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Trial record 3 of 3 for:    GM602

Phase 2A Study of GM 608 in Mild to Moderate Parkinson Disease (GAP-PD)

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ClinicalTrials.gov Identifier: NCT01850381
Recruitment Status : Completed
First Posted : May 9, 2013
Last Update Posted : August 8, 2017
Sponsor:
Collaborator:
Columbia University
Information provided by (Responsible Party):
Genervon Biopharmaceuticals, LLC

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

Condition or disease Intervention/treatment Phase
Parkinson's Disease Drug: GM608 Drug: Placebo Comparator Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: GM 608 in A Phase IIA Pilot Double-blinded, Randomized, Placebo Controlled Trial in Mild to Moderate Parkinson Disease
Study Start Date : June 2013
Actual Primary Completion Date : July 2014
Actual Study Completion Date : July 2014

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: GM608
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
For GM608: 320 mg/dose reconstituted with 6.4 mL bateriostatic saline. Administer 6.4 mL GM608 dosing solution by intravenous (IV) bolus over 1 min, on Monday, Wednesday, Friday for two consecutive weeks.
Other Names:
  • GM602
  • GM6
  • GM604
  • MNTF 6mer

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




Primary Outcome Measures :
  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. [ Time Frame: Baseline, week 2, week 6, week 12 ]

Secondary Outcome Measures :
  1. 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. [ Time Frame: Baseline, week 2, week 6 ]
  2. 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. [ Time Frame: Baseline, week 2, week 6, week 12 ]
  3. Time to the development of sufficient disability to require a change in symptomatic therapy. [ Time Frame: 12 weeks ]
  4. Proportion of subjects requiring additional symptomatic treatment due to disability. [ Time Frame: 12 weeks ]
  5. Change in Schwab & England ADL score from baseline to Week 2 (at visit 6 after dosing), 6, or 12. [ Time Frame: Baseline, week 2, week 6, week 12 ]
  6. Change in Hoehn & Yahr score (H&Y), Beck Depression Inventory (BDI), and Montreal Cognitive Assessment (MOCA) scores from baseline to Week 2 (at visit 6 after dosing), 6, or 12. [ Time Frame: Baseline, week 2, week 6, week 12 ]

Other Outcome Measures:
  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. [ Time Frame: 12 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   30 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • with mild-moderate idiopathic PD diagnosed based on UK 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 inhibitor, an anticholinergic, or amantadine, but not levodopa or dopamine agonist

Exclusion Criteria:

  • Patients with atypical parkinsonism (such as suspected PSP, MSA or CBD) and secondary parkinsonism (such as 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01850381


Locations
United States, New York
Columbia University Medical Center/NY Presbyterian Hospital
New York, New York, United States, 10032
Sponsors and Collaborators
Genervon Biopharmaceuticals, LLC
Columbia University
Investigators
Principal Investigator: Stanley Fahn, MD Columbia University Medical Center/NY Presbyterian Hospital

Responsible Party: Genervon Biopharmaceuticals, LLC
ClinicalTrials.gov Identifier: NCT01850381     History of Changes
Other Study ID Numbers: GBD 002
First Posted: May 9, 2013    Key Record Dates
Last Update Posted: August 8, 2017
Last Verified: August 2016

Keywords provided by Genervon Biopharmaceuticals, LLC:
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

Additional relevant MeSH terms:
Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases