Trial Outcomes & Findings for A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) (NCT NCT01886378)
NCT ID: NCT01886378
Last Updated: 2021-02-11
Results Overview
To evaluate the impact 24 weeks of treatment with UX007 has on exercise intolerance, the change from Baseline in time adjusted-AUC (AUC/time) for workload during 40-minute cycle ergometry tests at Week 24 were assessed using the generalized estimation equation (GEE) model. A cycle ergometer can measure the work performed by an individual over time during physical exercise, the work was measured every 10 minutes from 0 to 40 minutes at Baseline and Week 24. The GEE model included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. An increase in AUC is reflective of improved exercise tolerance; a negative change from Baseline indicates worsening.
COMPLETED
PHASE2
29 participants
Baseline, Week 24
2021-02-11
Participant Flow
Following the signing of informed consent at the Screening visit, each participant continued on current long-chain fatty acid oxidation disorder (LC-FAOD) management for a 4-week Run-in Period to establish a clinical baseline. Following completion of the 4-week Run-in Period, participants discontinued any use of medium chain triglycerides (MCT) and began treatment with UX007.
Participant milestones
| Measure |
UX007
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Overall Study
STARTED
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29
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Overall Study
Completed 24 Weeks of UX007 Treatment
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25
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Overall Study
COMPLETED
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24
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Overall Study
NOT COMPLETED
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5
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Reasons for withdrawal
| Measure |
UX007
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Overall Study
Adverse Event
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1
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Overall Study
Withdrawal by Subject
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4
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Baseline Characteristics
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Baseline characteristics by cohort
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Age, Continuous
|
12.06 years
STANDARD_DEVIATION 5.26 • n=93 Participants
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Age, Customized
0 - 1 years
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2 Participants
n=93 Participants
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Age, Customized
> 1 - 6 years
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13 Participants
n=93 Participants
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Age, Customized
> 6 -18 years
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8 Participants
n=93 Participants
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Age, Customized
> 18 years
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6 Participants
n=93 Participants
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Sex: Female, Male
Female
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12 Participants
n=93 Participants
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Sex: Female, Male
Male
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17 Participants
n=93 Participants
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Primary Analysis Set - Cycle Ergometry: the subset of participants in the primary analysis set (all enrolled participants who completed the 4 week Run-in Period and received at least one dose of UX007) who had at least one cycle ergometry test performed with any duration.
To evaluate the impact 24 weeks of treatment with UX007 has on exercise intolerance, the change from Baseline in time adjusted-AUC (AUC/time) for workload during 40-minute cycle ergometry tests at Week 24 were assessed using the generalized estimation equation (GEE) model. A cycle ergometer can measure the work performed by an individual over time during physical exercise, the work was measured every 10 minutes from 0 to 40 minutes at Baseline and Week 24. The GEE model included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. An increase in AUC is reflective of improved exercise tolerance; a negative change from Baseline indicates worsening.
Outcome measures
| Measure |
UX007
n=7 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in Time Adjusted-Area Under the Curve (AUC/Time) for Workload During Cycle Ergometry at Week 24
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423.594 watts
Standard Error 295.54
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Primary Analysis Set - Cycle Ergometry: the subset of participants in the primary analysis set (all enrolled participants who completed the 4 week Run-in Period and received at least one dose of UX007) who had at least one cycle ergometry test performed with any duration.
Change from baseline in time-adjusted-AUC for respiratory exchange ratio (RER) during cycle ergometry at Week 24, assessed using the GEE model, which included change from baseline as dependent variable, time as categorical variable, and adjusted for baseline measurement with compound symmetry covariance structure. RER during exercise is calculated as volume of carbon dioxide/volume of oxygen. RER measures whether carbohydrates or fats are being used as fuel. RER ≥1.0 indicates carbohydrates are the predominate fuel source. RER \<1.0 and RER \>0.70 indicates both fats and carbohydrates are the predominate fuel source. RER approximately =0.70 means fat is the predominant fuel source. RER would be expected to be lower, at similar exercise intensities, if a participant is able to utilize fat as an energy source. Therefore, an increase in RER (positive change from baseline) would suggest participants are still utilizing carbohydrates rather than fat, reflective a physiological response.
Outcome measures
| Measure |
UX007
n=7 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in Time-Adjusted-AUC for Respiratory Exchange Ratio (RER) During Cycle Ergometry at Week 24
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-0.011 respiratory exchange ratio
Standard Error 0.0132
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Primary Analysis Set - Cycle Ergometry: the subset of participants in the primary analysis set (all enrolled participants who completed the 4 week Run-in Period and received at least one dose of UX007) who had at least one cycle ergometry test performed with any duration.
To evaluate the impact of 24 weeks of treatment with UX007 on exercise intolerance, the change from Baseline in actual duration of exercise during 40-minute cycle ergometry tests at Week 24 was assessed using the GEE model. The GEE model included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. Duration of exercise is expected to increase as exercise tolerance improves.
Outcome measures
| Measure |
UX007
n=7 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in Actual Duration of Exercise During Cycle Ergometry at Week 24
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4.671 minutes
Standard Error 2.65
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PRIMARY outcome
Timeframe: Baseline (last assessment during the 4-week run-in period), Week 18Population: Primary Analysis Set - 12MWT: the subset of participants in the primary analysis set (all enrolled participants who completed the 4 week Run-in Period and received at least one dose of UX007) who had at least one 12MWT performed with any distance walked.
To evaluate the impact 18 weeks of treatment with UX007 has on muscle function, the change from Baseline in distance traveled during a 12MWT at Week 18 was assessed using the GEE model. The GEE model included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. Distance traveled during the 12MWT is expected to increase as muscle function increases.
Outcome measures
| Measure |
UX007
n=8 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in Distance Traveled During the 12-Minute Walk Test (12MWT) at Week 18
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181.37 meters
Standard Error 104.63
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PRIMARY outcome
Timeframe: Baseline (last assessment during the 4-week run-in period), Week 18Population: Primary Analysis Set - 12MWT: the subset of participants in the primary analysis set (all enrolled participants who completed the 4 week Run-in Period and received at least one dose of UX007) who had at least one 12MWT performed with any distance walked.
To evaluate the impact 18 weeks of treatment with UX007 has on muscle function, the change from Baseline of EEI during the 12MWT at Week 18 was assessed using the GEE model. The GEE model included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. EEI is quantified as the post-test heart rate minus the pre-test heart rate (in beats/min) divided by overall velocity, and is valued in beats/meter. A decrease in EEI when walking a similar distance or no change when walking longer distances, may indicate improved exercise tolerance.
Outcome measures
| Measure |
UX007
n=8 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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Change From Baseline in Energy Expenditure Index (EEI) During the 12MWT at Week 18
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-0.185 beats/meter
Standard Error 0.09
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PRIMARY outcome
Timeframe: Baseline (last assessment during the 4-week run-in period), Week 18Population: Primary Analysis Set - 12MWT: the subset of participants in the primary analysis set (all enrolled participants who completed the 4 week Run-in Period and received at least one dose of UX007) who had at least one 12MWT performed with any distance walked.
To evaluate the impact 18 weeks of treatment with UX007 has on muscle function, the change from Baseline in the percentage of the predicted distance traveled during the first 6 minutes (6MWT) of the 12MWT at Week 18 was assessed using the GEE model. A participant's mathematical formula to calculate their percent predicted (PP) distance walked in the 6MWT was based on their demographics at baseline. For participants \< 20 years old, the formula used was referenced from (Gieger, et. al. 2007) which calculated PP distance walked based on age, gender, and height. For participants \>= 20 years old, the formula used was referenced from (Gibbons, et. al. 2001) and calculated the PP distance walked based on age and gender. The GEE model included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. Percent predicted values are expected to increase as muscle function increases.
Outcome measures
| Measure |
UX007
n=8 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in Percentage of the Predicted 6-Minute Walk Test (6MWT) Distance Walked at Week 18
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12.44 % of predicted distance (in meters)
Standard Error 7.22
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Primary Analysis Set - SF-10: the subset of participants in the primary analysis set (all enrolled participants who completed the 4 week Run-in Period and received at least one dose of UX007) who had at least one SF-10 test performed.
