Clinical Trial Ceftriaxone in Subjects With ALS

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Merit E. Cudkowicz, MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT00349622
First received: July 5, 2006
Last updated: April 1, 2014
Last verified: April 2014
Results First Received: October 23, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Conditions: Amyotrophic Lateral Sclerosis
ALS
Interventions: Drug: ceftriaxone
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
Ceftriaxone is approved by the U.S. Food and Drug Administration (FDA) for treating bacterial infections but not for treating ALS. Subjects with ALS were enrolled in 58 institutions across in the US and Canada.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Participants were randomly assigned to receive treatment with ceftriaxone or placebo for at least 12 months. Two thirds of participants received ceftriaxone and one third received placebo. This is a blinded study, so neither participants nor study staff knew which treatment a participant is receiving.

Reporting Groups
  Description
Ceftriaxone

Two thirds of participants were assigned to 4 grams of ceftriaxone per day. This is a blinded study, so neither participants nor study staff will know which treatment a participant is receiving.

Ceftriaxone is a cephalosporin antibiotic and was administered intravenously via a central venous catheter twice a day.

Placebo

One third of participants were assigned to placebo, or an inactive substance. This is a blinded study, so neither participants nor study staff will know which treatment a participant is receiving.

Pediatric multivitamin solution was used as the placebo in this study and was administered intravenously via a central venous catheter twice a day.


Participant Flow:   Overall Study
    Ceftriaxone     Placebo  
STARTED     340     173  
COMPLETED     162     77  
NOT COMPLETED     178     96  
Death                 168                 86  
Lost to Follow-up                 5                 1  
Withdrawal by Subject                 5                 9  



  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
Ceftriaxone

Two thirds of participants were assigned to 4 grams of ceftriaxone per day. This is a blinded study, so neither participants nor study staff will know which treatment a participant is receiving.

Ceftriaxone is a cephalosporin antibiotic and was administered intravenously via a central venous catheter twice a day.

Placebo

One third of participants were assigned to placebo, or an inactive substance. This is a blinded study, so neither participants nor study staff will know which treatment a participant is receiving.

Pediatric multivitamin solution was used as the placebo in this study and was administered intravenously via a central venous catheter twice a day.

Total Total of all reporting groups

Baseline Measures
    Ceftriaxone     Placebo     Total  
Number of Participants  
[units: participants]
  340     173     513  
Age  
[units: years]
Mean ± Standard Deviation
     
Age at Screening     55.6  ± 10.4     54.8  ± 10.3     55.4  ± 10.4  
Gender  
[units: participants]
     
Female     131     72     203  
Male     209     101     310  
Ethnicity (NIH/OMB)  
[units: participants]
     
Hispanic or Latino     17     6     23  
Not Hispanic or Latino     320     165     485  
Unknown or Not Reported     3     2     5  
Race (NIH/OMB)  
[units: participants]
     
American Indian or Alaska Native     2     0     2  
Asian     6     5     11  
Native Hawaiian or Other Pacific Islander     1     0     1  
Black or African American     8     3     11  
White     320     163     483  
More than one race     2     0     2  
Unknown or Not Reported     1     2     3  
Region of Enrollment  
[units: participants]
     
United States     293     151     444  
Canada     47     22     69  
ALS Family History [1]
[units: participants]
     
Familial History of ALS     26     8     34  
No Known Familial History of ALS     307     161     468  
Unknown     7     4     11  
Site of Onset [2]
[units: participants]
     
Limb     257     137     394  
Bulbar     75     35     110  
Both     8     1     9  
Riluzole Use [3]
[units: participants]
     
On Riluzole     249     128     377  
Not on Riluzole     91     45     136  
Vital Capacity Percent Predicted [4]
[units: percent predicted based on age and heigh]
Mean ± Standard Deviation
  87.9  ± 16.6     91.1  ± 18.4     89.0  ± 17.3  
Time to Screening  
[units: years]
Mean ± Standard Deviation
     
Years from Symptom Onset to Screening     1.49  ± 0.68     1.50  ± 0.67     1.49  ± 0.68  
Years from Diagnosis to Screening     0.56  ± 0.49     0.58  ± 0.49     0.57  ± 0.49  
Years from Symptom Onset to Diagnosis     0.93  ± 0.55     0.92  ± 0.58     0.92  ± 0.56  
[1] Subjects were asked at screening whether or not they have a familial history of ALS.
[2] Indicates region of first symptoms
[3] At the screening visit, subjects were asked whether or not they were taking a continuous dose of riluzole.
[4] The vital capacity (lung capacity) for each subject was measured at screening.



  Outcome Measures
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1.  Primary:   Survival   [ Time Frame: From date of randomization until date of death, tracheostomy, or the initiation of permanent assisted ventilation (PAV). This was assessed at time of each participant's drug discontinuation and every 2 months thereafter for the life of the study (6 yrs) ]

2.  Primary:   Change From Baseline in ALS Functional Rating Scale, Revised (ALSFRS-R) at One Year   [ Time Frame: Every 8 weeks for one year ]

3.  Secondary:   Change in % Vital Capacity From Screening to One Year   [ Time Frame: Every 12 weeks for one Year ]

4.  Secondary:   Change From Baseline in Evaluation of Multiple Upper Extremity Muscles Using Hand Held Dynamometry at One Year   [ Time Frame: Every 12 weeks for one Year ]

5.  Secondary:   Change From Baseline in the ALS-Specific Quality of Life Scale (ALSQOL) at One Year   [ Time Frame: Every 12 weeks for one Year ]

6.  Secondary:   Change From Baseline in Evaluation of Multiple Lower Extremity Muscles Using Hand Held Dynamometry at One Year   [ Time Frame: Every 12 weeks for one Year ]


  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: Merit Cudkowicz
Organization: MGH
phone: 617-724-1873
e-mail: mcudkowicz@partners.org


No publications provided by Massachusetts General Hospital

Publications automatically indexed to this study:

Responsible Party: Merit E. Cudkowicz, MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT00349622     History of Changes
Other Study ID Numbers: U01NS049640-02, NINDS, U01NS049640-02, NINDS CRC
Study First Received: July 5, 2006
Results First Received: October 23, 2013
Last Updated: April 1, 2014
Health Authority: United States: Food and Drug Administration