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The Effect of Diflunisal on Familial Amyloidosis

This study has been completed.
Sponsor:
Collaborators:
Food and Drug Administration (FDA)
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
John L. Berk, Boston University
ClinicalTrials.gov Identifier:
NCT00294671
First received: February 21, 2006
Last updated: January 30, 2017
Last verified: January 2017
Results First Received: December 26, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions: Familial Amyloid Polyneuropathy
Familial Amyloidosis
Interventions: Drug: diflunisal
Other: 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
Participants were recruited between May 2006 and December 2010 from amyloid centers of excellence in Sweden (Umea), Italy (Pavia), United Kingdom (London), Japan (Matsumoto and Kumamoto), and the United States (New York, Minnesota, and Massachusetts).

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All enrolled participants were randomized to treatment groups.

Reporting Groups
  Description
Diflunisal Diflunisal 250 mg taken by mouth twice daily for 24 months
Placebo Placebo, an inactive substance, taken by mouth twice daily for 24 months

Participant Flow:   Overall Study
    Diflunisal   Placebo
STARTED   64   66 
COMPLETED   37   26 
NOT COMPLETED   27   40 
Lack of Efficacy                11                23 
Liver transplant                7                9 
Drug related adverse event (AE)                4                2 
Non-drug related AE                3                2 
Lost to Follow-up                1                3 
Non-compliance                1                0 
Death                0                1 



  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.
No text entered.

Reporting Groups
  Description
Diflunisal Diflunisal 250 mg taken by mouth twice daily for 24 months
Placebo Placebo, an inactive substance, taken by mouth twice daily for 24 months
Total Total of all reporting groups

Baseline Measures
   Diflunisal   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 64   66   130 
Age 
[Units: Years]
Mean (Standard Deviation)
 60.3  (11.7)   59.2  (12.2)   59.7  (11.9) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      21  32.8%      22  33.3%      43  33.1% 
Male      43  67.2%      44  66.7%      87  66.9% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      8  12.5%      6   9.1%      14  10.8% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      1   1.6%      5   7.6%      6   4.6% 
White      52  81.3%      50  75.8%      102  78.5% 
More than one race      3   4.7%      4   6.1%      7   5.4% 
Unknown or Not Reported      0   0.0%      1   1.5%      1   0.8% 
Region of Enrollment 
[Units: Participants]
     
United States   34   35   69 
United Kingdom   3   4   7 
Italy   10   11   21 
Japan   5   4   9 
Sweden   12   12   24 
NIS+7 score [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 51.6  (42.8)   59.0  (50)   55.3  (46.5) 
[1] Higher scores indicating greater neurologic deficits (0=no neurologic impairment, 270=no detectable peripheral neurologic function)
Kumamoto score [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 15.3  (10.8)   16.7  (13.5)   16.0  (12.2) 
[1] Higher scores indicating greater neurologic and end organ deficits (0=no neurologic deficits, 102=no detectable peripheral neurologic function)
Modified BMI [1] 
[Units: kg/M2 x g/L]
Mean (Standard Deviation)
 1024.4  (226.3)   1019  (255)   1021.7  (240.4) 
[1] The product of BMI and serum albumin (g/L). Lower scores indicate worse nutritional status.
SF-36 physical component score [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 35.9  (11.6)   34.8  (11)   35.4  (11.3) 
[1] Higher scores indicate greater physical quality of life, scale from 0 to 100 points
SF-36 mental component score [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 46.6  (14.1)   46.5  (11.8)   46.6  (12.9) 
[1] Higher scores indicate greater mental quality of life, scale 0-100 points


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Neurologic Impairment Score + 7 (NIS+7)   [ Time Frame: Baseline, 1 and 2 years ]

2.  Secondary:   Kumamoto Neurologic Scale;   [ Time Frame: Baseline, 1 and 2 years ]

3.  Secondary:   Modified Body Mass Index (mBMI);   [ Time Frame: Baseline, 1 and 2 years ]

4.  Secondary:   Quality of Life Questionnaire: SF-36 Physical Component Score   [ Time Frame: Baseline, 1 and 2 years ]

5.  Secondary:   Quality of Life Questionnaire: SF-36 Mental Component Score   [ Time Frame: Baseline, 1 and 2 years ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

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: Dr. John L. Berk
Organization: Amyloidosis Center, Boston Medical Center
phone: 617-638-4494
e-mail: jberk@bu.edu


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: John L. Berk, Boston University
ClinicalTrials.gov Identifier: NCT00294671     History of Changes
Other Study ID Numbers: R01NS051306 ( U.S. NIH Grant/Contract )
FD R 002532 ( Other Grant/Funding Number: FDA Office of Orphan Products Development (OOPD) )
Study First Received: February 21, 2006
Results First Received: December 26, 2013
Last Updated: January 30, 2017