Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study Evaluating Intepirdine (RVT-101) in Subjects With Mild to Moderate Alzheimer's Disease on Donepezil: MINDSET Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02585934
Recruitment Status : Completed
First Posted : October 26, 2015
Results First Posted : December 5, 2018
Last Update Posted : December 5, 2018
Sponsor:
Information provided by (Responsible Party):
Axovant Sciences Ltd.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Alzheimer's Disease
Interventions Drug: RVT-101
Drug: Placebo
Enrollment 1315
Recruitment Details  
Pre-assignment Details 2173 participants signed consent and were screened for participation. Of these, 1470 entered a 3-week single-blind placebo run-in period with treatment with placebo qd + stable dose of donepezil 5 or 10 mg qd. There was an overlap between the participants from the run-in period and the participants that were randomized during the treatment period.
Arm/Group Title RVT-101 Placebo
Hide Arm/Group Description

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets for up to 27 weeks

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet for up to 27 weeks

Period Title: Overall Study
Started 661 654
Safety Population [1] 656 651
Intent to Treat (ITT) [2] 643 633
Per Protocol (PP) [3] 603 582
Completed [4] 592 581
Not Completed 69 73
Reason Not Completed
Lost to Follow-up             2             2
Protocol Violation             14             11
Adverse Event             21             22
Physician Decision             3             2
Withdrawal by Subject             7             21
Lack of Efficacy             2             1
Death             2             0
Caregiver Withdrew Consent             7             7
Sponsor Termination             1             2
Another Reason             10             5
[1]
The Safety population included subjects who took at least 1 dose of double-blind study drug
[2]
Subjects took at least 1 dose of double-blind study drug and had at least 1 post-baseline assessment
[3]
Subjects in the ITT population who had no major protocol violations
[4]
Subjects in the ITT population who completed the study
Arm/Group Title RVT-101 Placebo Total
Hide Arm/Group Description

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets for up to 27 weeks

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet for up to 27 weeks

Total of all reporting groups
Overall Number of Baseline Participants 643 633 1276
Hide Baseline Analysis Population Description
ITT population
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 643 participants 633 participants 1276 participants
72.7
(50 to 85)
72.5
(50 to 86)
72.6
(50 to 86)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
< 74 years Number Analyzed 643 participants 633 participants 1276 participants
309
  48.1%
312
  49.3%
621
  48.7%
>/= 74 years Number Analyzed 643 participants 633 participants 1276 participants
334
  51.9%
321
  50.7%
655
  51.3%
< 65 years Number Analyzed 643 participants 633 participants 1276 participants
98
  15.2%
97
  15.3%
195
  15.3%
>/=65 years Number Analyzed 643 participants 633 participants 1276 participants
545
  84.8%
536
  84.7%
1081
  84.7%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 643 participants 633 participants 1276 participants
Female
386
  60.0%
394
  62.2%
780
  61.1%
Male
257
  40.0%
239
  37.8%
496
  38.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 643 participants 633 participants 1276 participants
Hispanic or Latino
120
  18.7%
108
  17.1%
228
  17.9%
Not Hispanic or Latino
518
  80.6%
519
  82.0%
1037
  81.3%
Unknown or Not Reported
5
   0.8%
6
   0.9%
11
   0.9%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race Number Analyzed 643 participants 633 participants 1276 participants
White
595
  92.5%
592
  93.5%
1187
  93.0%
Black or African American
12
   1.9%
10
   1.6%
22
   1.7%
Asian
27
   4.2%
20
   3.2%
47
   3.7%
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
1
   0.2%
1
   0.1%
Others
2
   0.3%
1
   0.2%
3
   0.2%
Not Applicable
3
   0.5%
5
   0.8%
8
   0.6%
Missing
4
   0.6%
4
   0.6%
8
   0.6%
BMI   [1] 
Mean (Full Range)
Unit of measure:  Kg/m^2
Number Analyzed 642 participants 633 participants 1275 participants
26.4
(17.2 to 56.3)
26.5
(15.2 to 45.3)
26.4
(15.2 to 56.3)
[1]
Measure Analysis Population Description: Data was missing for one subject
1.Primary Outcome
Title Alzheimer’s Disease Assessment Scale – Cognitive Subscale 11 Items (ADAS-Cog-11) Score Change From Baseline to Week 24
Hide Description The 11-item ADAS-Cog assesses a range of cognitive abilities including memory, comprehension, orientation in time and place, and spontaneous speech. The ADAS-Cog-11 total score range is from 0 to 70, with a higher score indicating more severe cognitive impairment.
Time Frame Baseline, 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population with data at both timepoints.
Arm/Group Title RVT-101 Placebo
Hide Arm/Group Description:

