Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SPK-8011 · 1 trial · 1 indication
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as adverse events that result in death, are life-threatening, require inpatient hospitalization or prolongation of existing hospitalization, result in persistent or significant disability or incapacity, are a congenital anomaly or birth defect, or are an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE is defined as an AE with an onset date on or following SPK-8011 administration. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Median peak FVIII activity up to Week 52
Steady-state FVIII activity measured by median FVIII levels at week 52 by OSA.
| Arm | Type | Description |
|---|---|---|
| SPK-8011 5x10^11 vg/kg | EXPERIMENTAL | Participants received a single intravenous (IV) infusion of SPK-8011 5x10\^11 vector genomes per kilogram (vg/kg) body weight. |
| SPK-8011 1x10^12 vg/kg | EXPERIMENTAL | Participants received a single IV infusion of SPK-8011 1x10\^12 vg/kg. |
| SPK-8011 2x10^12 vg/kg | EXPERIMENTAL | Participants received a single IV infusion of SPK-8011 2x10\^12 vg/kg. |
| SPK-8011 1.5x10^12 vg/kg | EXPERIMENTAL | Participants received a single IV infusion of SPK-8011 1.5x10\^12 vg/kg. |
| Name | Type | Description |
|---|---|---|
| SPK-8011 | GENETIC | A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene |
Inclusion Criteria: * Males age 18 years or older * Confirmed diagnosis of hemophilia A as evidenced by their medical history with baseline FVIII activity levels \<=2% * Have received \>150 exposure days (EDs) to FVIII concentrates or cryoprecipitate * Have no prior history of allergic reaction to ...
SPK-8011 is an investigational gene therapy being developed for the treatment of hemophilia A, a bleeding disorder caused by a deficiency in clotting factor VIII. It is designed to address the underlying genetic cause of the condition.
SPK-8011 is a gene therapy that works by delivering a functional copy of the gene responsible for producing clotting factor VIII. This aims to enable the body to produce the missing clotting protein, potentially reducing or eliminating the need for regular factor replacement infusions.
SPK-8011 is being developed by Roche Holding AG, a multinational healthcare company. Roche's stock is traded on the OTC market under the ticker symbol RHHBY.
SPK-8011 is in Phase 1 clinical development. It is an investigational therapy and has not yet been approved by regulatory authorities. The Phase 1 trial has been completed, and the therapy remains under investigation for safety and efficacy.
SPK-8011 has been studied in one completed Phase 1 clinical trial, identified as NCT03003533, titled 'A Gene Transfer Study for Hemophilia A.' This trial enrolled 25 male participants aged 18 years and older across the United States, Australia, Canada, Israel, and Thailand.
SPK-8011 is a distinct investigational gene therapy developed by Roche. It is not known to be the same as any other named gene therapy product. Its specific design and manufacturing process differentiate it from other approaches in development.