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Trial record 3 of 19 for:    Sod1 | Motor Neuron Disease

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

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.
 
ClinicalTrials.gov Identifier: NCT01083667
Recruitment Status : Completed
First Posted : March 10, 2010
Results First Posted : June 19, 2017
Last Update Posted : June 19, 2017
Sponsor:
Collaborator:
Muscular Dystrophy Association
Information provided by (Responsible Party):
Dr. Dale J. Lange, Weill Medical College of Cornell University

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
Enrollment 32
Recruitment Details Patient were recruited from all sites and referrals received from other physicians. Subjects also contacted sites from ClinicalTrials.gov posting
Pre-assignment Details  
Arm/Group Title Pyrimethamine
Hide Arm/Group Description

Open label. Only one arm will receive the intervention.

Pyrimethamine: Open Label, dose escalating,

Period Title: Overall Study
Started 32 [1]
Completed 22 [2]
Not Completed 10
[1]
enrolled
[2]
22 completed to visit 9, the last visit on study.
Arm/Group Title Pyrimethamine
Hide Arm/Group Description

Open label. Only one arm will receive the intervention.

Pyrimethamine: Open Label, dose escalating,

Overall Number of Baseline Participants 32
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants
<=18 years
0
   0.0%
Between 18 and 65 years
31
  96.9%
>=65 years
1
   3.1%
[1]
Measure Description: age of subjects enrolled
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 32 participants
48
(19 to 72)
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants
Female
16
  50.0%
Male
16
  50.0%
[1]
Measure Description: gender of subjects enrolled
Region of Enrollment   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 32 participants
United States 20
Italy 8
Germany 4
[1]
Measure Description: enrollment of subjects in US and Europe
1.Primary Outcome
Title Mean Change in SOD1 CSF
Hide Description Reported change in mean SOD1 CSf from baseline to visit 6 (week 18) and end of study for all subjects who completed the measure
Time Frame baseline, Visit 6 week 18, end of study
Hide Outcome Measure Data
Hide Analysis Population Description
24 subjects completed up to visit 6 and 21 subjects for final study visit. Reported is the change in SOD1 CSF from baseline to week 36.
Arm/Group Title Pyrimethamine
Hide Arm/Group Description:

Open label. Only one arm will receive the intervention.

Pyrimethamine: Open Label, dose escalating,

Overall Number of Participants Analyzed 22
Mean (95% Confidence Interval)
Unit of Measure: ng/ml
6.8
(3.4 to 10.3)
2.Secondary Outcome
Title Appel ALS Score
Hide Description an objective and timed measurement of strength and function of subjects including muscle testing, respiratory function and fine motor function, all summed together for a total value, and is measured at baseline, visit 2, visit 6 and end of study. The scale ranges from 30 in a healthy person to to 164 in a maximally impaired person; an increase in score indicates progression and is expected in disease progression.
Time Frame Week 0, 6, 18, and end of study
Hide Outcome Measure Data
Hide Analysis Population Description
22 subjects completed to visit 9 and had a final score for Appel Score. Reported is the mean change from Baseline to week 36
Arm/Group Title Pyrimethamine
Hide Arm/Group Description:

Open label. Only one arm will receive the intervention.

Pyrimethamine: Open Label, dose escalating,

Overall Number of Participants Analyzed 22
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: units on a scale
18
(12 to 24)
Time Frame AE's were collected over the course of the study and up to 30 days following the last dose taken.
Adverse Event Reporting Description

CTCAE version 4.0 was used to assess the severity of AE's experienced during the study. This uses a grading system (referring to the severity) to assess the AEs. The CTCAE v4.0 displays Grades 1-5 with unique clinical descriptions of severity for each AE based on this general guideline: G1 Mild AE, G2 Moderate AE, G3 Severe or medically significant but not immediately life-threatening, G4 Life-threatening, G5 Death related to AE.

SAE reporting per criteria od clinicaltrials.gov

 
Arm/Group Title Pyrimethamine
Hide Arm/Group Description

Open label. Only one arm will receive the intervention.

Pyrimethamine: Open Label, dose escalating,

All-Cause Mortality
Pyrimethamine
Affected / at Risk (%)
Total   0/32 (0.00%)    
Hide Serious Adverse Events
Pyrimethamine
Affected / at Risk (%) # Events
Total   1/32 (3.13%)    
Infections and infestations   
Pneumonia  1 [1]  1/32 (3.13%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
[1]
Patient admitted to the hospital with pneumonia while on study, course complicated by discovery of a congenital esophageal fistula which was repaired, patient discharged home and continued on the study. The event was not related to study medication.
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Pyrimethamine
Affected / at Risk (%) # Events
Total   14/32 (43.75%)    
Infections and infestations   
Upper respiratory infection  1 [1]  4/32 (12.50%)  4
Metabolism and nutrition disorders   
Nausea  1 [2]  14/32 (43.75%)  14
Diarrhea  1  6/32 (18.75%)  6
weight loss  1 [3]  3/32 (9.38%)  3
decreased appetite  1  2/32 (6.25%)  2
Musculoskeletal and connective tissue disorders   
Pain  1 [4]  5/32 (15.63%)  5
Nervous system disorders   
Headache  1 [5]  13/32 (40.63%)  13
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
[1]
URI infection and Pneumonia
[2]
GI nausea
[3]
greater than 5% was recorded
[4]
Pain following Spinal Tap
[5]
headache following spinal tap
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
Layout table for Results Point of Contact information
Name/Title: Dale Lange
Organization: HSS/WCMC
Phone: 6467978917
EMail: langed@hss.edu
Layout table for additonal information
Responsible Party: Dr. Dale J. Lange, Weill Medical College of Cornell University
ClinicalTrials.gov Identifier: NCT01083667    
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