A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish)
- Conditions
- Spinal Muscular Atrophy
- Registration Number
- JPRN-jRCT2080223666
- Lead Sponsor
- CHUGAI PHARMACEUTICAL CO., LTD.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 168
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
- 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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method efficacy<br>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 Outcome Measures
Name Time Method