In short
DT-DEC01 is an investigational cell therapy for Duchenne muscular dystrophy (DMD), a genetic disease that causes progressive muscle weakness. It is made by fusing normal donor muscle cells with a patient's own muscle cells. One Phase 1/2 trial (2024-519004-27-00) is currently authorised to evaluate its safety and efficacy in 20 patients with DMD.
Key points
- DT-DEC01 is an investigational cell therapy for Duchenne muscular dystrophy.
- The therapy combines normal donor myoblasts with the patient's own myoblasts.
- It is given by intraosseous injection (into the bone).
- One Phase 1/2 trial is authorised, planning to enrol 20 patients.
- The trial measures safety and functional outcomes such as the 6-minute walk test.
- DT-DEC01 has orphan designation from the European Medicines Agency.
What is DT-DEC01?
DT-DEC01 is an investigational cell therapy, also known by its full registry name ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor. It is a structurally diverse substance classified as a cell therapy. The product is developed by Dystrogen Therapeutics Technology Polska Sp. z o.o. and has no marketing authorisation, meaning it is still under investigation.
The European Medicines Agency (EMA) granted orphan designation (EU/3/18/2089) for DT-DEC01 on 19 November 2018 for the treatment of Duchenne muscular dystrophy. Orphan designation is given to treatments for rare diseases; at the time of designation, DMD affected fewer than 0.5 in 10,000 people in the European Union, equivalent to fewer than 26,000 people.
How does DT-DEC01 work?
DT-DEC01 is created by taking normal muscle cells (myoblasts) from a healthy donor and fusing them with myoblasts derived from the patient's own cells. Duchenne muscular dystrophy is caused by a lack of normal dystrophin, a protein that protects muscles from injury during contraction and relaxation. The fused cells are intended to introduce healthy myoblasts that can contribute to muscle repair. The exact mechanism by which DT-DEC01 may improve muscle function is being evaluated in the ongoing clinical trial.
Which conditions is DT-DEC01 studied for?
- Duchenne muscular dystrophy (DMD) — a genetic disease that gradually causes muscle weakness and wasting, mainly affecting boys, usually starting before age six. The weakness typically begins in the hips and legs before affecting the arms, chest and heart.
How is DT-DEC01 given?
DT-DEC01 is formulated as a suspension for injection and is administered by intraosseous use, meaning it is injected directly into the bone. Intraosseous administration allows the therapy to reach the bone marrow, where blood-forming stem cells reside.
What do the clinical trials show?
One clinical trial is currently authorised for DT-DEC01: a Phase 1/2, open-label, exploratory trial (EU CT number 2024-519004-27-00) evaluating safety and efficacy in patients with Duchenne muscular dystrophy. The trial plans to enrol 20 participants. As an open-label study, both researchers and participants know which treatment is being given.
What are the trials measuring?
The trial has two phases with similar objectives. The primary outcomes are the frequency and severity of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESI), and changes in functional assessments. For ambulatory patients (those who can walk), the trial measures the Six-Minute Walk Test (6MWT), timed functions from the NorthStar Ambulatory Assessment (NSAA) including supine to rise and 10-meter walk/run. For non-ambulatory patients, the trial uses the Performance of Upper Limb (PUL) 2.0 assessment. Both groups also undergo electromyography (EMG) to measure the duration of motor unit potential (MUP) in selected muscles of the upper and lower extremities. These assessments are performed at baseline and at months 1, 3, 6 and 12.
Who can take part in the trial?
The trial includes both ambulatory and non-ambulatory patients with Duchenne muscular dystrophy, with functional assessments adjusted to the stage of the disease. The registry record does not specify additional eligibility criteria such as age range or genetic confirmation of DMD.