NCT00035815

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
330

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Jun 2003

Longer than P75 for phase_3

Geographic Reach
2 countries

20 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 6, 2002

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 7, 2002

Completed
1.1 years until next milestone

Study Start

First participant enrolled

June 1, 2003

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2007

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2007

Completed
5.2 years until next milestone

Results Posted

Study results publicly available

February 15, 2013

Completed
Last Updated

February 15, 2013

Status Verified

February 1, 2013

Enrollment Period

4.2 years

First QC Date

May 6, 2002

Results QC Date

November 17, 2009

Last Update Submit

February 13, 2013

Conditions

Keywords

amyotrophic lateral sclerosisALSprogressive weaknessinsulin-like growth factor-1IGF-IMyotrophin

Outcome Measures

Primary Outcomes (1)

  • Rate of Change in Composite Manual Muscle Testing (MMT) Score

    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).

    Baseline and 24 months

Secondary Outcomes (2)

  • Number of Participants Alive and Tracheostomy-free at 24 Months

    baseline to 24 months

  • Rate of Change in ALS Functional Rating Scale.

    Baseline and 24 months

Study Arms (2)

IGF-1

ACTIVE COMPARATOR

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

Placebo

PLACEBO COMPARATOR

Placebo arm

Drug: Placebo

Interventions

0.05 mg per kg body weight given subcutaneously twice daily

Also known as: Mycotrophin
IGF-1

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.

Placebo

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Mayo Clinic in Scottsdale

Scottsdale, Arizona, 85259, United States

Location

California Pacific Medical Center

San Francisco, California, 94115, United States

Location

Mayo Clinic in Jacksonville

Jacksonville, Florida, 32224, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

University of Michigan Medical Center

Ann Arbor, Michigan, 48109, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Hennepin County Medical Center

Minneapolis, Minnesota, 55404, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of Mississippi

Jackson, Mississippi, 39216, United States

Location

Beth Israel Medical Center

New York, New York, 10003, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45219, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

University of Pennsylvania, Pennsylvania Hospital

Philadelphia, Pennsylvania, 19107, United States

Location

Methodist Hospital

Houston, Texas, 77030, United States

Location

West Virginia University

Morgantown, West Virginia, 26506, United States

Location

Froedtert and Medical College Clinics

Milwaukee, Wisconsin, 53226, United States

Location

University of Puerto Rico

San Juan, 00935, Puerto Rico

Location

Related Publications (2)

  • Howe CL, Bergstrom RA, Horazdovsky BF. Subcutaneous IGF-1 is not beneficial in 2-year ALS trial. Neurology. 2009 Oct 13;73(15):1247; author reply 1247-8. doi: 10.1212/WNL.0b013e3181b26ae6. No abstract available.

    PMID: 19822878BACKGROUND
  • Sorenson EJ, Windbank AJ, Mandrekar JN, Bamlet WR, Appel SH, Armon C, Barkhaus PE, Bosch P, Boylan K, David WS, Feldman E, Glass J, Gutmann L, Katz J, King W, Luciano CA, McCluskey LF, Nash S, Newman DS, Pascuzzi RM, Pioro E, Sams LJ, Scelsa S, Simpson EP, Subramony SH, Tiryaki E, Thornton CA. Subcutaneous IGF-1 is not beneficial in 2-year ALS trial. Neurology. 2008 Nov 25;71(22):1770-5. doi: 10.1212/01.wnl.0000335970.78664.36.

Related Links

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Insulin-Like Peptides

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Intercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Results Point of Contact

Title
Eric J. Sorenson, MD
Organization
Mayo Clinic

Study Officials

  • Eric Sorenson, M.D.

    Department of Neurology, Mayo Clinic

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER

Study Record Dates

First Submitted

May 6, 2002

First Posted

May 7, 2002

Study Start

June 1, 2003

Primary Completion

August 1, 2007

Study Completion

December 1, 2007

Last Updated

February 15, 2013

Results First Posted

February 15, 2013

Record last verified: 2013-02

Locations