Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Hoffmann-La Roche
Age
0–55
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Males and females, aged 2 to 55 years inclusive or below 7 months inclusive
Confirmed diagnosis of 5q-autosomal recessive SMA (Types 1 to 3), for patients aged 7 months or below clinical symptoms attributable to type 1 SMA and 2 SMN2 copies
Able and willing to provide informed consent and to comply with the study protocol. Alternatively, a legally authorized representative must be able to consent for the patient and assent must be given by the subject wherever possible.
Female patients of childbearing potential and male patients with a female partner of childbearing potential must agree with the required contraceptive methods as defined per protocol.
For patients aged 7 months or below, Gestational age of 37 to 42 weeks and not considered small for gestational age at birth
You may not be if
Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening
Concomitant or previous participation in a SMN2-targeting antisense oligonucleotide study within 12 months prior to screening
Concomitant or previous participation at any time in a gene therapy study
For patients aged 2-55 years, hospitalization for pulmonary event within the last 2 months or planned at the time of screening
Surgery for scoliosis in the last 6 months from screening or planned within 6 months from screening
Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system disease
Clinically relevant ECG abnormalities at screening or baseline; personal or family history (first degree relatives) of congenital long QT syndrome
Clinically significant abnormalities in laboratory test results at screening
Any concomitant disease or condition that could interfere with the conduct of the study, or pose an unacceptable risk to the subject in this study
Use of prohibited medications as per protocol within 90 days prior to randomization. Patients who are on inhaled corticosteroids, administered either through a nebulizer or an inhaler, are allowed.
Recently initiated treatment (within \<6 months prior to randomization) with oral salbutamol or another beta2-adrenergic agonist taken orally is not allowed. Patients who have been on oral salbutamol (or another beta2-adrenergic agonist) for at least 6 months before randomization are allowed. Use of inhaled beta2-adrenergic agonists is allowed.
For patients aged 7 months or below, patients requiring invasive ventilation or tracheostomy, presence of non-SMA related morbidities
