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A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy (MANATEE)
EU CTIS ID: 2023-506761-65-00
What this study is testing
Part 1 Ambulant Cohorts A-C: - Evaluation of the safety of RO7204239 in combination with risdiplam compared with placebo in combination with risdiplam - Evaluation of pharmacokinetic (PK) parameters for RO7204239 when administered in combination with risdiplam - Evaluation of PK parameters for risdiplam when administered in combination with RO7204239 - Evaluation of the immune response to RO7204239 in combination with risdiplam - Evaluation of the pharmacodynamic (PD) effects of RO7204239 in combination with risdiplam compared with placebo in combination with risdiplam - Evaluation of RO7204239 PK/PD effects Part 2: - Evaluation of the efficacy of RO7204239 in combination with risdiplam compared with placebo in combination with risdiplam
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Age at screening: - Part 1 Cohorts A (ambulant participants), B (ambulant participants), and D (non-ambulant participants): 5-10 years, inclusive; - Part 1 Cohort C (ambulant participants): 2-4 years, inclusive; - Part 2 (ambulant participants): 2-25 years, inclusive
- Participants who have a confirmed genetic diagnosis of 5q-autosomal recessive SMA
- 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
- Inclusion Criteria for Part 1 Cohorts A, B, and C and Part 2 only: 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 seconds as measures by the Timed 10-Meter Walk/Run Test [10MWRT] at screening
- Inclusion Criteria for Part 1 Cohort D only: Participants who are able to sit, defined by: - A score of 3 on Item 9 of the MFM32 (sitting without upper limb support while maintaining contact between the two hands for 5 seconds); - A score of at least 2 on Item 10 of the MFM32 (while seated, leaning forward to touch a tennis ball and sitting back again, either with or without upper limb support)
- Inclusion Criteria for Part 1 Cohort D only: Participants who are able to raise a standardized plastic cup with a 200g weight in it to the mouth, using both hands if necessary, defined by a score of 3 on the entry item of the Revised Upper Limb Module (RULM)
You likely can't join if
- 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
- Exclusion Criteria for Part 1 Cohort D only: Participants who have severe scoliosis (curvature > 40°) at screening based on the participant's most recent X-ray as performed per standard of care or scoliosis that would interfere with functional assessments, as confirmed by the clinical evaluator. An X-ray is not required if it is not clinically indicated (e.g., in participants with mild scoliosis)
- Exclusion Criteria for Part 1 Cohorts A and B only: Participants with contraindications for MRI scan (including, but not restricted to, claustrophobia, pacemaker, artificial heart valves, cochlear implants, presence of foreign metal objects in heart or body, including spinal rods, intracranial vascular clips, insulin pumps, etc.), difficulties maintaining a prolonged supine position, or any other clinical history or examination finding that would pose a potential hazard in combination with MRI
- Exclusion Criteria for Part 1 Cohort D only: Participants who are unable to adopt the correct position to ensure adequate quality of DXA scan acquisition, as determined by the DXA scan technologist
- Exclusion Criteria for Part 1 Cohort D only: For participants able to take steps only: Able to walk 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 seconds as measured by the timed 10MWRT at screening
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Australia
- Canada
- United Kingdom
- Japan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Australia; Canada; United Kingdom; Japan. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.