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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-002087-12 | EudraCT Number | ||
| 2023-506009-20-00 | EU Trial (CTIS) Number |
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A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
The study is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants aged from birth to 6 weeks who have been genetically diagnosed with SMA but are not yet presenting with symptoms. There will be a screening, treatment, open-label extension (OLE) and a follow-up. All participants will receive risdiplam orally once daily for 2 years followed by an OLE phase of at least 3 years and a follow-up (if applicable), for a total treatment duration of at least 5 years for each participant enrolled.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open-label Risdiplam | Experimental | Participants will be enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Risdiplam | Drug | Risdiplam will be administered orally. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support | The Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", 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. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. An exact binomial test was performed. If the lower limit of the two-sided 90% CI was above the 5% threshold, the primary objective of the study was considered achieved. | At Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Developing Clinically Manifested SMA | At Month 12 and 24 | |
| Time to Permanent Ventilation and/or Death | Up to 7 years | |
| Percentage of Participants Who Are Alive Without Permanent Ventilation |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nemours Children's Hospital | Orlando | Florida | 32837 | United States | ||
| Sydney Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40802943 | Derived | Finkel RS, Servais L, Vlodavets D, Zanoteli E, Mazurkiewicz-Beldzinska M, Jong YJ, Navas-Nazario A, Al-Muhaizea M, Araujo APQC, Nelson L, Wang Y, Jaber B, Gorni K, Kletzl H, Palfreeman L, Rabbia M, Summers D, Gaki E, Wagner KR, Fontoura P, Farrar MA, Bertini E; RAINBOWFISH Study Group. Risdiplam in Presymptomatic Spinal Muscular Atrophy. N Engl J Med. 2025 Aug 14;393(7):671-682. doi: 10.1056/NEJMoa2410120. |
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For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
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The study enrolled infants aged from birth to 6 weeks who were genetically diagnosed with SMA but were not yet presenting with symptoms. Study arms were based on the number of copies of the SMN2 gene.
Overall, 26 infants with spinal muscular atrophy (SMA) were enrolled in the study across 7 different sites in 7 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | 2 SMN2 Copies, Risdiplam | Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL. |
| FG001 | 3 SMN2 Copies, Risdiplam | Infants with 3 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL. |
| FG002 | >/=4 SMN2 Copies, Risdiplam | Infants with 4 or more copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 2 SMN2 Copies, Risdiplam | Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL. |
| BG001 | 3 SMN2 Copies, Risdiplam |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support | The Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", 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. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. An exact binomial test was performed. If the lower limit of the two-sided 90% CI was above the 5% threshold, the primary objective of the study was considered achieved. | Primary efficacy population included all infants in the intent-to-treat (ITT) population with two SMN2 copies (excluding the known SMN2 gene modifier mutation c.859G>C) and a baseline compound muscle action potential (CMAP) amplitude >/= 1.5 mV. | Posted | Number | 90% Confidence Interval | percentage of participants |
From baseline up to the clinical cut off date (a minimum of 12 months and a maximum of 3.5 years)
The safety population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 2 SMN2 Copies, Risdiplam | Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Communications | Hoffmann-La Roche | 800 821-8590 | genentech@druginfo.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 30, 2021 | Feb 9, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 12, 2021 | Feb 9, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| ID | Term |
|---|---|
| C000629884 | Risdiplam |
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Open-label, single arm
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| At Month 12 and 24 |
| Percentage of Participants Alive | At Month 12 and 24 |
| Percentage of Participants Who Achieve the Attainment Level of the Motor Milestones as Assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2) | HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking | At Month 12 and 24 |
| Percentage of Participants With Two Copies of the SMN2 Gene Sitting Without Support for 5 Seconds (Independent of the CMAP Value at Baseline). | Assessed in Item 22 of the BSID-III Gross Motor Scale. The BSID-III is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", 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. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. | At Month 12 |
| Percentage of Participants Sitting Without Support for 5 Seconds | Assessed with BSID-III Gross Motor Scale | At Month 24 |
| Percentage of Participants Sitting Without Support for 30 Seconds | Assessed with BSID-III Gross Motor Scale | At Month 12 and 24 |
| Percentage of Participants Standing for at Least 3 Seconds | Assessed with BSID-III Gross Motor Scale | At Month 24 |
| Percentage of Participants Walking (Takes at Least 3 Steps) | Assessed with BSID-III Gross Motor Scale | At Month 24 |
| Percentage of Participants Demonstrating the Ability to Achieve a Scaled Score on BSID-III Gross Motor Subtests Within 1.5 Standard Deviations of Chronological Reference Standard | Assessed through BSID-III Gross Motor Scale | At Month 24 and 42 |
| Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12 | The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. A positive change from baseline indicates an improvement. | Baseline, Month 12 |
| Percentage of Participants Who Achieve a Score of 40 or Higher, 50 or Higher, and 60 or Higher in the CHOP INTEND Motor Function Scale at Month 12 | The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. Data are presented with a two-sided 90% Clopper-Pearson (exact) CI for the proportion. | At Month 12 |
| Percentage of Participants Who Meet CHOP INTEND Stopping Criteria at Any Point | Up to Month 24 |
| Change From Baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) Score | At Month 60 |
| Number and Percentage of Participants Within 3rd Percentile of Normal Range for Weight-for-Age, Length/Height-for-Age and Weight-for-Length/Height | Based on the WHO Child Growth Standards (WHO 2019) | At Month 12, 24, 36, 48 and 60 |
| Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age | Based on the WHO Child Growth Standards (WHO 2019) | At Month 12 and 24 |
| Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height | At Month 12, 24, 36, 48 and 60 |
| Change From Baseline Percentiles for Head Circumference- For-age | At Month 12 and 24 |
| Change From Baseline in Chest Circumference | At Month 12 and 24 |
| Ratio Between Chest and Head Circumferences | At Month 12 and 24 |
| Percentage of Participants With the Ability to Swallow and to Feed Orally | At Month 12, 24, 36, 48 and 60 |
| Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitude | Measured by CMAP | At Month 12 and 24 |
| Measurement of Pharmacodynamic Marker Levels in Blood | Day 1, 56, 196, 364, 728 and at early withdrawal |
| Percentage of Participants With Adverse Events | Adverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5 | Up to 7 years |
| Ophthalmological Examination as Appropriate for Age | Up to 7 years |
| Plasma Concentration of Risdiplam and Its Metabolites to Characterize the PK Profile | Up to 7 years |
| Randwick |
| New South Wales |
| 2031 |
| Australia |
| Chr de La Citadelle | Liège | 4000 | Belgium |
| Hospital das Clinicas - FMUSP_X | São Paulo | São Paulo | 05403-000 | Brazil |
| Szpital Gdanskiego Uniwersytetu Medycznego | Gda?sk | 80-952 | Poland |
| Russian Children Neuromuscular Center of Veltischev | Moscow | Moscow Oblast | 125412 | Russia |
| Kaohsiung Medical University Chung-Ho Hospital | Kaohsiung City | 807 | Taiwan |
Infants with 3 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL.
| BG002 | >/=4 SMN2 Copies, Risdiplam | Infants with 4 or more copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL. |
| BG003 | Total | Total of all reporting groups |
| days |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| At Month 12 |
|
|
|
|
| Secondary | Percentage of Participants Developing Clinically Manifested SMA | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Time to Permanent Ventilation and/or Death | Not Posted | Up to 7 years | Participants |
| Secondary | Percentage of Participants Who Are Alive Without Permanent Ventilation | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Percentage of Participants Alive | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Percentage of Participants Who Achieve the Attainment Level of the Motor Milestones as Assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2) | HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Percentage of Participants With Two Copies of the SMN2 Gene Sitting Without Support for 5 Seconds (Independent of the CMAP Value at Baseline). | Assessed in Item 22 of the BSID-III Gross Motor Scale. The BSID-III is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", 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. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. | Intent-to-treat (ITT) population included all enrolled participants, regardless of whether they received risdiplam or not. Only participants with two copies of the SMN2 gene were included in this outcome measure. | Posted | Number | 90% Confidence Interval | percentage of participants | At Month 12 |
|
|
|
| Secondary | Percentage of Participants Sitting Without Support for 5 Seconds | Assessed with BSID-III Gross Motor Scale | Not Posted | At Month 24 | Participants |
| Secondary | Percentage of Participants Sitting Without Support for 30 Seconds | Assessed with BSID-III Gross Motor Scale | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Percentage of Participants Standing for at Least 3 Seconds | Assessed with BSID-III Gross Motor Scale | Not Posted | At Month 24 | Participants |
| Secondary | Percentage of Participants Walking (Takes at Least 3 Steps) | Assessed with BSID-III Gross Motor Scale | Not Posted | At Month 24 | Participants |
| Secondary | Percentage of Participants Demonstrating the Ability to Achieve a Scaled Score on BSID-III Gross Motor Subtests Within 1.5 Standard Deviations of Chronological Reference Standard | Assessed through BSID-III Gross Motor Scale | Not Posted | At Month 24 and 42 | Participants |
| Secondary | Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12 | The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. A positive change from baseline indicates an improvement. | ITT population included all enrolled participants, regardless of whether they received risdiplam or not. | Posted | Median | Full Range | score on a scale | Baseline, Month 12 |
|
|
|
| Secondary | Percentage of Participants Who Achieve a Score of 40 or Higher, 50 or Higher, and 60 or Higher in the CHOP INTEND Motor Function Scale at Month 12 | The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. Data are presented with a two-sided 90% Clopper-Pearson (exact) CI for the proportion. | ITT population included all enrolled participants, regardless of whether they received risdiplam or not. | Posted | Number | 90% Confidence Interval | percentage of participants | At Month 12 |
|
|
|
| Secondary | Percentage of Participants Who Meet CHOP INTEND Stopping Criteria at Any Point | Not Posted | Up to Month 24 | Participants |
| Secondary | Change From Baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) Score | Not Posted | At Month 60 | Participants |
| Secondary | Number and Percentage of Participants Within 3rd Percentile of Normal Range for Weight-for-Age, Length/Height-for-Age and Weight-for-Length/Height | Based on the WHO Child Growth Standards (WHO 2019) | Not Posted | At Month 12, 24, 36, 48 and 60 | Participants |
| Secondary | Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age | Based on the WHO Child Growth Standards (WHO 2019) | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height | Not Posted | At Month 12, 24, 36, 48 and 60 | Participants |
| Secondary | Change From Baseline Percentiles for Head Circumference- For-age | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Change From Baseline in Chest Circumference | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Ratio Between Chest and Head Circumferences | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Percentage of Participants With the Ability to Swallow and to Feed Orally | Not Posted | At Month 12, 24, 36, 48 and 60 | Participants |
| Secondary | Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitude | Measured by CMAP | Not Posted | At Month 12 and 24 | Participants |
| Secondary | Measurement of Pharmacodynamic Marker Levels in Blood | Not Posted | Day 1, 56, 196, 364, 728 and at early withdrawal | Participants |
| Secondary | Percentage of Participants With Adverse Events | Adverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5 | Not Posted | Up to 7 years | Participants |
| Secondary | Ophthalmological Examination as Appropriate for Age | Not Posted | Up to 7 years | Participants |
| Secondary | Plasma Concentration of Risdiplam and Its Metabolites to Characterize the PK Profile | Not Posted | Up to 7 years | Participants |
| 0 |
| 8 |
| 3 |
| 8 |
| 8 |
| 8 |
| EG001 | 3 SMN2 Copies, Risdiplam | Infants with 3 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL. | 0 | 13 | 0 | 13 | 12 | 13 |
| EG002 | >/=4 SMN2 Copies, Risdiplam | Infants with 4 or more copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng*hr/mL. | 0 | 5 | 1 | 5 | 4 | 5 |
| Gastroenteritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Soft tissue injury | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Jaundice neonatal | Pregnancy, puerperium and perinatal conditions | MedDRA 25.1 | Systematic Assessment |
|
| Cryptorchism | Congenital, familial and genetic disorders | MedDRA 25.1 | Systematic Assessment |
|
| Cystoid macular oedema | Eye disorders | MedDRA 25.1 | Systematic Assessment |
|
| Retinal pigmentation | Eye disorders | MedDRA 25.1 | Systematic Assessment |
|
| Retinal vascular disorder | Eye disorders | MedDRA 25.1 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Gastrointestinal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Regurgitation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Teething | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
|
| Malaise | General disorders | MedDRA 25.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
|
| Bronchiolitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Croup infectious | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Cytomegalovirus infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Exanthema subitum | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Gastroenteritis norovirus | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Gastrointestinal viral infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Impetigo | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Oral candidiasis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Otitis media acute | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Respiratory syncytial virus bronchitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Respiratory tract infection bacterial | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Respiratory tract infection viral | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Skin infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Suspected COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Varicella | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Viral rash | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Vulvovaginitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Accidental overdose | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Expired product administered | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Incorrect dose administered | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Intercepted medication error | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Limb injury | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Overdose | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Underdose | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
|
| Cardiac murmur | Investigations | MedDRA 25.1 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
|
| Hyperphosphatasaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
|
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
|
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Umbilical granuloma | Pregnancy, puerperium and perinatal conditions | MedDRA 25.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Eczema | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Papule | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
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The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
| Change from Baseline at Month 12 |
|
|
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| Score >=60 |
|