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A study to check the effect of satralizumab in children and adolescents living with Duchenne muscular dystrophy, to check if it is safe and also how it affects the different parts of the body and how it is eliminated from the body (SHIELD DMD)
An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy
- Muscle And Peripheral Nerve Disease
- Duchenne Muscular Dystrophy (DMD)
Please note that the recruitment status of the trial at your site may differ from the overall study status because some study sites may recruit earlier than others.
Trial Status:
Not yet recruiting
This trial runs in
Country
- United States
Trial Identifier:
NCT06450639 2024-512383-65-00 BN45398
Trial Summary
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy (DMD) age ≥ 8 to < 16 years old receiving corticosteroid therapy.
Hoffmann-La Roche
Sponsor
Phase 2
Phase
NCT06450639, BN45398, 2024-512383-65-00
Trial Identifier
Male
Gender
≥8 Years & ≤ 15 Years
Age
No
Healthy Volunteers
1. Why is this study needed?
Duchenne muscular dystrophy (DMD) is a rare genetic disorder that causes muscle weakness and wasting and leads to difficulty in movement. It is usually seen in boys. DMD affects muscles that control movement and muscles in the heart and lungs. DMD also makes bones weaker so people with DMD have a high risk of broken bones. Certain medicines called glucocorticoids are currently the standard of care for the treatment of DMD. However, these medicines have many unwanted effects, including making the bones even weaker. Additionally, there are currently no approved treatments for DMD that specifically target weakened and broken bones.
This study is testing a medicine called satralizumab (RO5333787) to check if it can have an effect on the muscles and the bones of people living with DMD. Satralizumab is an experimental medicine for DMD. This means health authorities (like the U.S. Food and Drug Administration and European Medicines Agency) have not approved satralizumab for the treatment of people with DMD. However, satralizumab is currently approved for the treatment of another disease called Neuromielitis Optica Spectrum Disorder (NMOSD). This study aims to find out the effects of satralizumab when given along with glucocorticoids in children with DMD.
2. Who can take part in the study?
Male children between 8 and 15 years of age with DMD weighing no more than 100 kilograms (kg) and taking daily corticosteroids can take part in the study. Children with and without a history of broken bones who may or may not be able to walk on their own can take part in this study.
Children cannot take part in this study if they have had a major surgery within 3 months before the study or have planned a surgery or procedure during this study. Children who have either received gene therapy or are receiving other experimental drugs and those who present other health conditions that could create a safety risk for them cannot take part in this study.
3. How does this study work?
Children will be screened to check if they are able to participate in the study. The screening will take place for up to 12 weeks before the start of the treatment.
Everyone who joins this study will be given satralizumab as an injection under the skin in the belly or thigh at Weeks 0, 2, 4, and every 4 weeks thereafter for 104 weeks. The doses of satralizumab will depend on the participant’s body weight. Participants will have regular blood tests and will be checked for unwanted effects throughout the study.
This is an open-label study. This means everyone involved, including the participant and the study doctor, will know the study treatment the participant has been given.
During this study, the study doctor will see the participants 12 times during their clinic visits. These visits will first occur every two weeks (Day 1, Week 2, and Week 4), then every four weeks for the following two visits (weeks 8 and 12), and then approximately every 3 to 4 months for the rest of the first year. For the second year, the visits will happen every 6 months. Study doctors will see how well the treatment is working and any unwanted effects participants may have. At the end the participants will have a follow-up visit after 12 weeks of completing the study treatment, during which the study doctor will check on the participant’s well-being.
Total time of participation in the study will be about 2 years. Participants have the right to stop study treatment and leave the study at any time, if they wish to do so. Participants will also occasionally receive follow-up calls to check on how they are doing. If they experience any unwanted effects or injury during the study, the clinical trial doctor will explain the options and discuss a plan for further treatment.
4. What are the main results measured in this study?
The main result measured in the study is to assess if the medicine changes the bone mineral density of the bones of the lower back. This will be measured using dual-energy X-ray absorptiometry (DEXA) scan, from the start of the study to Week 52. DEXA is a scan that measures bone density by passing high and low energy X-rays through the body.
Other key results measured in the study include:
- Number of participants with unwanted effects
- Changes in the rate at which bone structure is created and broken down
- Average number of new broken bones during the study
- Number of participants with new broken bones during the study
- Change in the time to rise from the floor in participants who can walk on their own
- How well the body processes satralizumab
- Number of participants who had their body’s defense system respond to satralizumab.
5. Are there any risks or benefits in taking part in this study?
Taking part in the study may or may not make participants feel better. But the information collected in the study can help other people with similar health conditions in the future.
The study involves some risks to the participant. These risks are generally not greater than those related to routine medical care or the natural progression of DMD. People interested in taking part will be informed about the risks and benefits, as well as any additional procedures or tests they may need to undergo. All details of the study will be described in an informed consent document. This includes information about possible effects and other options of treatment.
Risks associated with the study drug
Participants may have unwanted effects of the drug used in this study. These unwanted effects can be mild to severe, even life-threatening, and vary from person to person. During this study, participants will have regular check-ups to see if there are any unwanted effects.
Satralizumab
Participants will be told about the known unwanted effects of satralizumab, and possible unwanted effects based on human and laboratory studies or knowledge of similar medicines. Known unwanted effects include decreased white blood cell count, headache, joint pain, difficulty sleeping or falling asleep, hay fever, itching, and migraine.
Satralizumab will be given as an injection under the skin in the belly or thigh. Known unwanted effects of injection under the skin include irritation where the injection is given, such as swelling, rash, redness, itching or pain.
Trial Summary
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy (DMD) age ≥ 8 to < 16 years old receiving corticosteroid therapy.
Hoffmann-La Roche
Sponsor
Phase 2
Phase
NCT06450639, BN45398, 2024-512383-65-00
Trial Identifier
Satralizumab
Treatments
Duchenne Muscular Dystrophy
Condition
Official Title
A phase II multicenter, open-label study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in pediatric patients with Duchenne muscular dystrophy (SHIELD DMD)
Eligibility Criteria
Male
Gender
≥8 Years & ≤ 15 Years
Age
No
Healthy Volunteers
Inclusion Criteria
- Signed Informed Consent Form and Signed Assent Form when appropriate
- Male at birth
- A definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
- Age ≥ 8 and < 16 years at the time of signing Informed Consent Form
- Participants who are fracture-naive are additionally required to meet the following criteria:
- No history of prior low-trauma fractures before the baseline visit nor any radiological findings indicative of prevalent VF at the screening visit * Be ambulatory, defined as able to walk independently without assistive devices. - Age ≥ 8 to < 12 years old at the time of screening
- Participants with a prior history of low-trauma fractures are additionally required to meet the following criteria:
- Evidence of at least one prevalent vertebral compression fracture of Genant Grade 1 or higher (or radiographic signs of VF) or history of at least one low-trauma long-bone fracture (upper or lower extremity) * Ambulatory (see above definition) if age ≥ 8 to < 12. * Non-ambulatory if age ≥ 12 to <16
- Daily oral corticosteroids
Exclusion Criteria
- Major surgery (e.g. spinal surgery) within 3 months prior to Baseline or planned surgery or procedure that would interfere with the conduct of the study for any time during this study •Presence of any clinically significant illness
- Has serological evidence of current, chronic, or active human immunodeficiency virus, tuberculosis, hepatitis C, or hepatitis B infection •Has a symptomatic infection (e.g. upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to baseline
- Body weight > 100 kg •Treatment with prohibited therapies as defined by the protocol
- Has received a live or live attenuated virus vaccine within 6 weeks of the Baseline visit or expects to receive a vaccination during the first 3 months after Baseline.
- Has abnormal laboratory values considered clinically significant as defined by the protocol
- Any medical condition that might interfere with the evaluation of lumbar spine BMD, such as severe scoliosis or spinal fusion.
- Participant has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator
- Participant has an allergy or hypersensitivity to the study medication or to any of its constituents
Other protocol defined inclusion and exclusion criteria may apply
For the latest version of this information please go to www.forpatients.roche.com