Clinical Trial Results:
A multicenter, randomized, double-blind, placebo-controlled, multiple-dose study to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RO6885247 following 12 weeks of treatment in adult and pediatric patients with spinal muscular atrophy (MOONFISH).
Summary
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EudraCT number |
2014-002246-41 |
Trial protocol |
IT ES |
Global end of trial date |
23 Jul 2015
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Results information
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Results version number |
v1(current) |
This version publication date |
24 Jun 2017
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First version publication date |
24 Jun 2017
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
BP29420
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02240355 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
F. Hoffmann-La Roche AG
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Sponsor organisation address |
Grenzacherstrasse 124, Basel, Switzerland, CH4070
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Public contact |
F. Hoffmann-La Roche AG,, F. Hoffmann-La Roche AG,, +41 61 6878333, global.trial_information@roche.com
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Scientific contact |
F. Hoffmann-La Roche AG,, F. Hoffmann-La Roche AG,, +41 61 6878333, global.trial_information@roche.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
23 Jul 2015
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
23 Jul 2015
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
The primary objective of this study was to evaluate the safety and tolerability of 12 weeks of treatment with RO6885247 in adult and pediatric subjects with Spinal Muscular Atrophy (SMA)
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Protection of trial subjects |
All study subjects were required to read and sign an Informed Consent Form
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
17 Nov 2014
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Long term follow-up planned |
Yes
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Long term follow-up rationale |
Safety | ||
Long term follow-up duration |
2 Months | ||
Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Switzerland: 6
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Country: Number of subjects enrolled |
United Kingdom: 4
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Country: Number of subjects enrolled |
Italy: 6
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Worldwide total number of subjects |
16
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EEA total number of subjects |
10
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
7
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Adults (18-64 years) |
9
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
The study was conducted at 3 sites/centers in Switzerland, United Kingdom, and Italy. | ||||||||||||||||||||
Pre-assignment
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Screening details |
A total 16 subjects were enrolled in Part 1 (13 subjects in Cohort 1a and 3 subjects in Cohort 1b). Parts 2 and 3 were not conducted as the study was terminated early by the Sponsor. | ||||||||||||||||||||
Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Double blind | ||||||||||||||||||||
Roles blinded |
Subject, Investigator | ||||||||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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RO6885247 10 mg | ||||||||||||||||||||
Arm description |
Subjects with Spinal Muscular Atrophy received RO6885247 10 milligrams (mg) once daily (QD) | ||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||
Investigational medicinal product name |
RO6885247
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Oral solution
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Routes of administration |
Oral use
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Dosage and administration details |
Subjects received 10 mg once daily (QD) of oral solution of either RO6885247.
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Arm title
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RO6885247 20 mg | ||||||||||||||||||||
Arm description |
Subjects with Spinal Muscular Atrophy recieved RO6885247 20 mg once daily (QD). | ||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||
Investigational medicinal product name |
RO6885247
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Oral solution
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Routes of administration |
Oral use
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Dosage and administration details |
Subjects received 20 mg QD of oral solution of RO6885247.
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Arm title
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Placebo | ||||||||||||||||||||
Arm description |
Subjects with Spinal Muscular Atrophy received a matching placebo to RO6885247 dose once daily (QD) | ||||||||||||||||||||
Arm type |
Placebo | ||||||||||||||||||||
Investigational medicinal product name |
Placebo
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Oral solution
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Routes of administration |
Oral use
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Dosage and administration details |
Subjects received an oral solution of matching Placebo to RO6885247 QD.
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Baseline characteristics reporting groups
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Reporting group title |
RO6885247 10 mg
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Reporting group description |
Subjects with Spinal Muscular Atrophy received RO6885247 10 milligrams (mg) once daily (QD) | |||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
RO6885247 20 mg
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Reporting group description |
Subjects with Spinal Muscular Atrophy recieved RO6885247 20 mg once daily (QD). | |||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Placebo
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Reporting group description |
Subjects with Spinal Muscular Atrophy received a matching placebo to RO6885247 dose once daily (QD) | |||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
RO6885247 10 mg
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Reporting group description |
Subjects with Spinal Muscular Atrophy received RO6885247 10 milligrams (mg) once daily (QD) | ||
Reporting group title |
RO6885247 20 mg
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Reporting group description |
Subjects with Spinal Muscular Atrophy recieved RO6885247 20 mg once daily (QD). | ||
Reporting group title |
Placebo
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Reporting group description |
Subjects with Spinal Muscular Atrophy received a matching placebo to RO6885247 dose once daily (QD) |
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End point title |
Percentage of Subjects with Adverse Events (AEs) [1] | ||||||||||||||||
End point description |
An AE was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events. Safety population included all subjects who had received at least 1 dose of study medication assigned to treatment arms.
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End point type |
Primary
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End point timeframe |
Up to 8 months approximately
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive analysis was planned to be reported. |
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No statistical analyses for this end point |
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End point title |
Maximum Plasma Concentration (Cmax) at Day 1 and Day 28 [2] | ||||||||||||
End point description |
To evaluate the maximum plasma concentration (Cmax) in treated subjects blood. The pharmacokinetic (PK) analysis population included all subjects who received at least one dose of the study medication.
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End point type |
Secondary
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End point timeframe |
2, 4, 6, 8, 24 h Day 1, pre-dose, 2, 4, 6, 8 h Day28
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Notes [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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No statistical analyses for this end point |
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End point title |
Area Under Curve (AUC) Over the 24-hour Dosing Interval at Day 1 and Day 8 [3] | ||||||||||||
End point description |
AUC is a measure of the plasma concentration of a drug over time. The PK analysis population included all subjects who received at least one dose of the study medication.
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End point type |
Secondary
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End point timeframe |
2, 4, 6, 8, 24h Day1, pre-dose, 2, 4, 6, 8h Day 28
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Notes [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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No statistical analyses for this end point |
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End point title |
Steady-State Concentration at the End of a Dosing Interval (Ctrough) at Day 28 [4] | ||||||||
End point description |
To evaluate steady-state concentration of drug in blood at the end of dosing interval. The PK analysis population included all subjects who received at least one dose of the study medication.
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End point type |
Secondary
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End point timeframe |
pre-dose, 2, 4, 6, 8 h Day 28
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Notes [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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No statistical analyses for this end point |
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End point title |
Time to Maximum Observed Plasma Concentration (Tmax) at Day 1 and Day 28 [5] | ||||||||||||
End point description |
To evaluate the time to maximum observed plasma concentration. All treated subjects were analyzed for this endpoint.
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End point type |
Secondary
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End point timeframe |
2, 4, 6, 8, 24 h Day 1; pre-dose, 2, 4, 6, 8 h Day 28
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Notes [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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No statistical analyses for this end point |
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End point title |
Accumulation Ratio (Racc) at Day 28 [6] | ||||||||
End point description |
To evaluate the relationship between the dosing interval and the elimination rate constant. The PK analysis population included all subjects who received at least one dose of the study medication.
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End point type |
Secondary
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End point timeframe |
pre-dose, 2, 4, 6, 8 h Day 28
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Notes [6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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No statistical analyses for this end point |
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End point title |
Tmax Under Fasted and Fed Conditions [7] | ||||||||||||
End point description |
To evaluate the time to maximum observed plasma concentration in subjects in fed and fasted states. The PK analysis population included all subjects who received at least one dose of the study medication.
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End point type |
Secondary
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End point timeframe |
12h Day 14, pre-dose, 2, 4, 6, 8, 12, 24h Day 15
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Notes [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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No statistical analyses for this end point |
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End point title |
Cmax Under Fasted and Fed Conditions [8] | ||||||||||||
End point description |
To evaluate the maximum plasma concentration in treated subjects blood under both fed and fasted conditions.The PK analysis population included all subjects who received at least one dose of the study medication.
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End point type |
Secondary
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End point timeframe |
12h Day 14, pre-dose, 2, 4, 6, 8,12, 24 h Day 15
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Notes [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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No statistical analyses for this end point |
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End point title |
AUCtau Under Fasted and Fed Conditions [9] | ||||||||||||
End point description |
To evaluate drug concentration in subjects blood plasma over time under both fed and fasted states. The PK analysis population included all subjects who received at least one dose of the study medication.
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End point type |
Secondary
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End point timeframe |
12h Day 14, pre-dose, 2, 4, 6, 8, 12, 24h Day 15
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Notes [9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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No statistical analyses for this end point |
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End point title |
Survival of Motor Neuron (SMN) Protein Levels in Blood | ||||||||||||||||||||||||||||||||||||||||
End point description |
To evaluate SMN protein levels in subjects blood. All enrolled subjects. Here, n signifies the number of subjects who evaluable at each time point. Here, 9999 indicates arithmetic mean and standard deviation for time points where number of subjects evaluable is 0.
