Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS) Trial

Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS)

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.