Safety & Efficacy of SIG-001 Cell Therapy in Haemophilia A
Research type
Research Study
Full title
A Phase 1/2 Open-Label, Dose-Escalation, Safety, Tolerability, and Efficacy Study of SIG-001 in Adult Patients with Severe or Moderately-Severe Haemophilia A Without Inhibitors
IRAS ID
278684
Contact name
Rashid Kazmi
Contact email
Sponsor organisation
Sigilon Therapeutics Inc
Eudract number
2019-004210-33
Duration of Study in the UK
6 years, 0 months, 1 days
Research summary
This is a research study that is evaluating an experimental cell therapy product called SIG-001 for severe or moderately severe haemophilia A.
The appearance of SIG-001 is that of small spheres that are approximately 1.5mm or approximately the size of the tip of a ballpoint pen. The spheres are made of alginate and thousands of human cells. Alginate is a natural material that comes from seaweed and is already used in some foods and medical products. The human cells have been modified with the human gene for the FVIII protein and are designed to produce human FVIII. The alginate forms a protective layer around the cells and shields them. It may help the cells to avoid detection by the immune system and allow them to remain intact in the body. The cells are expected to continuously produce human FVIII in the abdominal cavity. The FVIII then goes into the blood, potentially resulting in an increase in FVIII levels.
This will be the first study of SIG-001 in humans. Trial Participants will have SIG-001 placed in their abdomen during laparoscopic surgery. Laparoscopic surgery is a type of surgical procedure in which the surgeon inserts narrow tubes through 2 or 3 small openings. A thin device with an attached light and camera and other surgical instruments can go through those tubes. This allows the surgeon to view the inside of the participant's abdomen, and, in this case, to place the SIG-001 spheres. The SIG-001 spheres will remain in the participant's body where it is hoped that they will produce FVIII.
The purpose of this study is to evaluate how the participant's bodies will respond to and tolerate SIG-001, and if SIG-001 can increase FVIII activity level. Study duration is approximately 63 months, which includes a circa 3-month screening period and 5-year follow-up.REC name
London - West London & GTAC Research Ethics Committee
REC reference
20/LO/0431
Date of REC Opinion
11 Jun 2020
REC opinion
Further Information Favourable Opinion