Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS) (SDALS-001)

This study has been completed.
Sponsor:
Collaborators:
ALS Therapy Alliance
State University of New York - Upstate Medical University
Information provided by (Responsible Party):
Nazem Atassi, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT01257581
First received: December 8, 2010
Last updated: December 3, 2014
Last verified: December 2014
Results First Received: March 10, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Amyotrophic Lateral Sclerosis
Interventions: Drug: creatine
Drug: tamoxifen

  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
No text entered.

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
Creatine 30gm

Creatine will be taken as a powder mixed into food or liquid twice a day. Volunteers in this arm will take a total of 30gm of creatine per day for 38 weeks. Volunteers will also take placebo capsules twice a day for 38 weeks.

This is a blinded study, so neither participants nor study staff will know which treatment a volunteer is receiving.

Creatine is a nutritional supplement and is not approved by the U.S. Food and Drug Administration (FDA) for treating ALS.

creatine: creatine monohydrate powder

Tamoxifen 40mg

Tamoxifen will be taken as capsules twice a day. Volunteers in this arm will take a total of 40mg of tamoxifen per day for 38 weeks. Volunteers will also take placebo powder twice a day for 38 weeks.

This is a blinded study, so neither participants nor study staff will know which treatment a volunteer is receiving.

Tamoxifen is approved by the U.S. Food and Drug Administration (FDA) for breast cancer treatment but is not approved for treating ALS.

tamoxifen: Tamoxifen citrate capsules

Tamoxifen 80mg

Tamoxifen will be taken as capsules twice a day. Volunteers in this arm will take a total of 80mg of tamoxifen per day for 38 weeks. Volunteers will also take placebo powder twice a day for 38 weeks.

This is a blinded study, so neither participants nor study staff will know which treatment a volunteer is receiving.

Tamoxifen is approved by the U.S. Food and Drug Administration (FDA) for breast cancer treatment but is not approved for treating ALS.

tamoxifen: Tamoxifen citrate capsules


Participant Flow:   Overall Study
    Creatine 30gm     Tamoxifen 40mg     Tamoxifen 80mg  
STARTED     22     21     17  
COMPLETED     17     18     11  
NOT COMPLETED     5     3     6  
Early Termination                 3                 1                 2  
Death                 2                 2                 4  



  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.
Efficacy outcomes are adjusted for baseline differences in time from symptom onset to diagnosis, site of symptom onset, and VC.

Reporting Groups
  Description
Creatine 30gm Creatine will be taken as a powder mixed into food or liquid twice a day. Volunteers in this arm will take a total of 30gm of creatine per day for 38 weeks. Volunteers will also take placebo capsules twice a day for 38 weeks.
Tamoxifen 40mg Tamoxifen will be taken as capsules twice a day. Volunteers in this arm will take a total of 40mg of tamoxifen per day for 38 weeks. Volunteers will also take placebo powder twice a day for 38 weeks.
Tamoxifen 80mg Tamoxifen will be taken as capsules twice a day. Volunteers in this arm will take a total of 80mg of tamoxifen per day for 38 weeks. Volunteers will also take placebo powder twice a day for 38 weeks.
Total Total of all reporting groups

Baseline Measures
    Creatine 30gm     Tamoxifen 40mg     Tamoxifen 80mg     Total  
Number of Participants  
[units: participants]
  22     21     17     60  
Age  
[units: years]
Mean (Standard Deviation)
  55.5  (11.4)     60.3  (10.6)     56.3  (11.3)     57.4  (11.1)  
Gender  
[units: participants]
       
Female     6     4     9     19  
Male     16     17     8     41  
Ethnicity (NIH/OMB)  
[units: participants]
       
Hispanic or Latino     0     0     0     0  
Not Hispanic or Latino     22     21     17     60  
Unknown or Not Reported     0     0     0     0  
Race (NIH/OMB)  
[units: participants]
       
American Indian or Alaska Native     0     1     0     1  
Asian     0     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0     0  
Black or African American     0     0     0     0  
White     22     20     17     59  
More than one race     0     0     0     0  
Unknown or Not Reported     0     0     0     0  
Region of Enrollment  
[units: participants]
       
United States     22     21     17     60  
Years from Symptom Onset to Screening  
[units: years]
Mean (Standard Deviation)
  2.8  (1.7)     1.9  (1.4)     2.0  (1.0)     2.3  (1.5)  
Years from Diagnosis to Screening  
[units: years]
Mean (Standard Deviation)
  1.3  (0.8)     0.9  (0.8)     1.0  (0.8)     1.1  (0.8)  
Years from Symptom Onset to Diagnosis  
[units: years]
Mean (Standard Deviation)
  1.6  (1.5)     0.9  (0.8)     1.0  (0.8)     1.2  (1.1)  
Baseline ALS Functional Rating Scale - Revised (ALSFRS-R) Total [1]
[units: Score on a scale]
Mean (Standard Deviation)
  35.82  (7.20)     36.19  (6.04)     34.76  (6.56)     35.65  (6.54)  
Baseline VC% Predicted Max [2]
[units: % of predicted max value]
Mean (Standard Deviation)
  90.64  (20.29)     86.90  (13.92)     81.71  (19.93)     86.80  (18.31)  
[1] The ALSFRS-R is a quickly administered (5 min) ordinal rating scale used to determine a subject's assessment of their capability and independence in 12 functional activities. There are 12 questions, graded by the subject 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing.
[2] The vital capacity (VC) (percent of predicted normal) was determined using the slow VC method. Vital Capacity is the maximum amount of air a person can expel from the lungs after a maximum inhalation. A subject's VC depends on their age, sex and height. The value is recorded as a percent of predicted normal.



  Outcome Measures
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1.  Primary:   Change in ALS Functional Rating Scale - Revised (ALSFRS-R)   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

2.  Secondary:   Vital Capacity/Pulmonary Function Testing   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

3.  Secondary:   Tracheostomy-free Survival   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

4.  Secondary:   Dose Adjustments   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

5.  Secondary:   Lab Abnormal Reports by Treatment Assignment   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

6.  Secondary:   Hand Held Dynamometry (HHD) Lower Z-score   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

7.  Secondary:   HHD Lower % Baseline   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

8.  Secondary:   HHD Upper Z-score   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

9.  Secondary:   HHD Upper % Baseline   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

10.  Secondary:   Accurate Test of Limb Isometric Strength (ATLIS) Lower Percentage of Predicted Normal (PPN)   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]

11.  Secondary:   ATLIS Upper Percentage of Predicted Normal (PPN)   [ Time Frame: 38 weeks of treatment followed by a telephone interview at 42 weeks. ]


  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:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Nazem Atassi, MD
Organization: Massachusetts General Hospital
phone: 617-643-1807
e-mail: natassi@partners.org


No publications provided


Responsible Party: Nazem Atassi, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT01257581     History of Changes
Other Study ID Numbers: SDALS-001
Study First Received: December 8, 2010
Results First Received: March 10, 2014
Last Updated: December 3, 2014
Health Authority: United States: Food and Drug Administration