Assessment of Preliminary Safety and Efficacy of DT-DEC01 Therapy in Duchenne Muscular Dystrophy Patients, up-to 12-months post administration

Topic:

Clinical Trials

Poster Number: 84

Author(s):

Ahlke Heydemann, PhD, University of Illinois at Chicago, Grzegorz Bieganski, MD, Poznan University of Medical Sciences, Jacek Wachowiak, MD, PhD, Department Pediatric Oncology, Hematology and Transplantology, Poznan University of Medical Sciences, Jaroslaw Czarnota, MD, Medpolonia Hospital, Poznan, Krzysztof Siemionow, MD, PhD, University of Illinois at Chicago, Adam Niezgoda, MD, PhD, Department of Neurology, Poznan University of Medical Sciences, Maria Siemionow, MD, PhD, University of Illinois at Chicago

Introduction:
Duchenne muscular dystrophy (DMD) is a lethal X-linked disease, caused by mutations of the dystrophin gene leading to progressive muscle degeneration and weakness.
We developed a novel DT-DEC01 therapy of Dystrophin Expressing Chimeric (DEC) cells created by ex-vivo fusion of human myoblasts derived from normal (allogeneic) donor and DMD-affected (autologous) patient.
This study determined preliminary safety and efficacy following administration of DT-DEC01 to DMD patients.
Methods:
Pilot, open label study. Intraosseous (systemic) administration of a single dose of DT-DEC01 therapy (2×10^6 cells / kg) in 5-18 years old boys (n = 3) with DMD (Bioethics Committee approval no.185/2022).
Adverse event (AE) and serious adverse event (SAE) were assessed. Efficacy was assessed by: 6MWT, NSAA, PUL, grip strength, electromyography (EMG) – Motor Unit Potentials (MUP) and step count.
Results: Average follow up was 9.6 months (range 7-12). No therapy related AE or SAE were reported during follow up. Efficacy assessments revealed:
Patient #1 (7-year-old ambulatory Deletion Exon 3-12): Improved 6MWD +9%, NSAA score +10%, 10-meter run +14%, maintained PUL test (100%), improved grip strength +40%,
EMG reveled increased MUP duration in: biceps +91%; deltoideus +28%, gastrocnemius +30%, rectus femoris +40%, increased step tracker activity.
Patient #2 (15-year-old non-ambulatory Deletion Exon 48-50: Improved PUL +15% compared to baseline. Improved grip strength in both hands +25%; improved biceps +59% and triceps +70% strength. EMG reveled: increased MUP duration in: biceps +140%, deltoideus +65%, gastrocnemius +30%, MUP maintained in rectus femoris.
Patient #3 (6-year-old nonsense mutation): Improved 6MWD +16%, NSAA score +8%, time raise from floor +5%, PUL test +22%. EMG reveled: increased MUP duration in: biceps +6%, rectus femoris +29%, gastrocnemius +5%, increased steps tracker activity.
Conclusions:
This study confirmed safety and preliminary efficacy of a single dose of DT-DEC01 therapy up to 12-months after intraosseous administration to both ambulatory and non-ambulatory DMD patients.