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Purpose: To evaluate the tolerability and efficacy of a novel formulation of topical nitroglycerin gel, MQX-503, in the treatment of Raynaud phenomenon (RP) in an “in-life” study.
Methods: This was a multi-center study enrolling subjects with a clinical diagnosis of primary or secondary RP. Enrollment occurred in 2 groups, before or after March 1, 2006. Subjects discontinued vasodilator therapies for 2 weeks, followed by a 2-week single-blind placebo run-in phase to assess baseline disease severity. Those with at least 5 Raynaud attacks in a 7-day period were randomly assigned to receive placebo or 1% MQX-503 in the 4-week double-blinded phase. Subjects used an electronic diary to record a daily Raynaud Condition Score (RCS), a composite self-assessment of the severity of RP rated on a scale of 0 (no attacks) to 10 (severe attacks). The primary outcome was the change in mean RCS at the target week (treatment week that matched the run-in period in terms of ambient temperature) compared to baseline. Analyses were performed on the intention to treat (ITT) population, and stratified by enrollment period and subtype of RP.
Results: Table 1 shows the change in mean RCS at the target week compared to baseline. In each population, MQX-503 improved the RCS more than placebo, with a statistically significant effect in the ITT population and in the subgroup of secondary RP. MQX-503 resulted in greater benefit during the early compared with late enrollment period, and in patients with primary compared with secondary RP.
Adverse events were not statistically different between the treatment and placebo groups.
Conclusions: MQX-503 is well-tolerated and more effective than placebo in the treatment of RP, with more pronounced effects during the winter season and in patients with primary disease.
Methods: This was a multi-center study enrolling subjects with a clinical diagnosis of primary or secondary RP. Enrollment occurred in 2 groups, before or after March 1, 2006. Subjects discontinued vasodilator therapies for 2 weeks, followed by a 2-week single-blind placebo run-in phase to assess baseline disease severity. Those with at least 5 Raynaud attacks in a 7-day period were randomly assigned to receive placebo or 1% MQX-503 in the 4-week double-blinded phase. Subjects used an electronic diary to record a daily Raynaud Condition Score (RCS), a composite self-assessment of the severity of RP rated on a scale of 0 (no attacks) to 10 (severe attacks). The primary outcome was the change in mean RCS at the target week (treatment week that matched the run-in period in terms of ambient temperature) compared to baseline. Analyses were performed on the intention to treat (ITT) population, and stratified by enrollment period and subtype of RP.
Results: Table 1 shows the change in mean RCS at the target week compared to baseline. In each population, MQX-503 improved the RCS more than placebo, with a statistically significant effect in the ITT population and in the subgroup of secondary RP. MQX-503 resulted in greater benefit during the early compared with late enrollment period, and in patients with primary compared with secondary RP.
| Number of Patients | Baseline RCS mean (SD) | Change in RCS mean (%) | p-value | ||||
| MQX | PLB | MQX | PLB | MQX | PLB | ||
| ITT | 111 | 108 | 3.34 (2.07) | 3.19 (1.93) | -0.49 (-14.8) | -0.03 (-0.8) | 0.02 |
| Early enrollment | 55 | 57 | 3.38 (2.10) | 3.68 (2.00) | -0.64 (-18.8) | -0.14 (-3.7) | 0.08 |
| Late enrollment | 56 | 51 | 3.30 (2.06) | 2.64 (1.70) | -0.35 (-10.6) | +0.10 (+3.5) | 0.13 |
| Primary RP | 32 | 37 | 3.60 (2.12) | 3.69 (2.05) | -0.77 (-21.3) | -0.22 (-6.0) | 0.26 |
| Secondary RP | 79 | 71 | 3.32 (2.14) | 2.82 (1.84) | -0.50 (-15.0) | +0.12 (+4.4) | 0.02 |
Adverse events were not statistically different between the treatment and placebo groups.
Conclusions: MQX-503 is well-tolerated and more effective than placebo in the treatment of RP, with more pronounced effects during the winter season and in patients with primary disease.
L. Chung, None; M. Baron, None; D. Collier, None; M. Csuka, None; D. Fiorentino, None; B. Gruber, None; V. Hsu, None; B. Kahaleh, None; R. Martin, None; M. Mayes, None; J. Pope, None; N. Rothfield, None; J. Shanahan, None; L. Shapiro, None; R. Simms, None; E. Smith, None; V. Steen, None; S. Sule, None; N. Sweiss, None; E. Matthews, MediQuest Therapeutics, Inc., 5; A. Gilbert, MediQuest Therapeutics, Inc., 5; F.J. Dechow, MediQuest Therapeutics, Inc., 1; MediQuest Therapeutics, Inc., 3; F.M. Wigley, MediQuest Therapeutics, Inc., 5.