Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS) Trial
Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS) Trial
The purpose of this multicenter study is to determine if insulin-like growth factor-1 (IGF-I)
 slows the progressive weakness in amyotrophic lateral sclerosis (ALS) patients. Study
 participants will be followed for 2 years once enrolled. They will receive either placebo or
 the active IGF-I. Examinations will take place at approximately 6-month intervals.
The objective of this trial was to determine whether IGF-1 (MyotrophinTM) slows progression
 of weakness in amyotrophic lateral sclerosis (ALS). Three hundred thirty patients with ALS
 from 20 medical centers participated in this double blind, placebo-controlled two-year study.
 Half the patients received IGF-1 and the other half received placebo. The drug will be
 administered twice a day.

 ALS is a neurodegenerative disorder that causes progressive muscle weakness and loss of motor
 neurons. IGF-1 is a neurotrophic factor essential for normal development of the nervous
 system and shows protection of motor neurons in animal models and cell culture systems. It is
 thought to block cell death pathways and promote muscle re-innervation and axonal growth and
 regeneration.
Inclusion Criteria

 Patients entering this study:

 -  Are between the ages of 18-80 years old.

 -  Legal residents of the United States or Canada.

 -  Have a history of a chronic onset of a progressive motor weakness of less than 24
 months duration.

 -  Fulfill El Escorial criteria of probable or definite ALS.

 -  If female, are surgically sterile, two years postmenopausal, or if of child-bearing
 potential, must be using a medically acceptable method of birth control and agree to
 continue use of this method for the duration of the study. Acceptable methods include
 a barrier method with spermicide, oral contraceptives (normal doses are acceptable;
 low dose oral contraceptives or contraceptive implants must be used with a barrier
 method), intrauterine device (IUD), or abstinence. Have a negative pregnancy test.

 -  Are able to comply with protocol requirements.

 -  Can provide written informed consent.

 -  Have a manual muscle testing score of less than 8.

 -  Have a forced vital capacity by pulmonary function testing *60% predicted.

 Exclusion Criteria:

 Patients entering this study will not:

 -  Have any of the following conditions:renal disease (Creatine > 2.0) or other active
 systemic disease

 -  Have any clinically significant abnormalities on the prestudy laboratory evaluation,
 physical examination, ECG, chest x-ray or ophthalmologic exam.

 -  Have any clinically significant medical condition (e.g., within six months of
 baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure)
 that, in the opinion of the investigator, would compromise the safety of patient.

 -  Have Type I or Type II diabetes.

 -  Have a history of cancer including melanoma with the exception of localized skin
 cancers (with no evidence of metastasis, significant invasion, or re-occurrence within
 three years of baseline) and carcinoma in-situ of the cervix (women only).

 -  Have used an investigational drug within 30 days of baseline visit.

 -  Have had a tracheostomy.

 -  Have a Beck's Depression Inventory score * 12.

 -  Have legal residency outside of the United States or Canada.

 -  Be pregnant or breast-feeding.