Hemophilia A study: SPK-8011QQ
Official title A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
ClinicalTrials.gov ID: NCT07226206
What this study is testing
What is SPK-8011QQ?
An investigational IV medicine being studied as a potential treatment for hemophilia A.
Also referred to as RO7878488.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study aims to check if the new treatment is safe and how well the body tolerates it.
- Who it's for
- The study is looking for adult men who have moderately severe to severe Hemophilia A.
- What's involved
- Participants will receive a one-time gene therapy injection and undergo medical tests to monitor their health.
- Time commitment
- The source text does not specify the total duration or the number of required visits.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older, men only
You may be able to join if
- 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...
- Have documented treatment for a minimum of 6 months prior to screening with either of the following: plasma coagulation factor VIII (FVIII)...
You likely can't join if
- 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...
- Have had prior treatment with a vector or gene transfer agent. Nucleic acid-based vaccines, such as the vaccine for coronavirus disease 2019...
- Are receiving an investigational drug concurrently or have received an investigational drug within 30 days or 5 half-lives of the last...
- Have a major surgical procedure planned in the 15-month period following SPK-8011QQ infusion
See the full eligibility criteria
- 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...
- 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...
- 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)...
- 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
- 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...
- 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)
- 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
- 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.
- 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...
- 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 study team makes the final eligibility decision.
Where it is enrolling
- Orange, California, United States
- San Francisco, California, United States
- Vallejo, California, United States
- New Haven, Connecticut, United States
- Newark, New Jersey, United States
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Orange, California, United States; San Francisco, California, United States; Vallejo, California, United States; New Haven, Connecticut, United States; Newark, New Jersey, United States; New York, New York, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.