Two-year data from a natural history study of Duchenne muscular dystrophy (DMD) in Chinese individuals


Clinical Trials

Poster Number: V408


xihua Li, PhD, Fudan University Children Hospital, Shanghai, China, Junlan Lv, MD, Beijing Children‘s Hospital, Capital Medical University, Beijing, China, Xingzhi Chang, MD, Peking University First Hospital, Beijing, China, Zhiqiang Wang, MD, The First Affiliated Hospital Of Fujian Medical University, China, Siqi Hong, MD, Children's Hospital of Chongqing Medical University, Chong Qing, China, Wenhua Zhu, MD, Huashan Hospital, Fudan University, Shanghai, China, Qi Shen, MD, Pfizer, Yangmei Zhou, PhD, Pfizer, Shanghai, China, Jia Fang, MS, Pfizer, Shanghai, China, Yingxu Gao, MS, Pfizer, Beijing, China, Caiping Jia, MS, Pfizer, Shanghai, China

Background: This abstract reports 2-year (y) data from a prospective natural history study of DMD in Chinese individuals (NCT03760029).
Methods: Chinese males (N=312, any age) with DMD were allocated as follows: Group 1: ambulatory, <6y (n=99); Group 2: ambulatory, ≥6y (n=177); Group 3: non-ambulatory, any age (n=36). Primary endpoints included age at failure to walk, North Star Ambulatory Assessment (NSAA, ambulatory individuals ≥3y old), Performance of Upper Limb (PUL, individuals ≥10y old), percent predicted forced vital capacity (%pFVC, individuals ≥6y old), and left ventricular ejection fraction (LVEF, individuals ≥6y old). Data are mean (standard deviation) unless specified. Results: Age at baseline was 4.0 (1.2), 9.0 (2.0), 12.7 (2.7), and 7.9 (3.4) y in Groups 1, 2, 3, and overall, respectively. Kaplan-Meier estimates of median (95% confidence interval) age at failure to walk was 14.4y (13.9, not evaluable) in Group 2, 11.1y (10.0, 11.4) in Group 3, and 13.1y (12.5, 14.1) overall. No event of failure to walk was reported in Group 1; all individuals in Group 3 reported failure to walk. At 2y, change from baseline in NSAA was 3.9 (4.7), -4.9 (5.2), and -2.1 (6.5) in Groups 1, 2, and all ambulatory individuals, respectively. Change from baseline in PUL was -2.8 (3.9) in Group 2, -2.9 (3.2) in Group 3, and -2.8 (3.7) in all assessed individuals. Change from baseline in %pFVC was -4.3% (16.0%), -14.1% (20.7%), and -5.7% (17.0%) in Groups 2, 3, and all assessed individuals, respectively. Change from baseline in LVEF was minimal in Groups 2, 3, and all assessed individuals (-0.7% [5.4%], -1.6% [7.6%], and -0.8% [5.8%], respectively). Conclusions: At 2y, DMD progression in this Chinese population is consistent with findings from previous studies. These results demonstrate a general decline of motor, pulmonary, and cardiac function compared with 1y. This study was sponsored by Pfizer.