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A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (Rainbowfish)

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ClinicalTrials.gov Identifier: NCT03779334
Recruitment Status : Active, not recruiting
First Posted : December 19, 2018
Results First Posted : March 5, 2024
Last Update Posted : March 15, 2024
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Muscular Atrophy, Spinal
Intervention Drug: Risdiplam
Enrollment 26
Recruitment Details Overall, 26 infants with spinal muscular atrophy (SMA) were enrolled in the study across 7 different sites in 7 countries.
Pre-assignment Details 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.
Arm/Group Title 2 SMN2 Copies, Risdiplam 3 SMN2 Copies, Risdiplam >/=4 SMN2 Copies, Risdiplam
Hide Arm/Group Description 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. 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. 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.
Period Title: Overall Study
Started 8 13 5
Primary Efficacy Population 5 0 0
Completed 0 0 0
Not Completed 8 13 5
Reason Not Completed
Withdrawal by Subject             3             0             0
Ongoing in Study             5             13             5
Arm/Group Title 2 SMN2 Copies, Risdiplam 3 SMN2 Copies, Risdiplam >/=4 SMN2 Copies, Risdiplam Total
Hide Arm/Group Description 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. 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. 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. Total of all reporting groups
Overall Number of Baseline Participants 8 13 5 26
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Days
Number Analyzed 8 participants 13 participants 5 participants 26 participants
22.8  (5.0) 28.9  (7.5) 31.2  (6.1) 27.5  (7.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 13 participants 5 participants 26 participants
Female
4
  50.0%
9
  69.2%
3
  60.0%
16
  61.5%
Male
4
  50.0%
4
  30.8%
2
  40.0%
10
  38.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 13 participants 5 participants 26 participants
Hispanic or Latino
3
  37.5%
0
   0.0%
0
   0.0%
3
  11.5%
Not Hispanic or Latino
5
  62.5%
12
  92.3%
5
 100.0%
22
  84.6%
Unknown or Not Reported
0
   0.0%
1
   7.7%
0
   0.0%
1
   3.8%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 13 participants 5 participants 26 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
   7.7%
2
  40.0%
3
  11.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
8
 100.0%
11
  84.6%
3
  60.0%
22
  84.6%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
   7.7%
0
   0.0%
1
   3.8%
1.Primary Outcome
Title 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
Hide Description 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.
Time Frame At Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title 2 SMN2 Copies, Risdiplam
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Measure Type: Number
Number (90% Confidence Interval)
Unit of Measure: percentage of participants
80.0
(34.26 to 98.98)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 2 SMN2 Copies, Risdiplam
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Exact Binomial Test
Comments Performance Criterion = 5%
2.Secondary Outcome
Title Percentage of Participants Developing Clinically Manifested SMA
Hide Description [Not Specified]
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
3.Secondary Outcome
Title Time to Permanent Ventilation and/or Death
Hide Description [Not Specified]
Time Frame Up to 7 years
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Percentage of Participants Who Are Alive Without Permanent Ventilation
Hide Description [Not Specified]
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Percentage of Participants Alive
Hide Description [Not Specified]
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Percentage of Participants Who Achieve the Attainment Level of the Motor Milestones as Assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2)
Hide Description HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
7.Secondary Outcome
Title Percentage of Participants With Two Copies of the SMN2 Gene Sitting Without Support for 5 Seconds (Independent of the CMAP Value at Baseline).
Hide Description 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.
Time Frame At Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title 2 SMN2 Copies, Risdiplam
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 8
Measure Type: Number
Number (90% Confidence Interval)
Unit of Measure: percentage of participants
87.5
(52.93 to 99.36)
8.Secondary Outcome
Title Percentage of Participants Sitting Without Support for 5 Seconds
Hide Description Assessed with BSID-III Gross Motor Scale
Time Frame At Month 24
Outcome Measure Data Not Reported
9.Secondary Outcome
Title Percentage of Participants Sitting Without Support for 30 Seconds
Hide Description Assessed with BSID-III Gross Motor Scale
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
10.Secondary Outcome
Title Percentage of Participants Standing for at Least 3 Seconds
Hide Description Assessed with BSID-III Gross Motor Scale
Time Frame At Month 24
Outcome Measure Data Not Reported
11.Secondary Outcome
Title Percentage of Participants Walking (Takes at Least 3 Steps)
Hide Description Assessed with BSID-III Gross Motor Scale
Time Frame At Month 24
Outcome Measure Data Not Reported
12.Secondary Outcome
Title 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
Hide Description Assessed through BSID-III Gross Motor Scale
Time Frame At Month 24 and 42
Outcome Measure Data Not Reported
13.Secondary Outcome
Title Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12
Hide Description 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.
Time Frame Baseline, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants, regardless of whether they received risdiplam or not.
Arm/Group Title 2 SMN2 Copies, Risdiplam 3 SMN2 Copies, Risdiplam >/=4 SMN2 Copies, Risdiplam
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 8 13 5
Median (Full Range)
Unit of Measure: score on a scale
Baseline Number Analyzed 8 participants 13 participants 5 participants
46.50
(35.0 to 52.0)
55.00
(44.0 to 62.0)
50.00
(44.0 to 52.0)
Change from Baseline at Month 12 Number Analyzed 8 participants 13 participants 4 participants
9.50
(-6.0 to 20.0)
8.00
(2.0 to 20.0)
16.00
(8.0 to 19.0)
14.Secondary Outcome
Title 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
Hide Description 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.
Time Frame At Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants, regardless of whether they received risdiplam or not.
Arm/Group Title 2 SMN2 Copies, Risdiplam 3 SMN2 Copies, Risdiplam >/=4 SMN2 Copies, Risdiplam
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 8 13 4
Measure Type: Number
Number (90% Confidence Interval)
Unit of Measure: percentage of participants
Score >=40
75.0
(40.03 to 95.36)
100
(79.42 to 100.00)
100
(47.29 to 100.00)
Score >=50
75.0
(40.03 to 95.36)
100
(79.42 to 100.00)
100
(47.29 to 100.00)
Score >=60
37.5
(11.11 to 71.08)
100
(79.42 to 100.00)
100
(47.29 to 100.00)
15.Secondary Outcome
Title Percentage of Participants Who Meet CHOP INTEND Stopping Criteria at Any Point
Hide Description [Not Specified]
Time Frame Up to Month 24
Outcome Measure Data Not Reported
16.Secondary Outcome
Title Change From Baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) Score
Hide Description [Not Specified]
Time Frame At Month 60
Outcome Measure Data Not Reported
17.Secondary Outcome
Title Number and Percentage of Participants Within 3rd Percentile of Normal Range for Weight-for-Age, Length/Height-for-Age and Weight-for-Length/Height
Hide Description Based on the WHO Child Growth Standards (WHO 2019)
Time Frame At Month 12, 24, 36, 48 and 60
Outcome Measure Data Not Reported
18.Secondary Outcome
Title Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age
Hide Description Based on the WHO Child Growth Standards (WHO 2019)
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
19.Secondary Outcome
Title Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height
Hide Description [Not Specified]
Time Frame At Month 12, 24, 36, 48 and 60
Outcome Measure Data Not Reported
20.Secondary Outcome
Title Change From Baseline Percentiles for Head Circumference- For-age
Hide Description [Not Specified]
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
21.Secondary Outcome
Title Change From Baseline in Chest Circumference
Hide Description [Not Specified]
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
22.Secondary Outcome
Title Ratio Between Chest and Head Circumferences
Hide Description [Not Specified]
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
23.Secondary Outcome
Title Percentage of Participants With the Ability to Swallow and to Feed Orally
Hide Description [Not Specified]
Time Frame At Month 12, 24, 36, 48 and 60
Outcome Measure Data Not Reported
24.Secondary Outcome
Title Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitude
Hide Description Measured by CMAP
Time Frame At Month 12 and 24
Outcome Measure Data Not Reported
25.Secondary Outcome
Title Measurement of Pharmacodynamic Marker Levels in Blood
Hide Description [Not Specified]
Time Frame Day 1, 56, 196, 364, 728 and at early withdrawal
Outcome Measure Data Not Reported
26.Secondary Outcome
Title Percentage of Participants With Adverse Events
Hide Description Adverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5
Time Frame Up to 7 years
Outcome Measure Data Not Reported
27.Secondary Outcome
Title Ophthalmological Examination as Appropriate for Age
Hide Description [Not Specified]
Time Frame Up to 7 years
Outcome Measure Data Not Reported
28.Secondary Outcome
Title Plasma Concentration of Risdiplam and Its Metabolites to Characterize the PK Profile
Hide Description [Not Specified]
Time Frame Up to 7 years
Outcome Measure Data Not Reported
Time Frame From baseline up to the clinical cut off date (a minimum of 12 months and a maximum of 3.5 years)
Adverse Event Reporting Description The safety population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not.
