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Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease

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ClinicalTrials.gov Identifier: NCT03033108
Recruitment Status : Completed
First Posted : January 26, 2017
Results First Posted : April 27, 2021
Last Update Posted : May 19, 2021
Sponsor:
Information provided by (Responsible Party):
Kubota Vision Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Stargardt Disease
Macular Atrophy
Intervention Drug: Emixustat
Enrollment 23
Recruitment Details Subjects were recruited at 6 sites in the United States from January to September 2017
Pre-assignment Details  
Arm/Group Title Emixustat Dose 1 Emixustat Dose 2 Emixustat Dose 3
Hide Arm/Group Description

lowest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

middle dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

highest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

Period Title: Overall Study
Started 7 9 7
Completed 7 8 7
Not Completed 0 1 0
Arm/Group Title Emixustat Dose 1 Emixustat Dose 2 Emixustat Dose 3 Total
Hide Arm/Group Description

lowest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

middle dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

highest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

Total of all reporting groups
Overall Number of Baseline Participants 7 9 7 23
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 7 participants 9 participants 7 participants 23 participants
53.6
(27 to 77)
54.3
(32 to 77)
46.3
(18 to 70)
51.6
(18 to 77)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 9 participants 7 participants 23 participants
Female
4
  57.1%
1
  11.1%
3
  42.9%
8
  34.8%
Male
3
  42.9%
8
  88.9%
4
  57.1%
15
  65.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 9 participants 7 participants 23 participants
Hispanic or Latino
1
  14.3%
0
   0.0%
0
   0.0%
1
   4.3%
Not Hispanic or Latino
6
  85.7%
9
 100.0%
7
 100.0%
22
  95.7%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 9 participants 7 participants 23 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
  11.1%
0
   0.0%
1
   4.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
  28.6%
1
  11.1%
1
  14.3%
4
  17.4%
White
5
  71.4%
7
  77.8%
6
  85.7%
18
  78.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants 9 participants 7 participants 23 participants
7 9 7 23
Number of ABCA4 gene mutations  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 9 participants 7 participants 23 participants
One mutation
1
  14.3%
2
  22.2%
2
  28.6%
5
  21.7%
Two mutations
6
  85.7%
7
  77.8%
5
  71.4%
18
  78.3%
1.Primary Outcome
Title Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram
Hide Description Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.
Time Frame Baseline and 1 month
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects with evaluable ERGs at both Baseline and Month 1
Arm/Group Title Emixustat Dose 1 Emixustat Dose 2 Emixustat Dose 3
Hide Arm/Group Description:

lowest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

middle dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

highest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

Overall Number of Participants Analyzed 6 7 6
Median (Full Range)
Unit of Measure: percent suppression
-12.23
(-41.0 to 54.7)
68.00
(1.0 to 91.5)
96.69
(64.9 to 100.0)
2.Secondary Outcome
Title Percentage of Subjects With Adverse Events, by Severity and Seriousness
Hide Description Assessment of safety profile
Time Frame 1 month
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Emixustat Dose 1 Emixustat Dose 2 Emixustat Dose 3
Hide Arm/Group Description:

lowest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

middle dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

highest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

Overall Number of Participants Analyzed 7 9 7
Measure Type: Count of Participants
Unit of Measure: Participants
Subjects with 1 or more adverse events
6
  85.7%
8
  88.9%
6
  85.7%
Subjects with 1 or more mild adverse events
5
  71.4%
5
  55.6%
4
  57.1%
Subjects with 1 or more moderate adverse events
1
  14.3%
2
  22.2%
2
  28.6%
Subjects with 1 or more severe adverse events
0
   0.0%
1
  11.1%
1
  14.3%
Subjects with 1 or more serious adverse events
0
   0.0%
0
   0.0%
0
   0.0%
Time Frame 1 month
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Emixustat Dose 1 Emixustat Dose 2 Emixustat Dose 3
Hide Arm/Group Description