To evaluate the impact treatment with UX007 has on functional disability and health in participants between 5 and 17 years of age, change from Baseline in the T-scores of the PHS-10 were assessed at Week 24 and analyzed using the GEE model. The GEE model included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. The SF-10 Health Survey for Children is a 10-item caregiver-completed assessment designed to measure children's health-related quality of life. The PHS-10 of the SF-10 is scored such that higher scores indicate more favorable functioning. The T-score based scoring signifies that scale scores are centered so that a score of 50 corresponds to the average score in a comprehensive sample of US population (scale scores are standardized to a mean of 50 and a standard deviation of 10).
Outcome measures
| Measure |
UX007
n=5 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in Physical Summary Score (PHS-10) of the Short Form 10 (SF-10) at Week 24
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2.16 T-score
Standard Error 2.16
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Primary Analysis Set - SF-10: the subset of participants in the primary analysis set (all enrolled participants who completed the 4 week Run-in Period and received at least one dose of UX007) who had at least one SF-10 test performed.
To evaluate the impact treatment with UX007 has on functional disability and health in participants between 5 and 17 years of age, changes from Baseline in the T-scores of the PSS-10 were assessed at Week 24 and analyzed using the GEE model. The GEE model included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. The PSS-10 of the SF-10 is scored such that higher scores indicate more favorable functioning. The T-score based scoring signifies that scale scores are centered so that a score of 50 corresponds to the average score in a comprehensive sample of US population (scale scores are standardized to a mean of 50 and a standard deviation of 10). Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of more favorable functioning/better health.
Outcome measures
| Measure |
UX007
n=5 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in Psychosocial Summary Score (PSS-10) of the SF10 at Week 24
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0.816 T-score
Standard Error 2.63
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Primary Analysis Set - SF-12: the subset of participants in the primary analysis set (those who completed the 4-week run-in period and received at least one dose of UX007) who had at least one SF-12 test performed.
Changes from baseline in T-scores as assessed by the PCS-12 Short-Form Health Survey, version 2 (SF-12v2) at Week 24 were assessed using the GEE model, which included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. PCS-12 scores were calculated from the individual responses to those questions that contribute to physical health. Raw scores range from 0 to 100 with higher scores indicating better health. The T-score based scoring signifies that scale scores are centered so that a score of 50 corresponds to the average score in the US general population (scale scores are standardized to a mean of 50 and a standard deviation of 10). Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of more favorable functioning/better health.
Outcome measures
| Measure |
UX007
n=5 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in the Physical Component Summary Scale (PCS-12) at Week 24
|
8.88 T-score
Standard Deviation 1.63
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Primary Analysis Set - SF-12: the subset of participants in the primary analysis set (those who completed the 4-week run-in period and received at least one dose of UX007) who had at least one SF-12 test performed.
Changes from baseline of T-scores as assessed by the MCS-12 of the SF-12v2 at Week 24 were assessed using the GEE model, which included the change from Baseline as the dependent variable, time as the categorical variable, and adjusted for Baseline measurement with compound symmetry covariance structure. MCS-12 scores were calculated from the individual responses to those questions that contribute to mental health. Raw scores range from 0 to 100 with higher scores indicating better health. The T-score based scoring signifies that scale scores are centered so that a score of 50 corresponds to the average score in the US general population (scale scores are standardized to a mean of 50 and a standard deviation of 10). Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of more favorable functioning/better health.
Outcome measures
| Measure |
UX007
n=5 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Change From Baseline in the Mental Component Summary Scale (MCS-12) at Week 24
|
9.7 T-score
Standard Error 4.0
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PRIMARY outcome
Timeframe: 18 months before and after UX007 initiationPopulation: Primary Analysis Set: participants who completed the 4-week run-in period and received at least one dose of UX007.
Major clinical events are defined as adverse events (AEs) resulting in hospitalizations, emergency room (ER) visits, and emergency intervention.
Outcome measures
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Annualized Event Rate of All Major Clinical Events Pre- and Post-Treatment With UX007
Pre-UX007
|
1.69 events/year
Standard Deviation 1.6081
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Annualized Event Rate of All Major Clinical Events Pre- and Post-Treatment With UX007
Post-UX007
|
0.877 events/year
Standard Deviation 1.142
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PRIMARY outcome
Timeframe: 18 months before and after UX007 initiationPopulation: Primary Analysis Set: participants who completed the 4-week run-in period and received at least one dose of UX007.
