Hemophilia APhase 1/2NCT07226206
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
A Phase 1/2 study testing SPK-8011QQ in people with Hemophilia A, run by Hoffmann-La Roche at 6 sites in United States.
Trial summary
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- SPK-8011QQ
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 6 sitesUnited States
- Study dates
- 8 Sep 2026to 30 Apr 2032
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Reference Study ID Number: XO46084 https://forpatients.roche.com/ No email attachments. contact
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry contact
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Yale School of Medicine
New Haven, Connecticut, 06510-3206
- Recruiting
New York Presbyterian Hospital - Weill-Cornell
New York, New York, 10065-4870
- Recruiting
Newark Beth Israel Medical Center
Newark, New Jersey, 07112-2027
- Recruiting
Center for Inherited Blood Disorders
Orange, California, 92868-4748
- Recruiting
University of California San Francisco
San Francisco, California, 94143-1222
- Recruiting
Kaiser Permanente
Vallejo, California, 94589-2441
About this study
Official title: A Phase 2b, Single-Arm, Open-Label, Multicenter Study of the Safety of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
- Conditions
- Hemophilia A
- Being tested
- SPK-8011QQ
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPK-8011QQDrug
Also called RO7878488
Participants will receive IV SPK-8011QQ
| Study group | Role | What they receive |
|---|---|---|
| SPK-8011QQ | Experimental | Participants will receive an intravenous (IV) infusion of SPK-8011QQ on Day 1 of the study. Receives: Drug: SPK-8011QQ |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of participants with adverse events (AEs)
Measured: 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 scale
Measured: Up to approximately 5 years
Incidence of adverse events of special interest (AESIs)
Measured: Up to approximately 5 years
Incidence of serious adverse events (SAEs)
Measured: Up to approximately 5 years
Incidence of treatment-related AEs
Measured: Up to approximately 5 years
Number of participants with abnormal laboratory values
Measured: Up to approximately 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2025
- Study start8 Sep 2026
- Primary completion30 Apr 2032 (estimated)
- Study completion30 Apr 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07226206
- Sponsor's protocol ID
- XO46084
- Other IDs
- SPK-8011QQ-201
- Status last verified
- Oct 2026
- First submitted
- 28 Oct 2025
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing