MQX-503, a novel formulation of nitroglycerin, improves the severity of Raynaud's phenomenon: a randomized, controlled trial.
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.
Chung
Lorinda
L
Stanford University, Stanford, California, and VA Palo Alto Health Care System, Palo Alto, CA 94304, USA. shauwei@stanford.edu
Shapiro
Lee
L
Fiorentino
David
D
Baron
Murray
M
Shanahan
Joseph
J
Sule
Sangeeta
S
Hsu
Vivien
V
Rothfield
Naomi
N
Steen
Virginia
V
Martin
Richard W
RW
Smith
Edwin
E
Mayes
Maureen
M
Simms
Robert
R
Pope
Janet
J
Kahaleh
Bashar
B
Csuka
M E
ME
Gruber
Barry
B
Collier
David
D
Sweiss
Nadera
N
Gilbert
Adam
A
Dechow
Frederick J
FJ
Gregory
Jeffrey
J
Wigley
Fredrick M
FM
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*
D014665:Vasodilator Agents; D005996:Nitroglycerin