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A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants (SUNFISH)
Muscle And Peripheral Nerve Disease Spinal Muscular Atrophy (SMA)
Basic Details
Study Summary
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of Risdiplam for 12 weeks and a confirmatory part (Part 2) of Risdiplam for 24 months.
Eligibility Criteria
- Confirmed diagnosis of 5q-autosomal recessive SMA
- Negative blood pregnancy test at screening and agreement to comply with measures to prevent pregnancy and restrictions on sperm donation
- For Part 1: Type 2 or 3 SMA ambulant or non-ambulant
- For Part 2: 1) Type 2 or 3 SMA non-ambulant; 2) RULM entry item A greater than or equal to 2; 3) ability to sit independently as assessed by item 9 of the MFM
- 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 administration of a SMN2-targeting antisense oligonucleotide, SMN2 splicing modifier or gene therapy either in a clinical study or as part of medical care
- 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 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
- 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 Risdiplam or to the constituents of its formulation
- Recent history (less than one year) of ophthalmological diseases
- Participants requiring invasive ventilation or tracheostomy
The source of the below information is public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc.. It has been summarised and edited into simpler language. For more information about this clinical study see the For Expert tab on the specific ForPatients page or follow these links to https://clinicaltrials.gov and/or https://euclinicaltrials.eu and/or https://www.isrctn.com.
The information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc., and has not been edited.
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Study results
LPS_BP39055_SUNFISH_interim results_January2022_English
(PDF, 1.2 MB)
LPS_BP39055_SUNFISH_interim results_January2021_English
(PDF, 0.6 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Brazil Portuguese
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Croatia Croatian
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 English
(PDF, 2.1 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 France French
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Germany German
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Italy Italian
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Netherlands Dutch
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Poland Polish
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Russia Russian
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Serbia Serbian
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Spain Spanish
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results Children September 2024 Turkey Turkish
(PDF, 5.4 MB)
LPS BP39055 SUNFISH Final Results September 2024 Brazil Portuguese
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 Croatia Croatian
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 English
(PDF, 3.2 MB)
LPS BP39055 SUNFISH Final Results September 2024 France French
(PDF, 1.0 MB)
LPS BP39055 SUNFISH Final Results September 2024 Germany German
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 Italy Italian
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 Netherlands Dutch
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 Poland Polish
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 Russia Russian
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 Serbia Serbian
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 Spain Spanish
(PDF, 0.9 MB)
LPS BP39055 SUNFISH Final Results September 2024 Turkey Turkish
(PDF, 0.9 MB)
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For the latest version of this information please go to www.forpatients.roche.com