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End point type |
Secondary
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End point timeframe |
Day -1, 28, 84, 114, 144, Early withdrawal
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No statistical analyses for this end point |
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End point title |
Change From Baseline in Compound Muscle Action Potential (CMAP) Parameter; Temperature | ||||||||||||||||||||||||
End point description |
CMAP was assessed by evaluating change from baseline in the following parameter- temperature. All enrolled subjects. Here, n indicates number of subjects evaluable at specified time points. Here, 99999 indicates mean and standard deviation at early withdrawal.
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End point type |
Secondary
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End point timeframe |
Baseline (Day-1), early withdrawal
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No statistical analyses for this end point |
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End point title |
Effect of RO6885247 on Electrical Impedance Myography [10] | ||||||||
End point description |
Data for this endpoint was not collected during the study.
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End point type |
Secondary
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End point timeframe |
Week 12
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Notes [10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: The endpoint was planned to report the values for reporting group (RO6885247 10 mg). |
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Notes [11] - No data for this endpoint are included as assessment was not performed in any of the subjects. |
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No statistical analyses for this end point |
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End point title |
In Vivo Splicing Modification of Survival of Motor Neuron 2 (SMN2) Messenger Ribonucleic Acid(mRNA) in Blood | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
To evaluate subjects SMN2 mRNA blood levels over time. All enrolled subjects. Here, n signifies the number of subjects who were evaluable at specified time points. Here, 99999 indicates arithmetic mean and standard deviation for the reporting groups where no subject was analysed.
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End point type |
Secondary
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End point timeframe |
Day -1, 1, 28, 56, 84, 114, 144, Early Withdrawal
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No statistical analyses for this end point |
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End point title |
Change From Baseline in CMAP Parameter; Distance | ||||||||||||||||||||||||
End point description |
CMAP was assessed by evaluating change from baseline in the following parameter- distance. All enrolled subjects. Here, n indicates number of subjects evaluable at specified time points. Here, 99999 indicates mean and standard deviation at early withdrawal.
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End point type |
Secondary
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End point timeframe |
Baseline (Day-1), Early withdrawal
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No statistical analyses for this end point |
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End point title |
Change From Baseline in CMAP Parameter; Distal Motor Latency | ||||||||||||||||||||||||
End point description |
CMAP was assessed by evaluating change from baseline in the following parameter- distal motor latency. All enrolled subjects. Here, n indicates number of subjects evaluable at specified time points. Here, 99999 indicates mean and standard deviation at early withdrawal.
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End point type |
Secondary
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End point timeframe |
Baseline (Day-1), Early Withdrawal
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No statistical analyses for this end point |
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End point title |
Change From Baseline in CMAP Paramter; Amplitude | ||||||||||||||||||||||||
End point description |
CMAP was assessed by evaluating change from baseline in the following parameter- Amplitude. All enrolled subjects. Here, n indicates number of subjects evaluable at specified time points. Here, 99999 indicates mean and standard deviation at early withdrawal.
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End point type |
Secondary
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End point timeframe |
Baseline (Day-1), Early Withdrawal
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No statistical analyses for this end point |
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End point title |
Change From Baseline in CMAP; Area | ||||||||||||||||||||||||
End point description |
CMAP was assessed by evaluating change from baseline in the following parameter- Area. All enrolled subjects. Here, n indicates number of subjects evaluable at specified time points. Here, 99999 indicates mean and standard deviation at early withdrawal.
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End point type |
Secondary
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End point timeframe |
Baseline (Day-1), Early Withdrawal
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
Up to 8 months approximately
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Adverse event reporting additional description |
The safety analysis population included all subjects who had received at least one dose of the study medication.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
18.0
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Reporting groups
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Reporting group title |
RO6885247 10 mg
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Reporting group description |
Subjects received a 10 mg dose of RO6885247 once daily (QD). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
RO6885247 20 mg
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Reporting group description |
Subjects received a 20 mg dose of RO6885247 once daily (QD). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Placebo
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Reporting group description |
Subjects received a placebo dose once daily (QD) | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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26 Sep 2014 |
Two exclusion criteria for females of child-bearing potential and hypersensitivity to RO6885247 or to the constituents of its formulation were updated, as a request from MHRA during review of original protocol (v1). |
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24 Dec 2014 |
1. Addition of a cohort of infant subjects with Spinal Muscular Atrophy Type 1, 2. Clarification on the food effect assessment in Cohort 1a, 3. Addition of staggered enrollment in Cohorts 1b and 2a; 4. Changes to the criteria for treatment discontinuation for skin AEs, to the AE reporting period, study medication intake on the days of site visits, SMN2 mRNA and SMN protein time points (Parts 1 and 2), and follow up calls of the first 4 weeks of the study. |
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Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
The study was put on hold by the sponsor as it did not reveal any notable safety issues. |