EUCTR2011-001718-32-IT进行中（未招募）不适用
A Phase 2, Open Label, Multi-Center, Single-Dose Pharmacokinetics, and Multiple Dose Study of the Safety, Efficacy and Tolerability of FBS0701 in a Pediatric Population with Transfusional Iron Overload - FBS0701/CTP-07
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 30
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. Parents willing and able to sign the approved informed consent for their children and patients between the ages of 6 and 17 willing and able to provide their assent (based on institutional guidelines). 2. Able to swallow whole capsules. 3. Age >6 and <18 years. 4. Transfusion-dependent patients who have transfusional iron overload requiring chronic treatment with deferoxamine, deferasirox, or deferiprone. A transfusion dependent patient is defined in this study as one with a minimum transfusion history totaling more than 20 units of packed red blood cells OR a calculated iron load based on transfusion history of 200 mg/kg AND a transfusion requirement of 7 or more transfusions per year; or, in the case of sickle cell anemia, be iron overloaded but receiving transfusion exchange therapy in lieu of transfusions. 5. Willing to discontinue all existing iron chelation therapies for a minimum period of one to five days prior to enrollment, for the initial PK period of 8 days (if applicable), and for up to 49 weeks if entering into the chronic dosing phase. 6. Patients able to have an MRI must have: a) liver iron concentration (LIC) =2 and <30 mg/g (dry weight, liver) b) cardiac MRI T2* >10 milliseconds (Note: Patients not able to have an MRI will be considered iron overloaded on the basis of serum ferritin only.) 7. Serum ferritin >500 ng/mL at Screening. 8. Mean of the previous three pre-transfusion hemoglobin concentrations =7.5 g/dL. 9. If appropriate depending on age, patient agrees to use an approved method of contraception from Screening and until 30 days after the last administration of the study drug. Agreed methods of contraception for the patient or the patient’s partner may include: condom; use of approved birth control pills, patches, implants or injections; use of diaphragm with vaginal spermicide; use of an intrauterine device (IUD); and/or surgical sterilization (vasectomy or tubal ligation at least six months prior to dosing). Patients practicing abstinence must agree to use an approved method of contraception should they become sexually active during the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. As a result of medical review, physical examination (including height and weight) or Screening investigations, the Principal Investigator considers the patient unfit for the study. 2. Iron overload from causes other than transfusional hemosiderosis. 3. Severe cardiac dysfunction. 4. Non-elective hospitalization within the 30 days prior to Baseline testing. 5. Evidence of clinically significant oral, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurologic, psychiatric, or skin disorder that contra-indicates dosing with FBS0701. 6. Evidence of significant renal insufficiency, e.g. serum creatinine above the upper limit of normal or proteinuria greater than 1 gm per day. 7. Known sensitivity to any ingredient in the FBS0701 formulation. 8. Platelet count below 100,000/µL or absolute neutrophil count less than 1500/mm3 at Screening. 9. ALT >180 IU/mL at Screening. 10. Use of any investigational agent within the 30 days prior to Baseline testing. 11. Pregnant or lactating females.
研究者
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