Studio di fase 3 per valutare l’efficacia di una dose singola di sgt-003 per maschi ambulanti con distrofia muscolare di Duchenne

Baseline Assessment

After joining the study, a series of measurements are taken before any treatment.

These include a review of medical history, a physical Examination, tests of muscle strength and walking ability, blood samples, an electrocardiogram (ECG) to check heart rhythm, and an echocardiogram (echo) to look at heart structure.

Random assignment

The Patient is assigned by chance to receive either the active product sgt-003 or an inactive solution called placebo (normal saline).

The study is double‑blind, meaning neither the Patient nor the clinical staff knows which one is given.

Intravenous infusion

On the same day as random assignment, a single intravenous infusion is administered.

The active product consists of a single dose of 100000000000000 units of sgt-003 given as a suspension for IV infusion.

The placebo consists of a matching infusion of normal saline (0.9% sodium chloride).

The infusion is delivered through a vein over a short period, typically lasting less than an hour.

Post‑infusion monitoring

After the infusion, vital signs such as blood pressure and heart rate are checked regularly for several hours.

Any immediate side effects are recorded before the Patient is allowed to leave the Clinic.

Discharge

Once the monitoring period is completed and no acute problems are observed, the Patient returns home.

The Patient is instructed to note any new symptoms and to report them to the study team if they occur.

Follow‑up visit at day 90

Approximately three months after the infusion, a scheduled visit is made.

Blood samples are taken to Measure the level of microdystrophin, a protein that the therapy is intended to produce.

Additional safety checks such as another ECG, echo, and routine laboratory tests are performed.

The Patient is asked about any adverse events that may have occurred since the infusion.

Long‑term follow‑up through day 540

Over the next 1.5 years, the Patient attends periodic visits, with the main Assessment at day 540.

Muscle function is evaluated using several tests: time to rise from a chair, stride speed measured by a wearable device, 10‑meter walk/run speed, and 4‑stair climb speed.

The North Star Ambulatory Assessment (nsaa) score, which rates overall walking ability, is recorded.

Lung function is measured by forced vital capacity (FVC), peak expiratory flow (PEF), and forced expiratory volume in one second (FEV1).

A quality‑of‑life Questionnaire (PODCI) is completed.

Throughout the entire 540‑day period, any adverse events, serious adverse events, or events of special interest are documented.

Study completion

After the day 540 assessments are finished, the participant’s involvement in the trial ends.

Final safety data are collected and the Patient may continue regular clinical care as advised by their own Physician.