Phase 2A Study of GM 608 in Mild to Moderate Parkinson Disease

NCT01850381 | Completed Phase 2

• 1 other identifier: GBD 002
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

GM608 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system. The study drug is an oligopeptide with a sequence identical to one of the active sites of human Motoneuronotrophic Factor and is manufactured by solid phase synthesis. Preclinical research indicates it to be a neuro-protective agent in animal models of PD, other neuro-degenerative diseases and stroke. This trial is designed to test proof of principle, i.e. determine if a 2-week treatment with this agent can restore the non-functioning nigral dopaminergic neurons in PD over a 3 month period, during which the placebo-treated arm is expected to have little or no worsening of the total UPDRS (Unified Parkinson's Disease Rating Scale)score compared to baseline. Study Objectives are:

1. 1.To compare the safety and tolerability of GM608 with placebo in a population of patients with early PD.
2. 2.To field test the study procedures for feasibility and efficiency
3. 3.To determine if there is any hint that injections of GM608 might slow the rate of clinical worsening of PD.

Conditions

Parkinson's Disease

Keywords

Parkinson Disease; Efficacy; Safety; Tolerability; Phase 2A; Pilot trial; human Motoneuronotrophic Factor; neuroprotective agent; neurodegenerative diseases; rate of clinical progression of PD; UPDRS; Proof of principle

Outcome Measures

Primary Outcomes (1)

• The change from the mean total UPDRS score of the combined screening and baseline visits to the total UPDRS score at the Week-12 visit, comparing treated with placebo.Compare safety and tolerability with placebo.

  Measure UPDRS and the change from the mean total UPDRS score of the combined screening and baseline visits to the total UPDRS score at the Week-12 visit, comparing treated with placebo.Compare safety and tolerability with placebo.

  Baseline, week 2, week 6, week 12

Secondary Outcomes (6)

• Change in total UPDRS between the mean screening-baseline visits and end of Week 2 (at visit 6 after dosing), and week 6, comparing the two arms of the study encompassing the entire cohort of 6 subjects.

  Baseline, week 2, week 6

• Change in UPDRS sub-scores (Mental, Activities of Daily Living, Motor) between the mean screening-baseline visits and ends of Week 2 (at visit 6 after dosing), 6, and 12, comparing the two arms of the study encompassing the entire cohort of 6 subjects.

  Baseline, week 2, week 6, week 12

• Time to the development of sufficient disability to require a change in symptomatic therapy.

  12 weeks

• Proportion of subjects requiring additional symptomatic treatment due to disability.

  12 weeks

• Change in Schwab & England ADL (activity of daily living) score from baseline to Week 2 (at visit 6 after dosing), 6, or 12.

  Baseline, week 2, week 6, week 12

Other Outcomes (1)

• Secondary analyses will consider a comparison of slopes using a mixed-model approach with treatment as a fixed effect and subject-specific slopes as a random effect.

  12 weeks

Study Arms (2)

GM608

EXPERIMENTAL

4 subjects will receive 320 mg of GM608. 6.4 mL of GM608 (50 mg/mL) will be administered intravenously as a slow bolus, once a day for 3 times a week for 2 weeks.

Drug: GM608

Placebo comparator

PLACEBO COMPARATOR

2 subjects will receive matching placebo (bacteriostatic saline). 6.4 mL Bacteriostatic saline will be administered intravenously as a slow bolus, once a day for 3 times a week for 2 weeks.

Drug: Placebo Comparator

Interventions

GM608 DRUG

For GM608: 320 mg/dose reconstituted with 6.4 mL bacteriostatic saline. Administer 6.4 mL GM608 dosing solution by intravenous (IV) bolus over 1 min, on Monday, Wednesday, Friday for two consecutive weeks.

Also known as: GM602, GM6, GM604, MNTF 6mer

GM608

Placebo Comparator DRUG

For Placebo: Administer 6.4 mL Bacteriostatic saline by intravenous (IV) bolus over 1 min, on Monday, Wednesday, Friday for two consecutive weeks.

Also known as: Bacteriostatic saline

Placebo comparator

Eligibility Criteria

• Age: 30 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• with mild-moderate idiopathic PD diagnosed based on UK (United Kingdom) PD Brain Bank criteria.
• Age \> 30
• Motor UPDRS Score ≥ 15
• Hoehn \& Yahr stage \<3
• Diagnosis of PD \<10 years
• Have fully completed informed consent form
• May be on antiparkinsonian medications of an MAO-B (monoamine oxidase -B) inhibitor, an anticholinergic, or amantadine, but not levodopa or dopamine agonist

You may not qualify if:

• Patients with atypical parkinsonism: such as suspected progressive supranuclear palsy (PSP), multiple system atrophy (MSA) or Corticobasal degeneration (CBD) and secondary parkinsonism such as normal-pressure hydrocephalus (NPH), drug-induced, or vascular parkinsonism.
• Patients with uncertainty as to having classical Parkinson disease, such as those who might have scans without evidence of dopaminergic deficit (SWEDDs)
• Patients not willing to give an informed consent
• Patients who are on a dopaminergic medication (levodopa or dopamine agonist)
• Presence of a medical or psychiatric comorbidity that can compromise participation in the study

Sponsors & Collaborators

• Genervon Biopharmaceuticals, LLC: lead
• Columbia University: collaborator

Study Sites (1)

Columbia University Medical Center/NY Presbyterian Hospital

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

Parkinson Disease; Neurodegenerative Diseases

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies

Study Officials

• Stanley Fahn, MD

  Columbia University Medical Center/NY Presbyterian Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 7, 2013
• First Posted: May 9, 2013
• Study Start: June 1, 2013
• Primary Completion: July 1, 2014
• Study Completion: July 1, 2014
• Last Updated: August 8, 2019

Record last verified: 2019-07

Locations

US (1)