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet

Overall Number of Participants Analyzed 584 577
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
0.39  (0.211) 0.75  (0.213)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RVT-101, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2249
Comments the threshold for statistical significance was p=0.05
Method Mixed Models Analysis
Comments Statistical Method: Mixed model for repeated measures
Method of Estimation Estimation Parameter least square mean difference
Estimated Value -0.36
Confidence Interval (2-Sided) 95%
-0.95 to 0.22
Estimation Comments [Not Specified]
2.Primary Outcome
Title Alzheimer’s Disease Cooperative Study - Activities of Daily Living (ADCS-ADL) Score Change From Baseline to Week 24
Hide Description The ADCS-ADL scale measures functional impairment in terms of activities of daily living. The score ranges from 0 to 78. The lower the score, the greater the impairment; higher scores indicate better (more desirable) function
Time Frame Baseline, 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population with data at both endpoints
Arm/Group Title RVT-101 Placebo
Hide Arm/Group Description:

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet

Overall Number of Participants Analyzed 588 575
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
-1.06  (0.289) -0.97  (0.293)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RVT-101, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8260
Comments the threshold for statistical significance was p=0.05
Method Mixed Models Analysis
Comments Statistical Method: Mixed model for repeated measures
Method of Estimation Estimation Parameter least square mean difference
Estimated Value -0.09
Confidence Interval (2-Sided) 95%
-0.90 to 0.72
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Clinical Global Impression of Change - Plus Caregiver Interview (CIBIC+) Score at Week 24
Hide Description The CIBIC+ assessment measures the global functioning of the subject. The CIBIC+ is scored as a seven-point categorical rating, ranging from a score of 1 (indicating “very much improved”), to a score of 4 (indicating “no change”), or to a score of 7 (indicating “very much worse.”) Lower CIBIC+ scores indicate better (more desirable) function
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population at the Week 24 timepoint
Arm/Group Title RVT-101 Placebo
Hide Arm/Group Description:

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet

Overall Number of Participants Analyzed 577 568
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
4.18  (0.037) 4.30  (0.037)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RVT-101, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0234
Comments the threshold for statistical significance was p=0.05
Method Mixed Models Analysis
Comments Statistical Method: Mixed model for repeated measures
Method of Estimation Estimation Parameter least square mean difference
Estimated Value -0.12
Confidence Interval (2-Sided) 95%
-0.22 to -0.02
Estimation Comments [Not Specified]
4.Secondary Outcome
Title The Dependence Scale (DS) Score Change From Baseline to Week 24
Hide Description The DS measures the amount of assistance patients with dementia require in performing daily activities. The scale consists of 13 items, representing a range of severity from mild to severe levels of dependency. The score range is from 0 to 15 with higher scores indicating greater dependency.
Time Frame Baseline, 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population at both timepoints
Arm/Group Title RVT-101 Placebo
Hide Arm/Group Description:

