Clinical trial NCT02240355 (phase 1, status: terminated)
Conditions: muscular atrophy, spinal
ICD codes: G12.9, G12.1, G12.0
Drugs: ro6885247, placebo
SMILES: CN1C(=O)C=C(N2CCC[C@@H](N)C2)N(CC2=C(C=CC=C2)C#N)C1=O
Reason stopped: 
    there was an unexpected toxicology finding observed in the 39 week monkey study. dosing was
    suspended, the study was put on hold and eventually terminated.
  
Eligibility criteria:

        Inclusion Criteria:

          -  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

        Exclusion Criteria:

          -  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