Long-term Safety Study of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy Who Previously Received SRP-9001

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What is this study about?

This clinical trial is focused on studying Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle weakness and degeneration. The treatment being evaluated is called Delandistrogene Moxeparvovec, also known by its code name SRP-9001. This treatment is a solution for injection or infusion, designed to help manage the symptoms of Duchenne Muscular Dystrophy.

The purpose of this study is to assess the long-term safety of Delandistrogene Moxeparvovec in participants who have previously received this treatment in an earlier clinical study. Participants will be monitored over a period of time to observe any potential side effects or adverse events that may occur after receiving the treatment. The study will also track changes in participants’ physical abilities, such as their ability to walk, run, and perform upper limb tasks, as well as their lung function and heart health.

Throughout the study, participants will undergo various assessments, including the North Star Ambulatory Assessment and Magnetic Resonance Imaging (MRI), to evaluate changes in their condition from the time before they received the treatment to five years after. This long-term follow-up is crucial to understanding the overall impact of Delandistrogene Moxeparvovec on individuals with Duchenne Muscular Dystrophy.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying that the participant has previously received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a prior clinical study.

The participant or their legal caregiver must demonstrate the ability to understand and comply with the study visit schedule and protocol requirements.

2 treatment administration

The study involves the administration of delandistrogene moxeparvovec, which is a solution for injection or infusion given intravenously.

This step is a follow-up to the initial treatment received in a previous study and does not involve new administration during this trial.

3 monitoring and follow-up

Participants are monitored for any treatment-emergent adverse events, serious adverse events, and adverse events of special interest.

The study aims to evaluate the long-term safety of delandistrogene moxeparvovec over a period of up to five years post-infusion.

4 assessment of physical abilities

Changes in physical abilities are assessed using various measures, including the North Star Ambulatory Assessment, time to rise from the floor, and time for a 10-meter walk or run.

These assessments are conducted from the pre-infusion baseline to five years post-infusion.

5 evaluation of respiratory and cardiac function

Changes in respiratory function are evaluated through measures such as forced vital capacity and peak expiratory flow percent predicted.

Cardiac function is assessed using cardiac magnetic resonance imaging (MRI) findings.

6 musculoskeletal evaluation

Musculoskeletal MRI findings are evaluated to assess any changes from the pre-infusion baseline to five years post-infusion.

7 completion of study

The study is estimated to conclude by November 15, 2030, with all assessments and evaluations completed by this date.

Who Can Join the Study?

  • The patient must have received delandistrogene moxeparvovec for Duchenne Muscular Dystrophy in a previous clinical study. This means they have already been treated with a specific medication for a muscle condition.
  • The patient must have a parent or legal caregiver, or be at least 18 years old and able to understand and follow the study visit schedule and all other study requirements. This means they need to be able to attend appointments and follow instructions.
  • The study is only for male patients. This means only boys or men can participate.

Who Cannot Join the Study?

  • Only males can participate in the study. Females are not eligible.
  • Participants must have a specific condition called Duchenne Muscular Dystrophy.
  • Participants must fall within certain age ranges, which are not specified here.
  • There are no specific exclusion criteria listed, but participants must meet the general requirements mentioned above.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Hospital Universitario Y Politecnico La Fe Valencia Spain

Other Sites

Site Name City Country Status
IRCCS Istituto Giannina Gaslini Genoa Italy
Universitair Ziekenhuis Gent Gent Belgium
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Ukdsvserarssfpejdywfz Emvek Aww Essen Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
29.05.2024
Germany Germany
Recruiting
29.05.2024
Italy Italy
Recruiting
29.05.2024
Spain Spain
Recruiting
29.05.2024

Trial locations

Investigated drugs:

SRP-9001 is a gene therapy being studied for its long-term safety and effectiveness. It is designed to help treat certain genetic conditions by delivering a specific gene to the body. This therapy aims to address the underlying cause of the condition by providing the body with the necessary genetic material to produce a missing or faulty protein.

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 loss of mobility. Over time, complications can arise in the heart and respiratory muscles, further impacting physical abilities. The progression of muscle deterioration varies among individuals, but it generally leads to significant physical disability.

Trial ID:
2023-505043-39-00
Protocol code:
SRP-9001-305
NCT ID:
NCT05967351
Trial Phase:
Therapeutic confirmatory (Phase III)

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