Phase 2 Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen in Amyotrophic Lateral Sclerosis (ALS)
18 Years
N/A
Both
Amyotrophic Lateral Sclerosis
Trial Information
Phase 2 Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen in Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS) is a rare, neurodegenerative disorder that results in
progressive wasting and paralysis of voluntary muscles. It is known that nerve cells called
motor neurons die in the brains and spinal cords of people with amyotrophic lateral
sclerosis (ALS). However, the cause of this cell death is unknown.
In this double blind, randomized, selection design trial, researchers will evaluate the
safety and effectiveness of creatine and tamoxifen in volunteers with ALS. 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. A Selection Design study
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
Selection Design study, each volunteer will take one active study drug (creatine 30gm,
tamoxifen 40mg, or tamoxifen 80mg) and one placebo.
Approximately 60 eligible volunteers with ALS will be recruited from multiple centers in the
US that belong to the Northeast ALS Consortium (NEALS). 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. After screening and
randomization, volunteers will be followed at weeks 4, 10, 18, 28 and week 38. A final
telephone interview will occur at week 42 (off study drug).
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 coumarin anticoagulants (warfarin sodium), rifampin, aminoglutethimide,
medroxyprogesterone, letrozole, or bromocriptine.
- Presence of any of the following clinical conditions: Clinical evidence of unstable
medical or psychiatric illness at the Screening Visit; 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; or Lactating or have a positive serum
pregnancy test at the Screening Visit.
- 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.
Type of Study:
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
Outcome Measure:
Decline in ALSFRS-R
Outcome Description:
Primary efficacy will be assessed by analyzing the mean rate of decline in the ALS Functional Rating Scale-Revised (ALSFRS-R) score over nine months.
Outcome Time Frame:
38 weeks of treatment followed by a telephone interview at 42 weeks.
Safety Issue:
No
Principal Investigator
Nazem Atassi, MD, MMSc
Investigator Role:
Principal Investigator
Investigator Affiliation:
Masaschusetts General Hospital, Boston, MA
Authority:
United States: Food and Drug Administration
Study ID:
SDALS-001
NCT ID:
NCT01257581
Start Date:
March 2011
Completion Date:
February 2013
Related Keywords:
- Amyotrophic Lateral Sclerosis
- ALS
- Lou Gehrig's Disease
- Amyotrophic Lateral Sclerosis
- Creatine
- Tamoxifen
- Selection Design
- Amyotrophic Lateral Sclerosis
- Sclerosis
- Motor Neuron Disease
Name | Location |
|---|---|
| University of Washington Medical Center | Seattle, Washington 98195-6043 |
| Medical College of Wisconsin | Milwaukee, Wisconsin 53226 |
| University of Kansas Medical Center | Kansas City, Kansas 66160-7353 |
| Carolinas Medical Center | Charlotte, North Carolina 28232-2861 |
| Massachusetts General Hospital | Boston, Massachusetts 02114-2617 |
| SUNY Upstate Medical University | Syracuse, New York 13210 |
| University of Massachusetts Medical Center | Worcester, Massachusetts 01605 |
| Washington University at St. Louis | Saint Louis, Missouri 63110 |
| Pennsylvania State University, Hershey Medical Center | Hershey, Pennsylvania 17033 |
