Open Clinical Trials | Comprehensive Hemophilia Treatment Center

We are committed to advancing hemophilia patient care and facilitating the development of novel treatments through research studies and clinical trials. The development of new strategies, including disease-modifying and curative therapies, form the basis of our progressive approach to hemophilia management for patients at Weill Cornell Medicine.

FIX-LONG Study: A Factor IX (FIX) Gene Transfer, Multi-center Evaluation of the LONG-term Safety and Efficacy Study of SPK-9001 (rAAV-Spark100-hFIX-Padua) in Individuals with Hemophilia B

Diseases:

Blood Disorders | Non-Malignant/Benign Hematology, Hemophilia, Hemophilia B

Location(s):

Upper East Side - Manhattan

Primary Investigator(s):

Catherine McGuinn, MD

Study Details

A Gene-Transfer, Open-Label, Dose-Escalation Study of SPK-8011 [Recombinant Adeno-Associated Viral Vector with B-Domain Deleted Human Factor VIII Gene] in Individuals with Hemophilia A