Trial · NCT02913482
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
NCT02913482, Phase 2, Completed, Hoffmann-La Roche; results first posted 2021-01-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy. Trials, record NCT02913482. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02913482
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
- NCT number
- NCT02913482
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 62 participants, actual
- Lead sponsor
- Hoffmann-La Roche
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Risdiplam
- First posted
- 2016-09-23
- Last updated
- 2024-08-07
- Other registries' IDs
- eudract:2016-000778-40
- Results first posted
- 2021-01-08
Source ClinicalTrials.gov
Registered outcomes33
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1: Selected Part 2 Dose of Risdiplam | Minimum of 2 weeks at steady state exposure | All safety, tolerability, PK and PD data available up to the clinical cut-off date of 5 January 2018, plus data that became available prior to the database snapshot on 6 February 2018 were included in the Internal Monitoring Committee (IMC) review. The IMC was responsible for selecting the dose for Part 2 of the study (pivotal dose). An external Independent Data Monitoring Committee (iDMC) reviewed data from Part 1 and confirmed the dose-selection decision of the IMC. The dose for Part 2 selected by the IMC was a dose that: 1.Was judged to be safe and well-tolerated, based on all available safety data from Part 1 and as confirmed by the iDMC; 2. Resulted in an exposure at steady-state below the exposure cap (mean value) of AUC0-24h,ss 2000 ng\*h/mL (adjusted for free-fraction, if required); 3. Resulted in an SMN protein increase that was expected to be clinically relevant. |
| Primary | Part 2: Percentage of Infants Who Are Sitting Without Support for at Least 5 Seconds as Assessed by Item 22 of the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) at Month 12 | Month 12 | The BSID-III is a standardized assessment commonly used to evaluate development across five domains in infants and young children aged between 1 and 42 months. The gross motor subscale of the BSID-III is evaluated based on the linear and hierarchical obtainment of motor skills, as seen in typically developing children. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22 is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. The assessment was video recorded at study sites and reviewed/scored by two independent raters. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. |
| Secondary | Part 2: Percentage of Infants Who Achieve a Score of 40 or Higher in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) at Month 12 | Month 12 | The CHOP-INTEND instrument was developed to evaluate motor function in infants with SMA from the ages of 1.4 to 37.9 months. It consists of 16 items, where each item assesses a specific motor task graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. The total score ranges from 0 to 64, with higher scores consistent with better motor function. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. |
| Secondary | Part 2: Percentage of Infants Achieving an Increase of >/= 4 Points in Their CHOP-INTEND Score From Baseline at Months 8 and 12 | Month 8, Month 12 | The CHOP-INTEND instrument was developed to evaluate motor function in infants with SMA from the ages of 1.4 to 37.9 months. It consists of 16 items, where each item assesses a specific motor task graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. The total score ranges from 0 to 64, with higher scores consistent with better motor function. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. |
| Secondary | Part 2: Percentage of Infants Achieving Head Control (Defined as a Score of >/= 3 for CHOP-INTEND Item 12) at Months 8, 12 and 24 | Month 8, Month 12, Month 24 | The CHOP-INTEND instrument was developed to evaluate motor function in infants with SMA from the ages of 1.4 to 37.9 months. It consists of 16 items, where each item assesses a specific motor task graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. The total score ranges from 0 to 64, with higher scores consistent with better motor function. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. |
| Secondary | Part 2: Change From Baseline in the Total Raw Score of the BSID-III Gross Motor Scale at Months 12 and 24 | Baseline, Month 12, Month 24 | The BSID-III is a standardized assessment commonly used to evaluate development across five domains in infants and young children aged between 1 and 42 months. The gross motor subscale of the BSID-III is evaluated based on the linear and hierarchical obtainment of motor skills, as seen in typically developing children. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). In this study the gross motor scale was assessed in a modified way compared with the standard administration. A total raw score was calculated by summing the item scores to give a maximum possible score of 72. |
| Secondary | Part 2: Percentage of Infants Who Achieve the Attainment Levels of a Subset of Motor Milestones Assessed in the Hammersmith Infant Neurological Examination Module 2 (HINE-2) at Month 8 | Month 8 | The HINE was designed to evaluate infants between 2 months and 24 months of age. It is a simple and standardized instrument that includes 26 items assessing different aspects of neurological examinations, such as cranial nerves, posture, movements, tone, and reflexes. In this study, Module 2 of the HINE (HINE-2) was assessed. The HINE-2 evaluates 8 developmental milestones (head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking) scored on a 3, 4, or 5-point scale, with 0 indicating inability to perform a task and a score of 2, 3, or 4 (depending on the task) indicating full milestone development. The total score is calculated by summing the item scores to give a maximum possible score of 26. This measure represents subset numbers at Month 8 for head control, ability to kick and rolling milestones only. |
| Secondary | Part 2: Percentage of Infants Who Achieve the Attainment Levels of the Motor Milestones Assessed in the Hammersmith Infant Neurological Examination Module 2 (HINE-2) at Month 12 | Month 12 | The HINE was designed to evaluate infants between 2 months and 24 months of age. It is a simple and standardized instrument that includes 26 items assessing different aspects of neurological examinations, such as cranial nerves, posture, movements, tone, and reflexes. In this study, Module 2 of the HINE (HINE-2) was assessed. The HINE-2 evaluates 8 developmental milestones (head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking) scored on a 3, 4, or 5-point scale, with 0 indicating inability to perform a task and a score of 2, 3, or 4 (depending on the task) indicating full milestone development. The total score is calculated by summing the item scores to give a maximum possible score of 26. |
| Secondary | Part 2: Percentage of Infants Who Achieve the Attainment Levels of the Motor Milestones Assessed in the Hammersmith Infant Neurological Examination Module 2 (HINE-2) at Month 24 | Month 24 | The HINE was designed to evaluate infants between 2 months and 24 months of age. It is a simple and standardized instrument that includes 26 items assessing different aspects of neurological examinations, such as cranial nerves, posture, movements, tone, and reflexes. In this study, Module 2 of the HINE (HINE-2) was assessed. The HINE-2 evaluates 8 developmental milestones (head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking) scored on a 3, 4, or 5-point scale, with 0 indicating inability to perform a task and a score of 2, 3, or 4 (depending on the task) indicating full milestone development. The total score is calculated by summing the item scores to give a maximum possible score of 26. |
| Secondary | Part 2: Percentage of Motor Milestone Responders as Assessed by HINE-2 at Months 12 and 24 | Month 12, Month 24 | The HINE-2 evaluates 8 developmental milestones scored on a 3, 4, or 5-point scale, with 0 indicating inability to perform a task and a score of 2, 3, or 4 (depending on the task) indicating full milestone development. The total score is calculated by summing the item scores to give a maximum possible score of 26. For the motor milestone responder definition, an improvement in a motor milestone is defined as at least a 2-point increase in ability to kick (or maximal score) or a 1-point increase in head control, rolling, sitting, crawling, standing, or walking. Worsening is similarly defined as a 2-point decrease in ability to kick (or lowest score) or a 1-point decrease in head control, rolling, sitting, crawling, standing, or walking. Voluntary grasp is excluded from the definition. An infant is classified as a responder if more motor milestones show improvement than show worsening. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 registered outcomesOutcomes33
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part 1: Selected Part 2 Dose of Risdiplam | Minimum of 2 weeks at steady state exposure | All participants in Part 1 who received at least one dose of risdiplam and had available data at the time of the data snapshot for selecting the Part 2 dose. | mg/kg | NUMBER |
| PRIMARY | Part 2: Percentage of Infants Who Are Sitting Without Support for at Least 5 Seconds as Assessed by Item 22 of the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) at Month 12 | Month 12 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Percentage of Infants Who Achieve a Score of 40 or Higher in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) at Month 12 | Month 12 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Percentage of Infants Achieving an Increase of >/= 4 Points in Their CHOP-INTEND Score From Baseline at Months 8 and 12 | Month 8, Month 12 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Percentage of Infants Achieving Head Control (Defined as a Score of >/= 3 for CHOP-INTEND Item 12) at Months 8, 12 and 24 | Month 8, Month 12, Month 24 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Change From Baseline in the Total Raw Score of the BSID-III Gross Motor Scale at Months 12 and 24 | Baseline, Month 12, Month 24 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. Only participants for whom data were collected are included in the analysis. | Scores on a Scale | MEAN |
| SECONDARY | Part 2: Percentage of Infants Who Achieve the Attainment Levels of a Subset of Motor Milestones Assessed in the Hammersmith Infant Neurological Examination Module 2 (HINE-2) at Month 8 | Month 8 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Percentage of Infants Who Achieve the Attainment Levels of the Motor Milestones Assessed in the Hammersmith Infant Neurological Examination Module 2 (HINE-2) at Month 12 | Month 12 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Percentage of Infants Who Achieve the Attainment Levels of the Motor Milestones Assessed in the Hammersmith Infant Neurological Examination Module 2 (HINE-2) at Month 24 | Month 24 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Percentage of Motor Milestone Responders as Assessed by HINE-2 at Months 12 and 24 | Month 12, Month 24 | The intent-to-treat (ITT) population is defined as all infants enrolled in Part 2 of the study, regardless of whether they received treatment or not. | Percentage of Participants | NUMBER |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 outcomesResults by group165
