PMID- 23609019
OWN - NLM
STAT- MEDLINE
DCOM- 20140207
LR  - 20211021
IS  - 1525-0024 (Electronic)
IS  - 1525-0016 (Print)
IS  - 1525-0016 (Linking)
VI  - 21
IP  - 6
DP  - 2013 Jun
TI  - Phase 1/2 open-label dose-escalation study of plasmid DNA expressing two isoforms 
      of hepatocyte growth factor in patients with painful diabetic peripheral 
      neuropathy.
PG  - 1279-86
LID - 10.1038/mt.2013.69 [doi]
AB  - This study aimed to evaluate the safety and preliminary efficacy of intramuscular 
      injections of plasmid DNA (VM202) expressing two isoforms of hepatocyte growth 
      factor (HGF) in subjects with painful diabetic peripheral neuropathy (PDPN). 
      Twelve patients in three cohorts (4, 8, and 16 mg) received two sets of VM202 
      injections separated by two weeks. Safety and tolerability were evaluated and the 
      visual analog scale (VAS), the short form McGill questionnaire (SF-MPQ), and the 
      brief pain inventory for patients with diabetic peripheral neuropathy (BPI-DPN) 
      measured pain level throughout 12 months after treatment. No serious adverse 
      events (AEs) were observed. The mean VAS was reduced from baseline by 47.2% (P = 
      0.002) at 6 months and by 44.1% (P = 0.005) at 12 months after treatment. The VAS 
      scores for the 4, 8, and 16 mg dose cohorts at 6 months follow-up decreased in a 
      dose-responsive manner, by 21% (P = 0.971), 53% (P = 0.014), and 62% (P = 0.001), 
      respectively. The results with the BPI-DPN and SF-MPQ showed patterns similar to 
      the VAS scores. In conclusion, VM202 treatment appeared to be safe, well 
      tolerated, and sufficient to provide long term symptomatic relief and improvement 
      in the quality of life in patients with PDPN.
FAU - Ajroud-Driss, Senda
AU  - Ajroud-Driss S
AD  - Department of Neurology, Northwestern University's Feinberg School of Medicine, 
      Chicago, Illinois 60611-3008, USA.
FAU - Christiansen, Mark
AU  - Christiansen M
FAU - Allen, Jeffrey A
AU  - Allen JA
FAU - Kessler, John A
AU  - Kessler JA
LA  - eng
GR  - R01 NS020013/NS/NINDS NIH HHS/United States
GR  - R01 NS020778/NS/NINDS NIH HHS/United States
GR  - UL1 TR000150/TR/NCATS NIH HHS/United States
PT  - Clinical Trial, Phase I
PT  - Clinical Trial, Phase II
PT  - Journal Article
PT  - Multicenter Study
PT  - Research Support, Non-U.S. Gov't
DEP - 20130423
PL  - United States
TA  - Mol Ther
JT  - Molecular therapy : the journal of the American Society of Gene Therapy
JID - 100890581
RN  - 0 (Protein Isoforms)
RN  - 67256-21-7 (Hepatocyte Growth Factor)
RN  - 9007-49-2 (DNA)
SB  - IM
MH  - Adult
MH  - Aged
MH  - DNA/genetics
MH  - Diabetic Neuropathies/*therapy
MH  - Endpoint Determination
MH  - Follow-Up Studies
MH  - *Genetic Therapy
MH  - Hepatocyte Growth Factor/*genetics/metabolism
MH  - Humans
MH  - Injections, Intramuscular
MH  - Male
MH  - Middle Aged
MH  - Pain/genetics
MH  - Pain Measurement/methods
MH  - Plasmids/*genetics
MH  - Prospective Studies
MH  - Protein Isoforms/genetics
MH  - Surveys and Questionnaires
PMC - PMC3677315
EDAT- 2013/04/24 06:00
MHDA- 2014/02/08 06:00
PMCR- 2014/06/01
CRDT- 2013/04/24 06:00
PHST- 2013/04/24 06:00 [entrez]
PHST- 2013/04/24 06:00 [pubmed]
PHST- 2014/02/08 06:00 [medline]
PHST- 2014/06/01 00:00 [pmc-release]
AID - S1525-0016(16)32622-3 [pii]
AID - 10.1038/mt.2013.69 [doi]
PST - ppublish
SO  - Mol Ther. 2013 Jun;21(6):1279-86. doi: 10.1038/mt.2013.69. Epub 2013 Apr 23.