Duoc-01 / Duke University Medical Center - LARVOL DELTA

DUOC for MS: Intrathecal Administration of DUOC-01 in Adults With Primary Progressive Multiple Sclerosis (clinicaltrials.gov) - P1 | N=20 | Active, not recruiting | Sponsor: Joanne Kurtzberg, MD | Recruiting ➔ Active, not recruiting | Trial completion date: Aug 2025 ➔ Feb 2026 | Trial primary completion date: Aug 2025 ➔ Feb 2026

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Multiple Sclerosis

Safety and feasibility of umbilical cord blood transplantation in children with neuronal ceroid lipofuscinosis: a retrospective study. (PubMed, Stem Cells Transl Med) - P1 | "Four also enrolled in clinical trial NCT02254863 and received intrathecal DUOC-01 cells posttransplant...Three of 4 CLN3 subjects stabilized Hamburg motor and language scores. While UCBT appears safe and feasible in these patients, given the variable expression and natural history, extended follow-up and further studies are needed to elucidate the potential impact of UCBT on clinical outcomes."

Journal • Retrospective data • Acute Graft versus Host Disease • Autoimmune Hemolytic Anemia • Bone Marrow Transplantation • CNS Disorders • Epilepsy • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Transplantation

DUOC-01: UCB Transplant of Inherited Metabolic Diseases with Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Joanne Kurtzberg, MD | Trial completion date: Oct 2024 ➔ Oct 2025 | Trial primary completion date: Oct 2024 ➔ Oct 2025

Trial completion date • Trial primary completion date • Bone Marrow Transplantation • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Solid Tumor • Transplantation

DUOC for MS: Intrathecal Administration of DUOC-01 in Adults With Primary Progressive Multiple Sclerosis (clinicaltrials.gov) - P1 | N=20 | Recruiting | Sponsor: Joanne Kurtzberg, MD | Trial completion date: Aug 2024 ➔ Aug 2025 | Trial primary completion date: Aug 2024 ➔ Aug 2025

Trial completion date • Trial primary completion date • CNS Disorders • Multiple Sclerosis

DUOC-01: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Joanne Kurtzberg, MD | Trial completion date: Oct 2023 ➔ Oct 2024 | Trial primary completion date: Oct 2023 ➔ Oct 2024

Trial completion date • Trial primary completion date • Bone Marrow Transplantation • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Solid Tumor • Transplantation

Cellular therapies: yesterday, today, and tomorrow. (PubMed, Stem Cells Dev) - "Indeed, different types of stem cells (e.g., hematopoietic, mesenchymal, neural, pluripotent), as well as different lymphoid and myeloid cell populations (e.g., TILs, CAR-Ts, CAR-NKs, DUOC-01) are being used in clinical settings or are being tested in clinical trials...Technical, ethical, and economic issues persist and they need to be addressed. Undoubtedly, exciting times of challenges and opportunities are coming ahead in the CT arena."

Journal • Hematological Disorders • Transplantation

DUOC-01: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Joanne Kurtzberg, MD | N=12 ➔ 40 | Trial completion date: Sep 2022 ➔ Oct 2023 | Trial primary completion date: Sep 2022 ➔ Oct 2023

Enrollment change • Trial completion date • Trial primary completion date • Bone Marrow Transplantation • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Solid Tumor • Transplantation

DUOC for MS: Intrathecal Administration of DUOC-01 in Adults With Primary Progressive Multiple Sclerosis (clinicaltrials.gov) - P1 | N=20 | Recruiting | Sponsor: Joanne Kurtzberg, MD | Not yet recruiting ➔ Recruiting | Initiation date: Nov 2021 ➔ Jan 2021

Enrollment open • Trial initiation date • CNS Disorders • Multiple Sclerosis

DUOC for MS: Intrathecal Administration of DUOC-01 in Adults With Primary Progressive Multiple Sclerosis (clinicaltrials.gov) - P1; N=20; Not yet recruiting; Sponsor: Joanne Kurtzberg, MD

Clinical • New P1 trial • CNS Disorders • Multiple Sclerosis

DUOC-01: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (clinicaltrials.gov) - P1; N=12; Recruiting; Sponsor: Joanne Kurtzberg, MD; Trial completion date: Sep 2020 ➔ Sep 2022; Trial primary completion date: Sep 2020 ➔ Sep 2022

Trial completion date • Trial primary completion date