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part 1: Selected Part 2 Dose of Risdiplam | Exploratory Part 1 - Risdiplam | 0.2 | mg/kg | ||
Part 2: Percentage of Infants Who Are Sitting Without Support for at Least 5 Seconds as Assessed by Item 22 of the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) at Month 12 | Confirmatory Part 2 - Risdiplam | 29.3 | 90% Confidence Interval 17.84 to 43.07 | Percentage of Participants | |
| Part 2: Percentage of Infants Who Achieve a Score of 40 or Higher in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) at Month 12 | Confirmatory Part 2 - Risdiplam | 56.1 | 90% Confidence Interval 42.13 to 69.38 | Percentage of Participants | |
| Part 2: Percentage of Infants Achieving an Increase of >/= 4 Points in Their CHOP-INTEND Score From Baseline at Months 8 and 12 | Confirmatory Part 2 - Risdiplam | Month 8 | 87.8 | 90% Confidence Interval 76.05 to 95.07 | Percentage of Participants |
| Part 2: Percentage of Infants Achieving an Increase of >/= 4 Points in Their CHOP-INTEND Score From Baseline at Months 8 and 12 | Confirmatory Part 2 - Risdiplam | Month 12 | 90.2 | 90% Confidence Interval 79.05 to 96.60 | Percentage of Participants |
| Part 2: Percentage of Infants Achieving Head Control (Defined as a Score of >/= 3 for CHOP-INTEND Item 12) at Months 8, 12 and 24 | Confirmatory Part 2 - Risdiplam | Month 8 | 46.3 | 90% Confidence Interval 32.87 to 60.23 | Percentage of Participants |
| Part 2: Percentage of Infants Achieving Head Control (Defined as a Score of >/= 3 for CHOP-INTEND Item 12) at Months 8, 12 and 24 | Confirmatory Part 2 - Risdiplam | Month 12 | 53.7 | 90% Confidence Interval 39.77 to 67.13 | Percentage of Participants |
| Part 2: Percentage of Infants Achieving Head Control (Defined as a Score of >/= 3 for CHOP-INTEND Item 12) at Months 8, 12 and 24 | Confirmatory Part 2 - Risdiplam | Month 24 | 70.7 | 90% Confidence Interval 56.93 to 82.16 | Percentage of Participants |
| Part 2: Change From Baseline in the Total Raw Score of the BSID-III Gross Motor Scale at Months 12 and 24 | Confirmatory Part 2 - Risdiplam | Baseline | 1.85 | Standard Deviation 1.61 | Scores on a Scale |
| Part 2: Change From Baseline in the Total Raw Score of the BSID-III Gross Motor Scale at Months 12 and 24 | Confirmatory Part 2 - Risdiplam | Change from Baseline at Month 12 | 7.21 | Standard Deviation 5.71 | Scores on a Scale |
1 to 10 of 165Source ClinicalTrials.gov
See all 165 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Exploratory Part 1 - Cohort 1 | 4 | |
| Overall Study | STARTED | Exploratory Part 1 - Cohort 2 | 17 | |
| Overall Study | STARTED | Confirmatory Part 2 - Risdiplam | 41 | |
| Overall Study | COMPLETED | Exploratory Part 1 - Cohort 1 | 3 | |
| Overall Study | COMPLETED | Exploratory Part 1 - Cohort 2 | 13 | |
| Overall Study | COMPLETED | Confirmatory Part 2 - Risdiplam | 36 | |
| Overall Study | NOT COMPLETED | Exploratory Part 1 - Cohort 1 | 1 | |
| Overall Study | NOT COMPLETED | Exploratory Part 1 - Cohort 2 | 4 | |
| Overall Study | NOT COMPLETED | Confirmatory Part 2 - Risdiplam | 5 |
Source ClinicalTrials.gov
Adverse events522
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Exploratory Part 1 - Cohort 1 | 1 | 4 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Exploratory Part 1 - Cohort 2 | 0 | 17 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Confirmatory Part 2 - Risdiplam | 1 | 41 | 2 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Exploratory Part 1 - Cohort 1 | 1 | 4 | 1 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Exploratory Part 1 - Cohort 2 | 0 | 17 | 0 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Confirmatory Part 2 - Risdiplam | 1 | 41 | 1 |
| other | Leukocytosis | Blood and lymphatic system disorders | Exploratory Part 1 - Cohort 1 | 0 | 4 | 0 |
| other | Leukocytosis | Blood and lymphatic system disorders | Exploratory Part 1 - Cohort 2 | 1 | 17 | 1 |
| other | Leukocytosis | Blood and lymphatic system disorders | Confirmatory Part 2 - Risdiplam | 0 | 41 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Exploratory Part 1 - Cohort 1 | 0 | 4 | 0 |
1 to 10 of 522Source ClinicalTrials.gov
See all 522 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Exploratory Part 1 - Cohort 1 | 1 | 4 |
| Total, all-cause mortality | Exploratory Part 1 - Cohort 2 | 3 | 17 |
| Total, all-cause mortality | Confirmatory Part 2 - Risdiplam | 3 | 41 |
| Total, other adverse events | Exploratory Part 1 - Cohort 1 | 3 | 4 |
| Total, other adverse events | Exploratory Part 1 - Cohort 2 | 17 | 17 |
| Total, other adverse events | Confirmatory Part 2 - Risdiplam | 40 | 41 |
| Total, serious adverse events | Exploratory Part 1 - Cohort 1 | 4 | 4 |
| Total, serious adverse events | Exploratory Part 1 - Cohort 2 | 13 | 17 |
| Total, serious adverse events | Confirmatory Part 2 - Risdiplam | 34 | 41 |
Source ClinicalTrials.gov
Papers7
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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