We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01083667
First Posted: March 10, 2010
Last Update Posted: June 19, 2017
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.
Collaborator:
Muscular Dystrophy Association
Information provided by (Responsible Party):
Dr. Dale J. Lange, Weill Medical College of Cornell University
Results First Submitted: February 17, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Familial Amyotrophic Lateral Sclerosis
Intervention: Drug: Pyrimethamine

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patient were recruited from all sites and referrals received from other physicians. Subjects also contacted sites from ClinicalTrials.gov posting

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
Pyrimethamine

Open label. Only one arm will receive the intervention.

Pyrimethamine: Open Label, dose escalating,


Participant Flow:   Overall Study
    Pyrimethamine
STARTED   32 [1] 
COMPLETED   22 [2] 
NOT COMPLETED   10 
[1] enrolled
[2] 22 completed to visit 9, the last visit on study.



  Baseline Characteristics
  Hide Baseline Characteristics

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
Pyrimethamine

Open label. Only one arm will receive the intervention.

Pyrimethamine: Open Label, dose escalating,


Baseline Measures
   Pyrimethamine 
Overall Participants Analyzed 
[Units: Participants]
 32 
Age [1] 
[Units: Participants]
Count of Participants
 
<=18 years      0   0.0% 
Between 18 and 65 years      31  96.9% 
>=65 years      1   3.1% 
[1] age of subjects enrolled
Age 
[Units: Years]
Mean (Full Range)
 48 
 (19 to 72) 
Sex: Female, Male [1] 
[Units: Participants]
Count of Participants
 
Female      16  50.0% 
Male      16  50.0% 
[1] gender of subjects enrolled
Region of Enrollment [1] 
[Units: Participants]
 
United States   20 
Italy   8 
Germany   4 
[1] enrollment of subjects in US and Europe


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Mean Change in SOD1 CSF   [ Time Frame: baseline, Visit 6 week 18, end of study ]

2.  Secondary:   Appel ALS Score   [ Time Frame: Week 0, 6, 18, and end of study ]


  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
No text entered.


  More Information
  Hide More Information

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: Dale Lange
Organization: HSS/WCMC
phone: 6467978917
e-mail: langed@hss.edu



Responsible Party: Dr. Dale J. Lange, Weill Medical College of Cornell University
ClinicalTrials.gov Identifier: NCT01083667     History of Changes
Other Study ID Numbers: 0903010259
First Submitted: December 17, 2009
First Posted: March 10, 2010
Results First Submitted: February 17, 2017
Results First Posted: June 19, 2017
Last Update Posted: June 19, 2017