Major clinical events are defined as AEs resulting in hospitalizations, ER visits, and emergency intervention.
Outcome measures
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Annualized Duration Rate of All Major Clinical Events Pre- and Post-Treatment With UX007
Pre-UX007
|
5.961 days/year
Standard Deviation 6.0783
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Annualized Duration Rate of All Major Clinical Events Pre- and Post-Treatment With UX007
Post-UX007
|
2.964 days/year
Standard Deviation 3.9733
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PRIMARY outcome
Timeframe: 18 months before and after UX007 initiationPopulation: Primary Analysis Set: participants who completed the 4-week run-in period and received at least one dose of UX007.
Rhabdomyolysis is a condition in which damaged skeletal muscle breaks down rapidly. Major rhabdomyolysis clinical events are defined as those AEs resulting in hospitalizations, ER visits, and emergency intervention.
Outcome measures
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Annualized Event Rate of Major Rhabdomyolysis Clinical Events Pre- and Post-Treatment With UX007
Pre-UX007
|
1.303 events/year
Standard Deviation 1.5007
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Annualized Event Rate of Major Rhabdomyolysis Clinical Events Pre- and Post-Treatment With UX007
Post-UX007
|
0.833 events/year
Standard Deviation 1.1513
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PRIMARY outcome
Timeframe: 18 months before and after UX007 initiationPopulation: Primary Analysis Set: participants who completed the 4-week run-in period and received at least one dose of UX007.
Rhabdomyolysis is a condition in which damaged skeletal muscle breaks down rapidly. Major rhabdomyolysis clinical events are defined as those AEs resulting in hospitalizations, ER visits, and emergency intervention.
Outcome measures
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Annualized Duration Rate of Major Rhabdomyolysis Clinical Events Pre- and Post-Treatment With UX007
Pre-UX007
|
3.949 days/year
Standard Deviation 4.3687
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Annualized Duration Rate of Major Rhabdomyolysis Clinical Events Pre- and Post-Treatment With UX007
Post-UX007
|
2.792 days/year
Standard Deviation 3.8452
|
PRIMARY outcome
Timeframe: 18 months before and after UX007 initiationPopulation: Primary Analysis Set: participants who completed the 4-week run-in period and received at least one dose of UX007.
Major hypoglycemia clinical events are defined as those AEs resulting in hospitalizations, ER visits, and emergency intervention.
Outcome measures
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Annualized Event Rate of Major Hypoglycemia Clinical Events Pre- and Post-Treatment With UX007
Pre-UX007
|
0.318 events/year
Standard Deviation 0.9053
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Annualized Event Rate of Major Hypoglycemia Clinical Events Pre- and Post-Treatment With UX007
Post-UX007
|
0.023 events/year
Standard Deviation 0.1224
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PRIMARY outcome
Timeframe: 18 months before and after UX007 initiationPopulation: Primary Analysis Set: participants who completed the 4-week run-in period and received at least one dose of UX007.
Major hypoglycemia clinical events are defined as those AEs resulting in hospitalizations, ER visits, and emergency intervention.
Outcome measures
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
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|---|---|
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Annualized Duration Rate of Major Hypoglycemia Clinical Events Pre- and Post-Treatment With UX007
Pre-UX007
|
1.414 days/year
Standard Deviation 4.3025
|
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Annualized Duration Rate of Major Hypoglycemia Clinical Events Pre- and Post-Treatment With UX007
Post-UX007
|
0.023 days/year
Standard Deviation 0.1224
|
PRIMARY outcome
Timeframe: 18 months before and after UX007 initiationPopulation: Primary Analysis Set: participants who completed the 4-week run-in period and received at least one dose of UX007.
Major cardiac clinical events are defined as those AEs resulting in hospitalizations, ER visits, and emergency intervention.
Outcome measures
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
|
|---|---|
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Annualized Event Rate of Major Cardiac Clinical Events Pre- and Post-Treatment With UX007
Pre-UX007
|
0.069 events/year
Standard Deviation 0.2728
|
|
Annualized Event Rate of Major Cardiac Clinical Events Pre- and Post-Treatment With UX007
Post-UX007
|
0.021 events/year
Standard Deviation 0.115
|
PRIMARY outcome
Timeframe: 18 months before and after UX007 initiationPopulation: Primary Analysis Set: participants who completed the 4-week run-in period and received at least one dose of UX007.
Major cardiac clinical events are defined as those AEs resulting in hospitalizations, ER visits, and emergency intervention.
Outcome measures
| Measure |
UX007
n=29 Participants
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
|
|---|---|
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Annualized Duration Rate of Major Cardiac Clinical Events Pre- and Post-Treatment With UX007
Pre-UX007
|
0.598 days/year
Standard Deviation 2.4054
|
|
Annualized Duration Rate of Major Cardiac Clinical Events Pre- and Post-Treatment With UX007
Post-UX007
|
0.149 days/year
Standard Deviation 0.8047
|
Adverse Events
UX007
Serious adverse events
| Measure |
UX007
n=29 participants at risk
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
|
|---|---|
|
Surgical and medical procedures
Infection Prophylaxis
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Cardiac disorders
Cardiomyopathy Acute
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Congenital, familial and genetic disorders
Talipes
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Gastrointestinal Disorder
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Gastrointestinal Haemorrhage
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Vomiting
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Reproductive system and breast disorders
Menorrhagia
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
37.9%
11/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Metabolism and nutrition disorders
Metabolic Disorder
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Gastroenteritis
|
20.7%
6/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Gastroenteritis Viral
|
20.7%
6/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Gastrointestinal Viral Infection
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Adenoviral Upper Respiratory Infection
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Adenovirus Infection
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Conjunctivitis
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Coxsackie Viral Infection
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Croup Infectious
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Otitis Media
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Otitis Media Acute
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Pneumonia Mycoplasmal
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Respiratory Tract Infection Viral
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Roseola
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Urinary Tract Infection
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Viral Upper Respiratory Tract Infection
|
3.4%
1/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
Other adverse events
| Measure |
UX007
n=29 participants at risk
UX007 dosing was titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants were followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic Naevus
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
General disorders
Pyrexia
|
31.0%
9/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
General disorders
Pain
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Psychiatric disorders
Anxiety
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Psychiatric disorders
Irritability
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Psychiatric disorders
Panic Attack
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Injury, poisoning and procedural complications
Arthropod Bite
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Injury, poisoning and procedural complications
Fall
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Injury, poisoning and procedural complications
Laceration
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Injury, poisoning and procedural complications
Procedural Pain
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Injury, poisoning and procedural complications
Stoma Site Pain
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Investigations
Blood Creatine Phosphokinase Increased
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Investigations
Carnitine Decreased
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Cardiac disorders
Tachycardia
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.8%
4/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus Congestion
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Nervous system disorders
Headache
|
31.0%
9/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Eye disorders
Eye Swelling
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Diarrhoea
|
55.2%
16/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Vomiting
|
44.8%
13/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Abdominal Pain
|
27.6%
8/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
13.8%
4/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Gastrointestinal Pain
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Gastrooesophageal Reflux Disease
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Abdominal Distension
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Constipation
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Flatulence
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Nausea
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Gastrointestinal disorders
Teething
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Skin and subcutaneous tissue disorders
Acne
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Skin and subcutaneous tissue disorders
Dermatitis Allergic
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
27.6%
8/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
17.2%
5/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
13.8%
4/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Metabolism and nutrition disorders
Dehydration
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
37.9%
11/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Ear Infection
|
17.2%
5/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Nasopharyngitis
|
17.2%
5/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Bronchitis
|
13.8%
4/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Gastroenteritis Viral
|
13.8%
4/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Gastrointestinal Viral Infection
|
13.8%
4/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Rhinitis
|
13.8%
4/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Viral Upper Respiratory Tract Infection
|
13.8%
4/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Conjunctivitis
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Gastroenteritis
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Sinusitis
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Urinary Tract Infection
|
10.3%
3/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Otitis Media
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
|
Infections and infestations
Respiratory Tract Infection Viral
|
6.9%
2/29 • From the first dose of UX007 through the Follow-up visit (Week 82) or 30 days following the last UX007 administration.
Events presented are treatment-emergent. An event was considered as treatment-emergent if it occurred on or after the date of the first treatment of UX007.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER