Clinical trial NCT00790582: A Multi-Center Controlled Screening Trial of Safety and Efficacy of Lithium Carbonate in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Official title: A Multi-Center Controlled Screening Trial of Safety and Efficacy of Lithium Carbonate in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Amyotrophic Lateral Sclerosis
Lou Gehrig's Disease
Lithium
Lithium Carbonate
Lead sponsor: Forbes Norris MDA/ALS Research Center
Sponsor class: OTHER
Collaborators: name: Muscular Dystrophy Association
First posted: 2008-11-13
Enrollment: 109
Sex: ALL
Minimum age (years): 21
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Clinical diagnosis of laboratory-supported probable, probable, or definite ALS
* Vital capacity of at least 75% of predicted
* Onset of weakness within 3 years prior to enrollment
* If patients are on riluzole, they must be on a stable dose for at least 30 days prior to baseline visit
* Women of childbearing potential must be surgically sterile or using an effective method of birth control and have a negative pregnancy test
* Willing and able to give informed consent

Exclusion Criteria:

* Diagnosis of other neurodegenerative disease
* Need tracheotomy ventilation or non-invasive ventilation 23 or more hours/day
* Clinically significant history of any unstable medical condition in past 30 days
* History of renal
* History of liver disease
* Current pregnancy or lactation
* Use of lithium within thirty days of enrollment
* Significantly limited mental capacity
* History of recent drug or alcohol abuse
* Use of any investigational drug within 30 days prior to enrollment

Primary outcomes:
measure: Change in Monthly Rate of Decline in ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Score - Revised)
timeFrame: Baseline, Month 1,3,4.5,6,7.5,9,10.5,12,13
Secondary outcomes:
measure: Vital Capacity
timeFrame: Screen, Baseline, Month 1,3,6,9,12
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: Los Angeles
country: United States
facility: UCLA Neuromuscular Research Center
state: California
city: Orange
country: United States
facility: UC Irvine MDA/ALS & Neuromuscular Center
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center
state: California
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Saint Louis
country: United States
facility: Washington University Department of Neurology
state: Missouri
city: Portland
country: United States
facility: Providence ALS Clinic
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania Neurological Institute
state: Pennsylvania
city: Houston
country: United States
facility: Methodist Neurological Institute
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Clinical Neurosciences Center
state: Utah
Study officials:
affiliation: California Pacific Medical Center
name: Robert G Miller, MD
role: STUDY_DIRECTOR
References:
citation: 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.