EUCTR2012-002499-15-IT进行中（未招募）不适用
A Phase 2, Open-Label, Ascending Dose Study to Evaluate the Effects of ACE-536 in Patients with ß-Thalassemia Intermedia
ACCELERON PHARMA INC0 个研究点开始时间: 2012年9月28日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Men or women 18 years of age. 2. Documented diagnosis of ß-thalassemia intermedia, with anemia requiring
- •no more than 6 transfusion events in the last year, with no transfusion events for 56 days prior to Cycle 1 Day 1 3. Prior splenectomy or spleen size < 18 cm in the longest diameter by abdominal ultrasound. 4. Mean hemoglobin concentration < 10.0 g/dL of 2 measurements (one performed within one day prior to Cycle 1 Day 1 and the other performed 7-28 days prior, not influenced by RBC transfusion within 7 days of measurement). 5. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) < 3 x upper limit of normal (ULN). 6. Serum creatinine = 1.5 x ULN. 7. Females of child bearing potential (defined as sexually mature women who have not undergone hysterectomy or bilateral oophorectomy, or are not naturally postmenopausal = 24 consecutive months) must have negative urine or blood pregnancy test prior to enrollment and use adequate birth control methods (abstinence, oral contraceptives, barrier method with spermicide, or surgical sterilization) during study participation. Males must agree to use a latex condom during any sexual contact with females of child-bearing potential while participating in the study and for 12 weeks following the last dose of ACE-536, even if he has undergone a successful vasectomy. Patients must be counseled concerning measures to be used to prevent pregnancy and potential toxicities prior to the first dose of ACE-536. 8. Patients are able to adhere to the study visit schedule, understand and comply with all protocol requirements. 9. Understand and able to provide written informed consent.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range: 0
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 50
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Any clinically significant pulmonary (including pulmonary hypertension), cardiovascular, endocrine, neurologic, hepatic, gastrointestinal, infectious or genitourinary disease considered by the investigator as not adequately controlled prior to Cycle 1 Day 1. 2. Folate deficiency. 3. Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B(HBV) or active infectious hepatitis C (HCV). 4. Known history of thromboembolic events = grade 3 according to the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) v.4.0 (current active minor version). 5. Ejection fraction < 50% by echocardiogram, MUGA or cardiac MRI. 6. Uncontrolled hypertension defined as systolic blood pressure (BP) = 150 mm Hg or diastolic BP = 95 mm Hg. 7. Heart failure class 3 or higher (New York Heart Association, NYHA, Appendix 1). 8. QTc > 450 msec on screening ECG. 9. Platelet count < 100 x109/L or > 1,000,000 x109/L. 10. Proteinuria = Grade 2. 11. Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1. 12. Treatment with another investigational drug or device, or approved therapy for investigational use = 28 days prior to Cycle 1 Day 1, or if the half-life of the previous investigational product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer. 13. Patients receiving or planning to receive hydroxyurea treatment. Patients must not have had hydroxyurea within 56 days of Cycle 1 Day 1. 14. Splenectomy with 56 days prior to Cycle 1 Day 1. 15. Major surgery (except splenectomy) within 28 days prior to Cycle 1 Day 1. Patients must have completely recovered from any previous surgery prior to Cycle 1 Day 1. 16. Iron chelation therapy if initiated within 56 days prior to Cycle 1 Day 1 17. Cytotoxic agents, systemic corticosteroids, immunosuppressants, or anticoagulant therapy such as warfarin or heparin within 28 days priorto Cycle 1 Day 1 (prophylactic aspirin up to 100 mg/d is permitted). 18. Pregnant or lactating females. 19. History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational drug.
研究者
相似试验
进行中（未招募）
1 期
A Phase 2 study with increasing doses of a new medication called ACE-536 in patients suffering from ß-Thalassemia IntermediaEUCTR2012-002499-15-GRAcceleron Pharma, Inc.50
进行中（未招募）
1 期
A Phase 1/2 Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VT-464 in Chemotherapy-Naïve Patients with Castration-Refractory Prostate CancerEUCTR2011-004103-20-GRViamet Pharmaceuticals, Inc.135
进行中（未招募）
不适用
A clinical study in patients with Fabry disease to assess the safety, tolerability, and the body processing of the medication PRX-102, which will be given as an infusioMedDRA version: 17.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersFabry diseaseEUCTR2012-004786-40-GBProtalix Ltd.18
进行中（未招募）
不适用
Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion to Adult Fabry PatientsMedDRA version: 16.1Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersFabry diseaseEUCTR2012-004786-40-ESProtalix Ltd.18
撤回
1 期
Study to evaluate the safety, tolerability and efficacy parameters of the drug PRX-102 administered by intravenous infusion every 2 weeks for 12 weeks, to adult patients with Fabry diseaseC10.228.140.163.100.435.825.200Fabry DiseaseRBR-97trz9Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da USP