Efficacy and Safety of FG-4592 for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome (MDS)
- Registration Number
- NCT03303066
- Lead Sponsor
- FibroGen
- Brief Summary
The objective of this study to evaluate the efficacy FG-4592 for the treatment of anemia in Chinese participants with lower risk MDS.
- Detailed Description
This is a Phase 2/3 trial with sequential Phase 2 and Phase 3 components. In each part, there is an up to 4 weeks screening period followed by a treatment period of 26 weeks and a 4 week follow-up period.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 43
- Diagnosis of primary MDS classified as very low, low or intermediate risk with <5% blasts (documented within 12 weeks prior to Day 1)
- Screening Hb <10 g/dL and ≥6g/dL
- Transfusion independent defined as no red blood cell transfusions within 12 weeks of Day 1
- Erythropoiesis-stimulating agent (ESA)-naïve (not within 30 days of Day 1)
- Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2
Key
- Diagnosis of secondary MDS
- Significant myelofibrosis (>2+fibrosis)
- Prior therapy with azacitidine, decitabine, antithymocyte globulin, cyclosporine, thalidomide, or lenalidomide within 12 weeks prior to Day 1
- Baseline erythropoietin level of >400 units (U)/liter (L)
- Clinically significant anemia due to non-MDS etiologies
Note: Other inclusion and exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Phase 3 Part: FG-4592 FG-4592 Fixed starting doses (different doses for lower body weight \& higher body weight) of FG-4592 administered orally TIW for up to 26 weeks; dose adjustments to Hb levels are allowed during the study. Phase 3 Part: Placebo Placebo Placebo (matching to FG-4592) administered orally TIW for up to 26 weeks. Phase 2 Part: FG-4592 FG-4592 Fixed starting doses (different doses for lower body weight \& higher body weight) of FG-4592 administered orally 3 times a week (TIW) for up to 26 weeks; dose adjustments to Hb levels are allowed during the study.
- Primary Outcome Measures
Name Time Method Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion 26 weeks Hemoglobin response to FG-4592 is defined as an increase in mean Hb of ≥1.0 grams (g)/deciliter (dL) from baseline within any 8-week period during the study without transfusion.
- Secondary Outcome Measures
Name Time Method Number of Participants With Hb Increase ≥1.0 g/dL From Baseline 26 weeks Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion.
Time to First Hb Response 26 weeks Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion.
Change From Baseline in Mean Hb in Weeks 25 to 27 Baseline, Weeks 25 to 27 Percentage of Participants With Mean Hb ≥10.0 g/dL Within Any 8-week Period During the Study Without Transfusion 26 weeks Number of Participants that Require Transfusions or Hb <6 g/dL up to Week 27 Up to Week 27 Mean Change From Baseline in Functional Assessment of Cancer Therapy - Anemia (FACT-An) and Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) Scores at Week 27 30 weeks Frame: Baseline, Week 27
Trial Locations
- Locations (38)
Lanzhou University Second Hospital
🇨🇳Lanzhou, Gansu, China
Zhuzhou Central Hospital
🇨🇳Zhuzhou, Guangdong, China
Second hospital of Hebei Medical University
🇨🇳Shijiazhuang, Hebei, China
Xiyuan Hospital, CACMS
🇨🇳Beijing, Beijing, China
Tongji Hospital, Tongji Medical College of HUST
🇨🇳Wuhan, Hubei, China
Hainan Central Hospital
🇨🇳Haikou, Hainan, China
Shanghai Tongji Hospital
🇨🇳Shanghai, Shanghai, China
Qilu Hospital of Shandong University
🇨🇳Jinan, Shandong, China
West China Hospital, Sichuan University
🇨🇳Chengdu, Sichuan, China
The First Affiliated Hospital of Xi'an Jiao Tong University Medical College
🇨🇳Xian, Shanxi, China
The First Affiliated Hospital of Bengbu Medical College
🇨🇳Bengbu, Anhui, China
The First Affiliated Hospital of Anhui Medical University
🇨🇳Hefei, Anhui, China
China-Japan Friendship Hospital
🇨🇳Beijing, Beijing, China
Peking Union Medical College Hospital
🇨🇳Beijing, Beijing, China
Peking University Third Hospital
🇨🇳Beijing, Beijing, China
Guangdong General Hospital
🇨🇳Guangzhou, Guangdong, China
Fujian Medical University Union Hospital
🇨🇳Fuzhou, Fujian, China
Tumor Hospital of Henan province
🇨🇳Zhengzhou, Henan, China
The Third Xiangya Hospital of Central South University
🇨🇳Changsha, Hunan, China
Union Hospital Affiliated Tongji Medical College Huazhong University of Science and Technolog
🇨🇳Wuhan, Hubei, China
The First Affiliated Hospital Of Soochow University
🇨🇳Suzhou, Jiangsu, China
Jiangsu Province Hospital
🇨🇳Nanjing, Jiangsu, China
Affiliated Hospital of Shandong University of Traditional Chinese Medicine
🇨🇳Jinan, Shandong, China
The Affiliated Hospital of Xuzhou Medical University
🇨🇳Jinan, Shandong, China
Huashan Hospital Affiliated to Fudan University
🇨🇳Shanghai, Shanghai, China
Shanghai Sixth People's Hospital
🇨🇳Shanghai, Shanghai, China
Shaanxi Provincial People's Hospital
🇨🇳Xian, Shanxi, China
Sichuan Provincial People's Hospital
🇨🇳Chengdu, Sichuan, China
Blood disease hospital of Chinese Academy of Medical Sciences
🇨🇳Tianjin, Tianjin, China
Tianjin Medical University General Hospital
🇨🇳Tianjin, Tianjin, China
The First Affiliated Hospital, Zhejiang University Medical College
🇨🇳Hangzhou, Zhejiang, China
Anhui Provincial Hospital
🇨🇳Hefei, Anhui, China
Nan Fang Hospital
🇨🇳Guangzhou, Guangdong, China
Zhongda Hospital Southeast University
🇨🇳Nanjing, Jiangsu, China
Northern Jiangsu People's Hospital
🇨🇳Yangzhou, Jiangsu, China
The Second Hospital of Tianjin Medical University
🇨🇳Tianjin, Tianjin, China
First Affiliated Hospital of Kunming Medical University
🇨🇳Kunming, Yunnan, China
Peking University First Hospital
🇨🇳Beijing, Beijing, China