title: MQX-503, a novel formulation of nitroglycerin, improves the severity of Raynaud's phenomenon: a randomized, controlled trial.
abstract: OBJECTIVE
  Raynaud's phenomenon (RP) affects 3-9% of the general population and >90% of patients with systemic sclerosis. Nitrates are often prescribed for the treatment of RP, but currently available formulations are limited by side effects, particularly headaches, dizziness, and skin irritation. The purpose of this study was to evaluate the tolerability and efficacy of a novel formulation of topical nitroglycerin, MQX-503, in the treatment of RP in an ambulatory setting.
  
  
  METHODS
  We conducted a multicenter, randomized, placebo-controlled study with a 2-week single-blind run-in period to determine baseline severity, followed by a 4-week double-blind treatment phase. Two hundred nineteen adult patients with a clinical diagnosis of primary or secondary RP received 0.9% MQX-503 gel or matching placebo during the treatment period. Gel was applied immediately before or within 5 minutes of the beginning of an episode of RP (maximum of 4 applications daily). End points included the change in the mean Raynaud's Condition Score (RCS; scale 0-10), the frequency and duration of episodes, and subjective assessments at the target week (the week during the treatment phase that most closely matched the run-in period in terms of ambient temperature) compared with baseline.
  
  
  RESULTS
  The mean (%) change in the RCS at the target week compared with baseline was significantly greater in the MQX-503 group (0.48 [14.3%]) than that in the placebo group (0.04 [1.3%]; P = 0.04). Changes in the frequency and duration of RP episodes and subjective assessments were not statistically different between the groups. MQX-503 had a side effect profile similar to that of placebo.
  
  
  CONCLUSIONS
  MQX-503 is well tolerated and more effective than placebo for the treatment of RP.
authors.affiliation: Stanford University, Stanford, California, and VA Palo Alto Health Care System, Palo Alto, CA 94304, USA. shauwei@stanford.edu
authors.forename: Lorinda
authors.initials: L
authors.lastname: Chung
authors.forename: Lee
authors.initials: L
authors.lastname: Shapiro
authors.forename: David
authors.initials: D
authors.lastname: Fiorentino
authors.forename: Murray
authors.initials: M
authors.lastname: Baron
authors.forename: Joseph
authors.initials: J
authors.lastname: Shanahan
authors.forename: Sangeeta
authors.initials: S
authors.lastname: Sule
authors.forename: Vivien
authors.initials: V
authors.lastname: Hsu
authors.forename: Naomi
authors.initials: N
authors.lastname: Rothfield
authors.forename: Virginia
authors.initials: V
authors.lastname: Steen
authors.forename: Richard W
authors.initials: RW
authors.lastname: Martin
authors.forename: Edwin
authors.initials: E
authors.lastname: Smith
authors.forename: Maureen
authors.initials: M
authors.lastname: Mayes
authors.forename: Robert
authors.initials: R
authors.lastname: Simms
authors.forename: Janet
authors.initials: J
authors.lastname: Pope
authors.forename: Bashar
authors.initials: B
authors.lastname: Kahaleh
authors.forename: M E
authors.initials: ME
authors.lastname: Csuka
authors.forename: Barry
authors.initials: B
authors.lastname: Gruber
authors.forename: David
authors.initials: D
authors.lastname: Collier
authors.forename: Nadera
authors.initials: N
authors.lastname: Sweiss
authors.forename: Adam
authors.initials: A
authors.lastname: Gilbert
authors.forename: Frederick J
authors.initials: FJ
authors.lastname: Dechow
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Gregory
authors.forename: Fredrick M
authors.initials: FM
authors.lastname: Wigley
mesh_terms: D000287:Administration, Topical; D000328:Adult; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D005996:Nitroglycerin / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D011928:Raynaud Disease / Q000188:drug therapy* / Q000473:pathology; D012720:Severity of Illness Index; D016037:Single-Blind Method; D014665:Vasodilator Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D014665:Vasodilator Agents; D005996:Nitroglycerin