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Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis (TUDCA-ALS)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00877604
First Posted: April 8, 2009
Last Update Posted: January 23, 2015
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.
Collaborators:
University of Palermo
Federico II University
Information provided by (Responsible Party):
Alberto Albanese, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Results First Submitted: October 31, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Single Group Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Amyotrophic Lateral Sclerosis
Interventions: Drug: tauroursodeoxycholic acid (TUDCA)
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
Recruitment is started on eightth of october 2009, in medical clinics (Besta Institute in Milan, University II department of Neurology of Naples, University of Palermo department of Neurology).

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
34 patients enrolled, 5 of whom dropped out during the lead in phase.

Reporting Groups
  Description
TUDCA

tauroursodeoxycholic acid di-hydrate

tauroursodeoxycholic acid (TUDCA): Oral route at the dose of 1 g b.i.d. (2 g daily) for 1 year

Placebo

excipient lactose

Placebo: identical placebo by oral route at the same dosing schedule composed of excipient lactose


Participant Flow:   Overall Study
    TUDCA   Placebo
STARTED   17   17 
COMPLETED   15   14 
NOT COMPLETED   2   3 



  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
TUDCA

tauroursodeoxycholic acid di-hydrate

tauroursodeoxycholic acid (TUDCA): Oral route at the dose of 1 g b.i.d. (2 g daily) for 1 year

Placebo

excipient lactose

Placebo: identical placebo by oral route at the same dosing schedule

Total Total of all reporting groups

Baseline Measures
   TUDCA   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 17   17   34 
Age 
[Units: Years]
Mean (Standard Deviation)
 54.0  (12.2)   58.2  (12.9)   56.1  (12.3) 
Gender 
[Units: Participants]
     
Female   6   7   13 
Male   11   10   21 
Region of Enrollment 
[Units: Participants]
     
Italy   17   17   34 


  Outcome Measures

1.  Primary:   The Proportion of Responder Patients in the Two Treatment Groups According the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS)-R Slope.   [ Time Frame: 1 year ]
  Hide Outcome Measure 1

Measure Type Primary
Measure Title The Proportion of Responder Patients in the Two Treatment Groups According the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS)-R Slope.
Measure Description Responder patients were defined as those subjects showing an improvement of at least 15% in the ALSFRS-R slope during the treatment period as compared to the lead-in period.
Time Frame 1 year  

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
TUDCA

tauroursodeoxycholic acid di-hydrate

tauroursodeoxycholic acid (TUDCA): Oral route at the dose of 1 g b.i.d. (2 g daily) for 1 year

Placebo

excipient lactose

Placebo: identical placebo by oral route at the same dosing schedule composed of excipient lactose


Measured Values
   TUDCA   Placebo 
Participants Analyzed 
[Units: Participants]
 15   14 
The Proportion of Responder Patients in the Two Treatment Groups According the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS)-R Slope. 
[Units: Participants]
 13   6 

No statistical analysis provided for The Proportion of Responder Patients in the Two Treatment Groups According the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS)-R Slope.



2.  Secondary:   Forced Vital Capacity (FVC) %   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

3.  Secondary:   SF-36 Quality of Life Rating Scale   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

4.  Secondary:   Time to Tracheostomy From Starting of Study Medication Dosing (if Appropriate)   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

5.  Secondary:   Survival Time From Starting of Study Medication Dosing (if Appropriate)   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

6.  Secondary:   ALSFRS-R at Study End   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

7.  Secondary:   Incidence and Severity of Adverse Events, and Their Relationship to Treatment   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

8.  Secondary:   Medical Research Council Scores for Right and Left Muscle Groups   [ Time Frame: 1 year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events
  Hide Serious Adverse Events

Time Frame No text entered.
Additional Description No text entered.

Reporting Groups
  Description
TUDCA

tauroursodeoxycholic acid di-hydrate

tauroursodeoxycholic acid (TUDCA): Oral route at the dose of 1 g b.i.d. (2 g daily) for 1 year

Placebo

excipient lactose

Placebo: identical placebo by oral route at the same dosing schedule composed of excipient lactose


Serious Adverse Events
    TUDCA   Placebo
Total, Serious Adverse Events     
# participants affected / at risk   0/15 (0.00%)   0/14 (0.00%) 




  Other Adverse Events
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Time Frame No text entered.
Additional Description No text entered.

Frequency Threshold
Threshold above which other adverse events are reported   0%  

Reporting Groups
  Description
TUDCA

tauroursodeoxycholic acid di-hydrate

tauroursodeoxycholic acid (TUDCA): Oral route at the dose of 1 g b.i.d. (2 g daily) for 1 year

Placebo

excipient lactose

Placebo: identical placebo by oral route at the same dosing schedule composed of excipient lactose


Other Adverse Events
    TUDCA   Placebo
Total, Other (not including serious) Adverse Events     
# participants affected / at risk   0/15 (0.00%)   0/14 (0.00%) 



  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
No text entered.


  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: Alberto Albanese
Organization: Fondazione Istituto Neurologico Carlo Besta
phone: +39 02 2392 4552
e-mail: alberto.albanese@unicatt.it


Publications:

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

Responsible Party: Alberto Albanese, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
ClinicalTrials.gov Identifier: NCT00877604     History of Changes
Other Study ID Numbers: TUDCA200701
EudraCT No.: 2007-001592-10
First Submitted: April 7, 2009
First Posted: April 8, 2009
Results First Submitted: October 31, 2014
Results First Posted: November 26, 2014
Last Update Posted: January 23, 2015