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Trial record 68 of 488 for:    ALS (Amyotrophic Lateral Sclerosis)

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
Sponsor:
Collaborators:
National Institute of Neurological Disorders and Stroke (NINDS)
ALS Association
Cephalon
Information provided by:
Mayo Clinic

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Amyotrophic Lateral Sclerosis
Interventions Drug: Insulin like growth factor, type 1
Drug: Placebo
Enrollment 330
Recruitment Details Subjects recruited from 20 medical centers from June 2003 to August 2005.
Pre-assignment Details Patients were randomized and initiated on treatment at the time of enrollment.
Arm/Group Title IGF-1 Placebo
Hide Arm/Group Description The insulin-like growth factor type 1 (IGF-1) arm was the active treatment group. They received 0.05 mg/kg body weight administered subcutaneously twice daily. Placebo group received the equal volume (based on kg of body weight) of the inert suspension vehicle in which the IGF-1 was suspended.
Period Title: Overall Study
Started 167 [1] 163 [1]
Completed 150 152
Not Completed 17 11
Reason Not Completed
Withdrawal by Subject             17             11
[1]
Enrollment began June 2003, accrual was for 2 years, follow-up period was 2 years.
Arm/Group Title IGF-1 Placebo Total
Hide Arm/Group Description The IGF-1 arm was the active treatment group. They received 0.05 mg/kg body weight administered subcutaneously twice daily. Placebo group received the equal volume (based on kg of body weight) of the inert suspension vehicle in which the IGF-1 was suspended. Total of all reporting groups
Overall Number of Baseline Participants 167 163 330
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 167 participants 163 participants 330 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
135
  80.8%
129
  79.1%
264
  80.0%
>=65 years
32
  19.2%
34
  20.9%
66
  20.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 167 participants 163 participants 330 participants
53.9  (12.2) 54.8  (11.2) 54.4  (11.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 167 participants 163 participants 330 participants
Female
57
  34.1%
63
  38.7%
120
  36.4%
Male
110
  65.9%
100
  61.3%
210
  63.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 167 participants 163 participants 330 participants
167 163 330
1.Primary Outcome
Title Rate of Change in Composite Manual Muscle Testing (MMT) Score
Hide Description 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).
Time Frame Baseline and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title IGF-1 Placebo
Hide Arm/Group Description:
The IGF-1 arm was the active treatment group. They received 0.05 mg/kg body weight administered subcutaneously twice daily.
Placebo group received the equal volume (based on kg of body weight) of the inert suspension vehicle in which the IGF-1 was suspended.
Overall Number of Participants Analyzed 167 163
Mean (Standard Deviation)
Unit of Measure: MMT units per month
0.44  (0.57) 0.39  (0.39)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection IGF-1, Placebo
Comments Analysis of MMT was calculated as a ratio of change from baseline to last follow-up to time to duration until last follow-up. For the patients that died during the study period, the last follow-up time was considered as the time of death with a zero score for MMT measurement. Analysis was performed using intention to treat approach. Comparison of rate of change in MMT scores between the placebo and IGF-1 group was made using two sample t-test or Wilcoxon rank sum test as appropriate.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.529
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
2.Secondary Outcome
Title Number of Participants Alive and Tracheostomy-free at 24 Months
Hide Description 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.
Time Frame baseline to 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title IGF-1 Placebo
Hide Arm/Group Description:
The IGF-1 arm was the active treatment group. They received 0.05 mg/kg body weight administered subcutaneously twice daily.
Placebo group received the equal volume (based on kg of body weight) of the inert suspension vehicle in which the IGF-1 was suspended.
Overall Number of Participants Analyzed 167 163
Measure Type: Number
Unit of Measure: participants
93 90
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection IGF-1, Placebo
Comments Patients who elected to proceed to tracheostomy were assessed on the day of their procedure. Subjects who continuously utilized NIPPV for greater than 10 days were assessed as being ventilator-dependent on the first day they began continuous NIPPV. Survival between groups was compared using the Cox-proportional Hazards model.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.415
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Cox Proportional Hazard
Estimated Value 1.04
Confidence Interval (2-Sided) 95%
0.77 to 1.4
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Rate of Change in ALS Functional Rating Scale.
Hide Description 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.
Time Frame Baseline and 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title IGF-1 Placebo
Hide Arm/Group Description:
The IGF-1 arm was the active treatment group. They received 0.05 mg/kg body weight administered subcutaneously twice daily.
Placebo group received the equal volume (based on kg of body weight) of the inert suspension vehicle in which the IGF-1 was suspended.
Overall Number of Participants Analyzed 167 163
Mean (Standard Deviation)
Unit of Measure: Units on a scale per month
2.5  (3.2) 2.2  (2.1)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection IGF-1, Placebo
Comments The final secondary outcome measure was the rate of change in the ALSFRS-r score. The ALSFRS-r was completed at each visit (randomization and then at 3, 6, 12, 18 and 24 months post-randomization). 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.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.321
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Time Frame 2 years of follow-up
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title IGF-1 Placebo
Hide Arm/Group Description The IGF-1 arm was the active treatment group. They received 0.05 mg/kg body weight administered subcutaneously twice daily. Placebo group received the equal volume (based on kg of body weight) of the inert suspension vehicle in which the IGF-1 was suspended.
All-Cause Mortality
IGF-1 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
IGF-1 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   18/167 (10.78%)      12/163 (7.36%)    
Vascular disorders     
Thrombotic events  [1]  18/167 (10.78%)  18 12/163 (7.36%)  12
Indicates events were collected by systematic assessment
[1]
Serious thrombotic events
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 3%
IGF-1 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   103/167 (61.68%)      92/163 (56.44%)    
Endocrine disorders     
Hypoglycemia   21/167 (12.57%)  21 9/163 (5.52%)  9
Eye disorders     
Change in visual acuity   23/167 (13.77%)  23 20/163 (12.27%)  20
Hepatobiliary disorders     
Hepatotoxicity   14/167 (8.38%)  14 8/163 (4.91%)  8
Metabolism and nutrition disorders     
Abnormal blood chemistries (other than liver)   10/167 (5.99%)  10 13/163 (7.98%)  13
Nervous system disorders     
Headache   22/167 (13.17%)  22 22/163 (13.50%)  22
Psychiatric disorders     
Depression   103/167 (61.68%)  103 92/163 (56.44%)  92
Respiratory, thoracic and mediastinal disorders     
Respiratory failure   55/167 (32.93%)  55 50/163 (30.67%)  50
Skin and subcutaneous tissue disorders     
Site reactions   88/167 (52.69%)  88 92/163 (56.44%)  92
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Eric J. Sorenson, MD
Organization: Mayo Clinic
Phone: 507 538-1037
Responsible Party: Eric Sorenson, M.D., Department of Neurology, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00035815     History of Changes
Other Study ID Numbers: 1461-01
R01NS042759 ( U.S. NIH Grant/Contract )
First Submitted: May 6, 2002
First Posted: May 7, 2002
Results First Submitted: November 17, 2009
Results First Posted: February 15, 2013
Last Update Posted: February 15, 2013