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)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

F. Hoffmann-La Roche AG

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05], Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Nervous System Diseases [C10]

Trial duration

26 Sep 2024 → ongoing

Decision date (initial)

2025-09-22

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

F. Hoffmann-La Roche AG

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Pharmacodynamic, Efficacy, Pharmacokinetic, Safety

To evaluate the efficacy of satralizumab in bone mineral density (BMD) as assessed by dual-energy X-ray absorptiometry (DEXA) in fracture-naïve participants

Secondary objectives 7

1. To evaluate the safety of satralizumab in DMD
2. To evaluate the efficacy of satralizumab in BMD as assessed by DEXA in all participants
3. To evaluate the efficacy of satralizumab in bone metabolism biomarkers in all participants
4. To evaluate the efficacy of satralizumab in the incidence of fractures
5. To characterize the pharmacokinetics of satralizumab
6. To evaluate the immunogenicity of satralizumab
7. To evaluate the efficacy of satralizumab in bone metabolism biomarkers in fracture naive participants

Conditions and MedDRA coding

Duchenne Muscular Dystrophy (DMD)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Age ≥ 8 and < 18 years at the time of signing Informed Consent Form
2. Male at birth
3. Group 2 participants are required to meet the following criteria: Be fracture naïve, defined as: No history of prior low-trauma fractures before the baseline visit nor any radiological findings indicative of prevalent vertebral fracture (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
4. Group 1 participants are required to meet the following criteria: – SDI ≤ 3 - Age ≥ 8 to < 18 years old at the time of screening – If ambulatory (defined as able to walk independently without assistive devices) the participant must have a prior history of fractures Prior history of low-trauma fracture defined as: evidence of at least one prevalent vertebral compression fracture of Genant Grade 1 or 2 (or radiographic signs of VF) or history of at least one low-trauma long bone fracture (upper or lower extremity) but no more than two events incurring in low-trauma fractures (at any anatomical site) OR – If non-ambulatory, characterized as being non-ambulatory for a minimum of 6 months with onset of non-ambulatory status defined as participant- or caregiver-reported age of continuous wheelchair use, approximated to the nearest month, and an NSAA walk score of "0" and inability to perform the 10MWR at the baseline visit, the participant can be with or without prevalent fractures at baseline
5. For ambulatory participants: Group 1 and 2: NSAA total score ≥ 16 as assessed at the screening visit
6. Daily oral corticosteroids for at least 12 months with a stable dose for at least 12 weeks prior to screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the study

Exclusion criteria 6

1. 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
2. Presence of any clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for the participant or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the subject’s ability to comply with the protocol required testing or procedures or compromise the subject’s wellbeing, safety, or clinical interpretability
3. Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection
4. Has a symptomatic infection (e.g. upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to baseline
5. History or laboratory evidence of coagulation disorders
6. Body weight at screening < 20 or > 100 kg

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 1. Change from baseline to Week 52 in LS BMD Z-score measured by DEXA

Secondary endpoints 14

1. 1. Incidence of treatment-emergent adverse events
2. 2. Incidence of serious adverse events
3. 3. Incidence of adverse events of special interest
4. 4. Clinically significant changes in vital signs and physical examination findings
5. 5. Clinically significant changes in safety laboratory assessments, ECGs
6. 6. Change from baseline to Weeks 24, 52 and 104 in LS BMD Z-score measured by DEXA
7. 7. Change from baseline to Weeks 24, and 52 and 104 in TBLH BMD Z-score measured by DEXA
8. 8. Change from baseline to Week 24, week 52 and week 104 in total hip BMD Z-score measured by DEXA
9. 9 . Change from baseline to Weeks 12, 24 and 52 in circulating bone metabolism biomarkers
10. 10 . Number of new low-trauma long-bone or vertebral fractures by Week 52 and week 104
11. 11 . Proportion of Participants with new low-trauma long-bone or vertebral fracture by Week 52 and week 104
12. 12. Summary of observed serum concentration of satralizumab at specified trough timepoints up to Week 104
13. 13. Population and individual estimates of PK parameters (e.g., apparent clearance and apparent volume of distribution) and secondary PK parameters (e.g., area under the concentration-time curve)
14. 14. Prevalence of ADAs at baseline and incidence of ADAs during the study

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

F. Hoffmann-La Roche AG

Sponsor organisation

F. Hoffmann-La Roche AG

Address

Grenzacherstrasse 124

City

Basel

Postcode

4058

Country

Switzerland

Scientific contact point

Organisation

F. Hoffmann-La Roche AG

Contact name

Trial Information System - TISL

Public contact point

Organisation

F. Hoffmann-La Roche AG

Contact name

Trial Information System - TISL

Third parties 11

Locations

4 EU/EEA countries · 12 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 68 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications