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Clinical Trial Ceftriaxone in Subjects With ALS

This study has been completed.
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
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 
Overall Participants Analyzed 
[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 
[1] Subjects were asked at screening whether or not they have a familial history of ALS.
Site of Onset [1] 
[Units: Participants]
     
Limb   257   137   394 
Bulbar   75   35   110 
Both   8   1   9 
[1] Indicates region of first symptoms
Riluzole Use [1] 
[Units: Participants]
     
On Riluzole   249   128   377 
Not on Riluzole   91   45   136 
[1] At the screening visit, subjects were asked whether or not they were taking a continuous dose of riluzole.
Vital Capacity Percent Predicted [1] 
[Units: Percent predicted based on age and heigh]
Mean (Standard Deviation)
 87.9  (16.6)   91.1  (18.4)   89.0  (17.3) 
[1] The vital capacity (lung capacity) for each subject was measured at screening.
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) 


  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


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

Responsible Party: Merit E. Cudkowicz, MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT00349622     History of Changes
Other Study ID Numbers: U01NS049640-02 ( US NIH Grant/Contract Award Number )
NINDS ( Other Identifier: NINDS )
NINDS CRC ( Other Identifier: NINDS Clinical Research Collaboration )
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