Spinal Muscular Atrophy
Conditions
Sponsors
CHUGAI PHARMACEUTICAL CO., LTD.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Type 2 or 3 SMA non-ambulant - Confirmed diagnosis of 5q-autosomal recessive SMA 1) revised upper limb module(RULM) entry item A greater than or equal to [>=] 2; 2) ability to sit independently as assessed by item 9 of the motor function measure (MFM) - Negative blood pregnancy test at screening and agreement to comply with measures to prevent pregnancy and restrictions on sperm donation
Exclusion criteria
Exclusion criteria: - Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening, or 5 half-lives of the drug, whichever is longer - Concomitant or previous participation at any time in a Survival of motor neuron (SMN)-2 targeting antisense oligonucleotide, SMN2 splicing modifier or gene therapy study - Any history of cell therapy - Hospitalization for a pulmonary event within the last 2 months or planned at time of screening - Surgery for scoliosis or hip fixation in the one year preceding screening or planned within the next 18 months - Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator - Presence of clinically significant electrocardiogram (ECG) abnormalities before study drug administration from average of triplicate measurement or cardiovascular disease indicating a safety risk for participants as determined by the Investigator - Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration - Any OCT 2 and MATE substrates within 2 weeks before dosing (including but not limited to: amantadine, cimetidine, memantine, amiloride, famotidine, metformin, pindolol, ranitidine, procainamide, varenicline, acyclovir, ganciclovir, oxaliplatin, cephalexin, cephradine, fexofenadine). - Recently initiated treatment (within less than [<] 6 months prior to randomization) with oral salbutamol or another beta 2-adrenergic agonist taken orally - Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or thioridazine, is not allowed - Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to RO7034067 or to the constituents of its formulation - Recent history (less than one year) of ophthalmological diseases
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy Part 2: Change from baseline in the total motor function measure 32 (MFM-32) score at Month 12 [ Time Frame: Baseline (Day -1) and Month 12 ] | — |
Secondary
| Measure | Time frame |
|---|---|
| safety efficacy confirmatory exploratory pharmacokinetics pharmacodynamics pharmacogenomics - Survival of Motor Neuron 2 (SMN2) Messenger Ribonucleic Acid (mRNA) Levels in Blood - Survival of Motor Neuron (SMN) Protein Levels in Blood - Change From Baseline in Total Score of Hammersmith Functional Motor Scale Expanded (HFMSE) at Month 12 - Change From Baseline in the Total Score of the Revised Upper Limb Module (RULM) at Month 12 - Percentage of Participants who Achieve Stabilization or Improvement (Defined as >= 0) in the Total Motor function measure (MFM) Score at Month 12 - Change From Baseline in the Best Sniff Nasal Inspiratory Pressure (SNIP) at Month 12 [ Time Frame: Baseline (Day-1) and Month 12 ] - Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 12 - Change From Baseline in Forced Vital Capacity (FVC) at Month 12 - Change From Baseline in the Peak Cough Flow (PCF) at Month 12 - Survival of Motor Neuron 2 (SMN2) Messenger Ribonucleic Acid (mRNA) Levels in Blood - Survival of Motor Neuron (SMN) Protein Levels in Blood | — |
Countries
Belgium, Brazil, Canada, China, Croatia, France, Italy, Japan, Poland, Russian Federation, Serbia, Spain, Turkey, United States
Outcome results
None listed