Study on the Effects of Satralizumab in Children and Adolescents with Duchenne Muscular Dystrophy

2 1 1

What is this study about?

This clinical trial is focused on studying the effects of a medication called Satralizumab in children and adolescents who have Duchenne Muscular Dystrophy (DMD). DMD is a genetic disorder characterized by progressive muscle weakness and degeneration. The medication being tested, Satralizumab, is given as a solution for injection under the skin. It is a type of protein that works by targeting specific pathways in the body to potentially help manage symptoms of DMD.

The purpose of this study is to evaluate how effective Satralizumab is in improving bone health, specifically looking at bone mineral density, which is a measure of bone strength. The study will also assess the safety of the medication and how it interacts with the body. Participants in the study will receive either Satralizumab or a placebo, and their progress will be monitored over a period of time to see how the treatment affects their condition.

Throughout the study, participants will undergo various assessments to track changes in their bone health and overall well-being. These assessments will include regular check-ups and tests to measure bone density and other health indicators. The study aims to provide valuable information on the potential benefits and safety of Satralizumab for young patients with Duchenne Muscular Dystrophy.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria such as age, gender, and medical history related to Duchenne Muscular Dystrophy (DMD).

Participants must be between 8 and 16 years old and meet certain health conditions, including being fracture-naive or having a history of low-trauma fractures.

2 initial assessment

An initial assessment is conducted to establish a baseline for various health parameters. This includes measuring bone mineral density (BMD) using a dual-energy X-ray absorptiometry (DEXA) scan.

Other assessments may include physical examinations, laboratory tests, and evaluations of current medication use.

3 medication administration

The medication satralizumab is administered as a subcutaneous injection. The frequency and dosage are determined by the study protocol and are monitored throughout the trial.

Participants continue their daily oral corticosteroids, maintaining a stable dose as required by the study.

4 ongoing monitoring

Participants are monitored for any treatment-emergent adverse events, serious adverse events, and changes in vital signs or laboratory assessments.

Regular follow-up visits are scheduled to assess the effects of the medication on bone mineral density and other health indicators.

5 midpoint evaluation

At weeks 24 and 52, changes in bone mineral density and other health markers are evaluated using DEXA scans and other tests.

The study also measures the presence of any new low-trauma fractures and changes in physical abilities.

6 final assessment

At the end of the study period, a comprehensive evaluation is conducted to assess the overall impact of satralizumab on the participants’ health.

This includes a final DEXA scan, laboratory tests, and assessments of any adverse events experienced during the trial.

Who Can Join the Study?

Must be between 8 and 16 years old at the time of signing the consent form.
Must be male at birth.
For participants who have never had a fracture:
- No history of fractures from minor injuries before the study starts.
- No signs of broken bones in the spine from X-rays at the start of the study.
- Must be able to walk independently without help.
- Must be between 8 and 12 years old at the start of the study.
For participants with a history of fractures:
- Must have at least one broken bone in the spine or a history of a broken long bone (like an arm or leg) from minor injuries, but no more than two such fractures.
- Must have a score of less than 3 on a specific fracture assessment.
- If between 8 and 12 years old, must be able to walk independently.
- If between 12 and 16 years old, must have been unable to walk for at least 6 months and use a wheelchair, with a specific walking score of “0” and unable to perform a 10-meter walk test at the start of the study.
For those who can walk: must be able to get up from the floor without help in less than 10 seconds during the initial assessment.
Must have been taking daily oral corticosteroids (a type of medication) for at least 12 months, with a stable dose for at least 12 weeks before the study starts, and the dose should remain constant during the study, except for changes due to weight.

Who Cannot Join the Study?

Participants who have had fractures before cannot join the study. A fracture is a break in a bone.
Only male participants are allowed in the study, so females cannot participate.
Participants who are not within the specified age range cannot join. The age range is not specified here, but it is important for eligibility.
Participants with medical conditions other than Duchenne Muscular Dystrophy (DMD) may not be eligible. DMD is a genetic disorder that affects muscles.
Participants who are considered part of a vulnerable population may not be eligible. Vulnerable populations include groups that may need special protection or care.

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
Rigshospitalet	Copenhagen	Denmark
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Instytut Centrum Zdrowia Matki Polki	Lodz	Poland
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego	Warsaw	Poland
Hospital Universitario Torrecardenas	Almeria	Spain
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Uniwersytecki Szpital Kliniczny W Poznaniu	Poznan	Poland
Udktfjonhkrpuw Cwygpoh Kuduffgot	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	26.11.2024
Italy	Not recruiting	26.11.2024
Poland	Not recruiting	26.11.2024
Spain	Not recruiting	26.11.2024

Trial locations

Satralizumab is a medication being studied for its potential benefits in children with Duchenne Muscular Dystrophy. The trial aims to assess how effective and safe this medication is, particularly in improving bone mineral density. This is measured using a special type of X-ray called dual-energy X-ray absorptiometry (DEXA). The study is focused on participants who have not experienced fractures before.

Investigated diseases:

Duchenne muscular dystrophy

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 muscle function. 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 disability. 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.

Trial ID:

2024-512383-65-00

Protocol code:

BN45398

Trial Phase:

Therapeutic exploratory (Phase II)

Other Trials to Consider

#1 Clinical Trials Search in Europe

Study on the Effects of Satralizumab in Children and Adolescents with Duchenne Muscular Dystrophy

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?