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RECRUITINGPHASE1/PHASE2

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

A Phase 2b, Single-Arm, Open-Label, Multicenter Study of the Safety of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

NCT07226206•Hoffmann-La Roche

View on ClinicalTrials.gov

Summary

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

Eligibility

Age

18 - No limit years

Sex

MALE

Healthy Volunteers

Inclusion 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 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
+1 more criteria

Exclusion Criteria

•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
•Are currently undergoing antiviral therapy for chronic hepatitis B or chronic hepatitis C
•Have an inherited or acquired bleeding disorder other than hemophilia A
+7 more criteria

Locations (1)

Kaiser Permanente

Vallejo, California, United States

Key Dates

Start Date

March 31, 2026

Primary Completion Date

July 31, 2031

Completion Date

July 31, 2031

Last Updated

March 12, 2026

Enrollment

ESTIMATED participants

Conditions

Hemophilia A

Interventions

SPK-8011QQ

DRUG

Contacts

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

CONTACT

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

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 12, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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