A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants with Spinal Muscular Atrophy

Conditions

Spinal Muscular Atrophy (SMA)

Brief summary

Plasma concentration of risdiplam and metabolite(s) as applicable at specified timepoints, AUC, Concentration at the end of a dosing interval to assess steady-state, Other PK parameters as appropriate, Risdiplam free fraction, Incidence and severity of adverse events, with severity determined according to the NCI CTCAE v5.0, Incidence and severity of serious adverse events, Incidence of treatment discontinuation due to adverse events, Incidence of abnormal laboratory values, Incidence of abnormal ECG values, Vital signs abnormalities, including body temperature, systolic and diastolic blood pressure, heart rate, respiratory rate, Physical examination, including detailed examination of the skin and mouth

Related papers

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Interventions

DRUGEvrysdi 0.75 mg/mL powder for oral solution

DRUGRO7034067

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Plasma concentration of risdiplam and metabolite(s) as applicable at specified timepoints, AUC, Concentration at the end of a dosing interval to assess steady-state, Other PK parameters as appropriate, Risdiplam free fraction, Incidence and severity of adverse events, with severity determined according to the NCI CTCAE v5.0, Incidence and severity of serious adverse events, Incidence of treatment discontinuation due to adverse events, Incidence of abnormal laboratory values, Incidence of abnormal ECG values, Vital signs abnormalities, including body temperature, systolic and diastolic blood pressure, heart rate, respiratory rate, Physical examination, including detailed examination of the skin and mouth	—

Measure

Time frame

Plasma concentration of risdiplam and metabolite(s) as applicable at specified timepoints, AUC, Concentration at the end of a dosing interval to assess steady-state, Other PK parameters as appropriate, Risdiplam free fraction, Incidence and severity of adverse events, with severity determined according to the NCI CTCAE v5.0, Incidence and severity of serious adverse events, Incidence of treatment discontinuation due to adverse events, Incidence of abnormal laboratory values, Incidence of abnormal ECG values, Vital signs abnormalities, including body temperature, systolic and diastolic blood pressure, heart rate, respiratory rate, Physical examination, including detailed examination of the skin and mouth

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Countries

Belgium, Germany, Italy, Netherlands, Norway, Poland

Outcome results

None listed