DTX101 / Ultragenyx - LARVOL DELTA

Gene Therapy for Hemophilia B: Results From the Phase1/2 101HEMB 1/2 Studies. (PubMed, Blood Adv) - P=N/A, P1/2 | "Elevated transaminase levels were asymptomatic and resolved with steroids in all subjects. The DTX101 program was halted for insufficient treatment response; however, from its completion, lessons can be learned regarding the design and execution of gene therapy clinical trials including additional optimization of transgene sequence and immunosuppression protocols."

Journal • P1/2 data • Fatigue • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B (clinicaltrials.gov) - P=N/A; N=6; Completed; Sponsor: Ultragenyx Pharmaceutical Inc; Active, not recruiting ➔ Completed; Trial completion date: Feb 2022 ➔ Nov 2021; Trial primary completion date: Feb 2022 ➔ Nov 2021

Clinical • Trial completion • Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B (clinicaltrials.gov) - P=N/A; N=6; Active, not recruiting; Sponsor: Ultragenyx Pharmaceutical Inc; Completed ➔ Active, not recruiting; Trial completion date: Oct 2017 ➔ Feb 2022; Trial primary completion date: Oct 2017 ➔ Feb 2022

Clinical • Enrollment closed • Trial completion date • Trial primary completion date