A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulant Patients With Spinal Muscular Atrophy

  • End date
    Dec 15, 2026
  • participants needed
  • sponsor
    Hoffmann-La Roche
Updated on 23 October 2022


Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an investigational anti-myostatin antibody that is designed to target myostatin. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. RO7204239 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and clinical outcomes for people living with SMA.

This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in children aged 2-10 years with spinal muscular atrophy (SMA) that are ambulant.

Condition Spinal Muscular Atrophy (SMA)
Treatment Placebo, Risdiplam, RO7204239
Clinical Study IdentifierNCT05115110
SponsorHoffmann-La Roche
Last Modified on23 October 2022


Yes No Not Sure

Inclusion Criteria

Participants who are 2 to 10 years of age inclusive at screening
Participants who have a confirmed genetic diagnosis of 5q-autosomal recessive SMA
Symptomatic SMA disease, as per investigator's clinical judgement
Participants who are ambulant, where ambulant is defined as able to walk/run unassisted (i.e., without the use of assistive devices such as canes, walking sticks, crutches, walkers, person/hand-held assistance, braces, orthoses, over the malleoli insoles or any other type of support) 10 meters in </= 30 as measured by the Timed 10-Meter Walk/Run Test [10MWRT] seconds at screening
Participants who have received previous SMA disease-modifying therapies may be included provided that: Onasemnogene abeparvovec was received at least 90 days prior to screening. Participants should be tapered off steroids prior to receiving risdiplam. In addition, participants should have normal levels of liver function tests, coagulatory parameters, platelets, and troponin-I at 90 days after administration of onasemnogene abeparvovec or at least 1 month after tapering off corticosteroids, whichever comes later; Nusinersen last dose was received at least 90 days prior to screening; Risdiplam is switched to the investigational medicinal product (IMP) provided by the site

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, with the exception of those who have completed a risdiplam study, or participated in a nusinersen or onasemnogene abeparvovec study
Receiving or have received previous administration of anti-myostatin therapies
For Part 1 participants aged 5 to 10 years only: contraindications for MRI scans including difficulties maintaining a prolonged supine position, or any other clinical history or examination finding that would pose a potential hazard in combination with MRI
Any history of cell therapy
Hospitalization for a pulmonary event within the last 2 months or planned hospitalization at the time of screening
Past surgery for scoliosis or hip fixation in the 6 months preceding screening or planned within the next 9 months (Part 1) or 21 months (Part 2)
Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases considered to be clinically significant
Clinically significant ECG abnormalities at screening from average of triplicate measurement, abnormal findings at echocardiography, or cardiovascular disease indicating a safety risk for participants at the time of screening
Any major illness within 1 month before screening
Received any multidrug and toxin extrusion (MATE1/2K) substrates within 2 weeks before screening
Hereditary fructose intolerance
Used any of the following medications within 90 days prior to screening: riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, probenecid, acetyl cholinesterase inhibitors, agents that could potentially increase or decrease muscle strength, and agents with known or presumed histone deacetylase (HDAC) inhibitory effect
Clinically significant abnormalities in laboratory test results at the time of screening
Ascertained or presumptive hypersensitivity to RO7204239 or risdiplam, or to the constituents of its formulations
Clinically relevant history of anaphylactic reaction requiring inotropic support
Any abnormal skin conditions, pigmentation or lesions in the area intended for SC injection (abdomen) and that would prevent visualization of potential injection site reactions to RO7204239
Immobilization, surgical procedures, fracture, or trauma to the upper or lower limbs within 90 days prior to screening
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