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet

Overall Number of Participants Analyzed 580 568
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
0.30  (0.070) 0.17  (0.071)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RVT-101, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2096
Comments the threshold for statistical significance was p=0.05
Method Mixed Models Analysis
Comments Statistical Method: Mixed model for repeated measures
Method of Estimation Estimation Parameter least square mean difference
Estimated Value 0.12
Confidence Interval (2-Sided) 95%
-0.07 to 0.32
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Neuropsychiatric Inventory (NPI) Score Change From Baseline to Week 24
Hide Description The NPI is a behavior rating scale composed of a 12-item structured interview of the caregiver that is scored from 0 to 144 (the higher the score, the greater the psychiatric disturbance). It assesses 12 behavioral disturbances occurring in dementia patients: delusions, hallucinations, agitation, dysphoria, anxiety, apathy, irritability, euphoria, disinhibition, aberrant motor activity, night-time behavior disturbances, and eating disturbances. Both the frequency and the severity of each behavior are determined.
Time Frame Baseline and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population at both timepoints
Arm/Group Title RVT-101 Placebo
Hide Arm/Group Description:

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet

Overall Number of Participants Analyzed 583 570
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
-0.08  (0.339) 0.06  (0.344)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RVT-101, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7650
Comments the threshold for statistical significance was p=0.05
Method Mixed Models Analysis
Comments Statistical Method: Mixed model for repeated measures
Method of Estimation Estimation Parameter least square mean difference
Estimated Value -0.14
Confidence Interval (2-Sided) 95%
-1.09 to 0.80
Estimation Comments [Not Specified]
6.Secondary Outcome
Title ADAS-Cog-13 Score Change From Baseline to Week 24
Hide Description 13-item ADAS-Cog assesses a range of cognitive abilities including memory, comprehension, orientation in time and place, and spontaneous speech. Most items are evaluated by tests, but some are dependent on clinician ratings on a 5-point scale. The ADAS-Cog-13 is the ADAS-Cog-11 with 2 additional items: delayed word recall and total digit cancellation. Scores for the ADAS-Cog-13 range from 0 to 85 with higher scores indicating greater dysfunction.
Time Frame Baseline, 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
ITT at both timepoints
Arm/Group Title RVT-101 Placebo
Hide Arm/Group Description:

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet

Overall Number of Participants Analyzed 583 576
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
0.26  (0.233) 0.64  (0.236)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RVT-101, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2472
Comments the threshold for statistical significance was p=0.05
Method Mixed Models Analysis
Comments Statistical Method: Mixed model for repeated measures
Method of Estimation Estimation Parameter least square mean difference
Estimated Value -0.38
Confidence Interval (2-Sided) 95%
-1.03 to 0.27
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Measurement of Concentrations of RVT-101 (Intepirdine) in Plasma
Hide Description Measurement collected at timepoints Week 6, Week 12, Week 18, and Week 24
Time Frame Week 6, Week 12, Week 18, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
PK population at specified timepoints: Week 6, Week 12, Week 18, and Week 24
Arm/Group Title RVT-101
Hide Arm/Group Description:

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets for up to 27 weeks

Overall Number of Participants Analyzed 656
Geometric Mean (95% Confidence Interval)
Unit of Measure: ng/mL
Summary Statistics at Week 6 Number Analyzed 563 participants
201.11
(191.88 to 210.78)
Summary Statistics at Week 12 Number Analyzed 579 participants
170.95
(164.40 to 177.76)
Summary Statistics at Week 18 Number Analyzed 454 participants
198.69
(189.36 to 208.48)
Summary Statistics at Week 24 Number Analyzed 562 participants
193.36
(184.60 to 202.53)
Time Frame Screening through post treatment (up to 33 weeks)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title RVT-101 Treatment Placebo Treatment Screening Period Run-In Period RVT-101 Post-Treatment Placebo Post-Treatment
Hide Arm/Group Description

RVT-101 adjunct to 5 mg or 10 mg donepezil

RVT-101: once daily, oral, 35 mg tablets for up to 27 weeks

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet for up to 27 weeks

Participants screened (0-4 weeks) prior to entering to the first dose of single-blind study medication (ie, prior to the Run-In period)

Placebo adjunct to 5 mg or 10 mg donepezil

Placebo: once daily, oral, pill manufactured to match RVT-101 35 mg tablet (for 3 weeks)

Subjects reported adverse events after completing the treatment period.

The Post-Treatment period - defined as up to 30 days post-last-dose

Subjects reported adverse events after completing the treatment period.

The Post-Treatment period - defined as up to 30 days post-last-dose

All-Cause Mortality
RVT-101 Treatment Placebo Treatment Screening Period Run-In Period RVT-101 Post-Treatment Placebo Post-Treatment
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/656 (0.46%)   2/651 (0.31%)   1/2173 (0.05%)   1/1470 (0.07%)   0/656 (0.00%)   1/651 (0.15%) 
Show Serious Adverse Events Hide Serious Adverse Events
RVT-101 Treatment Placebo Treatment Screening Period Run-In Period RVT-101 Post-Treatment Placebo Post-Treatment
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   40/656 (6.10%)   44/651 (6.76%)   12/2173 (0.55%)   14/1470 (0.95%)   3/656 (0.46%)   3/651 (0.46%) 
Blood and lymphatic system disorders             
Anaemia   0/656 (0.00%)  0/651 (0.00%)  1/2173 (0.05%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Cardiac disorders             
Myocardial infarction   1/656 (0.15%)  2/651 (0.31%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Atrial Fibrillation   1/656 (0.15%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Angina pectoris   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Angina unstable   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Atrioventricular block   0/656 (0.00%)  1/651 (0.15%)  1/2173 (0.05%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Cardiac arrest   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Cardiac failure congestive   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Cardio-respiratory arrest   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Mitral valve prolapse   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Pericardial effusion   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Right ventricular failure   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Sinus node dysfunction   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Cardiac failure   0/656 (0.00%)  0/651 (0.00%)  1/2173 (0.05%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Ear and labyrinth disorders             
Inner ear disorder   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Vertigo positonal   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Gastrointestinal disorders             
Duodenal vascular ectasia   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Pancreatitis acute   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Proctitis   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Small intestinal obstruction   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Upper gastrointestinal haemorrhage   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Volvulus   0/656 (0.00%)  0/651 (0.00%)  1/2173 (0.05%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
General disorders             
Chest pain   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Hypothermia   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Non-cardiac chest pain   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Asthenia   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Gait disturbance   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  1/656 (0.15%)  0/651 (0.00%) 
Hepatobiliary disorders             
Bile duct stone   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Cholecystitis   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Infections and infestations             
Diverticulitis   2/656 (0.30%)  2/651 (0.31%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Pneumonia   2/656 (0.30%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Urinary tract infection   1/656 (0.15%)  1/651 (0.15%)  1/2173 (0.05%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Appendicitis   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Cellulitis   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Gastroenteritis   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
lower respiratory tract infection   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Infected bite   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Sepsis   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  1/651 (0.15%) 
Injury, poisoning and procedural complications             
Fall   1/656 (0.15%)  3/651 (0.46%)  1/2173 (0.05%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Hip fracture   1/656 (0.15%)  3/651 (0.46%)  2/2173 (0.09%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Contusion   0/656 (0.00%)  1/651 (0.15%)  1/2173 (0.05%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Patella fracture   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Pelvic fracture   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Stoma obstruction   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Subdural haematoma   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Upper limb fracture   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Wrist fracture   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Humerus fracture   0/656 (0.00%)  0/651 (0.00%)  1/2173 (0.05%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Foot fracture   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Pubis fracture   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Investigations             
Haemoglobin decreased   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Metabolism and nutrition disorders             
Dehydration   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Musculoskeletal and connective tissue disorders             
Musculoskeletal pain   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)             
Basal cell carcinoma   2/656 (0.30%)  3/651 (0.46%)  0/2173 (0.00%)  3/1470 (0.20%)  0/656 (0.00%)  0/651 (0.00%) 