 
Arm/Group Title 2 SMN2 Copies, Risdiplam 3 SMN2 Copies, Risdiplam >/=4 SMN2 Copies, Risdiplam
Hide Arm/Group Description 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. 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. 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.
All-Cause Mortality
2 SMN2 Copies, Risdiplam 3 SMN2 Copies, Risdiplam >/=4 SMN2 Copies, Risdiplam
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/8 (0.00%)      0/13 (0.00%)      0/5 (0.00%)    
Hide Serious Adverse Events
2 SMN2 Copies, Risdiplam 3 SMN2 Copies, Risdiplam >/=4 SMN2 Copies, Risdiplam
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/8 (37.50%)      0/13 (0.00%)      1/5 (20.00%)    
Gastrointestinal disorders       
Constipation † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Infections and infestations       
Gastroenteritis † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 1/5 (20.00%)  1
Urinary tract infection † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  2
Injury, poisoning and procedural complications       
Femur fracture † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Soft tissue injury † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Pregnancy, puerperium and perinatal conditions       
Jaundice neonatal † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
1
Term from vocabulary, MedDRA 25.1
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
2 SMN2 Copies, Risdiplam 3 SMN2 Copies, Risdiplam >/=4 SMN2 Copies, Risdiplam
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/8 (100.00%)      12/13 (92.31%)      4/5 (80.00%)    
Blood and lymphatic system disorders       
Anaemia † 1  1/8 (12.50%)  1 1/13 (7.69%)  1 0/5 (0.00%)  0
Congenital, familial and genetic disorders       
Cryptorchism † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Eye disorders       
Cystoid macular oedema † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Retinal pigmentation † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Retinal vascular disorder † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Gastrointestinal disorders       
Abdominal pain † 1  1/8 (12.50%)  2 0/13 (0.00%)  0 1/5 (20.00%)  1
Constipation † 1  1/8 (12.50%)  2 3/13 (23.08%)  3 1/5 (20.00%)  1
Diarrhoea † 1  0/8 (0.00%)  0 4/13 (30.77%)  4 2/5 (40.00%)  2
Dyspepsia † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Gastrointestinal pain † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Gastrooesophageal reflux disease † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Regurgitation † 1  0/8 (0.00%)  0 1/13 (7.69%)  2 0/5 (0.00%)  0
Teething † 1  2/8 (25.00%)  2 6/13 (46.15%)  6 2/5 (40.00%)  2
Vomiting † 1  1/8 (12.50%)  2 2/13 (15.38%)  7 2/5 (40.00%)  2
General disorders       
Malaise † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Pyrexia † 1  1/8 (12.50%)  1 4/13 (30.77%)  7 2/5 (40.00%)  5
Infections and infestations       
Bronchiolitis † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Bronchitis † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 2/5 (40.00%)  3
COVID-19 † 1  2/8 (25.00%)  2 7/13 (53.85%)  7 0/5 (0.00%)  0
Conjunctivitis † 1  0/8 (0.00%)  0 2/13 (15.38%)  2 1/5 (20.00%)  1
Croup infectious † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Cytomegalovirus infection † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Ear infection † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Exanthema subitum † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Gastroenteritis † 1  1/8 (12.50%)  1 2/13 (15.38%)  2 2/5 (40.00%)  3
Gastroenteritis norovirus † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Gastrointestinal viral infection † 1  0/8 (0.00%)  0 2/13 (15.38%)  3 0/5 (0.00%)  0
Hand-foot-and-mouth disease † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Impetigo † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Influenza † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  2
Nasopharyngitis † 1  1/8 (12.50%)  1 3/13 (23.08%)  3 1/5 (20.00%)  2
Oral candidiasis † 1  1/8 (12.50%)  1 1/13 (7.69%)  2 0/5 (0.00%)  0
Otitis media acute † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Respiratory syncytial virus bronchitis † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Respiratory syncytial virus infection † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Respiratory tract infection † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Respiratory tract infection bacterial † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Respiratory tract infection viral † 1  2/8 (25.00%)  4 2/13 (15.38%)  2 0/5 (0.00%)  0
Rhinitis † 1  1/8 (12.50%)  1 2/13 (15.38%)  2 2/5 (40.00%)  5
Skin infection † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Suspected COVID-19 † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Tonsillitis † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Upper respiratory tract infection † 1  1/8 (12.50%)  1 1/13 (7.69%)  1 0/5 (0.00%)  0
Varicella † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 2/5 (40.00%)  2
Viral infection † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 1/5 (20.00%)  1
Viral rash † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Vulvovaginitis † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Injury, poisoning and procedural complications       
Accidental overdose † 1  0/8 (0.00%)  0 3/13 (23.08%)  3 0/5 (0.00%)  0
Arthropod bite † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Contusion † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Expired product administered † 1  1/8 (12.50%)  1 1/13 (7.69%)  1 0/5 (0.00%)  0
Fall † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Incorrect dose administered † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 1/5 (20.00%)  1
Intercepted medication error † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Limb injury † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Overdose † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Underdose † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Investigations       
Alanine aminotransferase increased † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Aspartate aminotransferase increased † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Cardiac murmur † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Metabolism and nutrition disorders       
Decreased appetite † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Hyperphosphatasaemia † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Hypoglycaemia † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 0/5 (0.00%)  0
Iron deficiency † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Vitamin D deficiency † 1  0/8 (0.00%)  0 2/13 (15.38%)  2 0/5 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Pain in extremity † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Pregnancy, puerperium and perinatal conditions       
Umbilical granuloma † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Cough † 1  0/8 (0.00%)  0 2/13 (15.38%)  2 1/5 (20.00%)  1
Epistaxis † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 1/5 (20.00%)  3
Nasal congestion † 1  1/8 (12.50%)  1 3/13 (23.08%)  3 1/5 (20.00%)  1
Rhinitis allergic † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Rhinorrhoea † 1  2/8 (25.00%)  2 0/13 (0.00%)  0 0/5 (0.00%)  0
Skin and subcutaneous tissue disorders       
Dermatitis † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Dermatitis atopic † 1  1/8 (12.50%)  1 0/13 (0.00%)  0 1/5 (20.00%)  1
Dermatitis contact † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
Dermatitis diaper † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Eczema † 1  1/8 (12.50%)  1 4/13 (30.77%)  5 1/5 (20.00%)  1
Erythema † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Papule † 1  2/8 (25.00%)  3 0/13 (0.00%)  0 0/5 (0.00%)  0
Rash † 1  1/8 (12.50%)  1 2/13 (15.38%)  2 0/5 (0.00%)  0
Rash maculo-papular † 1  0/8 (0.00%)  0 1/13 (7.69%)  1 0/5 (0.00%)  0
Skin discolouration † 1  0/8 (0.00%)  0 0/13 (0.00%)  0 1/5 (20.00%)  1
1
Term from vocabulary, MedDRA 25.1
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Communications
Organization: Hoffmann-La Roche
Phone: 800 821-8590
EMail: genentech@druginfo.com
Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03779334    
Other Study ID Numbers: BN40703
2018-002087-12 ( EudraCT Number )
First Submitted: December 17, 2018
First Posted: December 19, 2018
Results First Submitted: February 9, 2024
Results First Posted: March 5, 2024
Last Update Posted: March 15, 2024