A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

NCT07226206 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: XO46084SPK-8011QQ-201
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 2

Brief Summary

This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (6)

• 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 scale

  Up 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 AEs

  Up to approximately 5 years

• Number of participants with abnormal laboratory values

  Up to approximately 5 years

Study Arms (1)

SPK-8011QQ

EXPERIMENTAL

Participants will receive an intravenous (IV) infusion of SPK-8011QQ on Day 1 of the study.

Drug: SPK-8011QQ

Interventions

SPK-8011QQ DRUG

Participants will receive IV SPK-8011QQ

Also known as: RO7878488

SPK-8011QQ

Eligibility Criteria

• Age: 18 Years+
• Gender Eligibility Details: Male participants at least 18 years of age with moderately severe to severe hemophilia A
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify 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 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

You may not qualify 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 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (2)

Center for Inherited Blood Disorders

Orange, California, 92868-4748, United States

RECRUITING

Kaiser Permanente

Vallejo, California, 94589-2441, United States

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Central Study Contacts

Reference Study ID Number: XO46084 https://forpatients.roche.com/

CONTACT

888-662-6728 (U.S. and Canada); global-roche-genentech-trials@gene.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 28, 2025
• First Posted: November 10, 2025
• Study Start (Estimated): May 15, 2026
• Primary Completion (Estimated): July 31, 2031
• Study Completion (Estimated): July 31, 2031
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (2)