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

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

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

DRUG

GM604

GM604 treated group subject will receive a slow IV bolus injection (\~1min) of 6.4 mL (320mg @50 mg/mL=6.4 mL) for each dose. A total of 6 doses will be administered over two weeks (on Mondays, Wednesdays and Fridays for the first 2 weeks).

DRUG

Placebo comparator

Placebo comparator group subject will receive a slow IV bolus injection (\~1min) of 6.4 mL bacteriostatic saline for each dose. A total of 6 doses will be administered over two weeks (on Mondays, Wednesdays and Fridays for the first 2 weeks).

Sponsors & Collaborators

• Columbia University

  collaborator OTHER

• Massachusetts General Hospital

  collaborator OTHER

• Genervon Biopharmaceuticals, LLC

  lead INDUSTRY

Principal Investigators

• Hiroshi Mitsumoto, MD · Columbia Medical Center NY

• Merit Cudkowicz, MD · Massachusetts General Hospital

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2013-08-31

Primary Completion

2014-04-30

Completion

2014-07-31

Countries

• United States

Study Locations