| Primary | Part 1: Area Under the Plasma Concentration-Time Curve From Time Zero to Infinity (AUCinf) of Risdiplam and Its Metabolite (M1) | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | h*ng/mL | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG003 | Risdiplam M1 Metabolite - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| | | Title | Denominators | Categories |
|---|
| | | Title | Measurements |
|---|
| - OG000792± 20.0
- OG001987± 35.2
- OG002222± 36.6
- OG003
|
|
| | Group IDs | Group Description | Statistical Method | Statistical Comment | P-Value | P-Value Comment | Parameter Type | Parameter Value | Dispersion Type | Dispersion Value | Confidence Interval Sides | Confidence Interval % | CI Lower Limit | CI Upper Limit | CI Lower Limit Comment | CI Upper Limit Comment | Estimate Comment | Tested Non-Inferiority | Non-Inferiority Type | Non-Inferiority Comment | Other Analysis Description |
|---|
| | | | | | Ratio of Geometric Least Squares Means | 0.802 | | | 2-Sided | 90 | 0.627 | 1.03 | | | | | Other | | | | | | | |
|
| Primary | Area Under the Plasma Concentration-Time Curve From Time Zero to the Last Measurable Concentration (AUClast) of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | h*ng/mL | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Primary | Part 1: Maximum Observed Plasma Concentration (Cmax) of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | ng/mL | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Primary | Part 2: AUCinf of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | h*ng/mL | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Primary | Part 2: AUClast of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | h*ng/mL | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Primary | Part 2: Cmax of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | ng/mL | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 1: Time of the Maximum Observed Plasma Concentration (Tmax) of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Median | Full Range | hours (h) | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 1: Apparent Plasma Terminal Elimination Half-Life (t1/2) of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | h | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 1: Percentage of Area Under the Plasma Concentration-Time Curve Due to Extrapolation (%AUCextrap) of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Percentage (%) of AUCextrap | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 1: Terminal Elimination Rate Constant (λz=Lambda-Z) of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | h-1 | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 1: Apparent Total Clearance (CL/F) of Risdiplam | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | L/h | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| |
| Secondary | Part 1: Molecular Weight-Adjusted Metabolite-to-Parent Ratio for AUCinf of Risdiplam M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Ratio | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam M1 Metabolite - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| |
| Secondary | Part 1: Molecular Weight-Adjusted Metabolite-to-Parent Ratio for Cmax of Risdiplam M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Ratio | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam M1 Metabolite - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| |
| Secondary | Part 1: Molecular Weight-Adjusted Metabolite-to-Parent Ratio for AUClast of Risdiplam M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Ratio | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam M1 Metabolite - Mild Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam M1 Metabolite - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| |
| Secondary | Part 2: Tmax of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Median | Full Range | hours (h) | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 2: t1/2 of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | h | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 2: %AUCextrap of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Percentage (%) of AUCextrap | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 2: λz of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | h-1 | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Moderate Hepatic Impairment | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
|
| Secondary | Part 2: CL/F of Risdiplam and M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | L/h | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| |
| Secondary | Part 2: Molecular Weight-Adjusted Metabolite-to-Parent Ratio for AUCinf of Risdiplam M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Ratio | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam M1 Metabolite - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| |
| Secondary | Part 2: Molecular Weight-Adjusted Metabolite-to-Parent Ratio for Cmax of Risdiplam M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Ratio | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam M1 Metabolite - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| |
| Secondary | Part 2: Molecular Weight-Adjusted Metabolite-to-Parent Ratio for AUClast of Risdiplam M1 | | The PK population included all participants who received a dose of risdiplam and had evaluable PK data. A participant was excluded from the PK descriptive statistics and statistical analysis if the participant had an AE of vomiting that occurred at or before 2 times median time to maximum concentration or if they had any major protocol deviation(s) thought to impact PK analysis. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Ratio | | Predose, 1, 2, 4, 6, 8, 10, 12, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 312, 336, 360, 408, 456, 504, and 552 hours Postdose | | | | ID | Title | Description |
|---|
| OG000 | Risdiplam M1 Metabolite - Moderate Hepatic Impairment | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Risdiplam M1 Metabolite - Normal Hepatic Function | Participants with normal hepatic function received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. |
| |
| Secondary | Part 1 and Part 2: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. | The safety population included all participants who received a dose of risdiplam. | Posted | | Number | | Percentage of Participants | | Up to 31 Days | | | | ID | Title | Description |
|---|
| OG000 | Part 1 | Participants with mild hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG001 | Part 2 | Participants with moderate hepatic impairment received a single dose of 5 mg risdiplam orally as a drinking solution on Day 1 after an overnight fast of at least 8 hours. | | OG002 | Normal Hepatic Function Participants |
|