Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS) Trial
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|ClinicalTrials.gov Identifier: NCT00035815|
Recruitment Status : Completed
First Posted : May 7, 2002
Results First Posted : February 15, 2013
Last Update Posted : February 15, 2013
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Insulin like growth factor, type 1 Drug: Placebo||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||330 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS)|
|Study Start Date :||June 2003|
|Actual Primary Completion Date :||August 2007|
|Actual Study Completion Date :||December 2007|
Active Comparator: IGF-1
Insulin like growth factor, type 1 will be given 0.05 mg per kg body weight subcutaneously twice daily
Drug: Insulin like growth factor, type 1
0.05 mg per kg body weight given subcutaneously twice daily
Other Name: Mycotrophin
Placebo Comparator: Placebo
The placebo represented the inert suspension vehicle for the IGF-1. It was given as equal volume as the active drug based upon body weight, subcutaneously twice daily.
- Rate of Change in Composite Manual Muscle Testing (MMT) Score [ Time Frame: Baseline and 24 months ]The primary outcome measure was the rate of change in the MMT score. MMT involved the examination of 34 muscle groups with standard positioning. The final MMT score represented an average of the 34 muscles examined, and ranged from 10 to 0(10 normal strength, 0 paralyzed). The individual muscle score was based on the medical research council (MRC) grading scale (1-5) modified to a 10 point system corresponding to the MRC modifications of plus and minus (5, 5-,4+,4,4-,3+,3, 3-,2,1,0; with 5 being normal strength and 0 paralyzed).
- Number of Participants Alive and Tracheostomy-free at 24 Months [ Time Frame: baseline to 24 months ]Patients who elected to proceed to tracheostomy were assessed the month of their procedure. Subjects who continuously utilized non-invasive positive pressure ventilation for greater than 10 days were assessed as being ventilator-dependent on the first day they began continuous Non Invasive Positive Pressure Ventilation (NIPPV). All subjects were followed for the 24 month time period.
- Rate of Change in ALS Functional Rating Scale. [ Time Frame: Baseline and 24 months ]The final secondary outcome measure was the rate of change in the ALS Functional Rating Scale (ALSFRS-r) score. The ALSFRS-r was completed at each visit (randomization and then at 3, 6, 12, 18 and 24 months post-randomization). This is a scale from 0 to 48 assessing functional impairment in 12 clinically relevant areas in ALS. Forty-eight is normal with full function and zero is total loss of function in all clinical functions. As with the MMT scores a score of 0 was imputed on the day of death. Analysis of the ALSFRS-r scores as a secondary outcome was performed in similar manner as MMT score.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00035815
|Principal Investigator:||Eric Sorenson, M.D.||Department of Neurology, Mayo Clinic|