Efficacy and Safety of Tasimelteon Compared With Placebo in Totally Blind Subjects With Non-24-Hour Sleep-Wake Disorder

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Vanda Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01163032
First received: July 2, 2010
Last updated: October 15, 2014
Last verified: October 2014
Results First Received: August 8, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Non-24-Hour Sleep-Wake Disorder
Interventions: Drug: tasimelteon
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
*Various category for the Randomization Phase: 4 patients in each treatment group discontinued due to study termination by the sponsor and 1 patient in the tasimelteon group discontinued due to travel across multiple time zones

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Tasimelteon (Randomized)

20 mg tasimelteon capsules, PO daily for 6 months

tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months

Placebo (Randomized)

Placebo capsules, PO daily for 6 months

Placebo: Placebo capsules, PO daily for 6 months

Open Label Tasimelteon

20 mg tasimelteon capsules, PO daily for 6 months

tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months


Participant Flow for 2 periods

Period 1:   Randomization Phase
    Tasimelteon (Randomized)     Placebo (Randomized)     Open Label Tasimelteon  
STARTED     42     42     0 [1]
COMPLETED     32     30     0 [1]
NOT COMPLETED     10     12     0  
Withdrawal by Subject                 2                 3                 0  
Adverse Event                 3                 3                 0  
Protocol Violation                 0                 1                 0  
Various*                 5                 4                 0  
Unsatisfactory Therapeutic Effect                 0                 1                 0  
[1] Not applicable for Randomized Phase

Period 2:   Open Label Extension Phase
    Tasimelteon (Randomized)     Placebo (Randomized)     Open Label Tasimelteon  
STARTED     0 [1]   0 [1]   55 [2]
COMPLETED     0 [1]   0 [1]   39  
NOT COMPLETED     0     0     16  
Adverse Event                 0                 0                 3  
Withdrawal by Subject                 0                 0                 3  
Protocol Violation                 0                 0                 1  
Study Termination                 0                 0                 7  
Unsatisfactory Therapeutic Effect                 0                 0                 2  
[1] Not applicable for Open Label Extension Phase
[2] 3 randomized patients rolled into extension (2 randomized to tasimelteon and one to placebo)



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
52 patients entered the OLE Phase from screening and 3 patients rolled over after completing the Randomization Phase (2:tasimelteon; 1:placebo). For this analysis, 3 patients are included in the randomized arms and not the OLE. A separate analysis has been done for both age and gender for the 3 subjects (Rand to OLE).

Reporting Groups
  Description
Tasimelteon (Randomized)

20 mg tasimelteon capsules, PO daily for 6 months

tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months

Placebo (Randomized)

Placebo capsules, PO daily for 6 months

Placebo: Placebo capsules, PO daily for 6 months

Open Label Tasimelteon

20 mg tasimelteon capsules, PO daily for 6 months

tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months

Total Total of all reporting groups

Baseline Measures
    Tasimelteon (Randomized)     Placebo (Randomized)     Open Label Tasimelteon     Total  
Number of Participants  
[units: participants]
  42     42     52     136  
Age  
[units: years]
Mean ± Standard Deviation
  50.8  ± 12.63     50.7  ± 13.15     50.37  ± 13.17     50.6  ± 12.91  
Age, Customized [1]
[units: years]
Mean ± Standard Deviation
       
Rand to OLE     42.00  ± 5.66     54.00  ± NA [2]   NA  ± NA [3]   46.00  ± 8.00  
Gender, Customized [1]
[units: participants]
       
Rand to OLE (Female)     0     0     0     0  
Rand to OLE (Male)     2     1     0     3  
Gender  
[units: participants]
       
Female     18     17     25     60  
Male     24     25     27     76  
[1] N = 2 (tasimelteon randomized then went to OLE) and 1 (placebo randomized then went to OLE)
[2] N = 1; Cannot be calculated
[3] Not applicable



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Proportion of Patients Entrained as Assessed by Urinary aMT6   [ Time Frame: 1 month ]

2.  Primary:   Proportion of Patients With a Clinical Response: Entrainment of aMT6 and Score of ≥ 3 on N24CRS   [ Time Frame: 6 months ]

3.  Secondary:   Proportion of Patients Entrained as Assessed by Urinary Cortisol   [ Time Frame: 1 month ]

4.  Secondary:   Average Clinical Global Impression of Change (CGI-C)   [ Time Frame: Day 112 and 183 ]

5.  Secondary:   Proportion of Responders With a Combined Sleep/Wake Response for LQ-nTST (≥ 90 Minutes) and UQ-dTSD (≤ 90 Minutes)   [ Time Frame: 6 months ]

6.  Secondary:   Average Lower Quartile of Nights of Nighttime Total Sleep Time (LQ-nTST)   [ Time Frame: 6 months ]

7.  Secondary:   Average Upper Quartile of Days of Subjective Daytime Sleep Duration (UQ-dTSD)   [ Time Frame: 6 months ]

8.  Secondary:   Average Midpoint of Sleep (MoST)   [ Time Frame: 6 months ]

9.  Secondary:   Number of Patients With a Treatment Emergent Adverse Event (Open Label Extension Phase Only)   [ Time Frame: 6 months ]

10.  Other Pre-specified:   Proportion of Patients With a Clinical Response: Entrainment of aMT6 and Score of ≥ 2 on N24CRS   [ Time Frame: 6 months ]

11.  Other Pre-specified:   Proportion of Patients With a Clinical Response (Score of ≥ 3 on N24CRS)   [ Time Frame: 6 months ]

12.  Post-Hoc:   Proportion of Responders With a Combined Sleep/Wake Response for LQ-nTST (≥ 45 Minutes) and UQ-dTSD (≤ 45 Minutes)   [ Time Frame: 6 months ]

13.  Other Pre-specified:   Proportion of Patients With a Clinical Response (Score of ≥ 2 on N24CRS)   [ Time Frame: 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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:  
Name/Title: Marlene Dressman, Ph.D.
Organization: Vanda Pharmaceuticals Inc.
phone: 202-734-3462
e-mail: marlene.dressman@vandapharma.com


No publications provided


Responsible Party: Vanda Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01163032     History of Changes
Other Study ID Numbers: VP-VEC-162-3201
Study First Received: July 2, 2010
Results First Received: August 8, 2014
Last Updated: October 15, 2014
Health Authority: United States: Food and Drug Administration