NCT06149403 - A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

December 11, 2023

Primary Completion Date

March 31, 2028

Study Completion Date

March 31, 2031

Conditions

MPS-IH (Hurler Syndrome)

Interventions

GENETIC

Experimental: OTL-203

Experimental: OTL-203: Autologous CD34+ enriched cell fraction that contains hematopoietic stem and progenitor cells transduced ex vivo using lentiviral vector encoding the human IDUA gene

GENETIC

Active Comparator: Allo-HSCT

Active Comparator: Allogeneic hematopoietic stem cell transplantation

Trial Locations (5)

20131

Ospedale San Raffaele, Milan

55455

University of Minnesota, Pediatrics, Minneapolis

3584 CS

Princess Maxima Center, Utrecht

3584 CX

UMC Utrecht, Utrecht

M13 9WL

Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital, Manchester