Gene Therapy for Hemophilia A
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new gene therapy treatment called SPK-8011QQ for adult males with hemophilia A, a condition where blood doesn't clot properly, leading to excessive bleeding. The study aims to determine the safety and tolerability of this therapy. It targets those with severe or moderately severe hemophilia A who have been managing it with regular factor VIII infusions or have experienced several recent bleeding episodes. Participants will receive the treatment through an IV infusion. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.
Is there any evidence suggesting that this treatment is likely to be safe for humans?
Previous studies have shown that SPK-8011QQ, a gene therapy for hemophilia A, is safe, meaning it did not cause serious problems in patients. Research indicates that earlier versions of this therapy, such as SPK-8011, have been well-tolerated. The therapy aims to reduce bleeding in people with hemophilia A. Earlier studies reported no deaths and no development of FVIII inhibitors, which can stop the therapy from working. However, it is important to note that this therapy is still under investigation. These findings suggest that SPK-8011QQ could be a safe option, but more research is needed to confirm this.12345
Why do researchers think this study treatment might be promising?
Unlike the standard of care for hemophilia A, which often involves regular infusions of clotting factor VIII, SPK-8011QQ is a gene therapy aiming for a more lasting solution. Researchers are excited about SPK-8011QQ because it delivers a corrected gene directly to the liver cells, potentially enabling the body to produce its own factor VIII. This could reduce or even eliminate the need for frequent factor infusions, offering a significant improvement in quality of life for patients.
What evidence suggests that SPK-8011QQ might be an effective treatment for hemophilia A?
Research has shown that SPK-8011QQ, a gene therapy studied in this trial, could be promising for treating hemophilia A. Studies have found that this treatment can significantly improve blood clotting in patients. Long-term data suggest that a single treatment with SPK-8011QQ leads to a lasting reduction in bleeding for people with hemophilia A. The therapy uses a virus to deliver a healthy gene to cells, enabling them to produce the proteins needed to control bleeding. Initial findings also indicate that patients experienced fewer bleeding episodes after receiving the treatment. This early evidence supports its potential effectiveness in managing hemophilia A.13567
Who Is on the Research Team?
Clinical Trials
Principal Investigator
Hoffmann-La Roche
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive an intravenous (IV) infusion of SPK-8011QQ on Day 1 of the study
Follow-up
Participants are monitored for safety and tolerability after receiving the infusion
What Are the Treatments Tested in This Trial?
Interventions
- SPK-8011QQ
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants will receive an intravenous (IV) infusion of SPK-8011QQ on Day 1 of the study.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Hoffmann-La Roche
Lead Sponsor
Dr. Levi Garraway
Hoffmann-La Roche
Chief Medical Officer since 2019
MD from the University of Basel
Dr. Thomas Schinecker
Hoffmann-La Roche
Chief Executive Officer since 2023
PhD in Molecular Biology from New York University
Citations
Preclinical evaluation of SPK-8011QQ, an adeno- ...
Preclinical evaluation of SPK-8011QQ, an adeno-associated virus gene therapy for people with hemophilia A leveraging the dirloctocogene samoparvovec platform ...
NCT07226206 | A Gene Therapy Study of SPK-8011QQ in ...
A Phase 2b, Single-Arm, Open-Label, Multicenter Study of the Safety of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A. Conditions.
3.
hopeforhemophilia.org
hopeforhemophilia.org/industry-news/a-new-chapter-in-hemophilia-a-gene-therapy-spark-joins-roche-phase-2b-trial-announcedPhase 2b Hemophilia A Gene Therapy Clinical Trial
This next-generation gene therapy program is planned to be studied in a Phase 2b clinical trial sponsored by Roche and Genentech.
Roche to present new data from its broad and innovative ...
Data from innovative pipeline signals progress toward improved outcomes in haemophilia A, lymphoma, and multiple myeloma. Basel, 3 November ...
5.
biospace.com
biospace.com/press-releases/genentech-to-present-new-data-from-its-broad-and-innovative-hematology-portfolio-at-ash-2025Genentech to Present New Data From Its Broad and ...
SPK-8011QQ: Pre-clinical data on Genentech's next-generation investigational AAV gene therapy, show significantly enhanced hemostatic potency ...
6.
ashpublications.org
ashpublications.org/blood/article/146/Supplement%201/1068/555364/Preclinical-evaluation-of-SPK-8011QQ-an-adenoPreclinical evaluation of SPK-8011QQ, an adeno-associated ...
Furthering the learnings from the safety and durability of the SPK-8011 platform, our preclinical data collected to date support the ongoing ...
7.
hemophilianewstoday.com
hemophilianewstoday.com/news/long-term-data-reinforce-spk-8011-gene-therapy-potential-hem-a/Long-term Data Reinforce SPK-8011 Gene Therapy's ...
One-time treatment with the investigational gene therapy SPK-8011 led to sustained low bleed rates for people with hemophilia A in a Phase 1/2 clinical trial.
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