ICF
- Male sex assigned at birth
- Severe or moderately severe hemophilia A with factor VIII activity levels 3% or less, documented by a certified laboratory
- Documented treatment for at least 6 months prior to screening with either factor VIII prophylaxis or on-demand treatment with at least 5 breakthrough bleeds in the prior 6 months
- No history of hypersensitivity or anaphylaxis to any factor VIII product
- At least 150 exposure days to factor VIII protein products
- Negative inhibitor test against factor VIII (less than 0.6 Bethesda Units)
- Candidates with prior inhibitors tolerized with successful immune tolerance induction completed at least 5 years before screening and no inhibitor recurrence
- Negative anti-Spark200 antibodies confirmed by central lab
- Acceptable liver function: ALT, AST, ALP levels 2 times the upper limit of normal or less; INR less than 1.4; no cirrhosis or advanced liver disease
- Adequate kidney function with creatinine clearance 30 mL/min/1.73 m2 or higher; not on dialysis
- Platelet count 50,000 cells/µL or higher
- Negative HIV test unless stable on antiretroviral treatment with CD4 count over 200/mm3 and undetectable viral load
- Negative hepatitis B surface antigen test
- Positive hepatitis B surface antibody test, or negative with additional hepatitis B core antibody and DNA testing
- Negative hepatitis C antibody or positive antibody with negative RNA test
- Appropriate medical history and physical and lab evaluation for study inclusion
- Willing and able to comply with study visits, treatments, tests, and questionnaires
- Agree to contraception requirements described in the protocol
You will not qualify if you...
- Currently receiving antiviral therapy for chronic hepatitis B or C
- Having any other inherited or acquired bleeding disorder besides hemophilia A
- Known thrombophilia or signs of thromboembolic disease, or current treatment for thromboembolic disease
- Previous treatment with a vector or gene transfer agent (excluding nucleic acid-based vaccines)
- Currently on investigational drugs or received them within 30 days or 5 half-lives prior to study
- Planned major surgery in the 15 months following SPK-8011QQ infusion
- Unable or unwilling to receive blood products or standard treatment for life-threatening conditions
- Concurrent disease, treatment, or lab abnormalities interfering with study or safety
- History of malignancy within 5 years prior to screening except treated basal or squamous cell carcinoma of the skin
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
Center for Inherited Blood Disorders
Orange, California, United States, 92868-4748
Actively Recruiting
2
Kaiser Permanente
Vallejo, California, United States, 94589-2441
Actively Recruiting
Research Team
R
Reference Study ID Number: XO46084 https://forpatients.roche.com/
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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