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Phase 2 Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen in Amyotrophic Lateral Sclerosis (ALS) |
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Study Focus:
The purpose of this study is to evaluate the safety and effectiveness of creatine and tamoxifen in volunteers with ALS. |
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Disease:
Amyotrophic Lateral Sclerosis (ALS), Sporadic ALS, Familial ALS |
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Study Category:
Drug Trial |
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Study Status:
Closed |
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Study Results:
preliminary trial results in the analysis process, expected spring 2013 |
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Phase:
Phase II |
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Type:
Interventional Trial
with active agents/drugs only |
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Funding Source:
ALS Therapy Alliance (ATA) |
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Study Chair(s)/Principal Investigator(s):
Nazem Atassi, MD MMSc (Massachusetts General Hospital) |
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Clinicaltrials.gov ID:
NCT01257581 |
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Study ID:
SDALS-001 |
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NEALS Affiliated?
Yes
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Study Summary:
There are a large number of potential drugs that may improve the survival or slow down the disease progression in people with ALS. The current strategy is to test one drug at a time against placebo. "Selection Design" is a different type of study design that uses multiple drugs to screen against each other and picks the winner to take to a larger study. This design can speed the search for effective drugs to treat ALS. In this study, each volunteer will take one active study drug (creatine 30mg, tamoxifen 40mg, or tamoxifen 80mg) and one placebo.
Approximately 60 eligible volunteers with ALS will be recruited from multiple centers in the US. Volunteers will be randomly assigned equally to the three treatment arms: creatine 30gm/day, tamoxifen 40mg/day and tamoxifen 80mg/day. Volunteers will take study treatment for 38 weeks. Volunteers will have 7 in-person visits and 4 telephone visits during the study. |
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Participant Duration:
38 weeks on drug; 42 weeks total participation. |
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# of Subjects:
60 |
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Enrollment Start Date:
03/01/2011 |
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Posting Last Modified Date:
03/01/2013 |
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Date Study Added to alscortium.org:
10/15/2011 |
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| Eligibility Criteria |
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Gender:
Male & Female |
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Minimum Age:
18 |
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Min Vital Capacity (% predicted normal):
50% |
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Time since Symptom Onset:
Not applicable |
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Time since Diagnosis:
Less than 36 months |
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Can participants use Riluzole?
Yes |
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Coordinating Center
Contact Information |
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| Julie Berkley |
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| jberkley@partners.org |
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| 617-643-7429 |
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| Massachusetts General Hospital |
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149 13th Street RM 2274
Boston, Massachusetts 02129
UNITED STATES |
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Other Eligibility Criteria:
Inclusion Criteria:
• Familial or sporadic ALS.
• Disease duration from diagnosis no greater than 36 months at Screening Visit.
• Aged 18 years or older.
• Capable of providing informed consent and complying with trial procedures.
• Vital capacity (VC) equal to or more than 50% predicted normal value for gender, height and age at the Screening Visit.
• Not taking, or on a stable dose of riluzole (50mg bid) for at least 30 days prior to the Screening Visit.
• Women must not be able to become pregnant for the duration of the study (e.g., post menopausal for at least one year, surgically sterile, or practicing adequate birth control methods) for the duration of the study. Women of childbearing potential must have a negative serum pregnancy test at the Screening Visit and be non-lactating.
Exclusion Criteria:
• History of known sensitivity or intolerability to creatine monohydrate or tamoxifen citrate or to any other related compound.
• Prior exposure to creatine or tamoxifen within 30 days of the Screening Visit.
• Exposure to any investigational agent within 30 days of the Screening Visit.
• Use of any prohibited medication including but not limited to coumadin , rifampin, aminoglutethimide, medroxyprogesterone, letrozole, bromocriptine, Aminoglutethimide, Anastrazole, Bromocriptine, Colchicines, Conivaptan, Dabigatran etexilate, Duloxetine, Everolimus, Fluconazole, Rifamycin, Silodosin,Topotecan, or Vitamin K antagonists (warfarin).
• Presence of any of the following clinical conditions at the Screening Visit: Clinical evidence of unstable medical or psychiatric illness; Screening AST > 3 times the upper limit of normal or serum creatinine > 1.5 mg/dl (133 umol/L); Permanent assisted ventilation or mechanical ventilation; tracheostomy; or Lactating or have a positive serum pregnancy test.
• History of any of the following: blood clots including deep vein thrombosis, pulmonary embolism, and stroke, cataracts, renal problems, endometrial cancer, uterine sarcoma, or diabetes mellitus. |
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Site Contact Information (Please click to show/hide) |
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| Kansas |
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University of Kansas Medical Center
Department of Neurology, MSN 2012 3599 Rainbow Blvd
Kansas City, Kansas 66160
UNITED STATES |
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| Massachusetts |
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Massachusetts General Hospital
149 13th St, Room 2266
Charlestown, Massachusetts 02114
UNITED STATES |
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University of Massachusetts Medical Center
Department of Neurology / University Campus Room S5-710 55 Lake Ave. North
Worcester, Massachusetts 01655
UNITED STATES | |
| Missouri |
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Washington University at St. Louis
660 S. Euclid Ave. Box 8111 Neurology
St. Louis, Missouri 63110
UNITED STATES |
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| New York |
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SUNY Upstate Medical University
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750 East Adams Street 814 Jacobsen Hall
Syracuse, New York 13210
UNITED STATES |
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| North Carolina |
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Carolinas Medical Center
/ joanne.nemeth@carolinashealthcare.org
Neuroscience and Spine Institute 1010 Edgehill Street, North
Charlotte, North Carolina 28207
UNITED STATES |
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| Ohio |
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Cleveland Clinic Foundation
9500 Euclid Ave JJ36 Neurology
Cleveland, Ohio 44195
UNITED STATES |
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| Pennsylvania |
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Pennsylvania State University, Hershey Medical Center
Department of Neurology, EC037 30 Hope Drive
Hershey, Pennsylvania 17033
UNITED STATES |
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| Washington |
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University of Washington Medical Center
RR638 Health Sciences Building 1959 NE Pacific Street
Seattle, Washington 98195
UNITED STATES |
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| Wisconsin |
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Medical College of Wisconsin
Froedtert Memorial Lutheran Hospital 9200 W Wisconsin Ave.
Milwaukee, Wisconsin 53208
UNITED STATES |
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Familial ALS- ALS cases are inherited from 2 or more family members, affects 5-10% of ALS cases. Sporadic ALS- disease occurs at random with no clearly associated risk factors; most common form of ALS, affects approximately 90-95% ALS cases. Interventional trial- study where exposure, for example a drug, is assigned; used to determine the effectiveness of a treatment or intervention. With drugs and a placebo- One group gets the active treatment, the other gets the placebo. Everything else is held the same between the two groups, so that any difference in their outcome can be attributed to the active treatment. Observational study- study in which patients are observed and where no treatment is given; researcher has no control over the experiment because they are done in a more natural setting than an interventional trial. Trial Phase- objectives are different for each phase of a trial. - Phase I, drug is given to a small number of people to see if it is safe.
- Phase II begins to look at effectiveness of the drug.
- Phase III is considered the definitive test of whether a drug is effective, held at multiple research sites and much larger than the previous phases.
Vital Capacity- the maximum amount of air that can be expelled from the lungs after a maximum drawing of air into the lungs, measured by a spirometer and then scored as a percentage. |
