Clinical trial NCT03303066: Efficacy and Safety of FG-4592 for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome (MDS)
Official title: A Phase 2/3 Trial of FG-4592 for Treatment of Anemia in Subjects With Lower Risk Myelodysplastic Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Anemia
Myelodysplastic Syndrome
Keywords: Myelodysplastic Syndromes
Anemia
Hemoglobin (Hb)
Low Risk Myelodysplastic Syndrome
Lead sponsor: FibroGen
Sponsor class: INDUSTRY
Collaborators: name: AstraZeneca
First posted: 2017-10-05
Enrollment: 43
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Key Inclusion Criteria:

* 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 Exclusion Criteria:

* 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.

Primary outcomes:
measure: Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion
timeFrame: 26 weeks
Secondary outcomes:
measure: Number of Participants With Hb Increase ≥1.0 g/dL From Baseline
timeFrame: 26 weeks
measure: Time to First Hb Response
timeFrame: 26 weeks
measure: Change From Baseline in Mean Hb in Weeks 25 to 27
timeFrame: Baseline, Weeks 25 to 27
measure: Percentage of Participants With Mean Hb ≥10.0 g/dL Within Any 8-week Period During the Study Without Transfusion
timeFrame: 26 weeks
measure: Number of Participants that Require Transfusions or Hb <6 g/dL up to Week 27
timeFrame: Up to Week 27
measure: 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
timeFrame: 30 weeks
Other outcomes:
Locations:
city: Bengbu
country: China
facility: The First Affiliated Hospital of Bengbu Medical College
state: Anhui
city: Hefei
country: China
facility: Anhui Provincial Hospital
state: Anhui
city: Hefei
country: China
facility: The First Affiliated Hospital of Anhui Medical University
state: Anhui
city: Beijing
country: China
facility: China-Japan Friendship Hospital
state: Beijing
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
city: Beijing
country: China
facility: Peking University First Hospital
state: Beijing
city: Beijing
country: China
facility: Peking University Third Hospital
state: Beijing
city: Beijing
country: China
facility: Xiyuan Hospital, CACMS
state: Beijing
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital
state: Fujian
city: Lanzhou
country: China
facility: Lanzhou University Second Hospital
state: Gansu
city: Guangzhou
country: China
facility: Guangdong General Hospital
state: Guangdong
city: Guangzhou
country: China
facility: Nan Fang Hospital
state: Guangdong
city: Zhuzhou
country: China
facility: Zhuzhou Central Hospital
state: Guangdong
city: Haikou
country: China
facility: Hainan Central Hospital
state: Hainan
city: Shijiazhuang
country: China
facility: Second hospital of Hebei Medical University
state: Hebei
city: Zhengzhou
country: China
facility: Tumor Hospital of Henan province
state: Henan
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College of HUST
state: Hubei
city: Wuhan
country: China
facility: Union Hospital Affiliated Tongji Medical College Huazhong University of Science and Technolog
state: Hubei
city: Changsha
country: China
facility: The Third Xiangya Hospital of Central South University
state: Hunan
city: Nanjing
country: China
facility: Jiangsu Province Hospital
state: Jiangsu
city: Nanjing
country: China
facility: Zhongda Hospital Southeast University
state: Jiangsu
city: Suzhou
country: China
facility: The First Affiliated Hospital Of Soochow University
state: Jiangsu
city: Yangzhou
country: China
facility: Northern Jiangsu People's Hospital
state: Jiangsu
city: Jinan
country: China
facility: Affiliated Hospital of Shandong University of Traditional Chinese Medicine
state: Shandong
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
state: Shandong
city: Jinan
country: China
facility: The Affiliated Hospital of Xuzhou Medical University
state: Shandong
city: Shanghai
country: China
facility: Huashan Hospital Affiliated to Fudan University
state: Shanghai
city: Shanghai
country: China
facility: Shanghai Sixth People's Hospital
state: Shanghai
city: Shanghai
country: China
facility: Shanghai Tongji Hospital
state: Shanghai
city: Xian
country: China
facility: Shaanxi Provincial People's Hospital
state: Shanxi
city: Xian
country: China
facility: The First Affiliated Hospital of Xi'an Jiao Tong University Medical College
state: Shanxi
city: Chengdu
country: China
facility: Sichuan Provincial People's Hospital
state: Sichuan
city: Chengdu
country: China
facility: West China Hospital, Sichuan University
state: Sichuan
city: Tianjin
country: China
facility: Blood disease hospital of Chinese Academy of Medical Sciences
state: Tianjin
city: Tianjin
country: China
facility: The Second Hospital of Tianjin Medical University
state: Tianjin
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
state: Tianjin
city: Kunming
country: China
facility: First Affiliated Hospital of Kunming Medical University
state: Yunnan
city: Hangzhou
country: China
facility: The First Affiliated Hospital, Zhejiang University Medical College
state: Zhejiang
Study officials:
References: