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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00748501
• Date of registration: 04/09/2008
• Prospective Registration: No
• Primary sponsor: Sangamo Biosciences
• Public title: Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS) ALS
• Scientific title: A Phase 2 Repeat-Dosing Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis
• Date of first enrolment: September 2008
• Target sample size: 45
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00748501
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Ely Benaim, M.D.
• Affiliation: Sangamo Biosciences

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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.

Exclusion Criteria:

- 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.



Age minimum: 18 Years
Age maximum: 85 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: SB-509

Primary Outcome(s)

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). [Time Frame: 11 months]

Secondary Outcome(s)

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. [Time Frame: 11 months]

Secondary ID(s)

SB-509-0801

Source(s) of Monetary Support

Please refer to primary and secondary sponsors