Efficacy and Safety of DVC1-0101 for Intermittent Claudication Secondary to Peripheral Artery Disease: Study Protocol of a Randomized Phase IIb Trial
|Michiko Tanaka1*, Takuya Matsumoto2, Koichi Morisaki2, Ryoichi Kyuragi2, Yuriko Fujino1, Kumi Yoshida1, Yoshikazu Yonemitsu1 and Yoshihiko Maehara2|
|1R&D Laboratory for Innovative Biotherapeutics, Graduate School of Pharmaceutical Sciences, Kyushu University, Fukuoka, Japan|
|2Department of Surgery and Science, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan|
|Corresponding Author :||Michiko Tanaka RN
R&D Laboratory for Innovative Biotherapeutics
Graduate School of Pharmaceutical Sciences
Kyushu University, Rm 601, Station II for Collaborative Research
3-1-1 Maidashi, Higashi-ku, Fukuoka 812-8582, Japan
E-mail: [email protected]
|Received July 25, 2013; Accepted August 29, 2013; Published August 31, 2013|
|Citation: Tanaka M, Matsumoto T, Morisaki K, Kyuragi R, Fujino Y, et al. (2013) Efficacy and Safety of DVC1-0101 for Intermittent Claudication Secondary to Peripheral Artery Disease: Study Protocol of a Randomized Phase IIb Trial. J Clin Trials 3:138. doi:10.4172/2167-0870.1000138|
|Copyright: © 2013 Tanaka M, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.|
1.1. Background: We have developed a new gene transfer vector based on nontransmissible recombinant Sendai virus expressing the human fibroblast growth factor-2 gene (DVC1-0101) to treat peripheral arterial disease. A phase I/IIa open-label four dose-escalation clinical trial for critical limb ischemia was completed. We concluded that DVC1-0101 is safe and well tolerated, and resulted in significant improvement of limb function. We present the protocol of the next phase of our study.
1.2. Methods: We plan to conduct a phase IIb clinical trial, which will be a randomized, placebo-controlled, parallel design, single-dose blinded and single center clinical trial in Japan. This study will enroll 60 patients diagnosed with PAD with intermittent claudication. Subjects who meet eligibility criteria will be randomized to receive a single dose of either placebo, 5 × 109 ciu/limb of DVC1-0101, or 1 × 109 ciu/limb of DVC1-0101 administered by direct intramuscular injection. The participation length in this trial for subjects will be approximately 12 months with nine visits. The primary endpoints are to evaluate the efficacy of DVC1-0101 versus placebo on peak walking time, and to evaluate the safety and tolerability of two dosage levels of DVC1-0101. The secondary endpoints are 1) to evaluate the effect of DVC1-0101 on claudication onset time, measured by a treadmill test and quality of life, measured using the Walking Impairment Questionnaire, 2) to determine the effect of DVC1-0101 on qualifying limb hemodynamics, and 3) to explore the pharmacodynamics of DVC1-0101 by evaluating biomarkers.
1.3. Discussion: The results of this trial will provide insights into the potential of DVC1-0101 for improving walking activities. The results will also help with the design of a possible phase III study.