Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Muscular Dystrophy Association | OTHER |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a Phase II screening study of lithium carbonate in ALS. The purpose of this study is to find out if lithium carbonate is safe to be used in people with ALS and if it can slow the progression of the disease. Since there is no placebo in this study, all patients will be taking lithium carbonate.
This is a Phase II Screening study. There is no placebo (inactive or 'fake' drug) in this study, meaning that all participants will be taking lithium carbonate. The purpose of this study is to find out if lithium carbonate is safe to be used in people with ALS and if it can slow the progression of the disease.
A recent article was published in a highly regarded medical journal that showed a positive effect of lithium carbonate on an ALS type mouse. The researchers then studied a very small number of people with ALS, giving 16 people lithium carbonate with riluzole and giving 28 people only riluzole. The people who took lithium remained stronger for a considerably longer period of time. However, the study was very small and we cannot really tell if lithium works unless a larger study is performed. It is not well understood why lithium carbonate might be helpful but it is believed that it may play a role in protecting the motor nerves from the damage of ALS.
If you choose to participate, you will need to go to your study clinic for research study visits 7 times in one year and you will have 4 telephone interviews during that time. These visits and phone calls could take up to 17 hours in total.
Caution: Lithium is an FDA approved drug used for some psychiatric disorders. It is not FDA approved for ALS. Lithium has many potentially serious side effects and must only be taken under close supervision of your physician.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| lithium carbonate | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| lithium carbonate | Drug | The dosage of lithium carbonate will be adjusted for each individual. The maximum dose is 450mg/day. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Monthly Rate of Decline in ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Score - Revised) | This questionnaire has 12 questions about the patient's ability to complete certain daily activities. There are three questions each about the mouth area, arms, legs, and breathing. A normal score is 48 with each question scored at 0 (worst) to 4 (normal function). In research studies, the change in slope is measured in number of points changed per month. Maximum possible=48 (normal) Minimum=0 (severe dysfunction) | Baseline, Month 1,3,4.5,6,7.5,9,10.5,12,13 |
| Measure | Description | Time Frame |
|---|---|---|
| Vital Capacity | Vital Capacity (VC) is a breathing test in which the patient is asked to take a deep breath and then blow out all of the breath through a tube. The volume is measured in number of liters. It is converted to a percent predicted for the patient's age, height, and gender. | Screen, Baseline, Month 1,3,6,9,12 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Robert G Miller, MD | California Pacific Medical Center | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic | Scottsdale | Arizona | 85259 | United States | ||
| UCLA Neuromuscular Research Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 18250315 | Background | Fornai F, Longone P, Cafaro L, Kastsiuchenka O, Ferrucci M, Manca ML, Lazzeri G, Spalloni A, Bellio N, Lenzi P, Modugno N, Siciliano G, Isidoro C, Murri L, Ruggieri S, Paparelli A. Lithium delays progression of amyotrophic lateral sclerosis. Proc Natl Acad Sci U S A. 2008 Feb 12;105(6):2052-7. doi: 10.1073/pnas.0708022105. Epub 2008 Feb 4. |
Not provided
Not provided
This was an open label single-arm Phase II screening trail. All enrolled subjects (n=109) were treated. Their results were compared with placebo subjects from an historical database of ALS placebo patients from recent studies (n=249). Thus the number enrolled (n=109) does not equal the total number of subjects (n=358).
Enrolled subjects started lithium carbonate at 150 mg twice daily. If subjects did not tolerate a dose it was tapered by 150 mg increments to the maximum tolerated dosage.
Patients were enrolled between May 2008 and February 2009. Inclusion criteria were a diagnosis of probable or definite ALS (12), age 21-85 years, forced vital capacity (FVC) greater than or equal to 75% of predicted, and onset of weakness within 3 years. Patients taking riluzole were on a stable dose for at least 30 days.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Treatment | 109 subjects were assigned to treatment with lithium carbonate |
| FG001 | Control | 249 placebo subjects from previous clinical trials |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Los Angeles |
| California |
| 90095 |
| United States |
| UC Irvine MDA/ALS & Neuromuscular Center | Orange | California | 92868 | United States |
| California Pacific Medical Center | San Francisco | California | 94115 | United States |
| Kansas University Medical Center | Kansas City | Kansas | 66160 | United States |
| Washington University Department of Neurology | St Louis | Missouri | 63110 | United States |
| Providence ALS Clinic | Portland | Oregon | 97213 | United States |
| University of Pennsylvania Neurological Institute | Philadelphia | Pennsylvania | 19107 | United States |
| Methodist Neurological Institute | Houston | Texas | 77030 | United States |
| University of Utah Clinical Neurosciences Center | Salt Lake City | Utah | 84132 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Lithium Carbonate | |
| BG001 | Historical Controls | |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Number | participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Monthly Rate of Decline in ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Score - Revised) | This questionnaire has 12 questions about the patient's ability to complete certain daily activities. There are three questions each about the mouth area, arms, legs, and breathing. A normal score is 48 with each question scored at 0 (worst) to 4 (normal function). In research studies, the change in slope is measured in number of points changed per month. Maximum possible=48 (normal) Minimum=0 (severe dysfunction) | Posted | Mean | 95% Confidence Interval | points per month | Baseline, Month 1,3,4.5,6,7.5,9,10.5,12,13 |
|
|
| |||||||||||||||||||||||||||||
| Secondary | Vital Capacity | Vital Capacity (VC) is a breathing test in which the patient is asked to take a deep breath and then blow out all of the breath through a tube. The volume is measured in number of liters. It is converted to a percent predicted for the patient's age, height, and gender. | Posted | Mean | 95% Confidence Interval | Decline in the percent predicted/month | Screen, Baseline, Month 1,3,6,9,12 |
|
|
Adverse events were recorded for the duration of the trial (1 year).
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Lithium Carbonate | Treated Patients | 14 | 109 | 62 | 109 | 99 | 109 |
| EG001 | Historical Controls | Placebo Patients | 44 | 249 | 91 | 249 | 175 | 249 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Respiratory | Respiratory, thoracic and mediastinal disorders |
| |||
| Gastrointestinal | Gastrointestinal disorders |
| |||
| Other | General disorders |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastrointestinal | Gastrointestinal disorders |
| |||
| Cardiovascular | Cardiac disorders |
| |||
| Dermatological | Skin and subcutaneous tissue disorders |
| |||
| Ears, Nose, Throat | Ear and labyrinth disorders |
| |||
| Endocrine/Metabolic | Endocrine disorders |
| |||
| Genitourinary | Reproductive system and breast disorders |
| |||
| Musculoskeletal | Musculoskeletal and connective tissue disorders |
| |||
| Neurological | Nervous system disorders |
| |||
| Other | General disorders |
| |||
| Falls | Nervous system disorders |
| |||
| Psychological | Psychiatric disorders |
| |||
| Respiratory | Respiratory, thoracic and mediastinal disorders |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Giovanna Kushner | Forbes Norris MDA/ALS Center | 415-600-3983 | kushneg@cpmcri.org |
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D016651 | Lithium Carbonate |
| ID | Term |
|---|---|
| D002254 | Carbonates |
| D000468 | Alkalies |
| D007287 | Inorganic Chemicals |
| D002255 | Carbonic Acid |
| D017554 | Carbon Compounds, Inorganic |
| D018020 | Lithium Compounds |
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| 18 to 75 years |
|
| Male |
|
|