Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SPK-8016 · 1 trial · 17 indications
An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. SAEs were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
| Arm | Type | Description |
|---|---|---|
| SPK-8016 | EXPERIMENTAL | All participants who meet the eligibility criteria will receive an outpatient single intravenous (i.v.) administration of SPK-8016. |
| Name | Type | Description |
|---|---|---|
| SPK-8016 | GENETIC | adeno-associated viral vector |
Inclusion Criteria: 1. Be male and ≥18 years of age; 2. Have clinically severe hemophilia A, defined as: 1. \<1% (\<1 IU/dL) endogenous FVIII activity levels as historically documented by a certified laboratory or screening data results; OR 2. 1-2% (1-2 IU/dL) endogenous FVIII activity level...
SPK-8016 is an investigational gene therapy being studied for the treatment of hemophilia A, specifically to support evaluation in individuals with FVIII inhibitors. It is designed to address Factor VIII deficiency, a blood coagulation disorder. The drug is in Phase 1 clinical development and is not yet approved by regulatory authorities.
SPK-8016 targets the Factor VIII (FVIII) gene and protein. It is a gene therapy that aims to address Factor VIII deficiency, which is the underlying cause of hemophilia A. The therapy is designed to deliver a functional copy of the FVIII gene to help restore normal blood coagulation.
SPK-8016 is being developed by Roche Holding AG, a biopharmaceutical company. Roche's stock is traded under the ticker symbol RHHBY. The company is conducting clinical research on this gene therapy for hemophilia A.
SPK-8016 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The Phase 1 trial has been completed, and the drug remains under investigation for safety and dosing.
SPK-8016 has one completed clinical trial, identified as NCT03734588. This was a Phase 1 dose-finding study in patients with hemophilia A to support evaluation in individuals with FVIII inhibitors. The study enrolled 4 male participants aged 18 years and older in the United States.
SPK-8016 is a gene therapy specifically designed for hemophilia A, a condition caused by Factor VIII deficiency. It uses an Adeno-Associated Virus (AAV) vector to deliver the FVIII gene. The therapy is being studied to help patients who have developed inhibitors to standard Factor VIII replacement therapy.