Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Summary

A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.

Timeline

Start

2015-12-16

Primary completion

2017-10-18

Completion

2017-10-18

Outcome

Missed primary endpoint

Stopped (Business): “Sponsor decision; not due to any safety concerns related to DTX101.”

paper Levels failed to reach the 20 IU/dL target criteria; PMID 40197980 ↗

Publications

• Pipe S, Poma A, Rajasekhar A, Everington T, Sankoh S, Allen J, Cataldo J, Crombez E. Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies. Blood Adv. 2025 Jun 24;9(12):2980-2987. doi: 10.1182/bloodadvances.2024015184.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	DTX101	Gene therapy (AAV / viral vector)	1.6e+12 vg/kg	Intravenous
Subject	DTX101	Gene therapy (AAV / viral vector)	5e+12 vg/kg	Intravenous

Indications