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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00035815
Recruitment Status : Completed
First Posted : May 7, 2002
Results First Posted : February 15, 2013
Last Update Posted : February 15, 2013
Sponsor:
Collaborators:
National Institute of Neurological Disorders and Stroke (NINDS)
ALS Association
Cephalon
Information provided by:
Mayo Clinic

Tracking Information
First Submitted Date  ICMJE May 6, 2002
First Posted Date  ICMJE May 7, 2002
Results First Submitted Date  ICMJE November 17, 2009
Results First Posted Date  ICMJE February 15, 2013
Last Update Posted Date February 15, 2013
Study Start Date  ICMJE June 2003
Actual Primary Completion Date August 2007   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 13, 2013)
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).
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00035815 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 13, 2013)
  • 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.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS) Trial
Official Title  ICMJE Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS)
Brief Summary 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.
Detailed Description

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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • Drug: Insulin like growth factor, type 1
    0.05 mg per kg body weight given subcutaneously twice daily
    Other Name: Mycotrophin
  • Drug: 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.
Study Arms  ICMJE
  • Active Comparator: IGF-1
    Insulin like growth factor, type 1 will be given 0.05 mg per kg body weight subcutaneously twice daily
    Intervention: Drug: Insulin like growth factor, type 1
  • Placebo Comparator: Placebo
    Placebo arm
    Intervention: Drug: Placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 23, 2005)
330
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 2007
Actual Primary Completion Date August 2007   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Puerto Rico,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00035815
Other Study ID Numbers  ICMJE 1461-01
R01NS042759 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Eric Sorenson, M.D., Department of Neurology, Mayo Clinic
Study Sponsor  ICMJE Mayo Clinic
Collaborators  ICMJE
  • National Institute of Neurological Disorders and Stroke (NINDS)
  • ALS Association
  • Cephalon
Investigators  ICMJE
Principal Investigator: Eric Sorenson, M.D. Department of Neurology, Mayo Clinic
PRS Account Mayo Clinic
Verification Date February 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP