RecruitingRare disease

Study of SGT-003 Gene Therapy for Safety and Tolerability in Walking Males with Duchenne Muscular Dystrophy

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. The study will explore the effects of a new treatment called SGT-003, which is a type of gene therapy. This therapy involves using a specially designed virus, known as an adeno-associated virus, to deliver a gene called human microdystrophin into the body. The goal is to help the muscles produce a protein that is missing or not working properly in people with DMD.

The purpose of the study is to investigate the safety and tolerability of a single intravenous dose of SGT-003. Participants will receive the treatment through an infusion into a vein. The study will monitor participants over time to see how their bodies respond to the treatment and to check for any side effects. The study will also look at how the treatment affects the levels and distribution of the microdystrophin protein in the body.

Participants in the study will be ambulant males with DMD, meaning they are able to walk without assistance. The study will track various health indicators, such as muscle function and breathing capacity, to assess any changes from the start of the study. The trial will also examine how the body processes the gene therapy and any immune responses that may occur. The study is expected to continue for several years to gather comprehensive data on the treatment's effects.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
The study begins with an initial assessment to confirm eligibility. This includes verifying age, ability to walk without assistance, and a confirmed diagnosis of Duchenne muscular dystrophy (DMD).
Participants must have a stable dose of oral prednisone or deflazacort for at least 12 weeks before starting the study.
Step 2
Baseline measurements
Baseline measurements are taken to assess the current health status. These include tests for microdystrophin protein levels, muscle function, and lung capacity.
Participants will undergo assessments such as the North Star Ambulatory Assessment, 6-minute walk test, and various velocity and capacity tests.
Step 3
Administration of SGT-003
A single dose of SGT-003 is administered intravenously. This is a suspension for IV infusion designed to investigate safety and tolerability.
The administration is monitored closely to observe any immediate reactions.
Step 4
Monitoring and follow-up
Participants are monitored for treatment-emergent adverse events through Day 360. This includes regular check-ups and tests to track any changes in health status.
Secondary assessments include changes in microdystrophin levels, muscle function, and lung capacity, as well as monitoring for any laboratory or physical abnormalities.
Step 5
Final assessment
At the end of the study period, a final assessment is conducted to evaluate the overall impact of the treatment.
This includes a comprehensive review of all collected data to determine the safety and efficacy of the treatment.

Who can join the trial?

10 criteria

The participant must be a boy.
The participant's age should be between 4 and less than 7 years for Cohort 1, or between 7 and less than 12 years for Cohort 2.
The participant must be able to understand and follow all study procedures suitable for their age. A parent or legal guardian must also be able to understand and follow the study requirements.
The participant must be able to walk without needing any help or devices.
The participant must have a confirmed diagnosis of **Duchenne muscular dystrophy (DMD)**, which is a genetic disorder affecting muscles, and this must be confirmed by specific genetic testing.
The participant must test negative for **AAV antibodies**, which are proteins the body makes in response to certain viruses.
The participant must be on a stable dose of oral daily **prednisone** (at least 0.5 mg per kg of body weight per day) or **deflazacort** (0.75 mg per kg of body weight per day) for at least 12 weeks before joining the study. These are medications used to reduce inflammation and help with muscle strength.
The participant must meet specific time criteria for a 10-meter walk or run.
The participant must meet specific time criteria for rising from a lying down position.
The participant's body weight must be 50 kg or less.

Who cannot join the trial?

3 criteria

Only male participants are allowed, so females cannot participate.
Participants must have Duchenne muscular dystrophy, a condition that affects muscles, so those without this condition cannot join.
Participants must be within a specific age range, so those outside this range cannot participate.

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Verified sites

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Verified
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Italy

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Investigated drugs

SGT-003 is an investigational medication being studied for its potential to treat Duchenne Muscular Dystrophy. It is administered as a single intravenous dose. The study aims to evaluate how safe and tolerable this medication is for patients, as well as its potential effectiveness in treating the condition.

SGT-003

What is already known about the treatment

SGT-003 – SGT-003 is administered as a single intravenous dose and is currently being studied in clinical trials for its safety and tolerability. It is primarily being investigated for the treatment of Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration. The medication works at the molecular level by targeting specific genetic components to potentially improve muscle function. SGT-003 falls under the pharmacological classification of gene therapy agents, aiming to address the underlying genetic causes of the disease.

Investigated diseases

Duchenne muscular dystrophy – Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle weakness and degeneration. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. Symptoms usually begin in early childhood, with affected individuals experiencing difficulty in walking, running, and jumping. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to increased difficulty in movement. Over time, individuals may lose the ability to walk and require assistance with daily activities. The progression of muscle weakness can also affect the heart and respiratory muscles.