ARU-1801 (gene therapy)

Product Information

Scientific name: ARU-1801
Brand name: Ν/Α
RESPONSIBLE: Children’s Hospital Medical Center, Cincinnati, US

Clinical Trial/Study Information

Trial Name: Gene Transfer in Patients with SCD (MOMENTUM)
Code: NCT02186418
Phase: 1/2
Eligible patient diagnosis: : SCD (adults) (age 18-45)
No. of Patients enrolled: 7 [anticipated] (Last update: 17/11/2022)
Study Sites: 1 Sites per country

Completion date: June 2023
Scope of the Study / Aim: Increase therapeutic haemoglobin ; decrease annual VOCs ; evaluate RIC melphalan conditioning (less toxic than myeloablative approaches

Regulatory Information

Status: Not Authorised

Additional notable points:

• EMA: N/A
• FDA: N/A
• MHRA: N/A