Acoustic neuroma   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Breast cancer   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Hepatocellular carcinoma   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Lung neoplasm   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Malignant melanoma   0/656 (0.00%)  1/651 (0.15%)  1/2173 (0.05%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Meningioma   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Ovarian cancer   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Prostate cancer   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Squamous cell carcinoma   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Uterine leiomyoma   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Squamous cell carcinoma of skin   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Nervous system disorders             
Cerebrovascular Accident   0/656 (0.00%)  3/651 (0.46%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Syncope   2/656 (0.30%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Transient ischaemic attack   2/656 (0.30%)  1/651 (0.15%)  1/2173 (0.05%)  0/1470 (0.00%)  1/656 (0.15%)  0/651 (0.00%) 
Ischaemic stroke   2/656 (0.30%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Seizure   0/656 (0.00%)  2/651 (0.31%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Dementia   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Dizziness   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Headache   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Hypoxic-ischaemic encephalopathy   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Lethargy   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Status epilepticus   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Hydrocephalus   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  1/656 (0.15%)  0/651 (0.00%) 
Psychiatric disorders             
Aggression   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Delirium   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Mental status changes   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Mood altered   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Psychotic disorder   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Somatic symptom disorder   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Agitation   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Renal and urinary disorders             
Acute kidney injury   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Reproductive system and breast disorders             
Benign prostatic hyperplasia   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
invasive ductal breast carcinoma   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  1/651 (0.15%) 
Respiratory, thoracic and mediastinal disorders             
Dyspnoea   2/656 (0.30%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Acute respiratory failure   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Pneumonia aspiration   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Pulmonary embolism   1/656 (0.15%)  0/651 (0.00%)  1/2173 (0.05%)  1/1470 (0.07%)  0/656 (0.00%)  1/651 (0.15%) 
Pneumothorax spontaneous   0/656 (0.00%)  0/651 (0.00%)  0/2173 (0.00%)  1/1470 (0.07%)  0/656 (0.00%)  0/651 (0.00%) 
Vascular disorders             
Hypertension   0/656 (0.00%)  1/651 (0.15%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Poor peripheral circulation   1/656 (0.15%)  0/651 (0.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
RVT-101 Treatment Placebo Treatment Screening Period Run-In Period RVT-101 Post-Treatment Placebo Post-Treatment
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   191/656 (29.12%)   166/651 (25.50%)   0/2173 (0.00%)   0/1470 (0.00%)   0/656 (0.00%)   0/651 (0.00%) 
Gastrointestinal disorders             
Diarrhoea   13/656 (1.98%)  17/651 (2.61%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Nausea   17/656 (2.59%)  13/651 (2.00%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Infections and infestations             
Urinary tract infection   25/656 (3.81%)  26/651 (3.99%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Injury, poisoning and procedural complications             
Fall   37/656 (5.64%)  29/651 (4.45%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Musculoskeletal and connective tissue disorders             
Back pain   11/656 (1.68%)  15/651 (2.30%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Nervous system disorders             
Headache   17/656 (2.59%)  18/651 (2.76%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Dizziness   19/656 (2.90%)  12/651 (1.84%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Respiratory, thoracic and mediastinal disorders             
Nasopharyngitis   23/656 (3.51%)  19/651 (2.92%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Bronchitis   14/656 (2.13%)  10/651 (1.54%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Cough   15/656 (2.29%)  7/651 (1.08%)  0/2173 (0.00%)  0/1470 (0.00%)  0/656 (0.00%)  0/651 (0.00%) 
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
All proposed manuscripts, publications or abstracts must be reviewed and approved by the Sponsor 60 days prior to submission for publication. All confidential information identified by the Sponsor must be deleted prior to submission.
Results Point of Contact
Name/Title: Ilise Lombardo, MD
Organization: Axovant Sciences, Inc.
Phone: 646-822-8626
Responsible Party: Axovant Sciences Ltd.
ClinicalTrials.gov Identifier: NCT02585934     History of Changes
Other Study ID Numbers: RVT-101-3001
First Submitted: October 22, 2015
First Posted: October 26, 2015
Results First Submitted: September 30, 2018
Results First Posted: December 5, 2018
Last Update Posted: December 5, 2018