An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric patients with Spinal Muscular Atrophy

Conditions

Spinal Muscular Atrophy (SMA)

Brief summary

1. Incidence and severity of adverse events and serious adverse events with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events scale, Version 4.0, 2. Incidence of treatment discontinuations due to adverse events, 3. Incidence of abnormal vital signs, laboratory, and ECG abnormalities, 4. Incidence of clinically significant findings on ophthalmological examination, 5. Incidence of clinically significant findings on neurological examination, 6. Incidence of clinically significant findings in Physical examination, 7. Incidence of emergence or worsening of symptoms as measured by the Columbia-Suicide Severity Rating Scale (C-SSRS) (adult version for adults and adolescents, pediatric version for patients aged 6-11 years), 8. Mean plasma concentrations of Risdiplam and metabolites, 9. Peak plasma concentration (Cmax), 10. Area under the curve (AUC), 11. Concentration at the end of a dosing interval (Ctrough)

Detailed description

1. SMN mRNA levels in blood, 2. SMN protein levels in blood

Related papers

No related papers found yet.

Interventions

DRUGRO7034067

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
1. Incidence and severity of adverse events and serious adverse events with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events scale, Version 4.0, 2. Incidence of treatment discontinuations due to adverse events, 3. Incidence of abnormal vital signs, laboratory, and ECG abnormalities, 4. Incidence of clinically significant findings on ophthalmological examination, 5. Incidence of clinically significant findings on neurological examination, 6. Incidence of clinically significant findings in Physical examination, 7. Incidence of emergence or worsening of symptoms as measured by the Columbia-Suicide Severity Rating Scale (C-SSRS) (adult version for adults and adolescents, pediatric version for patients aged 6-11 years), 8. Mean plasma concentrations of Risdiplam and metabolites, 9. Peak plasma concentration (Cmax), 10. Area under the curve (AUC), 11. Concentration at the end of a dosing interval (Ctrough)	—

Measure

Time frame

1. Incidence and severity of adverse events and serious adverse events with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events scale, Version 4.0, 2. Incidence of treatment discontinuations due to adverse events, 3. Incidence of abnormal vital signs, laboratory, and ECG abnormalities, 4. Incidence of clinically significant findings on ophthalmological examination, 5. Incidence of clinically significant findings on neurological examination, 6. Incidence of clinically significant findings in Physical examination, 7. Incidence of emergence or worsening of symptoms as measured by the Columbia-Suicide Severity Rating Scale (C-SSRS) (adult version for adults and adolescents, pediatric version for patients aged 6-11 years), 8. Mean plasma concentrations of Risdiplam and metabolites, 9. Peak plasma concentration (Cmax), 10. Area under the curve (AUC), 11. Concentration at the end of a dosing interval (Ctrough)

—

Secondary

Measure	Time frame
1. SMN mRNA levels in blood, 2. SMN protein levels in blood	—

Countries

Belgium, France, Italy, Netherlands, Poland

Outcome results

None listed