lowest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

middle dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

highest dose of once-daily oral emixustat

Emixustat: Once daily, tablet for oral administration

All-Cause Mortality
Emixustat Dose 1 Emixustat Dose 2 Emixustat Dose 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/9 (0.00%)   0/7 (0.00%) 
Hide Serious Adverse Events
Emixustat Dose 1 Emixustat Dose 2 Emixustat Dose 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/9 (0.00%)   0/7 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Emixustat Dose 1 Emixustat Dose 2 Emixustat Dose 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   6/7 (85.71%)   8/9 (88.89%)   7/7 (100.00%) 
Eye disorders       
Delayed Dark Adaptation  1  1/7 (14.29%)  6/9 (66.67%)  4/7 (57.14%) 
Erythropsia  1  1/7 (14.29%)  3/9 (33.33%)  1/7 (14.29%) 
Vision blurred  1  2/7 (28.57%)  1/9 (11.11%)  1/7 (14.29%) 
Photophobia  1  1/7 (14.29%)  2/9 (22.22%)  0/7 (0.00%) 
Visual impairment  1  1/7 (14.29%)  1/9 (11.11%)  1/7 (14.29%) 
Blindness day  1  0/7 (0.00%)  1/9 (11.11%)  1/7 (14.29%) 
Chromatopsia  1  0/7 (0.00%)  0/9 (0.00%)  2/7 (28.57%) 
Night blindness  1  0/7 (0.00%)  1/9 (11.11%)  1/7 (14.29%) 
Xanthopsia  1  0/7 (0.00%)  1/9 (11.11%)  1/7 (14.29%) 
Asthenopia  1  1/7 (14.29%)  0/9 (0.00%)  0/7 (0.00%) 
Eye Pain  1  1/7 (14.29%)  0/9 (0.00%)  0/7 (0.00%) 
Eye Pruritis  1  1/7 (14.29%)  0/9 (0.00%)  0/7 (0.00%) 
Lenticular opacities  1  0/7 (0.00%)  0/9 (0.00%)  1/7 (14.29%) 
Visual acuity reduced  1  0/7 (0.00%)  0/9 (0.00%)  1/7 (14.29%) 
Infections and infestations       
Upper respiratory tract infections  1  0/7 (0.00%)  1/9 (11.11%)  0/7 (0.00%) 
Investigations       
Blood bilirubin increased  1  0/7 (0.00%)  1/9 (11.11%)  1/7 (14.29%) 
Blood creatine phosphokinase increased  1  0/7 (0.00%)  1/9 (11.11%)  1/7 (14.29%) 
Nervous system disorders       
Headache  1  0/7 (0.00%)  2/9 (22.22%)  2/7 (28.57%) 
Visual field defect  1  1/7 (14.29%)  0/9 (0.00%)  0/7 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Cough  1  1/7 (14.29%)  0/9 (0.00%)  0/7 (0.00%) 
Rhinorrheoea  1  1/7 (14.29%)  0/9 (0.00%)  0/7 (0.00%) 
Skin and subcutaneous tissue disorders       
Dermatitis allergic  1  0/7 (0.00%)  1/9 (11.11%)  0/7 (0.00%) 
Rash  1  0/7 (0.00%)  1/9 (11.11%)  0/7 (0.00%) 
1
Term from vocabulary, MedDRA 20.1
Indicates events were collected by systematic assessment
Small sample size and inherent, significant intersubject and intrasubject variability observed in electroretinography.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Clinical Trials Helpdesk
Organization: Kubota Vision Inc.
Phone: (206) 805-8310
EMail: clinicaltrials@kubotavision.com
Layout table for additonal information
Responsible Party: Kubota Vision Inc.
ClinicalTrials.gov Identifier: NCT03033108    
Other Study ID Numbers: 4429-204
First Submitted: January 20, 2017
First Posted: January 26, 2017
Results First Submitted: March 31, 2021
Results First Posted: April 27, 2021
Last Update Posted: May 19, 2021