Phase 2A Study of GM 608 in Mild to Moderate Parkinson Disease (GAP-PD)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01850381|
Recruitment Status : Completed
First Posted : May 9, 2013
Last Update Posted : August 8, 2017
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:
- To compare the safety and tolerability of GM608 with placebo in a population of patients with early PD.
- To field test the study procedures for feasibility and efficiency
- 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|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
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.
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.
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
- 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 ]
- 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 ]
- 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 ]
- Time to the development of sufficient disability to require a change in symptomatic therapy. [ Time Frame: 12 weeks ]
- Proportion of subjects requiring additional symptomatic treatment due to disability. [ Time Frame: 12 weeks ]
- 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 ]
- 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 ]
- 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 ]
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
|United States, New York|
|Columbia University Medical Center/NY Presbyterian Hospital|
|New York, New York, United States, 10032|
|Principal Investigator:||Stanley Fahn, MD||Columbia University Medical Center/NY Presbyterian Hospital|