GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)

• Status: Completed
• Trial end date: 2014-07-01
• Target enrollment: 0
• Participant gender: All

Summary

GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system. Neurological diseases are multisystem, multifactorial, and single target drugs are ineffective. Genervon's Master Regulators play a significant role in embryonic/fetal nervous system development and are potent disease modification drug candidates modulating many pathways including inflammation, apoptotic, and hypoxia. The study drug is an regulatory peptide with a sequence identical to one of the active sites of human Motoneuronotrophic Factor and is manufactured by solid phase synthesis. Pre-clinical research indicates it to be a neuro-protective agent in animal models of ALS, motorneuron diseases, PD, other neuro-degenerative diseases and stroke. GM604 controls and modulates over many known and significant ALS genes with positive effects interactively and dynamically through multiple pathways, and up to twenty-two biological processes, including neuro-protection, neurogenesis, neural development, neuronal signaling, neural transport, and other processes. GM6 is not a cocktail of drugs, but one master regulator peptide drug that functions through multiple pathways. Genervon hypothesized that studying the biomarkers of protein expressions of these ALS genes such as superoxide dismutase 1 (SOD1) and the protein expression of substances such as tau, neurofilament - heavy (NF-H), Cystatin C which were indications of degeneration of neuron in the CSF collected from ALS patients will provide information of the possible GM604's mechanisms of action in treating ALS. 1. This pilot trial is designed to test proof of principle, i.e. determine if a 2-week IV bolus treatment with this agent can (1) change ALS protein expression (target biomarkers and efficacy biomarkers) after treatment (2) have preliminary effects measures of ALS disease clinical progression.

Study Objectives are:

1. To test the safety and tolerability of GM604 in a population of ALS patients.

2. To test for changes in ALS biomarkers before and after treatment.

3. To determine preliminary effects of injections of GM604 on measures of ALS disease biomarkers and clinical progression

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genervon Biopharmaceuticals, LLC
• Collaborators: Columbia University; Massachusetts General Hospital

Criteria

Inclusion Criteria:

1. Patients with ALS: Familial and Sporadic ALS, with symptom onset < or equal to 24 months.

2. At least 18 years of age

3. Subjects meet the El Escorial criteria of definite criteria for a diagnosis of ALS.

4. Subjects can be on a stable dose of riluzole for at least a month or not taking or initiating riluzole for the duration of the trial.

5. Not on any experimental medication for the last 1 month or five times the half-life of experimental medication.

6. At screening, must have a Forced Vital Capacity (FVC) ≥ 65% of predicted capacity for age, height and gender.

7. Have fully completed informed consent form

8. Ability to comply with study procedures

9. Women of child-bearing age must be on birth control. Pregnancy test should be done in women in child bearing age.

10. Medically safe to have lumbar puncture to collect CSF

Exclusion Criteria:

1. History of liver disease, severe renal failure, diabetes, coronary heart disease, cancer

2. Clinically significant EKG abnormality at screening

3. Any comorbid condition which would make completion of the trial unlikely

4. FVC < 65%

5. Presence of a bleeding disorder

6. Allergy to local anesthetics

7. Problem with CSF pressure

8. Topical or other skin infection at the lumbar puncture site

9. BMI > 32 kg/m2

10. Medical or surgical conditions in which a lumbar puncture is contraindicated

11. Use of any anti-platelet or anticoagulant drugs, such as plavix, aggrenox, ticlid, warfarin or coumadin

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