Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Pfizer
Age
4–12
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing
Body weight between 15 and 50 kg
Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry
Ability to rise from floor within seven (7) seconds and ability to walk
You may not be if
Current exposure to systemic immunosuppressant agents other than glucocorticoids.
Prior exposure to any gene therapy agent, including exon-skipping and missense agents.
Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer.
Any injury which may impact functional testing per investigator's judgement. Previous injuries must be fully healed prior to consenting. Prior lower limb fractures must be fully healed and at least 3 months from injury date at screening.
Any planned surgeries which may impact physical activity and performance.
Presence or history of musculoskeletal or neurological disease in addition to DMD.
Any known allergies or skin reactions to stainless steel, versaflex, and silicon that may cause possible discomfort by wearable sensors.
Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, cancer, autoimmune or allergic disease that may interfere with the study conduct as per investigator's judgment, excluding untreated, asymptomatic, seasonal allergies at time of screening.
Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study.
