Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

NCT00748501 | Completed Phase 2

• 1 other identifier: SB-509-0801
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 6

Brief Summary

The purpose of the study is to evaluate the effects of the investigational drug, SB-509 on progression of the disease in subjects with ALS

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic Lateral Sclerosis,; ALS,; Lou Gehrig's Disease

Outcome Measures

Primary Outcomes (1)

• To evaluate the effect of SB-509 on progression of the disease in subjects with ALS, as measured by the ALS Functional Rating Scale -Revised (ALSFRS-R).

  11 months

Secondary Outcomes (1)

• To evaluate a) the effect of SB-509 on Forced Vital Capacity, Neurophysiologic Index, Manual Muscle Test, and survival. b) safety and tolerability of SB-509 in ALS. c) stem cell mobilization in subjects with ALS receiving SB-509.

  11 months

Study Arms (2)

Cohort 1

ACTIVE COMPARATOR

SB-509 drug administration via IM injection of neck, arms, and legs

Drug: SB-509

Cohort 2

ACTIVE COMPARATOR

SB-509 drug administration via IM injection of legs

Drug: SB-509

Interventions

SB-509 DRUG

Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90.

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female between the ages of 18 and 85 with clinical diagnosis of ALS
• Forced Vital Capacity (FVC) \> 60% of predicted
• Less than 3 years of ALS since the onset of the first symptom with clinical evidence of limb muscle atrophy and weakness.
• Subjects taking Riluzole must have been at a stable dose for at least 30 days with no evidence of toxicity
• Female of childbearing potential and male of child-creating potential must agree to use a medically acceptable physical barrier (condom, diaphragm, and cervical cap) through the treatment phase and for at least 30 days after the last study treatment.

You may not qualify if:

• Women who are pregnant or currently breast-feeding
• Dependent upon invasive or non-invasive artificial ventilation
• Patients with bulbar onset ALS or with other active neuromuscular/ neurodegenerative diseases.
• Type 1 or Type 2 diabetes.
• Evidence of chronic or active heart, liver, kidney, or lung diseases, or Age-related macular degeneration.
• Current or history of known immune or immunodeficiency disorders
• Patients with cognitive impairment with significant decision making incapacity, or major depression, or schizophrenia, or dementia (e.g. Alzheimer's disease).
• Malignancy or history of malignancy, except it has been in complete remission for at least 5 years
• Pre-cancerous conditions (e.g. Barrett's Esophagus, dysplasias) or benign tumors which have the potential for significant growth due to VEGF stimulation.

Sponsors & Collaborators

• Sangamo Therapeutics: lead

Study Sites (6)

Coordinated Clinical Research

La Jolla, California, 92037, United States

Location

University of California, Irvine; MDA ALS and Neuromuscular Center,

Orange, California, 92868, United States

Location

California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center

San Francisco, California, 94115, United States

Location

The University of Kansas Medical Center (KU)

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Ely Benaim, M.D.

  Sangamo Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 4, 2008
• First Posted: September 8, 2008
• Study Start: September 1, 2008
• Primary Completion: June 1, 2010
• Study Completion: June 1, 2010
• Last Updated: November 1, 2012

Record last verified: 2012-10

Locations

US (6)