Actively Recruiting
A Phase 2b, Single-Arm, Open-Label, Multicenter Study of the Safety of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
Led by Hoffmann-La Roche · Updated on 2026-05-26
5
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A, a condition characterized by low levels of factor VIII. This phase 1 and 2 study is sponsored by Hoffmann-La Roche and focuses on the effects of this gene therapy treatment in a specific patient group. The trial aims to understand the side effects and safety profile of SPK-8011QQ over an extended period. Participants will receive a single intravenous infusion of SPK-8011QQ on the first day of the study. The treatment involves delivering the gene therapy directly into the bloodstream. The study is open-label and conducted across multiple centers, with all participants receiving the experimental treatment. No placebo or comparison groups are involved in this single-arm trial. During the study, participants will be followed for up to approximately five years to monitor adverse events, including their incidence, severity, and any serious or treatment-related occurrences. Laboratory tests will be conducted regularly to detect any abnormal values. Participants will also complete questionnaires and attend scheduled visits to assess their health and response to the therapy throughout the study duration.
CONDITIONS
Brief 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
- 18 years of age or older at the time of signing the consent form
- Male sex assigned at birth
- Severe or moderately severe hemophilia A with factor VIII activity levels of 3% or less, confirmed by certified laboratory
- Documented treatment for at least 6 months prior to screening with plasma coagulation factor VIII prophylaxis or on-demand with at least 5 breakthrough bleeds in the last 6 months
- No history of hypersensitivity or anaphylaxis to any factor VIII product
- At least 150 exposure days to a factor VIII protein product
- Negative inhibitor screening test against factor VIII
- Prior inhibitors that were tolerized with successful immune tolerance induction completed at least 5 years prior and no inhibitor recurrence within 5 years
- Negative anti-Spark200 antibodies confirmed by central laboratory
- Acceptable liver function with ALT, AST, ALP less than or equal to 2 times upper limit of normal and INR less than 1.4
- No evidence of cirrhosis or advanced liver disease
- Adequate kidney function with creatinine clearance of at least 30 mL/min/1.73 m2
- Platelet count of at least 50,000 cells/µL
- Negative HIV test or stable HIV-positive status with controlled viral load and adequate CD4 count
- Negative hepatitis B surface antigen, with appropriate hepatitis B antibody and core antibody status
- Negative hepatitis C antibody or confirmed negative RNA test
- Appropriate medical history and physical and laboratory evaluation for study inclusion
- Willingness and ability to comply with study visits, treatment plans, laboratory tests, and questionnaires
- Agreement to follow contraception requirements as described in the protocol
You will not qualify if you...
- Currently undergoing antiviral therapy for chronic hepatitis B or C
- Having an inherited or acquired bleeding disorder other than hemophilia A
- Known thrombophilia or signs of thromboembolic disease or current treatment for such conditions
- Prior treatment with any vector or gene transfer agent
- Receiving or received investigational drugs within 30 days or 5 half-lives prior to study
- Planned major surgery within 15 months after SPK-8011QQ infusion
- Unable or unwilling to receive blood or blood products or standard-of-care treatment for life-threatening conditions
- Concurrent disease, treatment, or lab abnormalities that interfere with study participation 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
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to approximately 5 years
Participants receive an intravenous (IV) infusion of SPK-8011QQ on Day 1 of the study.
1 infusion visit and ongoing safety monitoring visits
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/
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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